Back to resultsStudy to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia
Primary Purpose
Friedreich Ataxia
Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
DT-216
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Friedreich Ataxia
Eligibility 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.
Sites / Locations
- ClinilabsRecruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Multiple Dose: DT-216
Multiple Dose: DT-216 matching placebo
Arm Description
Participants will be administered multiple doses of DT-216
Participants will be administered multiple doses of placebo
Outcomes
Primary Outcome Measures
Safety Measure
Frequency of treatment emergent adverse events (TEAEs)
Secondary Outcome Measures
Pharmacokinetic parameters
Maximum Plasma Concentration (Cmax) of DT-216
Pharmacokinetic parameters
Time to Maximum Plasma Concentration (Tmax) of DT-216
Pharmacokinetic parameters
Area Under the Concentration-time Curve (AUC) of DT-216
Full Information
NCT ID
NCT05573698
First Posted
October 6, 2022
Last Updated
March 26, 2023
Sponsor
Design Therapeutics, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT05573698
Brief Title
Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia
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
Study Type
Interventional
2. Study Status
Record Verification Date
March 2023
Overall Recruitment Status
Recruiting
Study Start Date
September 27, 2022 (Actual)
Primary Completion Date
July 11, 2023 (Anticipated)
Study Completion Date
September 13, 2023 (Anticipated)
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 multiple doses of intravenous DT-216 in adult patients with Friedrich Ataxia.
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
38 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Multiple Dose: DT-216
Arm Type
Experimental
Arm Description
Participants will be administered multiple doses of DT-216
Arm Title
Multiple Dose: DT-216 matching placebo
Arm Type
Placebo Comparator
Arm Description
Participants will be administered multiple doses of placebo
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)
Placebo
Intervention Description
Matching Placebo will be administered by intravenous (IV) injection
Primary Outcome Measure Information:
Title
Safety Measure
Description
Frequency of treatment emergent adverse events (TEAEs)
Time Frame
Up to approximately 60 days
Secondary Outcome Measure Information:
Title
Pharmacokinetic parameters
Description
Maximum Plasma Concentration (Cmax) of DT-216
Time Frame
Up to approximately 60 days
Title
Pharmacokinetic parameters
Description
Time to Maximum Plasma Concentration (Tmax) of DT-216
Time Frame
Up to approximately 60 days
Title
Pharmacokinetic parameters
Description
Area Under the Concentration-time Curve (AUC) of DT-216
Time Frame
Up to approximately 60 days
Other Pre-specified Outcome Measures:
Title
Pharmacodynamic parameters
Description
Frataxin expression
Time Frame
Up to approximately 60 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.
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Jennifer Schachtele
Phone
212-994-4567
Email
jschachtele@clinilabs.com
Facility Information:
Facility Name
Clinilabs
City
Eatontown
State/Province
New Jersey
ZIP/Postal Code
07724
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Joan Mallet
Phone
212-994-4567
Email
getinvolved@clinilabs.com
First Name & Middle Initial & Last Name & Degree
Eileen Thomas
Phone
212-994-4567
Email
getinvolved@clinilabs.com
12. IPD Sharing Statement
Learn more about this trial
Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia
We'll reach out to this number within 24 hrs