Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia

March 27, 2023 updated by: Design Therapeutics

A Phase 1a, Randomized, Double-blind, Placebo-controlled, Single Ascending Dose Study of DT-216 in Adult Patients With Friedreich Ataxia

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic and pharmacodynamic effects of intravenous DT-216 in adult patients with Friedreich Ataxia. This single ascending dose study is randomized, double-blind, placebo-controlled.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

39

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • New Jersey
      • Eatontown, New Jersey, United States, 07724
        • Clinilabs

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 55 years (Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Genetically confirmed diagnosis of Friedreich Ataxia with homozygous GAA repeat expansions
  • Body mass index (BMI) between 17 and 32 kg/m2
  • Stage 5.5 or less on the Functional Staging for Ataxia (FSA)

Exclusion Criteria:

  • Has any concomitant medical condition that in the opinion of the investigator, puts the participant at risk or precludes participant from completing the study
  • Has clinically significant abnormal laboratory results
  • Has significant cardiac disease
  • Received an investigational drug within 3 months of screening

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Sequential Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Single Dose: DT-216
DT-216 will be administered once
Drug: DT-216
DT-216 will be administered by intravenous (IV) injection
Experimental: Single Dose: DT-216 matching placebo
Placebo will be administered once
Drug: DT-216 matching Placebo
Placebo will be administered by intravenous (IV) injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Frequency of treatment adverse events (TEAEs)
Time Frame: Up to approximately 30 days
Frequency of treatment emergent adverse events (TEAEs)
Up to approximately 30 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Maximum Plasma Concentration (Cmax) of DT-216
Time Frame: Up to approximately 30 days
Maximum Plasma Concentration (Cmax) of DT-216
Up to approximately 30 days
Time to Maximum Plasma Concentration (Tmax) of DT-216
Time Frame: Up to approximately 30 days
Time to Maximum Plasma Concentration (Tmax) of DT-216
Up to approximately 30 days
Area Under the Concentration-time Curve (AUC) of DT-216
Time Frame: Up to approximately 30 days
Area Under the Concentration-time Curve (AUC) of DT-216
Up to approximately 30 days

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Frataxin expression
Time Frame: Up to approximately 30 days
Frataxin mRNA and protein expression measured in peripheral blood monocytes
Up to approximately 30 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Design Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 11, 2022

Primary Completion (Actual)

December 19, 2022

Study Completion (Actual)

December 19, 2022

Study Registration Dates

First Submitted

March 9, 2022

First Submitted That Met QC Criteria

March 9, 2022

First Posted (Actual)

March 17, 2022

Study Record Updates

Last Update Posted (Actual)

March 28, 2023

Last Update Submitted That Met QC Criteria

March 27, 2023

Last Verified

March 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • DTX-216-101

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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