SGT-212 Gene Therapy for Friedreich's Ataxia

(FALCON Trial)

Not yet recruiting at 1 trial location

Overseen By Solid Biosciences Clinical Trials

Age: 18 - 65

Sex: Any

Trial Phase: Phase 1

Sponsor: Solid Biosciences Inc.

Disqualifiers: Antibodies AAV9, BMI ≥33, Diabetes, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 5 years

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new gene therapy, SGT-212, for individuals with Friedreich's ataxia (FA), a rare genetic disorder affecting movement and coordination. The primary aim is to assess the treatment's safety and tolerability. Participants will receive the treatment through two methods: directly into a part of the brain and via an IV. This trial is suitable for those who developed FA symptoms at or before age 25 and have a confirmed diagnosis. As a Phase 1 trial, participants will be among the first to receive this treatment, aiding researchers in understanding its effects in people.

Will I have to stop taking my current medications?

The trial requires that if you are currently taking omaveloxolone, you must have been on a stable dose for 12 weeks and may need to stop if liver issues are detected. If you are not taking omaveloxolone, you should not resume it for 18 months after receiving SGT-212. For other medications, you should discuss with the study team.

Is there any evidence suggesting that SGT-212 is likely to be safe for humans?

Research shows that SGT-212, a gene therapy for Friedreich's ataxia, is being carefully monitored for safety and tolerability. The FDA approved the investigational new drug application (IND) for SGT-212, indicating that early safety tests were promising enough to warrant further study. This treatment uses an adeno-associated virus (AAV), a type of virus often used in gene therapy due to its safety in previous studies.

Although detailed safety data specific to SGT-212 is not yet available, the FDA has granted it fast track designation. This suggests confidence in its safety and potential effectiveness. Fast track designation is often given to treatments that show promise in early testing and address unmet medical needs.

As this is an early-phase trial, the main goal is to assess the safety and side effects of SGT-212. Researchers will closely monitor participants for any adverse reactions. This phase is crucial for understanding the treatment's tolerability and potential risks. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Friedreich's ataxia?

SGT-212 is unique because it offers a gene therapy approach to treating Friedreich's Ataxia, a condition traditionally managed with supportive care like physical therapy and medications to alleviate symptoms. Unlike standard treatments, SGT-212 targets the root cause by aiming to correct the genetic defect responsible for the disease. Researchers are particularly excited about this treatment because it involves a novel delivery method: a combination of bilateral intradentate infusion and systemic intravenous infusion, which potentially maximizes the therapy's reach and effectiveness. This innovative approach could provide significant, lasting benefits rather than just symptom management.

What evidence suggests that SGT-212 might be an effective treatment for Friedreich's ataxia?

Research has shown that SGT-212 is a promising new treatment for Friedreich's ataxia (FA). This therapy uses a virus to deliver a healthy version of the frataxin (FXN) gene into the body. Early results suggest it could help increase FXN levels, which are often low in people with FA. In this trial, participants will be divided into different cohorts based on their ambulatory status to receive SGT-212. The treatment targets the specific tissues affected by the disease, potentially slowing or altering its progression. Although more information is needed, SGT-212 has received FDA Fast Track status, highlighting its potential importance in treating FA. ^{1 3 4 6 7}

Are You a Good Fit for This Trial?

This trial is for individuals with Friedreich's Ataxia, a rare genetic movement disorder. Participants should be diagnosed with FA to qualify. Specific inclusion and exclusion criteria are not listed but typically involve age, disease severity, and overall health status.

Inclusion Criteria

My disability score for Friedreich's Ataxia is between 1 and 5.

My FA symptoms started when I was 25 or younger.

I have been diagnosed with Fanconi anemia both clinically and genetically.

Exclusion Criteria

Has a modified FARS (mFARS) score <20

Has uncontrolled diabetes as defined by a hemoglobin (Hb) A1c >9%

Has participated in recent interventional clinical studies or received any investigational therapy administered within 3 months or 5 half-lives (whichever is longer) prior to Screening

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive SGT-212 via dual intradentate nucleus (IDN) and intravenous (IV) administration

5 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

What Are the Treatments Tested in This Trial?

Interventions

• SGT-212

Trial Overview The study tests SGT-212 gene therapy in people with Friedreich's Ataxia. It's an early-stage (phase 1b) trial focusing on safety and how well patients tolerate the treatment. SGT-212 will be given through two methods: directly into the brain (IDN) and into a vein (IV).

How Is the Trial Designed?

Group I: Cohort 3 (Ambulatory and Non-Ambulatory) Experimental Treatment 1 Intervention

Participants will receive bilateral IDN followed by systemic IV infusion.

Group II: Cohort 2 (Ambulatory) Experimental Treatment 1 Intervention

Ambulatory participants will receive bilateral IDN infusion followed by systemic IV infusion.

Group III: Cohort 1 (Non-Ambulatory) Experimental Treatment 1 Intervention

Non-ambulatory participants will receive bilateral intradentate infusion (IDN) followed by systemic intravenous (IV) infusion.

Who Is Running the Clinical Trial?

Solid Biosciences Inc.

Lead Sponsor

Trials

4

Recruited

130+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT07180355

A Study of SGT-212 Gene Therapy in Friedreich's Ataxia For a candidate who is not actively taking omaveloxolone, at least 12 weeks have passed since the last dose and the candidate agrees not to resume ...

2. investors.solidbio.com investors.solidbio.com/news-releases/news-release-details/solid-biosciences-receives-fda-fast-track-designation-sgt-212

Solid Biosciences Receives FDA Fast Track Designation ... SGT-212 is a recombinant AAV-based gene replacement therapy for Friedreich's ataxia (FA) designed to deliver full-length human frataxin (FXN) via a dual route ...

3. curefa.org curefa.org/clinical-studies/a-study-of-sgt-212-gene-therapy-in-friedreichs-ataxia-falcon/

A Study of SGT-212 Gene Therapy in Friedreich's Ataxia ... The purpose of this study is to evaluate the safety and tolerability of SGT-212 in participants with Friedreich's ataxia (FA).

4. researchgate.net researchgate.net/publication/397257666_Abstract_4366785_Efficacy_and_Safety_of_a_Novel_AAV_FXN_Gene_Therapy_SGT-212_for_the_Treatment_of_Friedreich's_Ataxia

Efficacy and Safety of a Novel AAV FXN Gene Therapy ... Hypothesis: AAV-mediated gene replacement therapy can safely restore FXN expression in disease-relevant tissues and modify the course of FA.

5. firstwordpharma.com firstwordpharma.com/story/5962205

Solid Biosciences Reports First Quarter 2025 Financial ... SGT-212 is the first gene therapy candidate for FA to utilize a dual route of administration that was designed to promote restoration of ...

6. cgtlive.com cgtlive.com/view/solid-biosciences-friedreich-ataxia-gene-therapy-sgt-212-garners-fast-track-designation

Solid Biosciences' Friedreich Ataxia Gene Therapy SGT ... 1. Solid Biosciences receives FDA fast track designation for SGT-212 dual route of administration gene therapy for Friedreich's ataxia. News ...

7. neurologylive.com neurologylive.com/view/fda-clears-ind-trial-assessing-gene-therapy-sgt-212-friedreich-ataxia

FDA Clears IND for Trial Assessing Gene Therapy SGT ... The FDA has given clearance to Solid Biosciences' investigational new drug application (IND) for SGT-212, an adeno-associated virus (AAV)-based gene therapy ...