A Phase 2/3 interventional study of volanesorsen and Placebo in Familial Partial Lipodystrophy, sponsored by Akcea Therapeutics. Terminated at 12 sites in 7 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2021-10-18.
Sponsored by Akcea Therapeutics (part of Ionis Pharmaceuticals, Inc.) · Phase 2/3, Interventional, and Treatment
The purpose of this study is to evaluate the efficacy and safety of volanesorsen given for 52 weeks in a randomized treatment (RT) period in participants with familial partial lipodystrophy (FPL). Following the randomized treatment period, participants who did not enter the open-label extension (OLE) period went straight to the 13-week post-treatment (PT) follow-up period and participants who were entered in the OLE period continued to receive volanesorsen for another 52 weeks (Weeks 53 to 104). Following the Week 104 visit of the OLE period, participants had an option of continued dosing for up to an additional 52 weeks (Week 105 to 156). Participants who did not enter the OLE period went straight to a 13-week post-treatment follow-up period. Following the Week 104 OLE period, participants were entered a 13-week post-treatment follow-up period, if they did not choose the option for continued dosing.
20 studies on the registry are indexed under Lipodystrophy, Familial Partial; 5 are open to participants now.
This study's enrollment of 40 is above the median of 16 across 14 interventional studies indexed under Lipodystrophy, Familial Partial.
Browse Lipodystrophy, Familial Partial studies →Akcea Therapeutics is the lead sponsor of 15 studies on the registry; 1 is open to participants now.
Of its 9 completed or terminated interventional studies of FDA-regulated products, 8 (89%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Diagnosis of FPL is based on deficiency of subcutaneous body fat in a partial fashion assessed by physical examination and low skinfold thickness in anterior thigh by caliper measurement: men (less than or equal to [≤] 10 millimeters [mm]) and women (≤ 22 mm), and at least 1 of the following:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HbA1c
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Randomized Period: Volanesorsen-matching placebo as SC, QW for Weeks 1-52. Participants who received volanesorsen-matching placebo in RT period and not enter in OLE period went straight to 13-week PT follow-up period. Dose adjustment based on monitoring rules were allowed. OLE Period: Participants who received volanesorsen-matching placebo in RT period and completed RT period, were to receive 300 mg of volanesorsen as SC QW for 52 weeks (Weeks 53-104) in OLE period. Dose adjustment based on monitoring rules were allowed. After Week 104, participants had option of continuing treatment with 300 mg of volanesorsen as SC injection for up to additional 52 weeks (Weeks 105-156). Participants not entered in option for additional 52 weeks of dosing in OLE PT period went straight to 13-week PT follow-up period after completion of first 52 weeks (Weeks 53-104) of OLE. Participants entered in OLE PT period went straight to 13-week PT follow-up period after completion of Week 156 of OLE.
Drug: volanesorsen · Drug: Placebo
Randomized Period: 300 mg of volanesorsen as SC, QW for Weeks 1-52. Participants who received 300 mg of volanesorsen in RT period and did not enter in OLE period went straight to 13-week PT follow-up period. Dose adjustment based on monitoring rules were allowed. OLE Period: Participants who received volanesorsen in RT period and completed RT period, were to receive 300 mg of volanesorsen as SC QW for 52 weeks (Weeks 53-104) in OLE period. Dose adjustment based on monitoring rules were allowed. After Week 104, participants had option of continuing treatment with 300 mg of volanesorsen as SC injection for up to additional 52 weeks (Week 105-156). Participants who were not entered in option for additional 52 weeks of dosing in OLE PT period went straight to 13-week PT follow-up period after completion of first 52 weeks (Weeks 53-104) of OLE. Participants entered in OLE PT period went straight to 13-week PT follow-up period after completion of Week 156 of OLE.
Drug: volanesorsen
300 mg of volanesorsen administered subcutaneous (SC) injection, once-weekly (QW).
Also known as: ISIS 304801, IONIS-APOCIIIRx
Volanesorsen-matching placebo administered SC injection.
Randomized Treatment Period: Percent Change From Baseline to Month 3 in Fasting Triglycerides (TG)
Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 predose fasting assessment. Month 3 value was defined as the average of Week 12 and Week 13 fasting TG assessments of the randomized treatment period. The data was analyzed using an analysis of covariance (ANCOVA) model with the randomization stratification factor (diagnosis of disease with or without genetics and family history) and treatment group as factors and log-transformed baseline fasting TG as a covariate.
Time frame: Baseline to Month 3
Randomized Treatment Period: Percent Change From Baseline in Hepatic Steatosis as Assessed by Hepatic Fat Fraction Using Magnetic Resonance Imaging (MRI)
Baseline was defined as the last non-missing assessment prior to the first dose of study drug in the randomized treatment period. Randomized treatment period: Month 6 value was defined as Week 25 or Week 26 for MRI assessment and Month 12 was defined as Week 50 or Week 52 for MRI assessment. Hepatic steatosis is a reversible condition in which large vacuoles of triglyceride fat accumulate in the liver cells, causing nonspecific inflammation. Hepatic Steatosis was assessed by hepatic fat fraction using MRI.
Time frame: Baseline, Months 6 and 12
Open-Label Extension Period: Percent Change From Baseline in Hepatic Steatosis as Assessed by Hepatic Fat Fraction Using MRI
Baseline was defined as the last non-missing assessment prior to the first dose of study drug in the randomized treatment period. Open-label extension period: Month 6 value was defined as Week 77 or Week 78 for MRI assessment and Month 12 value was defined as Week 102 or Week 104 for MRI assessment. Hepatic steatosis is a reversible condition in which large vacuoles of triglyceride fat accumulate in the liver cells, causing nonspecific inflammation. Hepatic Steatosis was assessed by hepatic fat fraction using MRI.
