• Donate now
  • Monthly Giving
  • Support Calendar – What’s On
  • Contact us
  • About us
    • Our vision
    • What we do
    • Our Impact
    • Our team
    • Work for us
    • Volunteer
    • The DMD Registry
  • Celebrating our Action Duchenne Champions
  • Get Support
    • Recently diagnosed
    • Group Counselling Programme
    • Connect with others
      • Online support sessions
      • Support for 14-25 yrs ‘Yes I Can’
      • Support for 8-14 yrs ‘Turning Point’
    • Science on Tour 2023
    • Schools
    • Siblings
    • End of Life & Bereavement
  • International Conference
    • 2022 Recordings
  • News, Blogs & Webinars
    • News
    • Blogs
    • Webinar recordings
  • Challenge 79
  • Support Us
    • Make a Pledge
  • Shop
  •  0 items - Free
  • Menu
  • Skip to right header navigation
  • Skip to main content
  • Skip to secondary navigation
  • Skip to primary sidebar
  • Skip to footer

Before Header

  • BECOME A MEMBER
  • SHOP
  • My account
  •  0 items - Free

Action Duchenne

Header Right

  • About Us
    • Our vision
    • Our Strategy
    • What we do
    • Our Impact
    • Our team
    • Work for us
      • Volunteer for us
    • Volunteer
    • The DMD Registry
    • Action Duchenne Policies
  • Get Support
    • Mental Health Awareness Week 2025
    • Science on Tour
    • Support Calendar – What’s On
    • Support for you and your family
      • Time Out – A Space for Mums
      • Dads Against Duchenne
      • Grandparents Together
      • Open Space
      • Group Counselling Programme
    • Recently diagnosed
    • Connect with others
    • Support for 8-14 yrs ‘Turning Point’
    • Support for 14-25 yrs ‘Yes I Can’
    • Schools
    • Siblings
    • End of Life and Bereavement
  • AD Annual International Conference
    • SAVE THE DATE for the Action Duchenne Annual International Conference 2025
    • Highlights from the Annual Action Duchenne Annual International 2024
    • Annual International Conference 2023 Video Recordings
    • Annual International Conference 2022 Recordings
      • Adults with Duchenne
      • Growing up with Duchenne
      • The Duchenne Journey
      • What is new in Duchenne research?
  • News, Webinars and Blogs
    • News
    • Webinar Series 2025
      • Webinar Series 2025
      • Webinar recordings
    • Bite-Sized Duchenne Science Live
      • Facts about Duchenne muscular dystrophy
      • Signs and Symptoms of Duchenne Muscular Dystrophy
      • Diagnosis of Duchenne Muscular Dystrophy
      • Crucial Genetic Terminology
      • Genetics – Blueprint of Duchenne Muscular Dystrophy
      • How is Duchenne Muscular Dystrophy Inherited?
    • Blogs
  • Support Us
    • Help Make a Life Beyond Duchenne Possible – Every Month
    • Fundraising Events and Challenges
    • Take on a challenge for Duchenne
    • Shop
You are here: Home / News / Capricor Announces Initiation of HOPE-2 Clinical Trial Of CAP-1002 for Duchenne Muscular Dystrophy
Capricor Announces Initiation of HOPE-2 Clinical Trial Of CAP-1002 for Duchenne Muscular Dystrophy

Capricor Announces Initiation of HOPE-2 Clinical Trial Of CAP-1002 for Duchenne Muscular Dystrophy

May 1, 2018 by abzali123

Capricor Therapeutics has announced it has initiated the HOPE-2 clinical trial at UC Davis Medical Centre. The trial will test the safety and efficacy of Capricor’s novel cellular therapy, CAP-1002, in boys and young men with Duchenne muscular dystrophy, a devastating and fatal genetic disorder with limited treatment options and no cure.

Up to 84 boys and young men with Duchenne muscular dystrophy will be enrolled in HOPE-2, a Phase 2, randomised, double-blind, placebo-controlled trial that will test CAP-1002 in participants with advanced stages of Duchenne muscular dystrophy. The medical centre in Sacramento is the first site in the nation to begin enrolling and treating participants. Approximately 12-15 investigative sites are expected to participate in the trial.

“We are very pleased to begin this important clinical trial of CAP-1002,” said Craig McDonald, M.D., the national principal investigator for the HOPE-2 clinical trial and UC Davis professor and chair of its Department of Physical Medicine and Rehabilitation. “The HOPE-2 trial will study whether CAP-1002 can maintain or improve cardiac and skeletal muscle function. Because many of the participants are non-ambulatory, the study will focus primarily on the impact on arm mobility.”

