Multiple Dose: DT-216 for Friedreich Ataxia

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Friedreich AtaxiaDT-216 - Drug
Eligibility
18 - 55
All Sexes
What conditions do you have?
Select

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