Overview
Sponsor-declared trial summary
Phase
Therapeutic use (Phase IV)
Status
Authorised, recruitment pending
Participants planned
65
Countries
2
Sites
3
Friedreich's ataxia
To evaluate the effect of omaveloxolone on mitochondrial function in FA patients and compare it to untreated controls to identify a reliable biomarker of treatment effect.
Key facts
- Sponsor
- Fakultni Nemocnice V Motole
- Participant type
- Healthy volunteers, Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10]
- Decision date (initial)
- 2026-03-30
- Transition trial
- No
- Low-intervention
- Yes
- Rare-disease indication
- Yes
- Vulnerable population
- No
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Pharmacodynamic
To evaluate the effect of omaveloxolone on mitochondrial function in FA patients and compare it to untreated controls to identify a reliable biomarker of treatment effect.
Secondary objectives 2
- To compare mitochondrial biomarker profiles between treated FA patients and healthy controls.
- To investigate associations between changes in mitochondrial biomarkers and changes in clinical disease severity (e.g. mFARS, SARA, ADL)
Conditions and MedDRA coding
Friedreich's ataxia
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 8
- For the patients with FA group: Patients with genetically confirmed FA, eligible for treatment with omaveloxolone in accordance with the current approved Summary of Product Characteristics (SmPC), both ambulatory and non-ambulatory.
- For the healthy control group: Age- and gender-matched healthy controls without a history or clinical evidence of central or peripheral nervous system disease.
- Both male and female participants will be included.
- Age 18 or older.
- Ability and will to cooperate in the study.
- Only participants who have signed the informed consent form.
- Males and Females of childbearing potential willing to use highly effective method of contraception (hormonal contraception, intrauterine device or sexual abstinence) during the treatment period and for at least one month after the last dose of study drug.
- Patients should preferably be enrolled prior to initiation of omaveloxolone treatment. Patients who have already initiated omaveloxolone treatment may be included provided that: 1) a documented pre-treatment blood sample is available; 2) the date of treatment initiation is clearly recorded; 3) the pre-treatment sample was collected as part of routine clinical care or an ethically approved research protocol; 4) no study-specific procedures were performed prior to informed consent.
Exclusion criteria 6
- Participants who did not agree to participate in the study.
- For the patient with FA group: Hypersensitivity to omaveloxolone or any of the excipients used in Skyclarys.
- For the healthy control group: Controls with genetic risk of FA heterozygosity or homozygosity (i.e., healthy parents or siblings of patients).
- Participants with toxic abuse.
- Participants with other nervous system disease potentially influencing the results (i.e. Alzheimer or Parkinson disease etc).
- Patients with any contraindications to omaveloxolone as specified in the current approved SmPC.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Change in mitochondrial biomarkers (lipofuscin-like pigments, PINK1, ULK1, BNIP3L, TFEB, LC3, p62, GPX4, SLC7A11, and 4-HNE) from baseline to 6 and 12 months in patients with FA treated with omaveloxolone.
Secondary endpoints 2
- Between-group differences in mitochondrial biomarkers at the 6-month timepoint (treated FA patients vs. controls).
- Correlation of biomarker changes with changes in mFARS, SARA, and ADL scores over time.
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD11150682 · Product
- Active substance
- Omaveloxolone
- Pharmaceutical form
- CAPSULE, HARD
- Route of administration
- ORAL
- Max daily dose
- 150 mg milligram(s)
- Max total dose
- 150 mg milligram(s)
- Max treatment duration
- 24 Month(s)
- Authorisation status
- Authorised
- ATC code
- NOTASSIGN — -
- Marketing authorisation
- EU/1/23/1786/001
- MA holder
- BIOGEN NETHERLANDS B.V.
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/18/2037
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Fakultni Nemocnice V Motole
- Sponsor organisation
- Fakultni Nemocnice V Motole
- Address
- V Uvalu 84/1, Motol Motol
- City
- Prague
- Postcode
- 150 00
- Country
- Czechia
Scientific contact point
- Organisation
- Fakultni Nemocnice V Motole
- Contact name
- Martin Vyhnálek
Public contact point
- Organisation
- Fakultni Nemocnice V Motole
- Contact name
- Clinical Trial Department
Locations
2 EU/EEA countries · 3 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Czechia | Authorised, recruitment pending | 55 | 1 |
| Poland | Authorised, recruitment pending | 10 | 2 |
| Rest of world | — | 0 | — |
Investigational sites
Fakultni Nemocnice V Motole
Neurologická klinika 2. LF UK a FN Motol, V Uvalu 84/1, Motol, Prague
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 16 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol_2025-523881-26-00_CZ_ver01_15Sep2025 | 3 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_2025-523881-26-00_CZ_ver01_21Oct2025 | 1 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_2025-523881-26-00_PL_ver01_21Oct2025 | 2 |
| Recruitment arrangements (for publication) | K2_Recruitment material_recruitment flyer_PL | 1 |
| Recruitment arrangements (for publication) | K2_Recruitment material_website recruitment text_Gdansk | 1 |
| Recruitment arrangements (for publication) | K2_Recruitment material_website recruitment text_Warsaw | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_2025-523881-26-00_patient_CZ_ver01_15Sep2025_redacted | 2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_2025-523881-26-00_patient_PL_ver01_03Oct2025_redacted | 2 |
| Subject information and informed consent form (for publication) | L2_GDPR ICF_2025-523881-26-00_patient_CZ_ver01_29Jul2025_redacted | 2 |
| Subject information and informed consent form (for publication) | L2_GDPR ICF_2025-523881-26-00_patient_PL_ver01_29Jul2025_redacted | 2 |
| Subject information and informed consent form (for publication) | L2_GDPR Information_2025-523881-26-00_patient_CZ_ver01_29Jul2025_redacted | 2 |
| Subject information and informed consent form (for publication) | L2_GDPR Information_2025-523881-26-00_patient_PL_ver01_29Jul2025_redacted | 2 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC_2025-523881-26-00_CZ_Omaveloxolon_Skyclarys | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC_2025-523881-26-00_PL_Omaveloxolon_Skyclarys | 1 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2025-523881-26-00_CZ_ver01_15Sep2025 | 3 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_2025-523881-26-00_PL_ver01_15Sep2025 | 3 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2025-11-24 | Czechia | Acceptable 2026-03-30
|
2026-03-30 |