Linda Marbán, Ph.D., Capricor president and chief executive officer, said CAP-1002 is one of the very few clinical initiatives to focus on helping boys and young men whose ability to walk has been seriously impaired by the loss of muscle function that occurs as Duchenne muscular dystrophy progresses.

“We are thrilled to begin enrolling participants in HOPE-2 because we have seen the potential for improvements in muscle function in both pre-clinical studies and in our earlier HOPE-Duchenne trial,” she said. “We have also been granted the RMAT and orphan disease designations by the U.S. Food and Drug Administration (FDA). These designations will enable us to work closely with the FDA in finalizing the regulatory approval pathway for CAP-1002 and to receive expedited FDA reviews. We are hopeful that HOPE-2 may potentially be a registration trial.”

Dr. Marbán said CAP-1002 could be an important tool in the toolbox to treat Duchenne muscular dystrophy.

“While gene and other therapies have the potential to restore dystrophin expression and sustain muscle function, there will still be significant inflammation and fibrosis which can offset the restorative effects…CAP-1002 may work synergistically with the emerging disease-modifying therapies to control those additional pathological aspects of Duchenne muscular dystrophy because CAP-1002’s primary mechanism of action is immunomodulatory, meaning it can help balance inflammation in this chronic inflammatory disease.”

Capricor’s previous clinical trial, the HOPE-Duchenne trial, evaluated the safety and efficacy of a single dose of CAP-1002 in boys and young men with heart disease related to Duchenne muscular dystrophy. It found CAP-1002 was generally safe, well tolerated and demonstrated significant and sustained signals of improvement in cardiac and skeletal muscle function.

Participants in the HOPE-2 trial will be randomized to receive either placebo or CAP-1002, delivered intravenously every three months for a total of four administrations. Participants will be followed for a one-year period following randomization. An open label extension is planned if trial evidence suggests an appropriate risk/benefit profile of CAP-1002. For more information, please visit www.HOPE2Trial.com.

About CAP-1002

CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a unique population of cells that contains cardiac progenitor cells. CAP-1002 has been shown to exert potent immunomodulatory activity and stimulate cellular regeneration. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to approximately 140 human subjects across several clinical trials.

Share this:

Category: NewsTag: untagged

Previous Post: « Thank You – An announcement on the Early Access to Medicines Scheme for Raxone
Next Post: Action Duchenne co-funding new research bringing new hope for Duchenne muscular dystrophy »

Primary Sidebar

From our community

Mental Health Awareness Week: Alex’s Journal

Written By Alex Berbank 15 Minutes a Day I’ve really enjoyed getting into the frame of mind to look at my mental health. Before this week I wasn’t quite sure what to expect. My ideas of what looking at my own mental health would look like weren’t accurate. I thought there would be more softly …

Mental Health Awareness Week: Alex’s Journal

Mental Health Awareness Week Journal, Written by Alex Berbank Watch Alex’s Vlog for Mental Health Awareness Week Thursday’s Update: Meditation and Mindfulness So, to this point this week has been great. I’ve actually taken to the focus on my mental health and self improvement thing quite well and I hope I’ll keep these new ideas …

Mental Health Awareness Week – Alex’s Blog

Written by Alex Berbank Wednesday: Getting Into The Swing of Things The changes yesterday were about removing phones from certain times in the house, namely when eating, before bed and when me and my Fiancee are in the house together. The idea was that we would be more connected, talk more and just be a …

Footer

Action Duchenne
Wellesley House
Duke of Wellington Avenue Royal Arsenal
London
SE18 6SS

07535 498 506
info@actionduchenne.org 

 

 

 

 

 

 

 

 

Subscribe to our mailing list

Do you consent to receiving regular email updates? *
Email Format
  • Accessibility
  • Privacy Policy
  • Terms & Conditions

© Action Duchenne - Registered Charity No 1101971 - Scottish Charity No SC043852

Like most websites we use cookies to deliver a personalised service. To use the website as intended please accept cookies.
Privacy & Cookies Policy

Privacy Overview

This website uses cookies to improve your experience while you navigate through the website. Out of these, the cookies that are categorized as necessary are stored on your browser as they are essential for the working of basic functionalities of the website. We also use third-party cookies that help us analyze and understand how you use this website. These cookies will be stored in your browser only with your consent. You also have the option to opt-out of these cookies. But opting out of some of these cookies may affect your browsing experience.
Necessary
Always Enabled
Necessary cookies are absolutely essential for the website to function properly. This category only includes cookies that ensures basic functionalities and security features of the website. These cookies do not store any personal information.
Non-necessary
Any cookies that may not be particularly necessary for the website to function and is used specifically to collect user personal data via analytics, ads, other embedded contents are termed as non-necessary cookies. It is mandatory to procure user consent prior to running these cookies on your website.
SAVE & ACCEPT