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Multiple Dose: DT-216 for Friedreich Ataxia

Phase 1
Waitlist Available
Research Sponsored by Design Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Stage 5.5 or less on the Functional Staging for Ataxia (FSA) at screening.
Be between 18 and 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to approximately 60 days
Awards & highlights

Study Summary

This trial tests if a drug is safe & effective for treating Friedrich Ataxia in adults.

Eligible Conditions
  • Friedreich Ataxia

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
You have a mild or less severe stage of ataxia according to the Functional Staging for Ataxia (FSA) scale.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to approximately 60 days
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to approximately 60 days for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Safety Measure
Secondary outcome measures
Pharmacokinetic parameters
Other outcome measures
Pharmacodynamic parameters

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: Multiple Dose: DT-216Experimental Treatment1 Intervention
Participants will be administered multiple doses of DT-216
Group II: Multiple Dose: DT-216 matching placeboPlacebo Group1 Intervention
Participants will be administered multiple doses of placebo
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
DT-216
2022
Completed Phase 1
~80

Find a Location

Who is running the clinical trial?

Design TherapeuticsLead Sponsor
1 Previous Clinical Trials
39 Total Patients Enrolled
1 Trials studying Friedreich Ataxia
39 Patients Enrolled for Friedreich Ataxia
Design Therapeutics, Inc.Lead Sponsor
1 Previous Clinical Trials
39 Total Patients Enrolled
1 Trials studying Friedreich Ataxia
39 Patients Enrolled for Friedreich Ataxia

Media Library

Friedreich Ataxia Clinical Trial 2023: DT-216 Highlights & Side Effects. Trial Name: NCT05573698 — Phase 1
DT-216 (Unknown) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05573698 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Has the FDA provided its blessing to Multiple Dose: DT-216?

"Based on the limited amount of data collected thus far, our team at Power has assigned Multiple Dose: DT-216 a score of 1 for safety. This is largely due to it being in Phase 1 trials and having minimal evidence supporting efficacy or safety."

Answered by AI

Is this research team still searching for participants?

"Affirmative. Clinicaltrials.gov records suggest that this experiment began recruiting on September 27th 2022, and is currently in progress. 38 individuals are needed to join the trial at one medical centre."

Answered by AI

Are there certain participants that are more ideal for this research endeavor?

"To be eligible for this medical trial, Friedreich Ataxia patients need to between 18 and 55 years old. Furthermore, they must have a genetic confirmation of the condition with homozygous GAA repeat expansions on their record. Additionally, they should not exceed stage 5.5 on Functional Staging For Ataxia (FSA). Lastly, these potential participants will also require signing an informed consent form prior to enrollment into the study."

Answered by AI

How many participants are receiving treatment in this clinical trial?

"Affirmative. Evidenced on clinicaltrials.gov, this medical experiment was first advertised in September 27th 2022 and is actively looking for participants; 38 individuals will be accepted at a single trial site."

Answered by AI

Are octogenarians accepted into the clinical trial?

"Applicants for this medical trial must be over 18 years old and under 55 to fulfill the inclusion criteria."

Answered by AI

Who else is applying?

What portion of applicants met pre-screening criteria?
Did not meet criteria

Why did patients apply to this trial?

Because our country doctors are there in this research.
PatientReceived 2+ prior treatments
~13 spots leftby Mar 2025