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Cell Therapy
Cell Therapy for Duchenne Muscular Dystrophy (HOPE-3 Trial)
Phase 3
Recruiting
Led By Craig McDonald, MD
Research Sponsored by Capricor Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Adequate venous access for parenteral IP infusions and routine blood collection.
Performance of the Upper Limb test (PUL) entry item scores 2-6 and total PUL score less than or equal to 40. For Cohort A only: enrollment of patients with specific characteristics will be capped at no more than 10% of the total study population.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at months 1, 3, 6, 9, 12
Awards & highlights
HOPE-3 Trial Summary
This trial is testing a cell therapy called CAP-1002 for Duchenne muscular dystrophy. Boys and young men with the disease will be given the therapy or a placebo every 3 months for a year, then will have the option of receiving the therapy for another year.
Who is the study for?
This trial is for boys and young men at least 10 years old with genetically confirmed Duchenne muscular dystrophy (DMD). They must have been on stable glucocorticoids for a year, have up-to-date immunizations, and adequate venous access. Ambulatory participants should take more than 10 seconds to walk/run 10 meters; non-ambulatory ones should have lost independent walking between ages 10-18.Check my eligibility
What is being tested?
The HOPE-3 trial tests CAP-1002 cell therapy's effectiveness in improving muscle function in DMD patients. Participants are randomly assigned to receive either CAP-1002 or placebo every three months over a year, followed by an open-label extension where all get CAP-1002.See study design
What are the potential side effects?
Potential side effects of CAP-1002 may include reactions related to the infusion process, immune system responses due to the cells being introduced into the body, and possible discomfort at injection sites.
HOPE-3 Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have good veins for IV treatments and blood tests.
Select...
My upper limb function scores are within the required range for the study.
Select...
I have been diagnosed with Duchenne Muscular Dystrophy.
Select...
It takes me more than 10 seconds to walk or run 10 meters.
Select...
My DMD diagnosis was confirmed by genetic testing in a certified lab.
HOPE-3 Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ at months 1, 3, 6, 9, 12
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at months 1, 3, 6, 9, 12
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change in the upper limb function
Secondary outcome measures
Change in cardiac muscle function and structure by assessment of left ventricular ejection fraction
Change in cardiac muscle function and structure by assessment of left ventricular end-diastolic volume
Change in cardiac muscle function and structure by assessment of left ventricular end-systolic volume
+7 moreHOPE-3 Trial Design
2Treatment groups
Experimental Treatment
Placebo Group
Group I: CAP-1002Experimental Treatment1 Intervention
Cohort A: Approximatetly 29 subjects will receive CAP-1002A active treatment consisting of 150 million cardiosphere-derived cells (CDCs) via intravenous infusion every 3 months
Cohort B: Approximately 22 participants will receive CAP-1002B active treatment consisting of 150 million cardiosphere-derived cells (CDCs) via intravenous infusion every 3 months
Group II: PlaceboPlacebo Group1 Intervention
Cohort A: Approximately 29 subjects will receive a Placebo solution via intravenous infusion every 3 months
Cohort B: Approximately 22 participants will receive a Placebo solution via intravenous infusion every 3 months
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
CAP-1002
2018
Completed Phase 2
~90
Find a Location
Who is running the clinical trial?
Capricor Inc.Lead Sponsor
11 Previous Clinical Trials
386 Total Patients Enrolled
Craig McDonald, MDPrincipal InvestigatorUniversity of California, Davis
4 Previous Clinical Trials
647 Total Patients Enrolled
Mark AwadallaStudy DirectorCapricor Inc.
3 Previous Clinical Trials
73 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have good veins for IV treatments and blood tests.I am using effective birth control methods if I'm sexually active.It takes me more than 10 seconds to walk or run 10 meters.I lost the ability to walk on my own between ages 10 and 18.I cannot fully straighten my arms due to stiffness.I am a male at least 10 years old and can consent to the trial, or have a guardian's consent if under 18.My upper limb function scores are within the required range for the study.I have been on a stable dose of steroids for at least 6 months.I have had a respiratory illness in the last 30 days.I do not have the specific characteristics that would exclude me from Cohort B.I haven't started new growth hormone treatment in the last 3 months.I am planning to have leg surgery in the next 6 months and I can walk.I started using a nighttime breathing aid less than 30 days ago.I have not had cell therapy in the last 12 months.I have been diagnosed with Duchenne Muscular Dystrophy.It takes me more than 10 seconds to walk or run 10 meters.I am getting my treatment at a specialized center for Duchenne Muscular Dystrophy.I have a chronic lung condition not related to muscular dystrophy that needs treatment.I started treatment with specific medications recently.I am expecting to have chest or spine surgery within the next 6 months.My DMD diagnosis was confirmed by genetic testing in a certified lab.
Research Study Groups:
This trial has the following groups:- Group 1: CAP-1002
- Group 2: Placebo
Awards:
This trial has 1 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are there any know adverse effects of CAP-1002?
"CAP-1002's safety is well supported by clinical data, and it thus received a score of 3."
Answered by AI
How many humans are enrolled in this experiment?
"Yes, as of October 31st, 2022, this study is still recruiting patients according to the information provided on clinicaltrials.gov. This trial was first posted on June 22th, 2020 and is looking for a total of 68 participants from 8 different sites."
Answered by AI
Could you please summarize the findings of other research projects on CAP-1002?
"At the moment, there are 3 clinical trials underway that are studying CAP-1002. One of these trials is in Phase 3. Most of the clinical trials for CAP-1002 are located in Los Angeles, but there are a total of 14 different locations running these trials."
Answered by AI
Who else is applying?
What portion of applicants met pre-screening criteria?
Did not meet criteria
How many prior treatments have patients received?
0
Why did patients apply to this trial?
My son DMD treatment.
PatientReceived 1 prior treatment
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