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Small Molecule

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
Be between 18 and 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to approximately 30 days
Awards & highlights

Study Summary

This trial is studying an experimental drug to see if it's safe and effective for treating Friedreich's Ataxia, a rare neurological disease.

Eligible Conditions
  • Friedreich Ataxia

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to approximately 30 days
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to approximately 30 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
Frequency of treatment adverse events (TEAEs)
Secondary outcome measures
Area Under the Concentration-time Curve (AUC) of DT-216
Maximum Plasma Concentration (Cmax) of DT-216
Time to Maximum Plasma Concentration (Tmax) of DT-216
Other outcome measures
Frataxin expression

Trial Design

2Treatment groups
Experimental Treatment
Group I: Single Dose: DT-216 matching placeboExperimental Treatment1 Intervention
Placebo will be administered once
Group II: Single Dose: DT-216Experimental Treatment1 Intervention
DT-216 will be administered once
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
DT-216
2022
Completed Phase 1
~80
DT-216 matching Placebo
2022
Completed Phase 1
~40

Find a Location

Who is running the clinical trial?

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

Frequently Asked Questions

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

Does DT-216 pose any considerable risks to human health?

"There is limited evidence of safety and efficacy for DT-216, thus it was assigned a score of 1."

Answered by AI

Is there an opportunity to be involved in this investigation?

"In order to qualify for this clinical trial, participants must be between 18-55 years of age and have a diagnosis of friedreich ataxia. This particular research endeavour is seeking approximately 25 volunteers."

Answered by AI

Can new participants join this medical experiment?

"That is correct. Data hosted on clinicaltrials.gov confirms that this medical study, which first appeared on March 11th 2022, is actively enrolling subjects. Approximately 25 people need to be recruited from a single centre."

Answered by AI

How many subjects are actively participating in this exploration?

"Affirmative. Evidenced by the information on clinicaltrials.gov, this medical trial is actively seeking to enroll participants. It was first posted on March 11th 2022 and subsequently updated April 4th 2022; presently, 25 patients are being sought out at a single location."

Answered by AI

Is the minimum age requirement for this clinical research under 18?

"This research endeavour is accepting participants over the age of majority and younger than 55 years old."

Answered by AI

Who else is applying?

How old are they?
< 18
What site did they apply to?
Clinilabs
What portion of applicants met pre-screening criteria?
Met criteria
Recent research and studies
clinicaltrials.govStudy
Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia
2022. "Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05285540.
~13 spots leftby Apr 2025
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