Multiple Dose: DT-216 for Friedreich Ataxia

Phase-Based Progress Estimates
Friedreich AtaxiaDT-216 - Drug
18 - 55
All Sexes
What conditions do you have?

Study Summary

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of multiple doses of intravenous DT-216 in adult patients with Friedrich Ataxia.

Eligible Conditions
  • Friedreich Ataxia

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

1 Primary · 1 Secondary · Reporting Duration: Up to approximately 60 days

Day 60
Pharmacodynamic parameters
Pharmacokinetic parameters
Safety Measure

Trial Safety

Safety Progress

1 of 3

Trial Design

2 Treatment Groups

Multiple Dose: DT-216
1 of 2
Multiple Dose: DT-216 matching placebo
1 of 2

Experimental Treatment

Non-Treatment Group

38 Total Participants · 2 Treatment Groups

Primary Treatment: Multiple Dose: DT-216 · Has Placebo Group · Phase 1

Multiple Dose: DT-216
Experimental Group · 1 Intervention: DT-216 · Intervention Types: Drug
Multiple Dose: DT-216 matching placebo
PlaceboComparator Group · 1 Intervention: Placebo · Intervention Types: Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: up to approximately 60 days

Who is running the clinical trial?

Design TherapeuticsLead Sponsor
1 Previous Clinical Trials
25 Total Patients Enrolled
1 Trials studying Friedreich Ataxia
25 Patients Enrolled for Friedreich Ataxia

Eligibility Criteria

Age 18 - 55 · All Participants · 3 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have Friedreich Ataxia with homozygous GAA repeat expansions.
You are able and willing to sign informed consent form prior to study enrollment.