A Phase 3 interventional study of Idebenone and Idebenone in Friedreich's Ataxia, sponsored by Santhera Pharmaceuticals. Completed at 2 sites in United States. Open to participants aged 8 Years to 17 Years. Per ClinicalTrials.gov, last updated 2023-07-11.
Sponsored by Santhera Pharmaceuticals · Phase 3, Interventional, and Treatment
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Among 71 studies in Friedreich Ataxia with a site in United States — 18 open to participants now, 25 under way, 4 of them phase 3. Friedreich Ataxia · United States.
Among the 71 studies in Friedreich Ataxia with a site in United States, this one is the 11th largest by enrollment of the 49 interventional ones that give a count, is the only one that started in 2007, is one of 10 in phase 3, and was the 5th registered.
111 studies on the registry are indexed under Friedreich Ataxia; 28 are open to participants now.
Browse Friedreich Ataxia studies →18 of the 71 studies in Friedreich Ataxia with a site in United States are open now. Recruiting first, the newest first:
Biogen · Sites in United States
Ages 16+ · Women only
Larimar Therapeutics, Inc. · Sites in United States
Ages 12–40
Scott Barbuto · Sites in United States
Lexeo Therapeutics · Sites in United States
Ages 6+
Solid Biosciences Inc. · Sites in United States
Ages 18–40
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 18 Friedreich Ataxia studies recruiting now, 9 (50%) set no upper age limit and 9 (50%) stop at 75 or younger; 15 take children under 18; 3 take healthy volunteers.
This study: 8 Years to 17 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
71 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 18 · Phase 2 19 · Phase 3 12
First in 2001; this study’s year is marked. 1 started in 2007.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 70 is above the median of 30 across 76 interventional studies indexed under Friedreich Ataxia.
Median enrollment 32 across the 49 interventional studies, 65 across the 21 observational. Of the 43 that completed or were terminated, 16 (37%) have results posted.
This tab as a report to print or save as PDF → With an account.
39 of 71 have an industry lead sponsor. The 10 most frequent:
Santhera Pharmaceuticals is the lead sponsor of 27 studies on the registry; 1 is open to participants now.
Santhera Pharmaceuticals has 4 studies filed under Friedreich Ataxia — fewer than ten, so there is no comparison to draw.
6 of the studies in Friedreich Ataxia with a site in United States that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT06953583Phase 3BiogenA Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia | 2027-11-16 |
| NCT06447025Larimar Therapeutics, Inc.An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia | 2027-01 |
| NCT07869654Adjuvia Therapeutics, Inc.A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy Adults | 2027-05-27 |
| NCT07870590Graviton Bioscience CorporationGV101 for Friedreich's Ataxia (FA) Treatment | 2027-07 |
| NCT02316314Weill Medical College of Cornell UniversityCharacterization of the Cardiac Phenotype of Friedreich's Ataxia (FRDA) | 2027-08 |
Add all 6 to your calendar (.ics) → With an account.
Among the 71 studies in Friedreich Ataxia with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
This study is meant to assess the effectiveness of idebenone on neurological outcome measures in patients with Friedreich's Ataxia over a 6 months period.
The study involves 6 clinic visits and upon completion the possibility to join a 12 months extension study where all patients will receive high dose Idebenone.
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.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
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 top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
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.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
mid dose Idebenone
Drug: Idebenone
high dose Idebenone
Drug: Idebenone
Placebo
Drug: Placebo
Patients ≤ 45 kg/99 lbs: idebenone 450 mg/day; Patients \> 45 kg/99 lbs: idebenone 900 mg/day
Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day; Patients \> 45 kg/99 lbs: idebenone 2250 mg/day
Placebo was provided as film-coated tablets that were the same size, weight and appearance as the idebenone tablets.
Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 24
International Cooperative Ataxia Rating Scale (ICARS): ICARS consists of a one-hundred-point semi-quantitative scale based upon 19 simple testing manoeuvres compartmentalized into postural and stance disorders, limb ataxia, dysarthria and oculomotor disorders and has been previously used in this patient population with good inter-rater reliability. Minimum score: 0, maximum score 100, higher score indicates greater impairment Calculation details: ICARS score at week 24 minus ICARS score baseline
Time frame: baseline and 6 months
Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline to Week 24
To compare the efficacy of 6 months' treatment with 2 different doses of idebenone with that of placebo on neurological function as assessed by the Friedreich's Ataxia Rating Scale (FARS) FARS consists of a 25 manoeuvre exam along with 4 quantitative performance measures. The neurological exam covers bulbar function, upper limb coordination, lower limb coordination, peripheral nervous system function, deep tendon reflexes, stability and gait. The use of FARS has been recently validated as a sensitive scale for this population. The FARS exam scores can be added to make a total score ranging from 0 to 159. A higher score indicates a greater level of disability. Calculation details FARS score at week 24 minus FARS score baseline
Time frame: baseline and 6 months
Activities of Daily Living (ADL) of Friedreich's Ataxia Rating Scale (FARS), Change in ADL (Total Score ) From Baseline to Week 24
To compare the efficacy of 6 months' treatment with 2 different doses of idebenone with that of placebo on Activities of Daily Living (ADL) assessed by the ADL scale of the Friedreich's Ataxia Rating Scale (FARS) score 0 to 36, higher score indicates greater impairment
Time frame: baseline and 6 months
Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 12
To compare the efficacy of 3 months' treatment with 2 different doses of idebenone with that of placebo on neurological impairment as assessed by the International Cooperative Ataxia Rating Scale (ICARS) International Cooperative Ataxia Rating Scale (ICARS): ICARS consists of a one-hundred-point semi-quantitative scale based upon 19 simple testing manoeuvres compartmentalized into postural and stance disorders, limb ataxia, dysarthria and oculomotor disorders and has been previously used in this patient population with good inter-rater reliability. Minimum score: 0, maximum score 100, higher score indicates greater impairment Calculation details: ICARS score at week 12 minus ICARS score baseline
Time frame: baseline and 12 weeks
Absolute Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle
To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
Time frame: baseline and 6 months
Percent Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle
To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
Time frame: baseline and 6 months
Absolute Change From Baseline to Week 24 in Left Ventricular Mass Index (LVMI)
To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
Time frame: baseline and 6 months
Percent Change From Baseline to Week 24 in Left Ventricular Mass Index
To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
Time frame: baseline and 6 months
Absolute Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle
To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
Time frame: baseline and 6 months
Percent Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle
To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
Time frame: baseline and 6 months
Absolute Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS)
To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
Time frame: baseline and 6 months
Percent Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS)
To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
Time frame: baseline and 24 weeks
| Milestone | Mid Dose Idebenone | High Dose Idebenone | Placebo |
|---|---|---|---|
| Started | 22 | 24 | 24 |
| Completed | 22 | 22 | 24 |
| Not completed | 0 | 2 | 0 |
International Cooperative Ataxia Rating Scale (ICARS): ICARS consists of a one-hundred-point semi-quantitative scale based upon 19 simple testing manoeuvres compartmentalized into postural and stance disorders, limb ataxia, dysarthria and oculomotor disorders and has been previously used in this patient population with good inter-rater reliability. Minimum score: 0, maximum score 100, higher score indicates greater impairment Calculation details: ICARS score at week 24 minus ICARS score baseline
| ICARS points | Mid Dose Idebenone | High Dose Idebenone | Placebo |
|---|---|---|---|
| Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 24 | -2.5 ± 6.3 | -2.4 ± 4.8 | -1.3 ± 4.4 |
To compare the efficacy of 6 months' treatment with 2 different doses of idebenone with that of placebo on neurological function as assessed by the Friedreich's Ataxia Rating Scale (FARS) FARS consists of a 25 manoeuvre exam along with 4 quantitative performance measures. The neurological exam covers bulbar function, upper limb coordination, lower limb coordination, peripheral nervous system function, deep tendon reflexes, stability and gait. The use of FARS has been recently validated as a sensitive scale for this population. The FARS exam scores can be added to make a total score ranging from 0 to 159. A higher score indicates a greater level of disability. Calculation details FARS score at week 24 minus FARS score baseline
| FARS score | Mid Dose Idebenone | High Dose Idebenone | Placebo |
|---|---|---|---|
| Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline to Week 24 | -1.6 ± 6.52 | -1.2 ± 4.53 | 0.6 ± 5.91 |
To compare the efficacy of 6 months' treatment with 2 different doses of idebenone with that of placebo on Activities of Daily Living (ADL) assessed by the ADL scale of the Friedreich's Ataxia Rating Scale (FARS) score 0 to 36, higher score indicates greater impairment
| ADL points | Mid Dose Idebenone | High Dose Idebenone | Placebo |
|---|---|---|---|
| Activities of Daily Living (ADL) of Friedreich's Ataxia Rating Scale (FARS), Change in ADL (Total Score ) From Baseline to Week 24 | 0.2 ± 2.45 | 0.6 ± 3.13 | 1.0 ± 2.97 |
To compare the efficacy of 3 months' treatment with 2 different doses of idebenone with that of placebo on neurological impairment as assessed by the International Cooperative Ataxia Rating Scale (ICARS) International Cooperative Ataxia Rating Scale (ICARS): ICARS consists of a one-hundred-point semi-quantitative scale based upon 19 simple testing manoeuvres compartmentalized into postural and stance disorders, limb ataxia, dysarthria and oculomotor disorders and has been previously used in this patient population with good inter-rater reliability. Minimum score: 0, maximum score 100, higher score indicates greater impairment Calculation details: ICARS score at week 12 minus ICARS score baseline
| ICARS points | Mid Dose Idebenone | High Dose Idebenone | Placebo |
|---|---|---|---|
| Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 12 | -1.5 ± 5.35 | -1.6 ± 5.53 | -3.0 ± 3.52 |
To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
| cm | Mid Dose Idebenone | High Dose Idebenone | Placebo |
|---|---|---|---|
| Absolute Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle | -0.032 ± 0.096 | 0.007 ± 0.071 | 0.010 ± 0.097 |
To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
| % change | Mid Dose Idebenone | High Dose Idebenone | Placebo |
|---|---|---|---|
| Percent Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle | -3.32 ± 15.300 | 1.53 ± 12.560 | 2.65 ± 17.135 |
To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
| g/m2 | Mid Dose Idebenone | High Dose Idebenone | Placebo |
|---|---|---|---|
| Absolute Change From Baseline to Week 24 in Left Ventricular Mass Index (LVMI) | -3.06 ± 6.23 | -2.56 ± 4.27 | -1.26 ± 6.47 |
To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
| % change | Mid Dose Idebenone | High Dose Idebenone | Placebo |
|---|---|---|---|
| Percent Change From Baseline to Week 24 in Left Ventricular Mass Index | -4.5 ± 11.302 | -4.9 ± 8.713 | -1.7 ± 12.382 |
To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
| cm | Mid Dose Idebenone | High Dose Idebenone | Placebo |
|---|---|---|---|
| Absolute Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle | -0.018 ± 0.137 | -0.004 ± 0.112 | -0.038 ± 0.153 |
To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
| % change | Mid Dose Idebenone | High Dose Idebenone | Placebo |
|---|---|---|---|
| Percent Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle | -0.075 ± 13.379 | 0.12 ± 12.549 | -3.04 ± 14.651 |
To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
| cm | Mid Dose Idebenone | High Dose Idebenone | Placebo |
|---|---|---|---|
| Absolute Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS) | -0.014 ± 0.1983 | 0.021 ± 0.1474 | 0.063 ± 0.1952 |
To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography
| % change | Mid Dose Idebenone | High Dose Idebenone | Placebo |
|---|---|---|---|
| Percent Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS) | 1.3 ± 18.160 | 2.6 ± 13.084 | 7.8 ± 19.480 |
Non-serious events are listed at a 1% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Mid Dose Idebenone | — | 0/22 (0%) | 18/22 (81.8%) |
| High Dose Idebenone | — | 2/24 (8.3%) | 23/24 (95.8%) |
| Placebo | — | 0/24 (0%) | 21/24 (87.5%) |
| Event | Mid Dose Idebenone | High Dose Idebenone | Placebo |
|---|---|---|---|
| idiopathic thrombocytopenic purpuraBlood and lymphatic system disorders | 0/22 | 1/24 | 0/24 |
| chest painGeneral disorders | 0/22 | 1/24 | 0/24 |
| Event | Mid Dose Idebenone | High Dose Idebenone | Placebo |
|---|---|---|---|
| InfectionsInfections and infestations | 11/22 | 15/24 | 16/24 |
| Gastrointestinal disordersGastrointestinal disorders | 8/22 | 15/24 | 12/24 |
| Nervous system disordersNervous system disorders | 10/22 | 14/24 | 12/24 |
| General disordersGeneral disorders | 10/22 | 9/24 | 9/24 |
| Musculoskeletal & connective tissue disordersMusculoskeletal and connective tissue disorders | 7/22 | 10/24 | 7/24 |
| Age, Categorical(Participants) | Mid Dose Idebenone | High Dose Idebenone | Placebo | Total |
|---|---|---|---|---|
| <=18 years | 22 | 24 | 24 | 70 |
| Between 18 and 65 years | 0 | 0 | 0 | 0 |
| >=65 years | 0 | 0 | 0 | 0 |
| Age, Continuous(years) | Mid Dose Idebenone | High Dose Idebenone | Placebo | Total |
|---|---|---|---|---|
| Mean | 13.93 ± 2.54 | 13.44 ± 3.03 | 13.69 ± 2.77 | 13.68 ± 2.76 |
| Sex: Female, Male(Participants) | Mid Dose Idebenone | High Dose Idebenone | Placebo | Total |
|---|---|---|---|---|
| Female | 7 | 14 | 16 | 37 |
| Male | 15 | 10 | 8 | 33 |
| Region of Enrollment(participants) | Mid Dose Idebenone | High Dose Idebenone | Placebo | Total |
|---|---|---|---|---|
| United States | 22 | 24 | 24 | 70 |
The record gives a start of Dec 2007, a primary completion of Apr 2009 and a completion of Apr 2009. It does not say whether these dates are actual or estimated.
Results were first posted in Dec 2011, 2 years 8 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Jun 2023. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Idebenone