Limb Girdle Muscular DystrophyPhase 1NCT02050776
Stem Cell Therapy in Limb Girdle Muscular Dystrophy
A Phase 1 study testing Autologous bone marrow mononuclear cell transplantation in people with Limb Girdle Muscular Dystrophy, run by Neurogen Brain and Spine Institute at 1 site in India.
Trial summary
The purpose of this study was to study the effect of stem cell therapy on the course of the disease in patients with Limb Girdle Muscular Dystrophy. Read more about this study
- Who is it for?
- Ages 15 to 60 years
- Conditions the study is for
- Limb Girdle Muscular Dystrophy
- What is being tested?
- Autologous bone marrow mononuclear cell transplantation
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteIndia
- Study dates
- Dec 2008to Dec 2013
- 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 15 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- males and females
- age group of 15 years and above
- limb girdle muscular dystrophy diagnosed on the basis of clinical presentation
- Electromyographic and Nerve Conduction velocity findings
Who cannot join
- presence of respiratory distress
- presence of acute infections such as Human Immunodeficient Virus/Hepatitis B Virus/Hepatitis C Virus
- malignancies
- acute medical conditions such as respiratory infection, 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.
About this study
Official title: The Role of Cell Therapy in Modifying the Course of Limb Girdle Muscular Dystrophy- A Longitudinal 5-year Study
The purpose of this study was to study the effect of stem cell therapy on the course of the disease in patients with Limb Girdle Muscular Dystrophy.
- Conditions
- Limb Girdle Muscular Dystrophy
- Being tested
- Autologous bone marrow mononuclear cell transplantation
- Medical subject headings
- Muscular Dystrophies, Limb-Girdle
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous bone marrow mononuclear cell transplantationBiological
bone marrow derived mononuclear cells are administered intrathecally and intramuscularly in limb girdle muscular dystrophy patients
| Study group | Role | What they receive |
|---|---|---|
| Stem Cells | Experimental | autologous bone marrow mononuclear cell transplantation Receives: Biological: Autologous bone marrow mononuclear cell transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Functional Independence Measure
Measured: 6 months
Secondary outcomes (1)
Manual Muscle Testing
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jan 2014 (estimated)
- Study startDec 2008
- Primary completionMar 2013
- Study completionDec 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Limb Girdle Muscular 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
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Neurogen Brain and Spine Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Alok K Sharma, M.S., M.Ch, Principal investigator, Neurogen Brain and Spine Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02050776
- Sponsor's protocol ID
- NGBSI-07
- Status last verified
- Oct 2018
- First submitted
- 29 Jan 2014
- Last updated
- 25 Oct 2018
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Sharma A, Sane H, Badhe P, Gokulchandran N, Kulkarni P, Lohiya M, Biju H, Jacob VC. A clinical study shows safety and efficacy of autologous bone marrow mononuclear cell therapy to improve quality of life in muscular dystrophy patients. Cell Transplant. 2013;22 Suppl 1:S127-38. doi: 10.3727/096368913X672136. Epub 2013 Sep 10.
PubMed 24070109
Study at a glance
From the registry record, last updated by the sponsor on 25 Oct 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Role of Cell Therapy in Modifying the Course of Limb Girdle Muscular Dystrophy- A Longitudinal 5-year Study
- ClinicalTrials.gov ID
- NCT02050776
- Acronym / sponsor ID
- NGBSI-07
- Conditions
- Limb Girdle Muscular Dystrophy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Neurogen Brain and Spine InstituteUniversity, hospital or other
- Study start
- Dec 2008
- Primary completion
- Mar 2013 (actual)
- Study completion
- Dec 2013 (actual)
- First posted
- 31 Jan 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.
The purpose of this study was to study the effect of stem cell therapy on the course of the disease in patients with Limb Girdle Muscular Dystrophy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Stem Cells | Experimental | Biological: Autologous bone marrow mononuclear cell transplantationautologous bone marrow mononuclear cell transplantation |
Who may qualify
- Ages
- 15 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- males and females
- age group of 15 years and above
- limb girdle muscular dystrophy diagnosed on the basis of clinical presentation
- Electromyographic and Nerve Conduction velocity findings
Key exclusion criteria (5 listed)
- presence of respiratory distress
- presence of acute infections such as Human Immunodeficient Virus/Hepatitis B Virus/Hepatitis C Virus
- malignancies
- acute medical conditions such as respiratory infection, fever, hemoglobin less than 8, bleeding tendency, bone marrow disorder, left ventricular ejection fraction < 30%
- pregnancy or breastfeeding
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 |
|---|---|---|
| Autologous bone marrow mononuclear cell transplantationbone marrow derived mononuclear cells are administered intrathecally and intramuscularly in limb girdle muscular dystrophy patients | Biological | Stem Cells |
Primary outcome
Functional Independence Measure
Time frame: 6 months
1 secondary outcome
Manual Muscle Testing
Time frame: 6 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Oct 2018.
- Background: PubMed 24070109Sharma A, Sane H, Badhe P, Gokulchandran N, Kulkarni P, Lohiya M, Biju H, Jacob VC. A clinical study shows safety and efficacy of autologous bone marrow mononuclear cell therapy to improve quality of life in muscular dystrophy patients. Cell Transplant. 2013;22 Suppl 1:S127-38. doi: 10.3727/096368913X672136. Epub 2013 Sep 10.
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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.
Alok K Sharma, M.S., M.Ch principal investigator
Neurogen Brain and Spine Institute
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 Autologous bone marrow mononuclear cell transplantation in people with Limb Girdle Muscular Dystrophy, run by Neurogen Brain and Spine Institute at 1 site in India.
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 4 inclusion and 5 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: withdrawn, as of the sponsor's last update on 25 Oct 2018. 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 India. 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?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Functional Independence Measure (time frame: 6 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 NCT02050776, which also lists 1 linked publication.
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 25 Oct 2018.