A Phase 3 study of Omaveloxolone in participants with Friedreich’s Ataxia Ages 2 < 16 years

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Biogen Idec Research Limited

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

4 Dec 2025 → ongoing

Decision date (initial)

2025-11-03

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Biogen Idec Research Limited

External identifiers

EU CT number

2025-520896-13-00

ClinicalTrials.gov

NCT06953583

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacodynamic, Pharmacokinetic, Safety, Efficacy

Part 1
To evaluate the efficacy of omaveloxolone at Week 52

Part 2
To evaluate the safety and tolerability of long-term omaveloxolone use

Secondary objectives 2

1. Part 1 • To evaluate safety of omaveloxolone through Week 52 • To evaluate plasma concentration of omaveloxolone after single and multiple dose administration
2. Part 2 To evaluate the efficacy of omaveloxolone following long-term use

Conditions and MedDRA coding

Friedreich’s ataxia

Study design 5 periods

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-002487-PIP01-18

Plan to share IPD

No

IPD plan description

N/A

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

1. Part 1 • Aged 2 to < 16 years at the time of informed consent. •Diagnosed with genetically confirmed FA, i.e., homozygous for guanine-adenine-adenine (GAA) repeat expansion inintron-1 of the frataxin gene, or GAA repeat expansion in 1 allele and with point mutations or deletions, or other non-GAA expansion mutations in the other allele. •Symptomatic for FA as confirmed by clinician assessment a. Children 7 to < 16 years must also have an upright stability score (USS) score of 10 to ≤ 34 at baseline
2. Part 2 1. They have completed Part 1 RCT of the study and no discontinuation criteria (Section 8) have been met. 2. Safety and tolerability data from Part 1 RCT are supportive of continuation in the judgement of the Investigator.

Exclusion criteria 1

1. Key Exclusion Criteria: •Glycosylated hemoglobin A1C (HbA1c) > 11% •B-type natriuretic peptide (BNP) > 200 pg/mL •Ejection fraction (EF) < 40% (based on ECHO performed at Screening visit) •Clinically significant abnormal laboratory test results •Clinically significant cardiac disease except mild to moderate cardiomyopathy

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 3

1. Part 1 Primary Efficacy Endpoint •Change from baseline in USS (mFARS, Section E)
2. Secondary Efficacy Endpoints •Change from baseline in FA-HI •Change from baseline in mFARS •Change from baseline in PGI-S and CGI-S •Change from baseline in FA-ADL
3. Part 2 •Incidence of AEs and SAEs •Change from baseline in ECHO •Change from baseline in height, weight, and BMI •C-SSRS •Tanner scale

Secondary endpoints 2

1. Part 1 •Incidence of AEs and SAEs during the treatment period •Change from baseline in ECHO •Change from baseline in height, weight, and BMI •C-SSRS •Tanner scale •PK: Plasma concentrations of omaveloxolone by visit and timepoints
2. Part 2 Secondary Efficacy Endpoints: •Change from baseline in mFARS, including USS •Change from baseline in FA-ADL •Change from baseline in FA-HI •Change from baseline in PGI-S and CGI-S

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Biogen Idec Research Limited

Sponsor organisation

Biogen Idec Research Limited

Address

Building 5 Foundation Park, Roxborough Way Roxborough Way

City

Maidenhead

Postcode

SL6 3UD

Country

United Kingdom

Scientific contact point

Organisation

Biogen Idec Research Limited

Contact name

Clinical trial information desk

Public contact point

Organisation

Biogen Idec Research Limited

Contact name

Clinical trial information desk

Third parties 15

Locations

8 EU/EEA countries · 14 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 261 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

4 submissions — initial application plus substantial / non-substantial modifications