GV101 for Friedreich's Ataxia (FA) Treatment

NCT07870590 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: GBS-GV101-FA-201
• Study Type: interventional
• Target: 48
• Locations: 1 country
• Sites: 1

Brief Summary

This is a dose-ranging, randomized, double-blind, placebo-controlled 12-week Phase 2a trial with an up to two week loading dose period. Eligible participants will be sequentially randomized 3:1 into three parallel groups, low-dose, mid-dose, and high-dose, to receive GV101 or placebo once daily for 12 weeks. The low-dose group will receive 200 mg from Weeks 2-12 after a 100 mg loading dose at Week 1; the mid-dose group will receive 300 mg from Weeks 3-12 after loading doses of 100 mg and 200 mg at Weeks 1 and 2, respectively; and the high dose group will receive 400 mg from Weeks 3-12 after loading doses of 200 mg and 300 mg at Weeks 1 and 2, respectively. Target enrollment is 16 participants per dose group. The primary efficacy endpoint at 12 weeks is percent change in blood FXN-M protein in each dose group compared with placebo, the key secondary endpoints are change in hs-cTnI and mFARS score in each dose group compared with placebo. Data for exploratory, safety, PK, and PD endpoints will also be collected and analyzed. Down-dosing will be allowed in groups dosed with \> 200 mg daily. Participants who complete the 12-week trial are eligible to continue into a 52-week OLE at 200 mg until the first participant from the second dose group completes the 12-week trial, at which time all OLE participants will be asked to increase their dose (4-week window) to 300 mg per day. When the first participant from the third dose group completes the 12-week trial, all current OLE participants will be asked to increase their dose (4-week window) to 400 mg per day. Participants in the second dose group will start the OLE with a 1 week loading dose of 200 mg per day and be asked to increase to 300 mg per day after the first week. Participants in the third dose group will start the OLE with a 1 week loading dose of 200 mg per day and will be asked to increase to 300 mg per day after the first week and to 400 mg per day after the second week. The OLE extends initially for 52 weeks from the last participant's entry; an additional OLE or expanded access is anticipated after this OLE to provide seamless treatment until program cessation or drug approval. Safety and efficacy assessment will continue throughout the dosing period. Down dosing related to tolerance is allowed during the OLE.

Conditions

Friedreich's Ataxia

Outcome Measures

Primary Outcomes (1)

• Mean percent change in frataxin-M protein

  Mean percent change in frataxin-M protein in blood from baseline to Week 12 in each treated group as compared with placebo

  12 weeks

Study Arms (2)

GV101

ACTIVE COMPARATOR

200 mg, 300 mg, or 400 mg

Drug: GV101

Placebo

PLACEBO COMPARATOR

Matching GV101 placebo: 200 mg, 300 mg, or 400 mg

Drug: Placebo

Interventions

GV101 DRUG

ROCK2 inhibitor

GV101

Placebo DRUG

Matching GV101 placebo

Placebo

Eligibility Criteria

• Age: 16 Years - 40 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Be male or female aged 16 - 40 years, inclusive, at the time of informed consent.
• Have a genetically confirmed diagnosis of FA with homozygous GAA repeat expansions.
• Have mFARS score ≥20 and ≤80. The screening and baseline mFARS values must fall within this range; these two values also must be within 4.5 points of each other.
• Have an hs-c-troponin I above the laboratory defined 99th percentile at screening and at least 14 ng/L.
• Be able to ambulate with or without assistance (If a participant uses an assistive device for the walk test on V1, the same or similar assistive device must be used on all subsequent walk tests throughout the trial).
• Be able to sit upright.
• Be willing and able to comply with all study procedures.
• Heterosexually active women of child-bearing potential and males who are sexually active with female(s) of child-bearing potential must agree to highly effective contraception from screening until at least 30 days after the last dose of IMP for women and 90 days after the last dose of IMP for men. Acceptable highly effective methods include:
• Contraceptive implant.
• Intrauterine device (IUD), including hormonal or copper IUD.
• Bilateral salpingectomy or bilateral tubal occlusion (including tubal ligation).
• Vasectomized male partner(s) (6 months from vasectomy) with documented azoospermia.
• Provide written informed consent.

You may not qualify if:

• Be pregnant or lactating.
• Have cognitive impairment that may interfere with compliance with study procedures.
• Have swallowing impairment(s) that preclude IMP dosing.
• Have significant cardiac disease, apart from mild/moderate cardiomyopathy associated with FA, including but not limited to severe or unstable heart failure (NYHA Class III/IV), unstable angina or recent myocardial infarction, significant arrhythmias, left-sided structural heart disease, valvular disease, pericardial constriction, or restrictive or congestive cardiomyopathy.
• Have a history of significant QTcF prolongation or QTcF \> 470 ms (females) or \> 450 ms (males) at screening.
• Have other active neurological diseases.
• Have known untreated thyroid disease, or an abnormal thyroid panel at screening that is concerning for disease.
• Have moderate or severe hepatic or renal impairment, including but not limited to eGFR \< 60 mL/min/1.73 m² (by 2021 CKD-EPI) or creatinine clearance (CLcr- calculated by Cockcroft-Gault) \>1.5 ULN or AST/ALT or total bilirubin \>1.5x ULN at screening; retest allowed if abnormalities are potentially attributable to muscle injury.
• Have uncontrolled diabetes, defined as HbA1c \> 7.0%.
• Have albumin below LLN (screening).
• Have active infections or test positive for HIV, hepatitis B, or hepatitis C at screening.
• Have clinically significant hematology or biochemistry abnormalities or any laboratory result or medical condition that, in the investigator's judgment, would increase patient risk.
• Have a history of known or suspected drug or alcohol abuse within the last 2 years.
• Participants currently receiving omaveloxolone are not eligible. Participants who have previously received omaveloxolone must complete a minimum of 4 week washout period prior to screening. Other background medications or supplements intended to treat FA must be on stable doses for ≥30 days before screening and be intended to remain unchanged throughout the 12-week trial treatment period.
• Be taking medications with narrow therapeutic indices that are primarily metabolized by CYP2C8, CYP2C9, or CYP2C19 and cannot be reduced in dose; be taking medications with narrow therapeutic indices that are p-gp substrates. Be taking daily standard doses of strong inducers or inhibitors of CYP3A4 that cannot be modified.

Sponsors & Collaborators

• Graviton Bioscience Corporation: lead

Study Sites (1)

CLINiLABS

Eatontown, New Jersey, 07724, United States

Location

MeSH Terms

Conditions

Friedreich Ataxia

Condition Hierarchy (Ancestors)

Spinocerebellar Degenerations; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 6, 2026
• First Posted: October 13, 2026
• Study Start (Estimated): November 1, 2026
• Primary Completion (Estimated): July 1, 2027
• Study Completion (Estimated): October 1, 2027
• Last Updated: October 15, 2026

Record last verified: 2026-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)