|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
ClinicalTrials.gov |
|
Last refreshed on:
|
12 December 2020 |
|
Main ID: |
NCT03406780 |
|
Date of registration:
|
15/01/2018 |
|
Prospective Registration:
|
Yes |
|
Primary sponsor: |
|
|
Public title:
|
A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
HOPE-2 |
|
Scientific title:
|
A Phase 2, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Safety and Efficacy of Intravenous Delivery of Allogeneic Cardiosphere-Derived Cells in Subjects With Duchenne Muscular Dystrophy |
|
Date of first enrolment:
|
March 4, 2018 |
|
Target sample size:
|
18 |
|
Recruitment status: |
Completed |
|
URL:
|
https://clinicaltrials.gov/show/NCT03406780 |
|
Study type:
|
Interventional |
|
Study design:
|
Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
|
|
Phase:
|
Phase 2
|
|
|
Countries of recruitment
|
|
United States
| | | | | | | |
|
Contacts
|
|
Name:
|
Craig McDonald, MD |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
University of California, Davis |
| | |
|
Key inclusion & exclusion criteria
|
Inclusion Criteria:
1. Genetically confirmed DMD
2. Reduced upper arm strength as measured by the Performance of Upper Limb
3. Reduced ability to walk/run (if ambulatory)
4. Treatment with systemic glucocorticoids for at least 12 months and at a stable dose at
least 6 months prior to study participation, except for weight-based or
toxicity-related adjustments
5. Current and up-to-date immunizations
Exclusion Criteria:
1. Left ventricular ejection fraction < 35%
2. BMI > 45
3. Ambulant if = 18 years of age
4. Exon 44 skip-amenable mutation(s) in the DMD gene
5. Deletion mutation(s) encompassing exons 3-7 of the DMD gene
6. Percent-predicted forced vital capacity (FVC) < 35%
7. Chronic respiratory disease not related to DMD (for example, asthma, bronchitis, and
tuberculosis)
8. History of diabetes requiring treatment with metformin or insulin within 3 months
prior to randomization
9. Treatment with an FDA-approved exon skipping therapy for the treatment of DMD if on a
stable dose for less than 24 months prior to randomization
10. Treatment with human growth hormone (HGH) within 3 months prior to randomization,
unless on a stable dose for at least 24 months prior to randomization
11. Treatment with idebenone within 3 months prior to randomization
12. Treatment with a cell therapy product within 12 months prior to randomization
13. Treatment with an investigational product within 6 months prior to randomization
Age minimum:
10 Years
Age maximum:
N/A
Gender:
Male
|
|
Health Condition(s) or Problem(s) studied
|
|
Nervous System Diseases
|
|
Genetic Diseases, Inborn
|
|
Muscular Disorders, Atrophic
|
|
Neuromuscular Diseases
|
|
Muscular Dystrophies
|
|
Muscular Dystrophy, Duchenne
|
|
Genetic Diseases, X-Linked
|
|
Muscular Diseases
|
|
Intervention(s)
|
|
Biological: CAP-1002
|
|
Drug: Placebo
|
|
Primary Outcome(s)
|
|
Change in the mid-level (elbow) dimension of the Performance of the Upper Limb (PUL)
[Time Frame: Month 12]
|
|
Secondary Outcome(s)
|
|
Change in the mid-level (elbow) dimension of the PUL
[Time Frame: Months 3, 6, and 9]
|
|
Change in regional systolic left ventricular wall thickening as assessed by cardiac MRI
[Time Frame: Months 6 and 12]
|
|
Secondary ID(s)
|
|
CAP-1002-DMD-02
|
|
Source(s) of Monetary Support
|
|
Please refer to primary and secondary sponsors
|
|
Results
|
|
Results available:
|
|
|
Date Posted:
|
|
|
Date Completed:
|
|
|
URL:
|
|
|
|