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Study to Evaluate Multiple Ascending Dose and Multi-Dose of 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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05573698
Recruitment Status : Recruiting
First Posted : October 10, 2022
Last Update Posted : October 10, 2022
Sponsor:
Information provided by (Responsible Party):
Design Therapeutics

Study Description
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Brief Summary:
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of multiple doses of intravenous DT-216 in adult patients with Friedrich Ataxia.

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

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 38 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 1b, Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose and Multi-Dose Study of DT-216 in Adult Patients With Friedreich Ataxia
Actual Study Start Date : September 27, 2022
Estimated Primary Completion Date : July 11, 2023
Estimated Study Completion Date : September 13, 2023

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Multiple Dose: DT-216
Participants will be administered multiple doses of DT-216
 Drug: DT-216
DT-216 will be admininistered by intravenous (IV) injection
Placebo Comparator: Multiple Dose: DT-216 matching placebo
Participants will be administered multiple doses of placebo
 Drug: Placebo
Matching Placebo will be administered by intravenous (IV) injection


Outcome Measures
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Primary Outcome Measures :
  1. Safety Measure [ Time Frame: Up to approximately 60 days ]
    Frequency of treatment emergent adverse events (TEAEs)


Secondary Outcome Measures :
  1. Pharmacokinetic parameters [ Time Frame: Up to approximately 60 days ]
    Maximum Plasma Concentration (Cmax) of DT-216

  2. Pharmacokinetic parameters [ Time Frame: Up to approximately 60 days ]
    Time to Maximum Plasma Concentration (Tmax) of DT-216

  3. Pharmacokinetic parameters [ Time Frame: Up to approximately 60 days ]
    Area Under the Concentration-time Curve (AUC) of DT-216


Other Outcome Measures:
  1. Pharmacodynamic parameters [ Time Frame: Up to approximately 60 days ]
    Frataxin expression


Eligibility Criteria
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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.
  • Able and willing to sign informed consent form prior to study enrollment.
  • Stage 5.5 or less on the Functional Staging for Ataxia (FSA) at screening.

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.
Contacts and Locations
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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): NCT05573698


Contacts
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Contact: Jennifer Schachtele 212-994-4567 jschachtele@clinilabs.com

Locations
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United States, New Jersey
Clinilabs Recruiting
Eatontown, New Jersey, United States, 07724
Contact: Joan Mallet    212-994-4567    getinvolved@clinilabs.com   
Contact: Eileen Thomas    212-994-4567    getinvolved@clinilabs.com   
Sponsors and Collaborators
Design Therapeutics
More Information
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Responsible Party: Design Therapeutics
ClinicalTrials.gov Identifier: NCT05573698    
Other Study ID Numbers: DTX-216-102
First Posted: October 10, 2022    Key Record Dates
Last Update Posted: October 10, 2022
Last Verified: October 2022

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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