A Phase 3 interventional study of Givosiran and Placebo in Acute Hepatic Porphyria, Acute Intermittent Porphyria and Porphyria, Acute Intermittent, sponsored by Alnylam Pharmaceuticals. Completed at 36 sites in 18 countries. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2024-04-22.
Sponsored by Alnylam Pharmaceuticals · Phase 3, Interventional, and Treatment
The purpose of this study is to evaluate the effect of subcutaneous givosiran (ALN-AS1), compared to placebo, on the rate of porphyria attacks in patients with Acute Hepatic Porphyrias (AHP).
21 studies on the registry are indexed under Porphyria, Acute Intermittent; 3 are open to participants now.
This study's enrollment of 94 is above the median of 20 across 11 interventional studies indexed under Porphyria, Acute Intermittent.
Browse Porphyria, Acute Intermittent studies →Alnylam Pharmaceuticals is the lead sponsor of 84 studies on the registry; 19 are open to participants now.
Of its 18 completed or terminated interventional studies of FDA-regulated products, 15 (83%) 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.
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.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Givosiran 2.5 mg/kg administered subcutaneously (SC), monthly (QM), for 6 months during the 6-Month Double-blind (DB) Period, followed by givosiran 2.5 mg/kg or 1.25 mg/kg SC, QM for 29 months during the Open-label Extension (OLE) Period.
Drug: Givosiran · Drug: Placebo
Matching placebo (normal saline \[0.9% NaCl\]) was administered SC, QM, for 6 months during the 6-Month DB Period, followed by givosiran 2.5 mg/kg or 1.25 mg/kg SC, QM for 29 months during the OLE period.
Drug: Givosiran · Drug: Placebo
Givosiran by SC
Also known as: ALN-AS1, GIVLAARI
Matching placebo (normal saline \[0.9% NaCl\]) by SC
Annualized Rate of Porphyria Attacks in Participants With Acute Intermittent Porphyria (AIP)
Porphyria attacks were defined as meeting all of the following criteria: an acute episode of neurovisceral pain in the abdomen, back, chest, extremities and/or limbs, no other medically determined cause, and required treatment with intravenous (IV) dextrose or hemin, carbohydrates, or analgesics, or other medications such as antiemetics at a dose or frequency beyond the participant's usual daily porphyria management. The annualized rate of porphyria attacks is a composite endpoint which included porphyria attacks requiring hospitalization, urgent healthcare visit, or IV hemin administration at home.
Time frame: 6 months
The Pharmacodynamic (PD) Effect of Givosiran on Urine Levels of Delta-aminolevulinic Acid (ALA) in Participants With AIP
The PD effect of givosiran was evaluated by spot urine ALA levels normalized to spot urine creatinine levels.
Time frame: 3 and 6 months
The PD Effect of Givosiran on Urine Levels of Porphobilinogen (PBG) in Participants With AIP
The PD effect of givosiran was evaluated by spot urine PBG levels normalized to spot urine creatinine levels.
Time frame: 6 months
Annualized Rate of Hemin Administration in Participants With AIP
Annualized rate of hemin doses was evaluated as annualized days of hemin use.
Time frame: 6 months
Annualized Rate of Porphyria Attacks in Participants With AHP
Porphyria attacks were defined as meeting all of the following criteria: an acute episode of neurovisceral pain in the abdomen, back, chest, extremities and/or limbs, no other medically determined cause, and required treatment with intravenous (IV) dextrose or hemin, carbohydrates, or analgesics, or other medications such as antiemetics at a dose or frequency beyond the participant's usual daily porphyria management. The annualized rate of porphyria attacks is a composite endpoint which included porphyria attacks requiring hospitalization, urgent healthcare visit, or IV hemin administration at home.
Time frame: 6 months
Area Under the Curve (AUC) of the Change From Baseline in Weekly Mean Score of Daily Worst Pain as Measured by the Brief Pain Inventory-Short Form (BPI-SF) Numeric Rating Scale (NRS) in Participants With AIP
Participants rated worst daily pain score in an eDiary using the 11-point BPI-SF NRS, in which 0=no pain and 10=worst pain. Daily eDiary entries were averaged into a weekly (i.e. 7 day) score. The change from baseline in weekly mean scores is defined as the post baseline weekly mean score minus the baseline score. Lower scores indicate an improvement. The 6-month AUC was calculated based on change from baseline in weekly mean scores.