Time frame: Baseline, Months 6 and 12
Randomized Treatment Period: Change From Baseline in Hemoglobin A1c (HbA1c)
Baseline was defined as the last non-missing assessment prior to the first dose of study drug. Randomized treatment period: The Month 3 value was defined as Week 13, Month 6 value was defined as Week 26, Month 9 value was defined as Week 38 and Month 12 value was defined as Week 52.
Time frame: Baseline, Months 3, 6, 9, and 12
Open Label Extension Period: Change From Baseline in HbA1c
Baseline was defined as the last non-missing assessment prior to the first dose of study drug. Open-label extension period: Month 3 value was defined as Week 65, Month 6 value was defined as Week 78, Month 9 value was defined as Week 90 and Month 12 value was defined as Week 104.
Time frame: Baseline, Months 3, 6, 9, and 12
Randomized Treatment Period: Percentage of Participants Who Achieved Greater Than or Equal to (≥) 40% Reduction in Fasting Triglyceride and ≥ 30% Reduction of Hepatic Fat Fraction at Month 6
The baseline of TG is defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. The baseline of hepatic fat fraction is defined as the last non-missing assessment prior to the first dose of study drug. Randomized treatment period: Month 6 value was defined as average of Week 25 and Week 26 for fasting TG and Week 25 or Week 26 for hepatic fat fraction.
Time frame: Month 6
Randomized Treatment Period: Change From Baseline in Disease Burden Score
The Disease Burden Score is a questionnaire that allows participants to self-report their chronic conditions and then assess the degree to which each condition interferes with daily activities.
Time frame: From the first dose of study drug to Week 52
Open-Label Extension Period: Change From Baseline in Disease Burden Score
The Disease Burden Score is a questionnaire that allows participants to self-report their chronic conditions and then assess the degree to which each condition interferes with daily activities.
Time frame: From the first dose of study drug in open label extension period to Week 117
Randomized Treatment Period: Patient-Reported Pain
Patient-reported pain was assessed by rating pain symptoms at its worst and least for the last 24 hours, on average, and at the moment, with 0 as the lowest score (no pain) and 10 as the highest score (worst pain as you can imagine). Patient-reported pain was also assessed by rating pain symptoms (rate pain on average, rate pain right now) that interfered with general activity, interfered with mood, walking ability, normal work, relations with other people, sleep, and enjoyment of life, with 0 as the lowest score (did not interfered) and 10 as the highest score (completely interfered). The scores from each assessment time point were averaged for all of the below reported categories.
Time frame: From the first dose of study drug up to Week 52
Open Label Extension Period: Patient-Reported Pain
Patient-reported pain was assessed by rating pain symptoms at its worst and least for the last 24 hours, on average, and at the moment, with 0 as the lowest score (no pain) and 10 as the highest score (worst pain as you can imagine). Patient-reported pain was also assessed by rating pain symptoms (rate pain on average, rate pain right now) that interfered with general activity, interfered with mood, walking ability, normal work, relations with other people, sleep, and enjoyment of life, with 0 as the lowest score (did not interfered) and 10 as the highest score (completely interfered). The scores from each assessment time point were averaged for all of the below reported categories.
Time frame: From the first dose of study drug in open label extension period up to Week 117
Randomized Treatment Period: Patient-Reported Hunger
Patient-reported hunger was assessed by participants who completed a questionnaire about: how hungry you feel, how satisfied you feel, how full you feel, how much you think you can eat, like to eat something sweet, like to eat something salty, like to eat something savory and like to eat something fatty. Participants also rated the palatability of meals that included visual appeal, smell, taste, and aftertaste. Scores of 1-39 were categorized as mild, 40-69 as moderate, and 70-100 as severe. The scores from each assessment time point were averaged for all of the below reported categories.
Time frame: From the first dose of study drug up to Week 52
Open Label Extension Period: Patient-Reported Hunger
Patient-reported hunger was assessed by participants who completed a questionnaire about: how hungry you feel, how satisfied you feel, how full you feel, how much you think you can eat, like to eat something sweet, like to eat something salty, like to eat something savory and like to eat something fatty. Participants also rated the palatability of meals that included visual appeal, smell, taste, and aftertaste. Scores of 1-39 were categorized as mild, 40-69 as moderate, and 70-100 as severe. The scores from each assessment time point were averaged for all of the below reported categories.
Time frame: From the first dose of study drug in open label extension period up to Week 117
Randomized Treatment Period: Change From Baseline in Mean Short Form-36 (SF-36) Weighted Sum of Scores
The SF-36 Health Survey is a 36-item, patient-reported survey of patient health. SF-36 consists of 8 health dimensions,which are weighted sums of the questions in each section. SF-36 included 36 questions related to 8 health dimensions:physical functioning, physical role functioning, bodily pain, general health perceptions, vitality, social role functioning,emotional role functioning, and mental health. Each dimension was scored on a scale of 0 to 100 where, higher score = better quality of life. A positive change from Baseline indicates improvement.
Time frame: Baseline, Weeks 13, 26 and 52
Open-Label Extension Period: Change From Baseline in Mean SF-36 Weighted Sum of Scores
The SF-36 Health Survey is a 36-item, patient-reported survey of patient health. SF-36 consists of 8 health dimensions,which are weighted sums of the questions in each section. SF-36 included 36 questions related to 8 health dimensions:physical functioning, physical role functioning, bodily pain, general health perceptions, vitality, social role functioning,emotional role functioning, and mental health. Each dimension was scored on a scale of 0 to 100 where, higher score = better quality of life. A positive change from Baseline indicates improvement.
Time frame: Baseline, Weeks 65, 78 and 104
Randomized Treatment Period: Change From Baseline in Mean EQ-5D: Index Scores and Visual Analog Scale (VAS)
EQ-5D-5L is a standardized health-related quality of life questionnaire developed by EuroQol Group in order to provide a simple, generic measure of health for clinical and economic appraisal. EQ-5D-5L consists of two components: a health state profile and VAS. EQ-5D health state profile is comprised of 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 levels: 1=no problems, 2=slight problems, 3=moderate problems, 4=severe problems, and 5=extreme problems. The 5D-5L systems are converted into a single index utility score between 0 to 1, where higher score indicates a better health state. EQ-5D-5L- VAS is designed to rate the participant's current health state on a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A negative change from baseline indicates worsening. A positive change from baseline indicates improvement.
