Nervous System Diseases Clinical Trial
— HOPE-2Official 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
Verified date | June 2020 |
Source | Capricor Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
HOPE-2 is a double-blind clinical trial evaluating the safety and efficacy of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD). Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during a 12-month period.
Status | Completed |
Enrollment | 18 |
Est. completion date | March 10, 2020 |
Est. primary completion date | March 10, 2020 |
Accepts healthy volunteers | No |
Gender | Male |
Age group | 10 Years and older |
Eligibility |
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 |
Country | Name | City | State |
---|---|---|---|
United States | Children's Hospital Colorado | Aurora | Colorado |
United States | Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio |
United States | Children's Hospital Wisconsin | Milwaukee | Wisconsin |
United States | Nemours Children's Hospital | Orlando | Florida |
United States | University of California, Davis | Sacramento | California |
United States | Washington University | Saint Louis | Missouri |
United States | University of Utah | Salt Lake City | Utah |
Lead Sponsor | Collaborator |
---|---|
Capricor Inc. |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Change in the mid-level (elbow) dimension of the Performance of the Upper Limb (PUL) | The PUL includes functional tasks that relate to activities of daily living that are very important for quality of life. The PUL has been validated for the assessment of upper limb motor function in individuals with DMD. | Month 12 | |
Secondary | Change in the mid-level (elbow) dimension of the PUL | The PUL includes functional tasks that relate to activities of daily living that are very important for quality of life. The PUL has been validated for the assessment of upper limb motor function in individuals with DMD. | Months 3, 6, and 9 | |
Secondary | Change in regional systolic left ventricular wall thickening as assessed by cardiac MRI | Systolic thickening is thought to be a principal mechanism of cardiac output generation in people with DMD. | Months 6 and 12 |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT05086809 -
Investigation of an Updated Bone-anchored Sound Processor
|
N/A | |
Completed |
NCT05080777 -
Pilot Pragmatic Clinical Trial to Embed Tele-Savvy Into Health Care Systems
|
N/A | |
Recruiting |
NCT03222375 -
SQUEDâ„¢ Series 28.1 Home-use and Treatment of Autowave Reverberator of Autism
|
N/A | |
Completed |
NCT03295786 -
Clinical Study to Test the Safety of CDNF by Brain Infusion in Patients With Parkinson's Disease
|
Phase 1/Phase 2 | |
Completed |
NCT01915368 -
Determining Optimal Post-Stroke Exercise (DOSE)
|
N/A | |
Completed |
NCT01922258 -
Safety and Tolerability Study of Flexible Dosing of Brexpiprazole in the Treatment of Subjects With Agitation Associated With Dementia of the Alzheimer's Type
|
Phase 3 | |
Terminated |
NCT03270189 -
Effect of the Visual Information Change in Functional Dystonia
|
N/A | |
Active, not recruiting |
NCT03911388 -
HSV G207 in Children With Recurrent or Refractory Cerebellar Brain Tumors
|
Phase 1 | |
Completed |
NCT02798406 -
Combination Adenovirus + Pembrolizumab to Trigger Immune Virus Effects
|
Phase 2 | |
Suspended |
NCT04912115 -
Randomized, Double-Blind, Active Placebo-Controlled Study of Ketamine to Treat Levodopa-Induced Dyskinesia
|
Phase 2 | |
Completed |
NCT03994822 -
pRESET for Occlusive Stroke Treatment
|
N/A | |
Completed |
NCT03336645 -
Open-label Study of Midazolam Hydrochloride Oromucosal Solution (MHOS/SHP615) in Children With Status Epilepticus (Convulsive) in a Healthcare Setting in Japan
|
Phase 3 | |
Completed |
NCT04283253 -
Predictors of Response in Chronic Stroke
|
N/A | |
Not yet recruiting |
NCT06048523 -
Prospective Cohort Study of Neurogenetic Diseases
|
N/A | |
Completed |
NCT02684825 -
Detection of Silent Atrial Fibrillation aFter Ischemic StrOke
|
N/A | |
Active, not recruiting |
NCT02284126 -
Topical Vancomycin for Neurosurgery Wound Prophylaxis
|
Phase 3 | |
Completed |
NCT05815836 -
Precision Medicine in Stroke
|
||
Completed |
NCT00739518 -
Refinement and Assessment of New Magnetic Resonance Imaging Technologies for Neurological Exams
|
N/A | |
Recruiting |
NCT03678194 -
Treating Depression on a Day-to-day Basis: Development of a Tool for Physicians Based on a Smartphone Application
|
N/A | |
Recruiting |
NCT04698421 -
Collection of Biological Samples From Patients With Rare Neurological Diseases
|