Time frame: Baseline and 6 months
Average Change From Baseline in Weekly Mean Score of Daily Worst Pain as Measured by the Brief Pain Inventory-Short Form (BPI-SF) Numeric Rating Scale (NRS) in Participants With AIP
Participants rated worst daily pain score in an eDiary using the 11-point BPI-SF NRS, in which 0=no pain and 10=worst pain. Daily eDiary entries were averaged into a weekly (i.e. 7 day) score. The change from baseline in weekly mean scores is defined as the postbaseline weekly mean score minus the baseline score. Lower scores indicate an improvement.
Time frame: Baseline and 6 months
AUC of the Change From Baseline in Weekly Mean Score of Daily Worst Fatigue Score as Measured by the Brief Fatigue Inventory-Short Form (BFI-SF) NRS in Participants With AIP
Participants rated daily worst fatigue score in an eDiary using the 11-point BFI-SF NRS, in which 0=no fatigue and 10=worst fatigue. Daily eDiary entries were averaged into a weekly (i.e. 7 day) score. The change from baseline in weekly mean scores is defined as the post baseline weekly mean score minus the baseline score. Lower scores indicate an improvement. The 6-month AUC was calculated based on change from baseline in weekly mean scores.
Time frame: Baseline and 6 months
Average Change From Baseline in Weekly Mean Score of Daily Worst Fatigue Score as Measured by the Brief Fatigue Inventory-Short Form (BFI-SF) NRS in Participants With AIP
Participants rated daily worst fatigue score in an eDiary using the 11-point BFI-SF NRS, in which 0=no fatigue and 10=worst fatigue. Daily eDiary entries were averaged into a weekly (i.e. 7 day) score. The change from baseline in weekly mean scores is defined as the postbaseline weekly mean score minus the baseline score. Lower scores indicate an improvement.
Time frame: Baseline and 6 months
AUC of the Change From Baseline in Weekly Mean Score Daily Worst Nausea Score as Measured by NRS in Participants With AIP
Participants rated worst daily nausea score in an eDiary using an 11-point NRS, in which 0=no nausea and 10=worst nausea. Daily eDiary entries were averaged into a weekly (i.e. 7 day) score. The change from baseline in weekly mean scores is defined as the postbaseline weekly mean score minus the baseline score. Lower scores indicate an improvement. The 6-month AUC was calculated based on change from baseline in weekly mean scores.
Time frame: Baseline and 6 months
Average Change From Baseline in Weekly Mean Score Daily Worst Nausea Score as Measured by NRS in Participants With AIP
Participants rated worst daily nausea score in an eDiary using an 11-point NRS, in which 0=no nausea and 10=worst nausea. Daily eDiary entries were averaged into a weekly (i.e. 7 day) score. The change from baseline in weekly mean scores is defined as the postbaseline weekly mean score minus the baseline score. Lower scores indicate an improvement.
Time frame: Baseline and 6 months
Change From Baseline in the Physical Component Summary (PCS) of the 12-Item Short Form Survey (SF-12) in Participants With AIP
The SF-12 is a survey designed for use in patients with multiple chronic conditions. This 12-item scale can be used to assess the physical and mental health of respondents. 10 of the 12 questions are answered on a 5 point likert scale and 2 are answered on a 3 point likert scale. The questions are then scored and weighted into 2 subscales, physical health and mental health. Respondents can have a score that ranges from 0-100 with 100 being the best score and indicating high physical or mental health. A 3 point change in SF-12 score reflects a meaningful difference. A higher score indicates improvement.
Time frame: Baseline and 6 months
Participants with acute hepatic porphyrias (AHP) were enrolled at thirty-six sites in Australia, Bulgaria, Canada, Germany, Denmark, Spain, Finland, France, United Kingdom, Italy, Japan, Korea (the Republic of), Mexico, Netherlands, Poland, Sweden, Taiwan and the United States.