Time frame: Baseline, Weeks 13, 26 and 52
Open-Label Extension Period: Change From Baseline in Mean EQ-5D: Index and Visual Analog Scale (VAS) Scores
EQ-5D-5L is a standardized health-related quality of life questionnaire developed by EuroQol Group in order to provide a simple, generic measure of health for clinical and economic appraisal. EQ-5D-5L consists of two components: a health state profile and VAS. EQ-5D health state profile is comprised of 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 levels: 1=no problems, 2=slight problems, 3=moderate problems, 4=severe problems, and 5=extreme problems. The 5D-5L systems are converted into a single index utility score between 0 to 1, where higher score indicates a better health state. EQ-5D-5L- VAS is designed to rate the participant's current health state on a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A negative change from baseline indicates worsening. A positive change from baseline indicates improvement.
Time frame: Baseline, Weeks 65, 78 and 104
The study was conducted at 12 study centers in the United States, Russia, Brazil, Germany, Belgium, Canada, and Netherlands from 28 December 2015 to 13 November 2019.
| Milestone | Randomized Treatment Period: Placebo | Randomized Treatment Period: Volanesorsen | Randomized Post-Treatment Follow-up Period: Placebo | Randomized Post-Treatment Follow-up Period: Volanesorsen | Open-Label Extension Period (OLE and OLE Post-Treatment Follow-up): Placebo/Volanesorsen | Open-Label Extension Period (OLE and OLE Post-Treatment Follow-up): Volanesorsen/Volanesorsen |
|---|---|---|---|---|---|---|
| Started | 19 | 21 | 0 | 0 | 0 | 0 |
| Completed | 13 | 14 | 0 | 0 | 0 | 0 |
| Not completed | 6 | 7 | 0 | 0 | 0 | 0 |
| Withdrew: Investigator judgement | 1 | 0 | 0 | 0 | 0 | 0 |
| Withdrew: Voluntary withdrawal | 1 | 0 | 0 | 0 | 0 | 0 |
| Withdrew: Adverse event (ae) or serious adverse event (sae) | 0 | 4 | 0 | 0 | 0 | 0 |
| Withdrew: Other | 4 | 3 | 0 | 0 | 0 | 0 |
| Milestone | Randomized Treatment Period: Placebo | Randomized Treatment Period: Volanesorsen | Randomized Post-Treatment Follow-up Period: Placebo | Randomized Post-Treatment Follow-up Period: Volanesorsen | Open-Label Extension Period (OLE and OLE Post-Treatment Follow-up): Placebo/Volanesorsen | Open-Label Extension Period (OLE and OLE Post-Treatment Follow-up): Volanesorsen/Volanesorsen |
|---|---|---|---|---|---|---|
| Started | 0 | 0 | 7 | 9 | 0 | 0 |
| Completed | 0 | 0 | 6 | 7 | 0 | 0 |
| Not completed | 0 | 0 | 1 | 2 | 0 | 0 |
| Withdrew: Ae or sae | 0 | 0 | 1 | 0 | 0 | 0 |
| Withdrew: Other | 0 | 0 | 0 | 2 | 0 | 0 |
| Milestone | Randomized Treatment Period: Placebo | Randomized Treatment Period: Volanesorsen | Randomized Post-Treatment Follow-up Period: Placebo | Randomized Post-Treatment Follow-up Period: Volanesorsen | Open-Label Extension Period (OLE and OLE Post-Treatment Follow-up): Placebo/Volanesorsen | Open-Label Extension Period (OLE and OLE Post-Treatment Follow-up): Volanesorsen/Volanesorsen |
|---|---|---|---|---|---|---|
| Started | 0 | 0 | 0 | 0 | 12 | 12 |
| Completed | 0 | 0 | 0 | 0 | 1 | 3 |
| Not completed | 0 | 0 | 0 | 0 | 11 | 9 |
| Withdrew: Investigator judgement | 0 | 0 | 0 | 0 | 1 | 0 |
| Withdrew: Voluntary withdrawal | 0 | 0 | 0 | 0 | 3 | 1 |
| Withdrew: Ae or sae | 0 | 0 | 0 | 0 | 2 | 1 |
| Withdrew: Other | 0 | 0 | 0 | 0 | 5 | 7 |
| Milestone | Randomized Treatment Period: Placebo | Randomized Treatment Period: Volanesorsen | Randomized Post-Treatment Follow-up Period: Placebo | Randomized Post-Treatment Follow-up Period: Volanesorsen | Open-Label Extension Period (OLE and OLE Post-Treatment Follow-up): Placebo/Volanesorsen | Open-Label Extension Period (OLE and OLE Post-Treatment Follow-up): Volanesorsen/Volanesorsen |
|---|---|---|---|---|---|---|
| Started | 0 | 0 | 0 | 0 | 1 | 2 |
| Completed | 0 | 0 | 0 | 0 | 0 | 0 |
| Not completed | 0 | 0 | 0 | 0 | 1 | 2 |
| Withdrew: Other | 0 | 0 | 0 | 0 | 1 | 2 |
| Milestone | Randomized Treatment Period: Placebo | Randomized Treatment Period: Volanesorsen | Randomized Post-Treatment Follow-up Period: Placebo | Randomized Post-Treatment Follow-up Period: Volanesorsen | Open-Label Extension Period (OLE and OLE Post-Treatment Follow-up): Placebo/Volanesorsen | Open-Label Extension Period (OLE and OLE Post-Treatment Follow-up): Volanesorsen/Volanesorsen |
|---|---|---|---|---|---|---|
| Started | 0 | 0 | 0 | 0 | 12 | 12 |
| Completed | 0 | 0 | 0 | 0 | 11 | 11 |
| Not completed | 0 | 0 | 0 | 0 | 1 | 1 |
| Withdrew: Voluntary withdrawal | 0 | 0 | 0 | 0 | 1 | 0 |
| Withdrew: Other | 0 | 0 | 0 | 0 | 0 | 1 |
Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 predose fasting assessment. Month 3 value was defined as the average of Week 12 and Week 13 fasting TG assessments of the randomized treatment period. The data was analyzed using an analysis of covariance (ANCOVA) model with the randomization stratification factor (diagnosis of disease with or without genetics and family history) and treatment group as factors and log-transformed baseline fasting TG as a covariate.
| percent change | Randomized Treatment Period: Placebo | Randomized Treatment Period: Volanesorsen |
|---|---|---|
| Randomized Treatment Period: Percent Change From Baseline to Month 3 in Fasting Triglycerides (TG) | -21.64 (-60.85 to 17.57) | -88.47 (-133.56 to -43.38) |
Baseline was defined as the last non-missing assessment prior to the first dose of study drug in the randomized treatment period. Randomized treatment period: Month 6 value was defined as Week 25 or Week 26 for MRI assessment and Month 12 was defined as Week 50 or Week 52 for MRI assessment. Hepatic steatosis is a reversible condition in which large vacuoles of triglyceride fat accumulate in the liver cells, causing nonspecific inflammation. Hepatic Steatosis was assessed by hepatic fat fraction using MRI.
| percent change | Randomized Treatment Period: Placebo | Randomized Treatment Period: Volanesorsen |
|---|---|---|
| Percent Change at Month 6 | 2.83 (-23.46 to 29.12) | -22.86 (-52.07 to 6.34) |
| Percent Change at Month 12 | 1.46 (-30.49 to 33.42) | -51.87 (-87.87 to -15.87) |
Baseline was defined as the last non-missing assessment prior to the first dose of study drug in the randomized treatment period. Open-label extension period: Month 6 value was defined as Week 77 or Week 78 for MRI assessment and Month 12 value was defined as Week 102 or Week 104 for MRI assessment. Hepatic steatosis is a reversible condition in which large vacuoles of triglyceride fat accumulate in the liver cells, causing nonspecific inflammation. Hepatic Steatosis was assessed by hepatic fat fraction using MRI.
| percent change | Open-Label Extension Period: Placebo/Volanesorsen | Open-Label Extension Period: Volanesorsen/Volanesorsen |
|---|---|---|
| Percent Change at Month 6 | -18.4 ± 54.7 | -60.2 ± 43.6 |
| Percent Change at Month 12 | -93.5 ± 44.4 | -22.1 ± 47.5 |
Baseline was defined as the last non-missing assessment prior to the first dose of study drug. Randomized treatment period: The Month 3 value was defined as Week 13, Month 6 value was defined as Week 26, Month 9 value was defined as Week 38 and Month 12 value was defined as Week 52.
| percentage of HbA1c | Randomized Treatment Period: Placebo | Randomized Treatment Period: Volanesorsen |
|---|---|---|
| Change at Month 3 | -0.51 (-1.22 to 0.20) | -0.21 (-1.00 to 0.58) |
| Change at Month 6 | 0.06 (-1.02 to 1.15) | 0.26 (-0.98 to 1.50) |
| Change at Month 9 | 0.68 (-0.49 to 1.85) | 0.48 (-0.84 to 1.80) |
| Change at Month 12 | 0.48 (-0.49 to 1.45) | 0.28 (-0.78 to 1.35) |
Baseline was defined as the last non-missing assessment prior to the first dose of study drug. Open-label extension period: Month 3 value was defined as Week 65, Month 6 value was defined as Week 78, Month 9 value was defined as Week 90 and Month 12 value was defined as Week 104.
| percentage of HbA1c | Open-Label Extension Period: Placebo/Volanesorsen | Open-Label Extension Period: Volanesorsen/Volanesorsen |
|---|---|---|
| Change at Month 3 | 0.42 ± 1.54 | 0.91 ± 2.18 |
| Change at Month 6 | 0.35 ± 1.54 | -0.75 ± 0.35 |
| Change at Month 9 | 0.35 ± 1.06 | -0.05 ± 0.64 |
| Change at Month 12 | 0.00 ± 0.71 | 0.30 ± 0.99 |
The baseline of TG is defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. The baseline of hepatic fat fraction is defined as the last non-missing assessment prior to the first dose of study drug. Randomized treatment period: Month 6 value was defined as average of Week 25 and Week 26 for fasting TG and Week 25 or Week 26 for hepatic fat fraction.
| percentage of participants | Randomized Treatment Period: Placebo | Randomized Treatment Period: Volanesorsen |
|---|---|---|
| Randomized Treatment Period: Percentage of Participants Who Achieved Greater Than or Equal to (≥) 40% Reduction in Fasting Triglyceride and ≥ 30% Reduction of Hepatic Fat Fraction at Month 6 | 5.3 | 42.9 |
The Disease Burden Score is a questionnaire that allows participants to self-report their chronic conditions and then assess the degree to which each condition interferes with daily activities.
No measurements were reported for this outcome.
The Disease Burden Score is a questionnaire that allows participants to self-report their chronic conditions and then assess the degree to which each condition interferes with daily activities.
No measurements were reported for this outcome.
