Duchenne Muscular DystrophyPhase 1NCT05514249
Treatment of a Single Patient With CRD-TMH-001
A Phase 1 study testing CRD-TMH-001 in people with Duchenne Muscular Dystrophy, run by Cure Rare Disease, Inc at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 28 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- CRD-TMH-001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- 31 Aug 2022to 12 Oct 2022
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 28 years
- Men only
- No healthy volunteers
- Adult
Who may be able to join
- Completion of informed consent
- Confirmation of genetic mutation
- Confirmation of absence of elevated AAV9 NAbs
Who cannot join
- Any significant medical issue(s) (past or current) that would, in the opinion of the Principal Investigator (PI), prevent this patient from being dosed.
Trial locations and contacts
1 site in 1 country.
United States1 site
UMass Chan Medical School
Worcester, Massachusetts, 01655
About this study
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.
Read the 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.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- CRD-TMH-001
- Keywords
- Duchenne, Muscular Dystrophy, CRISPR, gene-editing
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CRD-TMH-001Drug
Participant will receive a single dose of CRD-TMH-001 administered via intravenous injection.
| Study group | Role | What they receive |
|---|---|---|
| Single patient | Experimental | Single dose of CRD-TMH-001 administered by IV Receives: Drug: CRD-TMH-001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Aug 2022
- Study start31 Aug 2022
- Primary completion12 Oct 2022
- Study completion12 Oct 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cure Rare Disease, Inc
- Sponsor type
- University, hospital or other
- Collaborators
- University of Massachusetts, Worcester
- Responsible party
- Sponsor
- Investigators
- Brenda Wong, MD, Principal investigator, UMass Chan Medical SchoolMedical Affairs, Study director, Cure Rare Disease
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05514249
- Sponsor's protocol ID
- CS-CRD-TMH-001
- Status last verified
- Oct 2026
- First submitted
- 11 Aug 2022
- Last updated
- 20 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of a Single Patient With CRD-TMH-001
- ClinicalTrials.gov ID
- NCT05514249
- Acronym / sponsor ID
- CS-CRD-TMH-001
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne, Muscular Dystrophy, CRISPR, gene-editing
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Cure Rare Disease, IncUniversity, hospital or other
- Collaborators
- University of Massachusetts, Worcester
- Study start
- 31 Aug 2022 (actual)
- Primary completion
- 12 Oct 2022 (actual)
- Study completion
- 12 Oct 2022 (actual)
- First posted
- 24 Aug 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single patient | Experimental | Drug: CRD-TMH-001Single dose of CRD-TMH-001 administered by IV |
Who may qualify
- Ages
- 18 to 28 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- Completion of informed consent
- Confirmation of genetic mutation
- Confirmation of absence of elevated AAV9 NAbs
Key exclusion criteria (1 listed)
- Any significant medical issue(s) (past or current) that would, in the opinion of the Principal Investigator (PI), prevent this patient from being dosed.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CRD-TMH-001Participant will receive a single dose of CRD-TMH-001 administered via intravenous injection. | Drug | Single patient |
Primary outcome
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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Brenda Wong, MD principal investigator
UMass Chan Medical School
Medical Affairs study director
Cure Rare Disease
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CRD-TMH-001 in people with Duchenne Muscular Dystrophy, run by Cure Rare Disease, Inc at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To assess the safety of CRD-TMH-001 (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Oct 2026.