Multiple clinical trials spanning Phase 1 through Phase 4 have evaluated idebenone in neurodegenerative and neuromuscular conditions, including Friedreich's ataxia, Duchenne muscular dystrophy, Leber's hereditary optic neuropathy, Parkinson's disease, and migraine. Sponsored by pharmaceutical companies like Santhera Pharmaceuticals alongside governmental and academic institutions such as the National Institute of Neurological Disorders and Stroke (NINDS) and university medical centers, these trials encompass completed, active, withdrawn, and terminated protocols.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT04534023 | ACTIVE_NOT_RECRUITING | Rapid Eye Movement Sleep Behavior Disorder; Synucleinopathy |
Ruijin Hospital |
2020-08-31 | PHASE2 |
| NCT05987397 | UNKNOWN | Post Stroke Epilepsy |
Xiangya Hospital of Central South University |
2023-07-05 | PHASE4 |
| NCT04151472 | RECRUITING | Migraine Disorders; Headache Disorders |
Second Affiliated Hospital, Zhejiang University, School of Medicine |
2021-12-08 | PHASE3 |
| NCT04071639 | UNKNOWN | Huntington Disease |
Second Affiliated Hospital, Zhejiang University, School of Medicine |
2020-03-12 | PHASE1 |
| NCT05411978 | UNKNOWN | Migraine |
Beijing Tiantan Hospital |
2022-05-09 | PHASE4 |
| NCT04669158 | COMPLETED | Non Alcoholic Steatohepatitis; Fibrosis |
Stanford University |
2021-07-30 | PHASE1; PHASE2 |
| NCT04152655 | WITHDRAWN | REM Sleep Behavior Disorder; Parkinson Disease |
Second Affiliated Hospital, Zhejiang University, School of Medicine |
2020-01-01 | PHASE2; PHASE3 |
| NCT02774005 | COMPLETED | Leber's Hereditary Optic Neuropathy (LHON) |
Santhera Pharmaceuticals |
2016-05 | PHASE4 |
| NCT03727295 | UNKNOWN | Oxidative Stress is an Important Cause of Parkinson's Disease |
Second Affiliated Hospital of Soochow University |
2019-01-01 | PHASE4 |
| NCT02814019 | TERMINATED | Duchenne Muscular Dystrophy (DMD) |
Santhera Pharmaceuticals |
2016-09 | PHASE3 |
| NCT03603288 | TERMINATED | Duchenne Muscular Dystrophy |
Santhera Pharmaceuticals |
2018-07-04 | PHASE3 |
| NCT01854359 | COMPLETED | Multiple Sclerosis; Primary Progressive Multiple Sclerosis |
National Institute of Allergy and Infectious Diseases (NIAID) |
2013-03-12 | PHASE1; PHASE2 |
| NCT00950248 | COMPLETED | Primary Progressive Multiple Sclerosis |
National Institute of Allergy and Infectious Diseases (NIAID) |
2009-11-01 | PHASE1; PHASE2 |
| NCT02887443 | COMPLETED | Drug-Drug Interaction |
Santhera Pharmaceuticals |
2016-09 | PHASE1 |
| NCT01027884 | COMPLETED | Muscular Dystrophy, Duchenne; Ambulatory Care |
Santhera Pharmaceuticals |
2009-07 | PHASE3 |
| NCT01303406 | COMPLETED | Friedreich's Ataxia |
Santhera Pharmaceuticals |
2011-04 | PHASE3 |
| NCT00887562 | COMPLETED | MELAS Syndrome |
Michio Hirano |
2009-05 | PHASE2 |
| NCT00993967 | COMPLETED | Freidreich's Ataxia |
Santhera Pharmaceuticals |
2007-06 | PHASE3 |
| NCT01303406 | Completed | Friedreich''s Ataxia |
Santhera Pharmaceuticals |
2011-04 | Phase 3 |
| NCT00758225 | COMPLETED | Duchenne Muscular Dystrophy |
Santhera Pharmaceuticals |
2008-09 | PHASE2 |
| NCT00697073 | COMPLETED | Friedreich's Ataxia |
Santhera Pharmaceuticals |
2008-07 | PHASE3 |
| NCT00747487 | COMPLETED | Leber's Hereditary Optic Neuropathy |
Santhera Pharmaceuticals |
2007-11 | PHASE2 |
| NCT00905268 | COMPLETED | Friedreich's Ataxia |
Santhera Pharmaceuticals |
2006-04 | PHASE3 |
| NCT00537680 | COMPLETED | Friedreich's Ataxia |
Santhera Pharmaceuticals |
2007-12 | PHASE3 |
| NCT00758225 | Completed | Duchenne Muscular Dystrophy |
Santhera Pharmaceuticals |
2008-09 | Phase 2 |
| NCT00229632 | COMPLETED | Friedreich Ataxia |
National Institute of Neurological Disorders and Stroke (NINDS) |
2005-09-27 | PHASE2 |
| NCT00747487 | Completed | Leber''s Hereditary Optic Neuropathy |
Santhera Pharmaceuticals |
2007-11 | Phase 2 |
| NCT00654784 | COMPLETED | Duchenne Muscular Dystrophy (DMD) |
Santhera Pharmaceuticals |
2005-10 | PHASE2 |
| NCT00229632 | Completed | Friedreich Ataxia |
National Institute of Neurological Disorders and Stroke (NINDS)|National Institutes of Health Clinical Center (CC) |
2005-09-27 | Phase 2 |
| NCT00078481 | COMPLETED | Friedreich Ataxia |
National Institute of Neurological Disorders and Stroke (NINDS) |
2004-02 | PHASE1 |
| NCT00015808 | COMPLETED | Friedreich Ataxia |
National Institute of Neurological Disorders and Stroke (NINDS) |
2001-05 | PHASE1 |
| NCT01495715 | WITHDRAWN | Leber's Hereditary Optic Neuropathy |
Santhera Pharmaceuticals |
PHASE3 |
(data from https://clinicaltrials.gov, updated on 2025-11-25)
Check the
Idebenone
product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.
Mechanism and Biochemical Profile
Appendix RUO and cGMP Quality Standards
| Quality Dimension | RUO (Research Use Only) | cGMP (Current Good Manufacturing Practice) |
|---|---|---|
| Clinical Applicability | Prohibited in human clinical trials or medical diagnostics. | Mandatory for human clinical trials (Phase I–III) and therapies. |
| Regulatory Status | Non-regulated grade; exempt from drug manufacturing laws. | Legally enforced by health authorities (e.g., FDA, EMA, NMPA). |
| Facility Environment | Unclassified analytical or research laboratories. | Validated Cleanrooms (ISO Class 5–8) with continuous monitoring. |
| Quality Control | Basic purity and activity testing. | Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma). |
| Process Validation | Basic equipment calibration; no process validation required. | Full qualification (IQ/OQ/PQ) and complete batch records. |
| Quality Assurance | Vendor self-declared without required formal QMS. | Mandatory QA/QC unit, Change Control, CAPA, and vendor audits. |
| Regulatory Impact | High risk of IND rejection if used as a critical raw material. | Required for IND/NDA filings, supported by Drug Master Files (DMF). |
Footnotes
Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).
Note: Technical data last updated: Sep 1, 2026.