Study to Evaluate DT-216 in Adult Patients With Friedreic... | NCT05285540

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

NCT05285540

Sponsor

Design Therapeutics, Inc.

Status

Completed

Last Update Posted

Mar 28, 2023Actual

Enrollment

39Actual

Phase

Phase 1

Conditions

Friedreich Ataxia

Interventions

DT-216
DT-216 matching Placebo

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Mar 2023

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Not provided

Start Date

Mar 11, 2022Actual

Primary Completion Date

Dec 19, 2022Actual

Completion Date

Dec 19, 2022Actual

First Submitted Date

Mar 9, 2022

First Submission Date that Met QC Criteria

Mar 9, 2022

First Posted Date

Mar 17, 2022Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Mar 27, 2023

Last Update Posted Date

Mar 28, 2023Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

39Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Single Dose: DT-216	Experimental	DT-216 will be administered once	Drug: DT-216
Single Dose: DT-216 matching placebo	Experimental	Placebo will be administered once	Drug: DT-216 matching Placebo

Interventions

Name	Type	Description	Arm Group Labels	Other Names
DT-216	Drug	DT-216 will be administered by intravenous (IV) injection	Single Dose: DT-216

Outcomes Module

Primary Outcomes

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

Secondary Outcomes

Measure	Description	Time Frame
Maximum Plasma Concentration (Cmax) of DT-216	Maximum Plasma Concentration (Cmax) of DT-216	Up to approximately 30 days
Time to Maximum Plasma Concentration (Tmax) of DT-216	Time to Maximum Plasma Concentration (Tmax) of DT-216

Other Outcomes

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

Eligibility Module

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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

55 Years

Standard Ages

Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Clinilabs		Eatontown	New Jersey	07724	United States

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D005621	Friedreich Ataxia

Ancestor Terms

ID	Term
D013132	Spinocerebellar Degenerations
D002526	Cerebellar Diseases
D001927	Brain Diseases
D002493	Central Nervous System Diseases

Browse Leaves

Not provided

Browse Branches

Not provided