Health TrialsbyHealthPanel

A Study of SGT-212 Gene Therapy in Friedreich's Ataxia

ConditionFriedreich's Ataxia (FA)
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18-40
SponsorSolid Biosciences Inc.

About this trial

This is a phase 1b, first in-human, open-label, dose-finding study investigating the safety and tolerability of SGT-212 in participants with Friedreich's ataxia (FA). It will be delivered via dual intradentate nucleus (IDN) and intravenous (IV) administration to participants with FA.

All participants will receive SGT-212 and will be enrolled in the study for approximately 5 years.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Has history of FA symptom onset ≤25 years of age

Has a clinical and genetic diagnosis of FA

Has a staging score of ≥1 but <6 on the Friedreich's Ataxia Rating Scale (FARS) Functional Disability Staging Score

For a candidate who is currently taking omaveloxolone, has been on a stable dose for 12 weeks, expects to continue taking omaveloxolone at that dose throughout the study, and is willing to stop taking omaveloxolone at the direction of the Investigator or Sponsor's Medical Monitor if evidence of transaminitis or synthetic liver dysfunction is detected during the study

Disqualifiers

Antibodies against adeno-associated virus serotype 9 (AAV9)

Has a modified FARS (mFARS) score <20

Has a body weight ≤25 kilogram (kg) or has body mass index (BMI) ≥33 kg/m²

Has a contraindication to endomyocardial biopsy (EMB) or cardiac catheterization

Trial design

Design model

Parallel

Treatments tested in this trial

  • SGT-212

    Drug

    Adeno-associated virus serotype AAVhu68 containing a codon-optimized complementary DNA (cDNA)

Treatment groups

10 Participants
are divided into 3 treatment groups
Group A: Cohort 1 (Non-Ambulatory)Experimental treatment 1 intervention
Group B: Cohort 2 (Ambulatory)Experimental treatment 1 intervention
Group C: Cohort 3 (Ambulatory and Non-Ambulatory)Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Incidence and Severity of Treatment Emergent Adverse Events (TEAEs)

Time frame
Month 12

Secondary outcomes

1

Incidence and Severity of TEAEs

Time frame
Months 18 and 60
2

Incidence and Severity of Treatment-emergent Serious adverse events (SAEs)

Time frame
Months 18 and 60
3

Number of Treatment-emergent deaths

Time frame
Months 18 and 60
4

Change from Baseline in Columbia-Suicide Severity Rating Scale (C-SSRS)

Time frame
Baseline, Months 18 and 60

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Solid Biosciences Inc.

Lead sponsor

Source ClinicalTrials.gov
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