DystrophyPhase 1NCT02208713
Intramuscular Transplantation of Muscle Derived Stem Cell and Adipose Derived Mesenchymal Stem Cells in Patients With Facioscapulohumeral Dystrophy (FSHD)
A Phase 1 study testing Intramuscular injection in people with Dystrophy, run by Royan Institute at 1 site in Iran.
Trial summary
Facioscapulohumeral Dystrophy is a Autosomal dominant inherited dystrophy with the prevalence of 1:20,000 and it is the third most common dystrophy after the dystrophinopathies and myotonic dystrophy. Read more about this study
- Who is it for?
- Ages 18 to 50 years
- Conditions the study is for
- Dystrophy
- What is being tested?
- Intramuscular injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 1 siteIran
- Study dates
- May 2014to Dec 2017
- Phase
- Phase 1
- 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 18 to 50 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Age: 18-50
- Both gender
- Weakness of face muscle
- FSHD phenotype positive
- Genetic Test confirmation for FSHD
Who cannot join
- Co - morbidities: Heart \& respiratory diseases,malignancy, rheumatologic disorders
- Progressive form of disease
- Not able to sign the consent
Trial locations and contacts
1 site in 1 country.
Iran1 site, 1 recruiting
- Recruiting
Royan Institute
Tehran
Nasser Aghdami, MD,PhD, (+98)212356000 ext. 516, nasser.aghdami@royaninstitute.org
Leila Arab, MD, (+98)23562000 ext. 414, Leara91@gmail.com
About this study
Official title: Intramuscular Transplantation of Autologous Muscle Derived Stem Cell(MDSC) and Adipose Derived Mesenchymal Stem Cells (AD-MSC) in Patients With Facioscapulohumeral Dystrophy (FSHD), Phase I Clinical Trial
Facioscapulohumeral Dystrophy is a Autosomal dominant inherited dystrophy with the prevalence of 1:20,000 and it is the third most common dystrophy after the dystrophinopathies and myotonic dystrophy. The symptoms including: Pain, facial weakness, scapular fixator, humeral, truncal, pelvic girdle and lower-extremity weakness, High frequency hearing loss, Retinal telangiectasia . The existing treatments are not effective so, cell therapy is a new hope to improve patients' quality of life. Therefore, We design this clinical trial to evaluate the safety and feasibility of stem cell transplantation.
Read the detailed description
In this study, we select 15 patients with FSHD based on eligibility criteria. All the patients underwent physical examination, laboratory evaluations, EMG-NCV, muscle sonography and muscle MRI. Then, a sample of patient's muscle is taken from Biceps Femoralis to isolate and culture of MDSC. The AD-MSC is prepared from Royan Adipose Tissue Bank. The patient is admitted in general hospital, and the cell suspensions are injected into biceps, triceps and trapezoids muscles by neurologists. After transplantation, the patients will be under observation for 5 hours and will be discharged if no side effect happen. All the patients will be followed at 1,2,4,6 and 12 months after cell injection.
- Conditions
- Dystrophy
- Being tested
- Intramuscular injection
- Keywords
- FSHD muscle derived stem cell adipose derived mesenchymal stem cell intramuscular injection
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Intramuscular injectionBiological
Also called Intramuscular cell transplantation
Intramuscular injection of stem cells in patients with FSHD.
| Study group | Role | What they receive |
|---|---|---|
| Stem cell recipient | Experimental | The patients with FSHD who underwent muscle derived stem cell and Adipose derived mesenchymal stem cell with intramuscular injection. Receives: Biological: Intramuscular injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Myalsia
Evaluation the presence of myalsia 1month after cell injection.
Measured: 1month
Mass formation
Evaluation the probability of mass formation 6 months after cell injection.
Measured: 6 months
Secondary outcomes (3)
Hematoma
Evaluation the presence of hematoma 1 month after injection.
Measured: 1 month
Muscle bulk
Evaluation the muscle bulk changes with sonography and MRI 6 months after transplantation.
Measured: 6 months
CPK
Evaluation the decrease of CPK 1 month after cell transplantation.