Patient-reported pain was assessed by rating pain symptoms at its worst and least for the last 24 hours, on average, and at the moment, with 0 as the lowest score (no pain) and 10 as the highest score (worst pain as you can imagine). Patient-reported pain was also assessed by rating pain symptoms (rate pain on average, rate pain right now) that interfered with general activity, interfered with mood, walking ability, normal work, relations with other people, sleep, and enjoyment of life, with 0 as the lowest score (did not interfered) and 10 as the highest score (completely interfered). The scores from each assessment time point were averaged for all of the below reported categories.
| score on a scale | Randomized Treatment Period: Placebo | Randomized Treatment Period: Volanesorsen |
|---|---|---|
| Rate Pain at its Worst Last 24 Hours | 3.28 ± 2.79 | 3.57 ± 2.72 |
| Rate Pain at its Least Last 24 Hours | 2.45 ± 2.46 | 2.59 ± 2.43 |
| Rate Pain on Average | 3.08 ± 2.62 | 3.16 ± 2.47 |
| Rate Pain Right Now | 2.72 ± 2.59 | 2.96 ± 2.50 |
| General Activity | 2.43 ± 2.80 | 2.83 ± 2.57 |
| Interfere With Mood | 2.31 ± 2.88 | 2.98 ± 2.59 |
| Walking Ability | 2.35 ± 2.96 | 2.79 ± 2.59 |
| Normal Work | 2.37 ± 2.84 | 2.89 ± 2.56 |
| Relations With Other People | 2.23 ± 2.79 | 2.62 ± 2.67 |
| Sleep | 2.75 ± 2.95 | 2.73 ± 2.75 |
| Enjoyment of Life | 2.42 ± 2.82 | 2.68 ± 2.62 |
Patient-reported pain was assessed by rating pain symptoms at its worst and least for the last 24 hours, on average, and at the moment, with 0 as the lowest score (no pain) and 10 as the highest score (worst pain as you can imagine). Patient-reported pain was also assessed by rating pain symptoms (rate pain on average, rate pain right now) that interfered with general activity, interfered with mood, walking ability, normal work, relations with other people, sleep, and enjoyment of life, with 0 as the lowest score (did not interfered) and 10 as the highest score (completely interfered). The scores from each assessment time point were averaged for all of the below reported categories.
| score on a scale | Open-Label Extension Period: Placebo/Volanesorsen | Open-Label Extension Period: Volanesorsen/Volanesorsen |
|---|---|---|
| Rate Pain at its Worst Last 24 Hours | 1.70 ± 1.18 | 3.82 ± 2.97 |
| Rate Pain at its Least Last 24 Hours | 0.95 ± 1.04 | 2.78 ± 2.60 |
| Rate Pain on Average | 1.35 ± 1.22 | 3.16 ± 2.72 |
| Rate Pain Right Now | 1.28 ± 1.18 | 3.49 ± 2.99 |
| General Activity | 1.03 ± 1.45 | 3.28 ± 2.90 |
| Interfere With Mood | 1.27 ± 1.46 | 3.23 ± 2.94 |
| Walking Ability | 1.05 ± 1.50 | 3.51 ± 2.83 |
| Normal Work | 1.08 ± 1.40 | 3.48 ± 2.98 |
| Relations With Other People | 1.08 ± 1.48 | 3.18 ± 3.09 |
| Sleep | 1.51 ± 1.83 | 3.13 ± 3.35 |
| Enjoyment of Life | 1.23 ± 1.65 | 3.33 ± 2.94 |
Patient-reported hunger was assessed by participants who completed a questionnaire about: how hungry you feel, how satisfied you feel, how full you feel, how much you think you can eat, like to eat something sweet, like to eat something salty, like to eat something savory and like to eat something fatty. Participants also rated the palatability of meals that included visual appeal, smell, taste, and aftertaste. Scores of 1-39 were categorized as mild, 40-69 as moderate, and 70-100 as severe. The scores from each assessment time point were averaged for all of the below reported categories.
| score on a scale | Randomized Treatment Period: Placebo | Randomized Treatment Period: Volanesorsen |
|---|---|---|
| How Hungry You Feel | 29.4 ± 20.7 | 29.6 ± 14.4 |
| How Satisfied You Feel | 56.8 ± 22.4 | 57.5 ± 15.1 |
| How Full You Feel | 62.6 ± 20.5 | 56.6 ± 18.4 |
| How Much You Think You Can Eat | 33.6 ± 22.1 | 36.8 ± 15.4 |
| Like to Eat Something Sweet | 71.0 ± 20.8 | 54.8 ± 27.1 |
| Like to Eat Something Salty | 66.9 ± 21.7 | 69.0 ± 20.2 |
| Like to Eat Something Savory | 65.2 ± 21.4 | 66.8 ± 21.9 |
| Like to Eat Something Fatty | 77.9 ± 18.4 | 75.9 ± 22.2 |
| Visual Appeal | 28.6 ± 20.5 | 34.5 ± 21.0 |
| Smell | 23.4 ± 16.2 | 25.4 ± 15.5 |
| Taste | 25.5 ± 18.3 | 29.1 ± 14.5 |
| Aftertaste | 58.9 ± 27.5 | 51.0 ± 24.6 |
| Palatability | 31.3 ± 20.1 | 32.2 ± 15.1 |
Patient-reported hunger was assessed by participants who completed a questionnaire about: how hungry you feel, how satisfied you feel, how full you feel, how much you think you can eat, like to eat something sweet, like to eat something salty, like to eat something savory and like to eat something fatty. Participants also rated the palatability of meals that included visual appeal, smell, taste, and aftertaste. Scores of 1-39 were categorized as mild, 40-69 as moderate, and 70-100 as severe. The scores from each assessment time point were averaged for all of the below reported categories.
| score on a scale | Open-Label Extension Period: Placebo/Volanesorsen | Open-Label Extension: Volanesorsen/Volanesorsen |
|---|---|---|
| How Hungry You Feel | 29.9 ± 23.3 | 33.2 ± 23.2 |
| How Satisfied You Feel | 54.2 ± 23.3 | 56.3 ± 20.2 |
| How Full You Feel | 59.7 ± 22.2 | 52.9 ± 24.4 |
| How Much You Think You Can Eat | 34.0 ± 21.8 | 34.9 ± 21.5 |
| Like to Eat Something Sweet | 73.7 ± 25.4 | 57.9 ± 24.0 |
| Like to Eat Something Salty | 65.7 ± 28.1 | 61.9 ± 22.1 |
| Like to Eat Something Savory | 65.4 ± 24.7 | 62.7 ± 30.5 |
| Like to Eat Something Fatty | 80.7 ± 18.1 | 68.0 ± 30.4 |
| Visual Appeal | 14.9 ± 16.1 | 35.3 ± 25.9 |
| Smell | 13.1 ± 14.3 | 29.6 ± 21.6 |
| Taste | 13.6 ± 15.1 | 32.9 ± 25.0 |
| Aftertaste | 48.1 ± 38.6 | 56.9 ± 29.3 |
| Palatability | 24.4 ± 23.4 | 37.3 ± 20.6 |
The SF-36 Health Survey is a 36-item, patient-reported survey of patient health. SF-36 consists of 8 health dimensions,which are weighted sums of the questions in each section. SF-36 included 36 questions related to 8 health dimensions:physical functioning, physical role functioning, bodily pain, general health perceptions, vitality, social role functioning,emotional role functioning, and mental health. Each dimension was scored on a scale of 0 to 100 where, higher score = better quality of life. A positive change from Baseline indicates improvement.
| score on a scale | Randomized Treatment Period: Placebo | Randomized Treatment Period: Volanesorsen |
|---|---|---|
| Vitality: Change at Week 13 | -1.14 ± 7.33 | -0.21 ± 5.64 |
| Vitality: Change at Week 26 | -0.25 ± 9.00 | -0.79 ± 6.89 |
| Vitality: Change at Week 52 | -0.85 ± 6.11 | -3.30 ± 8.20 |
| Physical Functioning: Change at Week 13 | -0.29 ± 4.81 | -0.41 ± 3.91 |
| Physical Functioning: Change at Week 26 | -0.64 ± 6.49 | 0.51 ± 5.29 |
| Physical Functioning: Change at Week 52 | -3.55 ± 4.05 | -2.76 ± 5.25 |
| Bodily Pain: Change at Week 13 | -0.46 ± 7.97 | 0.75 ± 6.26 |
| Bodily Pain: Change at Week 26 | -1.98 ± 12.24 | 0.19 ± 4.27 |
| Bodily Pain: Change at Week 52 | -0.29 ± 6.35 | -2.55 ± 8.48 |
| General Health Perceptions: Change at Week 13 | -0.55 ± 5.57 | -0.58 ± 4.04 |
| General Health Perceptions: Change at Week 26 | -1.59 ± 5.00 | 0.54 ± 7.25 |
| General Health Perceptions: Change at Week 52 | -1.50 ± 6.74 | -1.48 ± 4.53 |
| Physical Role Functioning: Change at Week 13 | -0.52 ± 5.44 | -0.64 ± 4.61 |
| Physical Role Functioning: Change at Week 26 | -2.06 ± 10.81 | 0.75 ± 4.70 |
| Physical Role Functioning: Change at Week 52 | -0.32 ± 2.02 | -2.00 ± 7.05 |
| Emotional Role Functioning: Change at Week 13 | -1.88 ± 4.63 | 0.75 ± 6.85 |
| Emotional Role Functioning: Change at Week 26 | -2.90 ± 6.44 | 0.23 ± 7.50 |
| Emotional Role Functioning : Change at Week 52 | -1.00 ± 5.94 | -5.42 ± 5.80 |
| Social Role Functioning: Change at Week 13 | -3.86 ± 9.85 | 1.07 ± 4.02 |
| Social Role Functioning: Change at Week 26 | -5.01 ± 9.07 | -0.67 ± 3.21 |
| Social Role Functioning: Change at Week 52 | 2.87 ± 4.89 | -2.79 ± 7.14 |
| Mental Health: Change at Week 13 | 0.00 ± 6.67 | -0.19 ± 7.91 |
| Mental Health: Change at Week 26 | -2.18 ± 7.71 | 1.05 ± 6.76 |
| Mental Health: Change at Week 52 | -0.37 ± 6.66 | -1.75 ± 6.13 |
The SF-36 Health Survey is a 36-item, patient-reported survey of patient health. SF-36 consists of 8 health dimensions,which are weighted sums of the questions in each section. SF-36 included 36 questions related to 8 health dimensions:physical functioning, physical role functioning, bodily pain, general health perceptions, vitality, social role functioning,emotional role functioning, and mental health. Each dimension was scored on a scale of 0 to 100 where, higher score = better quality of life. A positive change from Baseline indicates improvement.