| Milestone | Placebo 6-Month DB | Givosiran 2.5 mg/kg 6-Month DB | Placebo/Givosiran | Givosiran/Givosiran |
|---|---|---|---|---|
| Started | 46 | 48 | 0 | 0 |
| Completed | 46 | 48 | 0 | 0 |
| Not completed | 0 | 0 | 0 | 0 |
| Milestone | Placebo 6-Month DB | Givosiran 2.5 mg/kg 6-Month DB | Placebo/Givosiran | Givosiran/Givosiran |
|---|---|---|---|---|
| Started | 0 | 0 | 46 | 47 |
| Completed | 0 | 0 | 38 | 41 |
| Not completed | 0 | 0 | 8 | 6 |
| Withdrew: Adverse event | 0 | 0 | 2 | 0 |
| Withdrew: Death | 0 | 0 | 0 | 1 |
| Withdrew: Lost to follow-up | 0 | 0 | 1 | 0 |
| Withdrew: Physician decision | 0 | 0 | 1 | 0 |
| Withdrew: Pregnancy | 0 | 0 | 1 | 1 |
| Withdrew: Withdrawal by subject | 0 | 0 | 3 | 3 |
| Withdrew: Patient desired lower dose | 0 | 0 | 0 | 1 |
Porphyria attacks were defined as meeting all of the following criteria: an acute episode of neurovisceral pain in the abdomen, back, chest, extremities and/or limbs, no other medically determined cause, and required treatment with intravenous (IV) dextrose or hemin, carbohydrates, or analgesics, or other medications such as antiemetics at a dose or frequency beyond the participant's usual daily porphyria management. The annualized rate of porphyria attacks is a composite endpoint which included porphyria attacks requiring hospitalization, urgent healthcare visit, or IV hemin administration at home.
| annualized attack rate | Placebo | Givosiran 2.5 mg/kg |
|---|---|---|
| Annualized Rate of Porphyria Attacks in Participants With Acute Intermittent Porphyria (AIP) | 12.52 (9.35 to 16.76) | 3.22 (2.25 to 4.59) |
The PD effect of givosiran was evaluated by spot urine ALA levels normalized to spot urine creatinine levels.
| mmol/mol creatinine (Cr) | Placebo | Givosiran 2.5 mg/kg |
|---|---|---|
| Month 3 | 19.965 ± 1.475 | 1.756 ± 1.413 |
| Month 6 | 23.150 ± 2.534 | 4.013 ± 2.352 |
The PD effect of givosiran was evaluated by spot urine PBG levels normalized to spot urine creatinine levels.
| mmol/mol Cr | Placebo | Givosiran 2.5 mg/kg |
|---|---|---|
| The PD Effect of Givosiran on Urine Levels of Porphobilinogen (PBG) in Participants With AIP | 49.110 ± 4.959 | 12.906 ± 4.642 |
Annualized rate of hemin doses was evaluated as annualized days of hemin use.
| annualized rate of use | Placebo | Givosiran 2.5 mg/kg |
|---|---|---|
| Annualized Rate of Hemin Administration in Participants With AIP | 29.71 (18.41 to 47.94) | 6.77 (4.20 to 10.92) |
Porphyria attacks were defined as meeting all of the following criteria: an acute episode of neurovisceral pain in the abdomen, back, chest, extremities and/or limbs, no other medically determined cause, and required treatment with intravenous (IV) dextrose or hemin, carbohydrates, or analgesics, or other medications such as antiemetics at a dose or frequency beyond the participant's usual daily porphyria management. The annualized rate of porphyria attacks is a composite endpoint which included porphyria attacks requiring hospitalization, urgent healthcare visit, or IV hemin administration at home.
| annualized attack rate | Placebo | Givosiran 2.5 mg/kg |
|---|---|---|
| Annualized Rate of Porphyria Attacks in Participants With AHP | 12.26 (9.22 to 16.29) | 3.35 (2.37 to 4.74) |
Participants rated worst daily pain score in an eDiary using the 11-point BPI-SF NRS, in which 0=no pain and 10=worst pain. Daily eDiary entries were averaged into a weekly (i.e. 7 day) score. The change from baseline in weekly mean scores is defined as the post baseline weekly mean score minus the baseline score. Lower scores indicate an improvement. The 6-month AUC was calculated based on change from baseline in weekly mean scores.
| score on a scale*week | Placebo | Givosiran 2.5 mg/kg |
|---|---|---|
| Area Under the Curve (AUC) of the Change From Baseline in Weekly Mean Score of Daily Worst Pain as Measured by the Brief Pain Inventory-Short Form (BPI-SF) Numeric Rating Scale (NRS) in Participants With AIP | 5.286 (-23.048 to 11.145) | -11.514 (-29.181 to 3.040) |
Participants rated worst daily pain score in an eDiary using the 11-point BPI-SF NRS, in which 0=no pain and 10=worst pain. Daily eDiary entries were averaged into a weekly (i.e. 7 day) score. The change from baseline in weekly mean scores is defined as the postbaseline weekly mean score minus the baseline score. Lower scores indicate an improvement.
| score on a scale | Placebo | Givosiran 2.5 mg/kg |
|---|---|---|
| Average Change From Baseline in Weekly Mean Score of Daily Worst Pain as Measured by the Brief Pain Inventory-Short Form (BPI-SF) Numeric Rating Scale (NRS) in Participants With AIP | 0.245 (-1.020 to 0.470) | -0.506 (-1.309 to 0.143) |
Participants rated daily worst fatigue score in an eDiary using the 11-point BFI-SF NRS, in which 0=no fatigue and 10=worst fatigue. Daily eDiary entries were averaged into a weekly (i.e. 7 day) score. The change from baseline in weekly mean scores is defined as the post baseline weekly mean score minus the baseline score. Lower scores indicate an improvement. The 6-month AUC was calculated based on change from baseline in weekly mean scores.
| score on a scale*week | Placebo | Givosiran 2.5 mg/kg |
|---|---|---|
| AUC of the Change From Baseline in Weekly Mean Score of Daily Worst Fatigue Score as Measured by the Brief Fatigue Inventory-Short Form (BFI-SF) NRS in Participants With AIP | -4.208 ± 4.689 | -11.148 ± 4.501 |
Participants rated daily worst fatigue score in an eDiary using the 11-point BFI-SF NRS, in which 0=no fatigue and 10=worst fatigue. Daily eDiary entries were averaged into a weekly (i.e. 7 day) score. The change from baseline in weekly mean scores is defined as the postbaseline weekly mean score minus the baseline score. Lower scores indicate an improvement.
| score on a scale | Placebo | Givosiran 2.5 mg/kg |
|---|---|---|
| Average Change From Baseline in Weekly Mean Score of Daily Worst Fatigue Score as Measured by the Brief Fatigue Inventory-Short Form (BFI-SF) NRS in Participants With AIP | -0.182 ± 0.209 | -0.502 ± 0.200 |
Participants rated worst daily nausea score in an eDiary using an 11-point NRS, in which 0=no nausea and 10=worst nausea. Daily eDiary entries were averaged into a weekly (i.e. 7 day) score. The change from baseline in weekly mean scores is defined as the postbaseline weekly mean score minus the baseline score. Lower scores indicate an improvement. The 6-month AUC was calculated based on change from baseline in weekly mean scores.
| score on a scale | Placebo | Givosiran 2.5 mg/kg |
|---|---|---|
| AUC of the Change From Baseline in Weekly Mean Score Daily Worst Nausea Score as Measured by NRS in Participants With AIP | -4.011 ± 3.453 | 1.481 ± 3.310 |
Participants rated worst daily nausea score in an eDiary using an 11-point NRS, in which 0=no nausea and 10=worst nausea. Daily eDiary entries were averaged into a weekly (i.e. 7 day) score. The change from baseline in weekly mean scores is defined as the postbaseline weekly mean score minus the baseline score. Lower scores indicate an improvement.
| score on a scale | Placebo | Givosiran 2.5 mg/kg |
|---|---|---|
| Average Change From Baseline in Weekly Mean Score Daily Worst Nausea Score as Measured by NRS in Participants With AIP | -0.181 ± 0.154 | 0.067 ± 0.147 |
The SF-12 is a survey designed for use in patients with multiple chronic conditions. This 12-item scale can be used to assess the physical and mental health of respondents. 10 of the 12 questions are answered on a 5 point likert scale and 2 are answered on a 3 point likert scale. The questions are then scored and weighted into 2 subscales, physical health and mental health. Respondents can have a score that ranges from 0-100 with 100 being the best score and indicating high physical or mental health. A 3 point change in SF-12 score reflects a meaningful difference. A higher score indicates improvement.
