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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05285540
Recruitment Status : Completed
First Posted : March 17, 2022
Last Update Posted : March 28, 2023
Sponsor:
Information provided by (Responsible Party):
Design Therapeutics, Inc.

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.

Condition or disease Intervention/treatment Phase
Friedreich Ataxia Drug: DT-216 Drug: DT-216 matching Placebo Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 39 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 1a, Randomized, Double-blind, Placebo-controlled, Single Ascending Dose Study of DT-216 in Adult Patients With Friedreich Ataxia
Actual Study Start Date : March 11, 2022
Actual Primary Completion Date : December 19, 2022
Actual Study Completion Date : December 19, 2022


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




Primary Outcome Measures :
  1. Frequency of treatment adverse events (TEAEs) [ Time Frame: Up to approximately 30 days ]
    Frequency of treatment emergent adverse events (TEAEs)


Secondary Outcome Measures :
  1. Maximum Plasma Concentration (Cmax) of DT-216 [ Time Frame: Up to approximately 30 days ]
    Maximum Plasma Concentration (Cmax) of DT-216

  2. 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

  3. 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


Other Outcome Measures:
  1. Frataxin expression [ Time Frame: Up to approximately 30 days ]
    Frataxin mRNA and protein expression measured in peripheral blood monocytes



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years to 55 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05285540


Locations
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United States, New Jersey
Clinilabs
Eatontown, New Jersey, United States, 07724
Sponsors and Collaborators
Design Therapeutics, Inc.
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Responsible Party: Design Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT05285540    
Other Study ID Numbers: DTX-216-101
First Posted: March 17, 2022    Key Record Dates
Last Update Posted: March 28, 2023
Last Verified: March 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Ataxia
Cerebellar Ataxia
Friedreich Ataxia
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinocerebellar Degenerations
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases