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Duchenne Muscular DystrophyPhase 1/2NCT03067831

Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy

A Phase 1/2 study testing Stem Cells in people with Duchenne Muscular Dystrophy, run by Stem Cells Arabia at 1 site in Jordan.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial summary

This study is single arm, single center trial to study the safety and efficacy of bone marrow-derived autologous specific populations of stem cells and mesenchymal stem cells for the treatment of Duchenne Muscular Dystrophy (DMD). Read more about this study

Who is it for?
Ages 4 to 25 years
Conditions the study is for
Duchenne Muscular Dystrophy
What is being tested?
Stem Cells
Could I receive a placebo?
No, everyone receives an active treatment
How many people
20 participants
Where
1 siteJordan
Study dates
Sep 2015to Dec 2021
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 4 to 25 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Age group of 3-25 years
  • Duchenne muscular dystrophy diagnosed on the basis of clinical presentation

Who cannot join

  • Respiratory Distress
  • Acute infections such as Human Immunodeficient Virus/Hepatitis B Virus/Hepatitis C Virus malignancies
  • Acute medical conditions such as respiratory infections, fever, hemoglobin less than 8 bleeding tendency, bone marrow disorder, left ventricular ejection fraction < 30%
  • Pregnancy or breastfeeding

Trial locations and contacts

1 site in 1 country.

Adeeb AlZoubi, Ph.D. contact

00962795337575

adeebalzoubi@stemcellsarabia.net

Jordan1 site, 1 recruiting

  • Stem Cells of Arabia

    Amman, 11953

    Adeeb AlZoubi, PhD, adeebalzoubi@stemcellsarabia.net

    Adeeb AlZoubi, PhD

    Recruiting

Other trials in Jordan

About this study

Official title: Safety and Efficacy of Purified Autologous Bone Marrow-Derived Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy.

This study is single arm, single center trial to study the safety and efficacy of bone marrow-derived autologous specific populations of stem cells and mesenchymal stem cells for the treatment of Duchenne Muscular Dystrophy (DMD).

Read the detailed description

Duchenne muscular dystrophy (DMD) is a genetically determined X-linked disease. The manifestation of muscle weakness typically starts around the age of 4-5 in males and deteriorates fast. Typically muscle loss occurs first in the upper legs and pelvis followed by muscles of the upper arms. It is caused by a mutation in the gene for the protein dystrophin. Dystrophin is crucial to maintain the muscle fiber cell membrane.

Currently, there is no cure for muscular dystrophy. Corrective surgery, braces, and physical therapy may help with some of the symptoms. Assisted ventilation might be required in patients with weakness of breathing muscles. Medications prescribed include steroids to slow muscle degeneration, anti-convulsants to control seizures and muscle activity, and immunosuppressants to delay damage to muscle cells.

For decades, research has been conducted to find an effective therapy for Duchenne muscular dystrophy (DMD). Stem cell based therapy is considered to be one of the most promising methods for treating muscular dystrophies.

Stem cell based therapies for the treatment of Duchenne muscular dystrophy (DMD) can proceed via two strategies. The first is autologous stem cell transfer involving cells from a patient with Duchenne muscular dystrophy (DMD) that are genetically altered in vitro to restore dystrophin expression and are subsequently re-implanted. The second is allogenic stem cell transfer, containing cells from an individual with functional dystrophin, which are transplanted into a dystrophic patient.

Herein, the investigators describe a method for the treatment of Duchenne muscular dystrophy (DMD) using autologous bone marrow derived specific populations of stem cells and mesenchymal stem cells transplanted in patients with Duchenne muscular dystrophy (DMD).

Being tested
Stem Cells
Keywords
stem cells
Medical subject headings
Muscular Dystrophy, Duchenne

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Stem CellsBiological

    Transplantation of purified autologous bone marrow-derived stem cells.

Study groups in NCT03067831
Study groupRoleWhat they receive
Stem CellsExperimentalTransplantation of purified autologous bone marrow-derived stem cells.
Receives: Biological: Stem Cells

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Improvement in muscle strength using Kinetics Muscle testing or MMT

    Measured: 12 months

Secondary outcomes (1)

  1. Brooke and Vignos Scale

    Measured: 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted1 Mar 2017
  2. Study startSep 2015
  3. Primary completionNov 2021 (estimated)
  4. Study completionDec 2021 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
20 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03067831
Sponsor's protocol ID
SCA-DMD1
Status last verified
Mar 2020
First submitted
25 Feb 2017
Last updated
17 Mar 2020
Results posted
No
Data monitoring committee
Yes
Individual data sharing
Undecided

Publications

  • Sienkiewicz D, Kulak W, Okurowska-Zawada B, Paszko-Patej G, Kawnik K. Duchenne muscular dystrophy: current cell therapies. Ther Adv Neurol Disord. 2015 Jul;8(4):166-77. doi: 10.1177/1756285615586123.

    PubMed 26136844

  • Carletti B, Piemonte F, Rossi F. Neuroprotection: the emerging concept of restorative neural stem cell biology for the treatment of neurodegenerative diseases. Curr Neuropharmacol. 2011 Jun;9(2):313-7. doi: 10.2174/157015911795596603.

    PubMed 22131940

  • Farini A, Villa C, Manescu A, Fiori F, Giuliani A, Razini P, Sitzia C, Del Fraro G, Belicchi M, Meregalli M, Rustichelli F, Torrente Y. Novel insight into stem cell trafficking in dystrophic muscles. Int J Nanomedicine. 2012;7:3059-67. doi: 10.2147/IJN.S30595. Epub 2012 Jun 20.

    PubMed 22787400

  • Maclean S, Khan WS, Malik AA, Anand S, Snow M. The potential of stem cells in the treatment of skeletal muscle injury and disease. Stem Cells Int. 2012;2012:282348. doi: 10.1155/2012/282348. Epub 2011 Dec 19.

    PubMed 22220178

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Stem Cells in people with Duchenne Muscular Dystrophy, run by Stem Cells Arabia at 1 site in Jordan.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 17 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Jordan. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

Contact Adeeb AlZoubi, Ph.D. (00962795337575, adeebalzoubi@stemcellsarabia.net), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Improvement in muscle strength using Kinetics Muscle testing or MMT (time frame: 12 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03067831, which also lists 4 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Mar 2020.

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