Friedreich's AtaxiaPhase 2NCT07870590
GV101 for Friedreich's Ataxia (FA) Treatment
A Phase 2 study testing GV101 in people with Friedreich's Ataxia, run by Graviton Bioscience Corporation at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 16 to 40 years
- Conditions the study is for
- Friedreich's Ataxia
- What is being tested?
- GV101
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 48 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2026to Oct 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 to 40 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Show all 13Show fewer
- 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.
Who cannot join
- 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.
Show all 21Show fewer
- 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.
- Be taking dimethylfumerate or other medicinal products or supplements intended to raise FXN currently, within the past 45 days or within 5 half-lives of the medicinal product, whichever is longer
- Have participated in any other interventional clinical trial or received any investigational drug in any other clinical trial within 30 days or 5 half-lives of the IMP, whichever is longer, prior to the Baseline visit.
- Have undergone prior gene therapy for FA.
- Have previously documented mitochondrial respiratory chain disease.
- Have scheduled surgery for scoliosis or pes cavus during the clinical trial period.
- Current or prior active suicidal ideation with intent or plan or suicidal behavior/attempt (within 6 months) at screening or between screening and baseline.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
CLINiLABS
Eatontown, New Jersey, 07724
Magdy Shenouda, MD, 212-994-4567, getinvolved@clinilabs.com
About this study
Official title: A Dose-Ranging, Randomized, Double-Blind, Placebo-Controlled Phase 2a Study With an Open-Label Extension to Evaluate Safety, Pharmacodynamics (PD) and Efficacy of GV101 in Friedreich's Ataxia
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.
Read more
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
- Being tested
- GV101, Placebo
- Medical subject headings
- Friedreich Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GV101Drug
ROCK2 inhibitor
PlaceboDrug
Matching GV101 placebo
| Study group | Role | What they receive |
|---|---|---|
| GV101 | Active comparator | 200 mg, 300 mg, or 400 mg Receives: Drug: GV101 |
| Placebo | Placebo comparator | Matching GV101 placebo: 200 mg, 300 mg, or 400 mg Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Oct 2026
- Study startNov 2026 (estimated)
- Primary completionJul 2027 (estimated)
- Study completionOct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Friedreich's AtaxiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Graviton Bioscience Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07870590
- Sponsor's protocol ID
- GBS-GV101-FA-201
- Status last verified
- Oct 2026
- First submitted
- 6 Oct 2026
- Last updated
- 15 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Dose-Ranging, Randomized, Double-Blind, Placebo-Controlled Phase 2a Study With an Open-Label Extension to Evaluate Safety, Pharmacodynamics (PD) and Efficacy of GV101 in Friedreich's Ataxia
- ClinicalTrials.gov ID
- NCT07870590
- Acronym / sponsor ID
- GBS-GV101-FA-201
- Conditions
- Friedreich's Ataxia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- Graviton Bioscience CorporationIndustry (pharma, biotech, devices)
- Study start
- Nov 2026 (estimated)
- Primary completion
- Jul 2027 (estimated)
- Study completion
- Oct 2027 (estimated)
- First posted
- 13 Oct 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| GV101 | Active comparator | Drug: GV101200 mg, 300 mg, or 400 mg |
| Placebo | Placebo comparator | Drug: PlaceboMatching GV101 placebo: 200 mg, 300 mg, or 400 mg |
Who may qualify
- Ages
- 16 to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (13 listed)
- 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).
Key exclusion criteria (21 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GV101ROCK2 inhibitor | Drug | GV101 |
| PlaceboMatching GV101 placebo | Drug | Placebo |
Primary outcome
Mean percent change in frataxin-M protein
Time frame: 12 weeks
Mean percent change in frataxin-M protein in blood from baseline to Week 12 in each treated group as compared with placebo
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Oct 2026.
- Individual participant dataSharing planned: No
Related trials for Friedreich's Ataxia
Not yet recruitingObservational study
A Study to Learn More About Friedrich's Ataxia in People Living in the Gulf Cooperation Council (GCC) Region of the World
Friedreich's Ataxia
RecruitingObservational study
Clinical Course Of Disease In Participants With FA-CM
Friedreich Ataxia, Cardiomyopathy
Not yet recruitingPhase 1
A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy Adults
Friedreich Ataxia, Cardiomyopathies
RecruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia
Friedreich Ataxia, Friedreich's Ataxia
RecruitingPhase 2
An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia
Friedreich Ataxia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing GV101 in people with Friedreich's Ataxia, run by Graviton Bioscience Corporation at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 15 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mean percent change in frataxin-M protein (time frame: 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Oct 2026.