| score on a scale | Placebo | Givosiran 2.5 mg/kg |
|---|---|---|
| Change From Baseline in the Physical Component Summary (PCS) of the 12-Item Short Form Survey (SF-12) in Participants With AIP | 1.431 ± 1.220 | 5.369 ± 1.169 |
Collected over From the first dose of study drug through completion of the OLE Period (up to 39 months).. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Placebo 6-Month DB | 0/46 (0%) | 4/46 (8.7%) | 37/46 (80.4%) |
| Givosiran 2.5 mg/kg 6-Month DB | 0/48 (0%) | 10/48 (20.8%) | 43/48 (89.6%) |
| Placebo/Givosiran | 0/46 (0%) | 17/46 (37%) | 44/46 (95.7%) |
| Givosiran/Givosiran | 1/48 (2.1%) | 20/48 (41.7%) | 47/48 (97.9%) |
| All Givosiran | 1/94 (1.1%) | 37/94 (39.4%) | 91/94 (96.8%) |
| Event | Placebo 6-Month DB | Givosiran 2.5 mg/kg 6-Month DB | Placebo/Givosiran | Givosiran/Givosiran | All Givosiran |
|---|---|---|---|---|---|
| Pulmonary embolismRespiratory, thoracic and mediastinal disorders | 0/46 | 0/48 | 1/46 | 3/48 | 4/94 |
| Device related infectionInfections and infestations | 2/46 | 1/48 | 0/46 | 0/48 | 0/94 |
| Blood homocysteine increasedInvestigations | 0/46 | 0/48 | 2/46 | 0/48 | 2/94 |
| Chronic kidney diseaseRenal and urinary disorders | 0/46 | 2/48 | 0/46 | 0/48 | 0/94 |
| Chronic kidney diseaseRenal and urinary disorders | 0/46 | 0/48 | 0/46 | 2/48 | 2/94 |
| PyrexiaGeneral disorders | 1/46 | 1/48 | 0/46 | 0/48 | 0/94 |
| Escherichia urinary tract infectionInfections and infestations | 1/46 | 0/48 | 0/46 | 0/48 | 0/94 |
| SepsisInfections and infestations | 1/46 | 0/48 | 0/46 | 0/48 | 0/94 |
| Septic shockInfections and infestations | 1/46 | 0/48 | 0/46 | 0/48 | 0/94 |
| Fractured sacrumInjury, poisoning and procedural complications | 1/46 | 0/48 | 0/46 | 0/48 | 0/94 |
| Event | Placebo 6-Month DB | Givosiran 2.5 mg/kg 6-Month DB | Placebo/Givosiran | Givosiran/Givosiran | All Givosiran |
|---|---|---|---|---|---|
| NauseaGastrointestinal disorders | 0/46 | 0/48 | 12/46 | 22/48 | 34/94 |
| Injection site reactionGeneral disorders | 0/46 | 0/48 | 14/46 | 16/48 | 30/94 |
| NasopharyngitisInfections and infestations | 0/46 | 0/48 | 11/46 | 14/48 | 25/94 |
| FatigueGeneral disorders | 0/46 | 0/48 | 13/46 | 12/48 | 25/94 |
| NauseaGastrointestinal disorders | 5/46 | 13/48 | 0/46 | 0/48 | 0/94 |
| HeadacheNervous system disorders | 0/46 | 0/48 | 7/46 | 13/48 | 20/94 |
| Upper respiratory tract infectionInfections and infestations | 0/46 | 0/48 | 12/46 | 7/48 | 19/94 |
| Urinary tract infectionInfections and infestations | 0/46 | 0/48 | 10/46 | 9/48 | 19/94 |
| Abdominal painGastrointestinal disorders | 0/46 | 0/48 | 8/46 | 6/48 | 14/94 |
| VomitingGastrointestinal disorders | 0/46 | 0/48 | 8/46 | 7/48 | 15/94 |
Safety Analysis Set (SAS) consisted of all participants who received at least one dose of study drug.
| Age, Continuous(years) | Placebo | Givosiran | Total |
|---|---|---|---|
| Mean | 37.4 ± 10.5 | 40.1 ± 12.1 | 38.8 ± 11.4 |
| Sex: Female, Male(Participants) | Placebo | Givosiran | Total |
|---|---|---|---|
| Female | 41 | 43 | 84 |
| Male | 5 | 5 | 10 |
| Ethnicity (NIH/OMB)(Participants) | Placebo | Givosiran | Total |
|---|---|---|---|
| Hispanic or Latino | 3 | 5 | 8 |
| Not Hispanic or Latino | 42 | 42 | 84 |
| Unknown or Not Reported | 1 | 1 | 2 |
| Race/Ethnicity, Customized(Participants) | Placebo | Givosiran | Total |
|---|---|---|---|
| Asian | 7 | 8 | 15 |
| Native Hawaiian or Other Pacific Islander | 1 | 0 | 1 |
| Black or African American | 1 | 0 | 1 |
| White | 34 | 39 | 73 |
| More than One Race | 1 | 0 | 1 |
| Unknown or Not Reported | 2 | 1 | 3 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — Access to Anonymized individual participant data that support these results is made available 12 months after study completion and not less than 12 months after the product and indication have been approved in the US and/or the EU. Data will be provided contingent upon the approval of a research proposal and the execution of a data sharing agreement. Requests for access to data can be submitted via the website www.vivli.org.
Ran from Nov 2017 to primary completion in Jan 2019, 14 months.
Results were first posted in Feb 2020, 1 year after primary completion.
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This study is completed, as verified in May 2022. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Givosiran
Porphyria, Acute Intermittent →