| score on a scale | Open-Label Extension Period: Placebo/Volanesorsen | Open-Label Extension Period: Volanesorsen/Volanesorsen |
|---|---|---|
| Vitality: Change at Week 65 | 4.46 ± 6.30 | -0.42 ± 13.88 |
| Vitality: Change at Week 78 | -2.98 ± 12.61 | -2.97 ± NA |
| Vitality: Change at Week 104 | 2.97 ± NA | -5.94 ± NA |
| Physical Functioning: Change at Week 65 | -4.79 ± 6.77 | -1.37 ± 5.81 |
| Physical Functioning: Change at Week 78 | -3.83 ± 5.41 | 0.00 ± NA |
| Physical Functioning: Change at Week 104 | 0 ± NA | -1.91 ± NA |
| Bodily Pain: Change at Week 65 | -2.42 ± 3.42 | 0.40 ± 7.13 |
| Bodily Pain: Change at Week 78 | -7.26 ± 19.39 | -11.29 ± NA |
| Bodily Pain: Change at Week 104 | 6.45 ± NA | -10.49 ± NA |
| General Health Perceptions: Change at Week 65 | 2.38 ± 3.36 | -2.38 ± 4.42 |
| General Health Perceptions: Change at Week 78 | -2.86 ± 7.40 | -4.75 ± NA |
| General Health Perceptions: Change at Week 104 | 0 ± NA | -4.75 ± NA |
| Physical Role Functioning: Change at Week 65 | 0 ± NA | -0 ± 6.22 |
| Physical Role Functioning: Change at Week 78 | -10.11 ± 14.29 | -2.25 ± NA |
| Physical Role Functioning: Change at Week 104 | 0 ± NA | -4.50 ± NA |
| Emotional Role Functioning: Change at Week 65 | -1.74 ± 2.46 | -3.48 ± 11.37 |
| Emotional Role Functioning: Change at Week 78 | 1.74 ± 2.46 | -10.45 ± NA |
| Emotional Role Functioning: Change at Week 104 | 3.48 ± NA | -10.45 ± NA |
| Social Role Functioning: Change at Week 65 | 5.02 ± 7.09 | -2.15 ± 7.58 |
| Social Role Functioning: Change at Week 78 | -2.15 ± 7.09 | 0 ± NA |
| Social Role Functioning: Change at Week 104 | 0 ± NA | 0 ± NA |
| Mental Health: Change at Week 65 | -5.23 ± 11.10 | -1.12 ± 13.67 |
| Mental Health: Change at Week 78 | 6.54 ± 9.25 | -15.70 ± NA |
| Mental Health: Change at Week 104 | 2.62 ± NA | 0 ± NA |
EQ-5D-5L is a standardized health-related quality of life questionnaire developed by EuroQol Group in order to provide a simple, generic measure of health for clinical and economic appraisal. EQ-5D-5L consists of two components: a health state profile and VAS. EQ-5D health state profile is comprised of 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 levels: 1=no problems, 2=slight problems, 3=moderate problems, 4=severe problems, and 5=extreme problems. The 5D-5L systems are converted into a single index utility score between 0 to 1, where higher score indicates a better health state. EQ-5D-5L- VAS is designed to rate the participant's current health state on a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A negative change from baseline indicates worsening. A positive change from baseline indicates improvement.
| score on a scale | Randomized Treatment Period: Placebo | Randomized Treatment Period: Volanesorsen |
|---|---|---|
| Index Score: Change at Week 13 | 0.05 ± 0.10 | -0.02 ± 0.06 |
| Index Score: Change at Week 26 | -0.11 ± 0.19 | -0.02 ± 0.12 |
| Index Score: Change at Week 52 | -0.08 ± 0.10 | -0.05 ± 0.07 |
| EQ VAS Score: Change at Week 13 | -2 ± 13 | -2 ± 15 |
| EQ VAS Score: Change at Week 26 | -11 ± 17 | -2 ± 14 |
| EQ VAS Score: Change at Week 52 | -13 ± 18 | -4 ± 16 |
EQ-5D-5L is a standardized health-related quality of life questionnaire developed by EuroQol Group in order to provide a simple, generic measure of health for clinical and economic appraisal. EQ-5D-5L consists of two components: a health state profile and VAS. EQ-5D health state profile is comprised of 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 levels: 1=no problems, 2=slight problems, 3=moderate problems, 4=severe problems, and 5=extreme problems. The 5D-5L systems are converted into a single index utility score between 0 to 1, where higher score indicates a better health state. EQ-5D-5L- VAS is designed to rate the participant's current health state on a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A negative change from baseline indicates worsening. A positive change from baseline indicates improvement.
| score on a scale | Open-Label Extension Period: Placebo/Volanesorsen | Open-Label Extension Period: Volanesorsen/Volanesorsen |
|---|---|---|
| Index Score: Change at Week 65 | — | -0.06 ± 0.08 |
| Index Score: Change at Week 78 | -0.02 ± 0.03 | -0.07 ± NA |
| Index Score: Change at Week 104 | 0.00 ± NA | -0.27 ± NA |
| EQ VAS Score: Change at Week 65 | — | -4 ± 16 |
| EQ VAS Score: Change at Week 78 | -15 ± 22 | -6 ± NA |
| EQ VAS Score: Change at Week 104 | 2 ± NA | 0 ± NA |
Collected over From first dose of study drug to end of follow-up period [Up to Week 169]. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Randomized Treatment Period: Placebo | 0/19 (0%) | 3/19 (15.8%) | 18/19 (94.7%) |
| Randomized Treatment Period: Volanesorsen | 0/21 (0%) | 6/21 (28.6%) | 21/21 (100%) |
| Randomized Post-Treatment Follow-up: Placebo | 0/7 (0%) | 1/7 (14.3%) | 4/7 (57.1%) |
| Randomized Post-Treatment Follow-up: Volanesorsen | 0/9 (0%) | 1/9 (11.1%) | 5/9 (55.6%) |
| Open-Label Extension Period (OLE and OLE Post-Treatment Follow-up): Placebo/Volanesorsen | 0/12 (0%) | 4/12 (33.3%) | 11/12 (91.7%) |
| Open-Label Extension Period (OLE and OLE Post-Treatment Follow-up): Volanesorsen/Volanesorsen | 0/12 (0%) | 4/12 (33.3%) | 8/12 (66.7%) |
