Treatment of a Single Patient With CRD-TMH-001

August 31, 2022 updated by: Cure Rare Disease, Inc
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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

The objective of the study is to assess the safety and preliminary efficacy of CRD-TMH-001 after intravenous administration for a period of 1 year with long-term follow-up out to 15 years.

Study Type

Interventional

Enrollment (Anticipated)

1

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Massachusetts
      • Worcester, Massachusetts, United States, 01655
        • UMass Chan Medical School

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 28 years (Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

Inclusion Criteria:

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

Exclusion Criteria:

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Single patient
Single dose of CRD-TMH-001 administered by IV
Drug: CRD-TMH-001
Participant will receive a single dose of CRD-TMH-001 administered via intravenous injection.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To assess the safety of CRD-TMH-001
Time Frame: 1 year
To assess the safety and tolerability of the therapeutic by measuring both serious and non-serious adverse events.
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Cure Rare Disease, Inc

Collaborators

University of Massachusetts, Worcester

Investigators

  • Principal Investigator: Brenda Wong, MD, UMass Chan Medical School
  • Study Director: Medical Affairs, Cure Rare Disease

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 31, 2022

Primary Completion (Anticipated)

September 1, 2023

Study Completion (Anticipated)

September 1, 2023

Study Registration Dates

First Submitted

August 11, 2022

First Submitted That Met QC Criteria

August 22, 2022

First Posted (Actual)

August 24, 2022

Study Record Updates

Last Update Posted (Actual)

September 1, 2022

Last Update Submitted That Met QC Criteria

August 31, 2022

Last Verified

August 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CS-CRD-TMH-001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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