Not currently recruiting at University of California Health

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

a study on Cerebellar Ataxia Friedreich's Ataxia

Summary

Eligibility
for people ages 18-40 (full criteria)
Location
at UCLA
Dates
study started
study ends around
Principal Investigator
by Susan Perlman, MA (ucla)

Description

Summary

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.

Official Title

A Phase 1b First-in-Human, Open-Label, Dose-Finding Trial to Evaluate the Safety and Tolerability of SGT-212 Delivered Via Dual Intradentate Nucleus (IDN) and Intravenous (IV) Administration to Participants With Friedreich's Ataxia (FA)

Keywords

Friedreich's Ataxia (FA), SGT-212, Gene Therapy, Frataxin, Repeat Expansion, Neuromuscular, Cardiac, Ataxia, Movement Disorder, Rare disease, AAV, AAVhu68, Friedreich Ataxia, Movement Disorders, Rare Diseases, Ambulatory, Ambulatory and Non-Ambulatory

Eligibility

You can join if…

Open to people ages 18-40

  • 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
  • Is willing to agree to the following rules for use of omaveloxolone (Skyclarys):
    1. 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
    2. 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 omaveloxolone during the 18-month period after SGT-212 infusion NOTE: The use of any other approved or investigational medicinal product for the treatment of FA should be discussed with the study team.

You CAN'T join if...

  • 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
  • Is unable to undergo cardiac and brain MRI with contrast, including hypersensitivity to gadolinium contrast agent, presence of a non-MRI-compatible cardiac pacemaker, presence of a non-MRI-compatible implantable cardiac defibrillator, or physical condition (e.g., contractures)
  • 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
  • Has received gene therapy at any time
  • Has contraindications to receiving corticosteroids
  • Has any contraindication to the surgical procedures involved with IDN infusion of SGT-212
  • Has any known cardiac disease not related to FA including known obstructive coronary artery disease (CAD)
  • Other Inclusion/Exclusion criteria to be applied as per protocol.

Locations

  • The University of California, Los Angeles (UCLA) not yet accepting patients
    Los Angeles California 90095 United States
  • The Ohio State University accepting new patients
    Columbus Ohio 43210 United States
  • The Children's Hospital of Philadelphia (CHOP) not yet accepting patients
    Philadelphia Pennsylvania 19104 United States

Lead Scientist at University of California Health

  • Susan Perlman, MA (ucla)
    HS Clinical Professor, Neurology, Medicine. Authored (or co-authored) 213 research publications

Details

Status
accepting new patients at some sites,
but this study is not currently recruiting here
Start Date
Completion Date
(estimated)
Sponsor
Solid Biosciences Inc.
ID
NCT07180355
Phase
Phase 1 research study
Study Type
Interventional
Participants
Expecting 10 study participants
Last Updated