Treatment of a Single Patient With CRD-TMH-001
Single patient trial for rare Duchenne muscular dystrophy mutation
Plain English Summary
Treatment of a Single Patient With CRD-TMH-001 is a Phase 1 clinical trial sponsored by Cure Rare Disease, Inc studying Duchenne Muscular Dystrophy. Tests a gene-editing therapy called CRD-TMH-001 for a specific rare mutation of Duchenne muscular dystrophy. This trial is for a single patient with a confirmed genetic mutation for Duchenne muscular dystrophy. Participation involves receiving one dose of the therapy through an IV injection and ongoing safety monitoring for one year. Currently, there are no approved alternative gene-editing therapies for this specific mutation. The trial aims to enroll 1 participants.
Official 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.
Who Can Participate
Here is what you need to know about eligibility for this trial. Patients with a confirmed genetic mutation causing Duchenne muscular dystrophy can join. Patients must not have pre-existing antibodies that could interfere with the therapy. Patients must not have any other significant medical issues that could be worsened by the treatment. This trial is studying Duchenne Muscular Dystrophy, so participants generally need a confirmed diagnosis.
What They're Measuring
The primary goal is to see if CRD-TMH-001 is safe for the patient over one year, meaning doctors will closely monitor for any adverse reactions. The specific primary outcome measures are: To assess the safety of CRD-TMH-001 (1 year). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.
About This Phase
This trial is in Phase 1, the first major stage of clinical testing. Phase 1 trials typically involve 20-100 participants and focus on safety, dosage levels, and side effects. The primary goal is not to test whether the treatment works but to establish that it is safe enough for further testing. About 70% of Phase 1 drugs advance to Phase 2. If successful, the treatment will proceed to Phase 2 efficacy testing.
Why This Trial Matters
This trial addresses a critical unmet need for patients with a rare genetic mutation of Duchenne muscular dystrophy, for whom few treatment options exist. This research targets Duchenne Muscular Dystrophy, where improved treatment options are needed.
Investor Insight
This trial represents an early-stage investment in a novel gene-editing approach for a rare disease, with potential for future development if safety and efficacy are demonstrated. Phase 1 trials have approximately a 10% chance of eventually gaining FDA approval.
Is This Trial Right for Me?
Ask your doctor about the specific risks and benefits of CRD-TMH-001 for your condition. Understand the commitment to regular medical check-ups and monitoring for a full year. Discuss any concerns about potential side effects or the treatment process with the research team. The trial is being conducted at 1 site. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.
AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.
Study Design
- Study Type: INTERVENTIONAL
- Allocation: NA
- Model: SINGLE_GROUP
- Masking: NONE
- Enrollment: 1 participants
Interventions
- DRUG: CRD-TMH-001 — Participant will receive a single dose of CRD-TMH-001 administered via intravenous injection.
Primary Outcomes
- To assess the safety of CRD-TMH-001 (1 year)
Full Eligibility Criteria
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.
Trial Locations
- UMass Chan Medical School, Worcester, Massachusetts, United States
Frequently Asked Questions
What is clinical trial NCT05514249?
NCT05514249 is a Phase 1 INTERVENTIONAL study titled "Treatment of a Single Patient With CRD-TMH-001." It is currently unknown and is sponsored by Cure Rare Disease, Inc. The trial targets enrollment of 1 participants.
What conditions does NCT05514249 study?
This trial investigates treatments for Duchenne Muscular Dystrophy. The primary condition under study is Duchenne Muscular Dystrophy.
What treatments are being tested in NCT05514249?
The interventions being studied include: CRD-TMH-001 (DRUG). Participant will receive a single dose of CRD-TMH-001 administered via intravenous injection.
What does Phase 1 mean for NCT05514249?
Phase 1 trials are the first stage of testing a new treatment in humans. They focus on safety, dosage, and side effects, usually involving 20-100 healthy volunteers or patients.
What is the current status of NCT05514249?
This trial is currently "Unknown." It started on 2022-08-31. The estimated completion date is 2023-09.
Who is sponsoring NCT05514249?
NCT05514249 is sponsored by Cure Rare Disease, Inc. The sponsor is responsible for funding, designing, and overseeing the clinical trial.
How many people can participate in NCT05514249?
The trial aims to enroll 1 participants. The trial status is unknown.
How is NCT05514249 designed?
This is a interventional study, uses na allocation, follows a single_group design, employs none masking.
What are the primary outcomes being measured in NCT05514249?
The primary outcome measures are: To assess the safety of CRD-TMH-001 (1 year). These are the main endpoints researchers use to determine whether the treatment is effective.
Where is NCT05514249 being conducted?
This trial is being conducted at 1 site, including Worcester, Massachusetts (United States).
Where can I find official information about NCT05514249?
The official record for NCT05514249 is available on ClinicalTrials.gov at https://clinicaltrials.gov/study/NCT05514249. This government database provides the most up-to-date and detailed information about the trial.
What is NCT05514249 testing in simple terms?
Tests a gene-editing therapy called CRD-TMH-001 for a specific rare mutation of Duchenne muscular dystrophy. This trial is for a single patient with a confirmed genetic mutation for Duchenne muscular dystrophy.
Why is this trial significant?
This trial addresses a critical unmet need for patients with a rare genetic mutation of Duchenne muscular dystrophy, for whom few treatment options exist.
What are the potential risks of participating in NCT05514249?
The main risk is that the therapy may not be safe, potentially causing unexpected side effects. Side effects could include reactions to the intravenous injection or unforeseen consequences of gene editing. As with any clinical trial, participants are closely monitored and can withdraw at any time.
Should I consider participating in NCT05514249?
Ask your doctor about the specific risks and benefits of CRD-TMH-001 for your condition. Understand the commitment to regular medical check-ups and monitoring for a full year. Discuss any concerns about potential side effects or the treatment process with the research team. Always discuss clinical trial participation with your healthcare provider to determine if it is appropriate for your specific situation.
What does NCT05514249 signal from an investment perspective?
This trial represents an early-stage investment in a novel gene-editing approach for a rare disease, with potential for future development if safety and efficacy are demonstrated. This is a Phase 1 trial, which is in early development stages.
What happens if the treatment in this trial doesn't work?
Participation involves receiving one dose of the therapy through an IV injection and ongoing safety monitoring for one year. Participants in clinical trials always have the right to withdraw and pursue alternative treatments. The study team will help transition patients to other available options.
Related Conditions
More Duchenne Muscular Dystrophy Trials
This analysis is AI-generated and does not constitute medical advice. Always consult your healthcare provider before making decisions about clinical trial participation.