Treatment of a Single Patient With CRD-TMH-001 | NCT05514249

Studies
Treatment of a Single Patient With CRD-TMH-001

NCT05514249

Sponsor

Cure Rare Disease, Inc

Status

Unknown status

Last Update Posted

Sep 1, 2022Actual

Enrollment

1Estimated

Phase

Phase 1

Conditions

Duchenne Muscular Dystrophy

Interventions

CRD-TMH-001

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

Aug 2022

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Active, not recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Aug 31, 2022Actual

Primary Completion Date

Sep 2023Estimated

Completion Date

Sep 2023Estimated

First Submitted Date

Aug 11, 2022

First Submission Date that Met QC Criteria

Aug 22, 2022

First Posted Date

Aug 24, 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

Aug 31, 2022

Last Update Posted Date

Sep 1, 2022Actual

Name	Class
University of Massachusetts, Worcester	OTHER

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 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.

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

1Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Single patient	Experimental	Single dose of CRD-TMH-001 administered by IV	Drug: CRD-TMH-001

Interventions

Name	Type	Description	Arm Group Labels	Other Names
CRD-TMH-001	Drug	Participant will receive a single dose of CRD-TMH-001 administered via intravenous injection.	Single patient

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

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.

Accepts Healthy Volunteers

Sex

Male

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

28 Years

Standard Ages

Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Brenda Wong, MD	UMass Chan Medical School	Principal Investigator
Medical Affairs	Cure Rare Disease	Study Director

Locations

Facility	Status	City	State	ZIP	Country	Contacts
UMass Chan Medical School		Worcester	Massachusetts	01655	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
D020388	Muscular Dystrophy, Duchenne
D009136	Muscular Dystrophies

Ancestor Terms

ID	Term
D020966	Muscular Disorders, Atrophic
D009135	Muscular Diseases
D009140	Musculoskeletal Diseases
D009468	Neuromuscular Diseases

Browse Leaves

Not provided

Browse Branches

Not provided