Measured: 1month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Aug 2014 (estimated)
- Study startMay 2014
- Primary completionNov 2017 (estimated)
- Study completionDec 2017 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Royan Institute
- Sponsor type
- Other government
- Responsible party
- Sponsor
- Investigators
- Hamid Gourabi, PhD, Study chair, Head of Royan InstituteNasser Aghdami, MD,PhD, Study director, Head of Department of Regenerative Medicine & Cell therapy center of Royan InstituteMahdi Vahid Dastjerdi, MD, Study director, Scientific Board of BouAli Hospital, Azad UniversityLeila Arab, MD, Principal investigator, Department of Regenerative Medicine & cell therapy of Royan Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02208713
- Sponsor's protocol ID
- Royan-Nerve-007
- Status last verified
- Apr 2017
- First submitted
- 4 Aug 2014
- Last updated
- 27 Apr 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 27 Apr 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intramuscular Transplantation of Autologous Muscle Derived Stem Cell(MDSC) and Adipose Derived Mesenchymal Stem Cells (AD-MSC) in Patients With Facioscapulohumeral Dystrophy (FSHD), Phase I Clinical Trial
- ClinicalTrials.gov ID
- NCT02208713
- Acronym / sponsor ID
- Royan-Nerve-007
- Conditions
- Dystrophy
- Keywords
- FSHD muscle derived stem cell adipose derived mesenchymal stem cell intramuscular injection
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 21 participants (estimated)
- Lead sponsor
- Royan InstituteOther government
- Study start
- May 2014 (actual)
- Primary completion
- Nov 2017 (estimated)
- Study completion
- Dec 2017 (estimated)
- First posted
- 5 Aug 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Facioscapulohumeral Dystrophy is a Autosomal dominant inherited dystrophy with the prevalence of 1:20,000 and it is the third most common dystrophy after the dystrophinopathies and myotonic dystrophy. The symptoms including: Pain, facial weakness, scapular fixator, humeral, truncal, pelvic girdle and lower-extremity weakness, High frequency hearing loss, Retinal telangiectasia . The existing treatments are not effective so, cell therapy is a new hope to improve patients' quality of life. Therefore, We design this clinical trial to evaluate the safety and feasibility of stem cell transplantation.
Detailed description
In this study, we select 15 patients with FSHD based on eligibility criteria. All the patients underwent physical examination, laboratory evaluations, EMG-NCV, muscle sonography and muscle MRI. Then, a sample of patient's muscle is taken from Biceps Femoralis to isolate and culture of MDSC. The AD-MSC is prepared from Royan Adipose Tissue Bank. The patient is admitted in general hospital, and the cell suspensions are injected into biceps, triceps and trapezoids muscles by neurologists. After transplantation, the patients will be under observation for 5 hours and will be discharged if no side effect happen. All the patients will be followed at 1,2,4,6 and 12 months after cell injection.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Stem cell recipient | Experimental | Biological: Intramuscular injectionThe patients with FSHD who underwent muscle derived stem cell and Adipose derived mesenchymal stem cell with intramuscular injection. |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- Age: 18-50
- Both gender
- Weakness of face muscle
- FSHD phenotype positive
- Genetic Test confirmation for FSHD
Key exclusion criteria (3 listed)
- Co - morbidities: Heart \& respiratory diseases,malignancy, rheumatologic disorders
- Progressive form of disease
- Not able to sign the consent
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Intramuscular injectionAlso: Intramuscular cell transplantationIntramuscular injection of stem cells in patients with FSHD. | Biological | Stem cell recipient |
Primary outcome
Myalsia
Time frame: 1month
Evaluation the presence of myalsia 1month after cell injection.
Mass formation
Time frame: 6 months
Evaluation the probability of mass formation 6 months after cell injection.
3 secondary outcomes
Hematoma
Time frame: 1 month
Muscle bulk
Time frame: 6 months
CPK
Time frame: 1month
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Apr 2017.
- Related InfoLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Hamid Gourabi, PhD study chair
Head of Royan Institute
Nasser Aghdami, MD,PhD study director
Head of Department of Regenerative Medicine & Cell therapy center of Royan Institute
Mahdi Vahid Dastjerdi, MD study director
Scientific Board of BouAli Hospital, Azad University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Intramuscular injection in people with Dystrophy, run by Royan Institute at 1 site in Iran.
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 5 inclusion and 3 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 27 Apr 2017. 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 Iran. 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 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.
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 Nasser Aghdami, MD,PhD ((+98)23562000 ext. 516, nasser.aghdami@royaninstitute.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Myalsia (time frame: 1month). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 27 Apr 2017.