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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06214507




Registration number
Help: Registration number
NCT06214507
Ethics application status
Help: Ethics application status
Date submitted
Help: Date submitted
17/11/2023
Date registered
Help: Date registered
19/01/2024
Date last updated
Help: Date last updated
19/10/2026

Titles & IDs
Public title
Danon Disease Natural History Study
Scientific title
An Observational Study of Genetic Cardiomyopathy, Danon Disease
Additional trial identifiers [1] 0 0
LAMPLIGHT-NH
Additional trial identifiers [2] 0 0
RP-NI-A501-0223
Universal Trial Number (UTN)
Trial acronym
Related trial records

Health condition
Health condition(s) studied:
Danon Disease 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of the intervention or exposure
Other interventions - No intervention

Prospective Cohort -

Retrospective Cohort -


Other interventions: No intervention
No intervention
Intervention code [1] 0 0
Other interventions
Description of the comparator or control
Control group

Outcomes
Primary outcome [1] 0 0
Left Ventricular Mass Index (LVMI) by echocardiogram
Timepoint [1] 0 0
Month 12, 24, 36
Secondary outcome [1] 0 0
Cardiac biomarkers (hsTnI)
Timepoint [1] 0 0
Month 12, 24, 36 (when available)
Secondary outcome [2] 0 0
Cardiac biomarkers (NT-proBNP, BNP)
Timepoint [2] 0 0
Month 12, 24, 36 (when available)
Secondary outcome [3] 0 0
NYHA class
Timepoint [3] 0 0
Month 12, 24, 36
Secondary outcome [4] 0 0
KCCQ-12
Timepoint [4] 0 0
Month 12, 24, 36
Secondary outcome [5] 0 0
PedsQL
Timepoint [5] 0 0
Month 12, 24, 36
Secondary outcome [6] 0 0
Event free survival with events defined as death, heart transplant, mechanical circulatory support (MCS) or heart failure hospitalization
Timepoint [6] 0 0
Month 12, 24, 36
Secondary outcome [7] 0 0
Six Minute Walk Test
Timepoint [7] 0 0
Month 12, 24, 36
Secondary outcome [8] 0 0
CBC
Timepoint [8] 0 0
At enrollment
Secondary outcome [9] 0 0
Serum Chemistry
Timepoint [9] 0 0
At enrollment
Secondary outcome [10] 0 0
ECG
Timepoint [10] 0 0
Month 12, 24, 36
Secondary outcome [11] 0 0
Serum chemistry
Timepoint [11] 0 0
At enrollment
Secondary outcome [12] 0 0
Serum chemistry
Timepoint [12] 0 0
At enrollment
Secondary outcome [13] 0 0
Serum chemistry
Timepoint [13] 0 0
At enrollment
Secondary outcome [14] 0 0
Serum chemistry
Timepoint [14] 0 0
At enrollment
Secondary outcome [15] 0 0
Serum chemistry
Timepoint [15] 0 0
At enrollment
Secondary outcome [16] 0 0
Anti-AAV9 titer
Timepoint [16] 0 0
Month 12, 24, 36

Eligibility
Key inclusion criteria
Prospective Cohort:

1. Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene by a CLIA-certified genetic testing laboratory
2. Patient or parent/legal guardian are capable and willing to provide signed informed consent
3. Age = 8 years at enrollment

Female Prospective Cohort:
4. Evidence of left ventricular hypertrophy in the 12 months prior to or at enrollment.

Retrospective (only) Cohort:
5. Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene by a CLIA-certified genetic testing laboratory
6. Patient or parent/legal guardian are capable and willing to provide signed informed consent, as required by local regulations
7. Age = 8 years at enrollment
8. Prior cardiac transplantation or prior mechanical circulatory support
9. At least 30 days of retrospective medical records available prior to cardiac transplantation or mechanical circulatory support

Female Retrospective (only) Cohort:
10. Prior evidence of left ventricular hypertrophy.

Key
Minimum age
8 Years
Maximum age
No limit
Sex
Both males and females
Healthy volunteers
No
Key exclusion criteria
All Cohorts:

1. Concurrent enrollment in any other clinical investigation involving use of an investigational agent for any condition at time of enrollment to this study that could confound interpretation of this study
2. Previous treatment with a gene therapy

Prospective Cohort:
3. Prior mechanical circulatory support at time of enrollment to this study
4. Prior cardiac transplantation at time of enrollment to this study

Female patients:
5. Age >51 years at enrollment

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
20/12/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/03/2030
Actual
Sample size
Target
60
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Monash Health - Clayton
Recruitment postcode(s) [1] 0 0
3168 - Clayton
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Michigan
Country [6] 0 0
United States of America
State/province [6] 0 0
Missouri
Country [7] 0 0
United States of America
State/province [7] 0 0
New York
Country [8] 0 0
United States of America
State/province [8] 0 0
Texas
Country [9] 0 0
Germany
State/province [9] 0 0
München
Country [10] 0 0
Israel
State/province [10] 0 0
Ramat Gan
Country [11] 0 0
Italy
State/province [11] 0 0
Florence
Country [12] 0 0
Spain
State/province [12] 0 0
Madrid
Country [13] 0 0
Spain
State/province [13] 0 0
Barcelona
Country [14] 0 0
United Kingdom
State/province [14] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Rocket Pharmaceuticals Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Clinical Information
Address 0 0
Country 0 0
Phone 0 0
646-627-0033
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided


Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT06214507