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

Primary Purpose

Friedreich Ataxia

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
DT-216
DT-216 matching Placebo
Sponsored by
Design Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Friedreich Ataxia

Eligibility Criteria

18 Years - 55 Years (Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Clinilabs

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Single Dose: DT-216

Single Dose: DT-216 matching placebo

Arm Description

DT-216 will be administered once

Placebo will be administered once

Outcomes

Primary Outcome Measures

Frequency of treatment adverse events (TEAEs)
Frequency of treatment emergent adverse events (TEAEs)

Secondary Outcome Measures

Maximum Plasma Concentration (Cmax) of DT-216
Maximum Plasma Concentration (Cmax) of DT-216
Time to Maximum Plasma Concentration (Tmax) of DT-216
Time to Maximum Plasma Concentration (Tmax) of DT-216
Area Under the Concentration-time Curve (AUC) of DT-216
Area Under the Concentration-time Curve (AUC) of DT-216

Full Information

First Posted
March 9, 2022
Last Updated
March 27, 2023
Sponsor
Design Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT05285540
Brief Title
Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia
Official Title
A Phase 1a, Randomized, Double-blind, Placebo-controlled, Single Ascending Dose Study of DT-216 in Adult Patients With Friedreich Ataxia
Study Type
Interventional

2. Study Status

Record Verification Date
March 2023
Overall Recruitment Status
Completed
Study Start Date
March 11, 2022 (Actual)
Primary Completion Date
December 19, 2022 (Actual)
Study Completion Date
December 19, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Design Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Friedreich Ataxia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Sequential Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
39 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Single Dose: DT-216
Arm Type
Experimental
Arm Description
DT-216 will be administered once
Arm Title
Single Dose: DT-216 matching placebo
Arm Type
Experimental
Arm Description
Placebo will be administered once
Intervention Type
Drug
Intervention Name(s)
DT-216
Intervention Description
DT-216 will be administered by intravenous (IV) injection
Intervention Type
Drug
Intervention Name(s)
DT-216 matching Placebo
Intervention Description
Placebo will be administered by intravenous (IV) injection
Primary Outcome Measure Information:
Title
Frequency of treatment adverse events (TEAEs)
Description
Frequency of treatment emergent adverse events (TEAEs)
Time Frame
Up to approximately 30 days
Secondary Outcome Measure Information:
Title
Maximum Plasma Concentration (Cmax) of DT-216
Description
Maximum Plasma Concentration (Cmax) of DT-216
Time Frame
Up to approximately 30 days
Title
Time to Maximum Plasma Concentration (Tmax) of DT-216
Description
Time to Maximum Plasma Concentration (Tmax) of DT-216
Time Frame
Up to approximately 30 days
Title
Area Under the Concentration-time Curve (AUC) of DT-216
Description
Area Under the Concentration-time Curve (AUC) of DT-216
Time Frame
Up to approximately 30 days
Other Pre-specified Outcome Measures:
Title
Frataxin expression
Description
Frataxin mRNA and protein expression measured in peripheral blood monocytes
Time Frame
Up to approximately 30 days

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
55 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Facility Information:
Facility Name
Clinilabs
City
Eatontown
State/Province
New Jersey
ZIP/Postal Code
07724
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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