| Event | Randomized Treatment Period: Placebo | Randomized Treatment Period: Volanesorsen | Randomized Post-Treatment Follow-up: Placebo | Randomized Post-Treatment Follow-up: Volanesorsen | Open-Label Extension Period (OLE and OLE Post-Treatment Follow-up): Placebo/Volanesorsen | Open-Label Extension Period (OLE and OLE Post-Treatment Follow-up): Volanesorsen/Volanesorsen |
|---|---|---|---|---|---|---|
| ConstipationGastrointestinal disorders | 0/19 | 0/21 | 1/7 | 0/9 | 0/12 | 0/12 |
| Medical device site inflammationGeneral disorders | 0/19 | 0/21 | 0/7 | 1/9 | 0/12 | 0/12 |
| Abdominal painGastrointestinal disorders | 0/19 | 1/21 | 0/7 | 0/9 | 1/12 | 0/12 |
| PancreatitisGastrointestinal disorders | 0/19 | 1/21 | 0/7 | 0/9 | 0/12 | 1/12 |
| Pancreatitis acuteGastrointestinal disorders | 1/19 | 0/21 | 0/7 | 0/9 | 1/12 | 0/12 |
| Anaphylactic reactionImmune system disorders | 0/19 | 0/21 | 0/7 | 0/9 | 1/12 | 0/12 |
| DehydrationMetabolism and nutrition disorders | 0/19 | 0/21 | 0/7 | 0/9 | 0/12 | 1/12 |
| Atrioventricular block completeCardiac disorders | 0/19 | 0/21 | 0/7 | 0/9 | 1/12 | 0/12 |
| Systemic inflammatory response syndromeGeneral disorders | 0/19 | 0/21 | 0/7 | 0/9 | 1/12 | 0/12 |
| Blood creatinine increasedInvestigations | 0/19 | 0/21 | 0/7 | 0/9 | 0/12 | 1/12 |
| Event | Randomized Treatment Period: Placebo | Randomized Treatment Period: Volanesorsen | Randomized Post-Treatment Follow-up: Placebo | Randomized Post-Treatment Follow-up: Volanesorsen | Open-Label Extension Period (OLE and OLE Post-Treatment Follow-up): Placebo/Volanesorsen | Open-Label Extension Period (OLE and OLE Post-Treatment Follow-up): Volanesorsen/Volanesorsen |
|---|---|---|---|---|---|---|
| Injection site erythemaGeneral disorders | 0/19 | 13/21 | 0/7 | 0/9 | 7/12 | 1/12 |
| Injection site pruritusGeneral disorders | 0/19 | 11/21 | 0/7 | 0/9 | 3/12 | 0/12 |
| Injection site swellingGeneral disorders | 0/19 | 8/21 | 0/7 | 0/9 | 6/12 | 2/12 |
| Injection site painGeneral disorders | 3/19 | 7/21 | 0/7 | 0/9 | 4/12 | 1/12 |
| NauseaGastrointestinal disorders | 2/19 | 4/21 | 0/7 | 0/9 | 3/12 | 4/12 |
| SinusitisInfections and infestations | 0/19 | 0/21 | 2/7 | 0/9 | 1/12 | 0/12 |
| NasopharyngitisInfections and infestations | 1/19 | 6/21 | 0/7 | 0/9 | 1/12 | 1/12 |
| HypoglycaemiaMetabolism and nutrition disorders | 5/19 | 3/21 | 0/7 | 0/9 | 2/12 | 2/12 |
| Pain in extremityMusculoskeletal and connective tissue disorders | 5/19 | 2/21 | 0/7 | 0/9 | 0/12 | 1/12 |
| Injection site bruisingGeneral disorders | 0/19 | 3/21 | 0/7 | 0/9 | 3/12 | 0/12 |
Full Analysis Set (FAS) included all participants who were randomized and received at least one dose of study drug in the randomized treatment period, and who had a baseline fasting triglyceride (TG) assessment.
| Age, Continuous(years) | Randomized Treatment Period: Placebo | Randomized Treatment Period: Volanesorsen | Total |
|---|---|---|---|
| Mean | 48 ± 12 | 46 ± 10 | 47 ± 11 |
| Sex: Female, Male(Participants) | Randomized Treatment Period: Placebo | Randomized Treatment Period: Volanesorsen | Total |
|---|---|---|---|
| Female | 14 | 15 | 29 |
| Male | 5 | 6 | 11 |
| Ethnicity (NIH/OMB)(Participants) | Randomized Treatment Period: Placebo | Randomized Treatment Period: Volanesorsen | Total |
|---|---|---|---|
| Hispanic or Latino | 1 | 1 | 2 |
| Not Hispanic or Latino | 18 | 20 | 38 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race/Ethnicity, Customized(Participants) | Randomized Treatment Period: Placebo | Randomized Treatment Period: Volanesorsen | Total |
|---|---|---|---|
| Race — White | 17 | 20 | 37 |
| Race — Asian | 1 | 1 | 2 |
| Race — Other Race | 1 | 0 | 1 |
| Fasting Triglycerides(milligrams per deciliter (mg/dL)) | Randomized Treatment Period: Placebo | Randomized Treatment Period: Volanesorsen | Total |
|---|---|---|---|
| Mean | 1290.95 ± 1296.19 | 1241.31 ± 1090.83 | 1264.89 ± 1177.40 |
| Hepatic Fat Fraction(percentage (Hepatic Fat Fraction)) | Randomized Treatment Period: Placebo | Randomized Treatment Period: Volanesorsen | Total |
|---|---|---|---|
| Mean | 17.00 ± 7.52 | 18.10 ± 8.41 | 17.57 ± 7.88 |
| Hemoglobin A1c(percentage of HbA1c) | Randomized Treatment Period: Placebo | Randomized Treatment Period: Volanesorsen | Total |
|---|---|---|---|
| Mean | 8.25 ± 1.13 | 7.84 ± 1.62 | 8.04 ± 1.40 |
| Short Form-36 (SF-36) Weighted Sum of Scores(score on a scale) | Randomized Treatment Period: Placebo | Randomized Treatment Period: Volanesorsen | Total |
|---|---|---|---|
| Mean | 48.51 ± 12.83 | 46.31 ± 12.83 | 47.38 ± 12.45 |
2 further baseline measures are reported on the registry.
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Dec 2015 to primary completion in Jun 2018, 30 months.
Results were first posted in Oct 2021, 3 years 3 months after primary completion. A results delay was requested in Jan 2020.
How these figures are worked out, and which studies are left out of them.
This study is terminated, as verified in Sep 2021. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: ISIS 304801
Lipodystrophy, Familial Partial →