NCT05514249

Brief Summary

The study is a single patient study intended to understand the effects of a gene-editing therapeutic to treat a rare mutation of Duchenne muscular dystrophy.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
1

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Aug 2022

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 11, 2022

Completed
13 days until next milestone

First Posted

Study publicly available on registry

August 24, 2022

Completed
7 days until next milestone

Study Start

First participant enrolled

August 31, 2022

Completed
1 year until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2023

Completed
Last Updated

September 1, 2022

Status Verified

August 1, 2022

Enrollment Period

1 year

First QC Date

August 11, 2022

Last Update Submit

August 31, 2022

Conditions

Duchenne Muscular Dystrophy

Keywords

DuchenneMuscular DystrophyCRISPRgene-editing

Outcome Measures

Primary Outcomes (1)

  • To assess the safety of CRD-TMH-001

    To assess the safety and tolerability of the therapeutic by measuring both serious and non-serious adverse events.

    1 year

Study Arms (1)

Single patient

EXPERIMENTAL

Single dose of CRD-TMH-001 administered by IV

Drug: CRD-TMH-001

Interventions

CRD-TMH-001DRUG

Participant will receive a single dose of CRD-TMH-001 administered via intravenous injection.

Single patient

Eligibility Criteria

Age18 Years - 28 Years
Sexmale
Healthy VolunteersNo
Age GroupsAdult (18-64)

You may qualify if:

  • Completion of informed consent
  • Confirmation of genetic mutation
  • Confirmation of absence of elevated AAV9 NAbs

You may not qualify if:

  • \- Any significant medical issue(s) (past or current) that would, in the opinion of the Principal Investigator (PI), prevent this patient from being dosed.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

UMass Chan Medical School

Worcester, Massachusetts, 01655, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, DuchenneMuscular Dystrophies

Condition Hierarchy (Ancestors)

Muscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Brenda Wong, MD

    UMass Chan Medical School

    PRINCIPAL INVESTIGATOR

  • Medical Affairs

    Cure Rare Disease

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Masking Details
Single patient clinical trial
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 11, 2022

First Posted

August 24, 2022

Study Start

August 31, 2022

Primary Completion

September 1, 2023

Study Completion

September 1, 2023

Last Updated

September 1, 2022

Record last verified: 2022-08

Data Sharing

IPD Sharing
Will not share

Locations

US(1)