Muscular DystrophyPhase 1/2mdpNCT01834066
Study Safety and Efficacy of Bone Marrow Derived Autologous Cells for the Treatment of Muscular Dystrophy.
A Phase 1/2 study testing Stem Cell in people with Muscular Dystrophy and Duchenne Muscular Dystrophy,, run by Chaitanya Hospital, Pune at 1 site in India.
Trial summary
This Study is single arm, single centre trial to check the safety and efficacy of Bone Marrow derived autologous cell(100 million per dose) for the patient with Duchenne Muscular Dystrophy. Read more about this study
- Who is it for?
- Ages 6 to 25 yearsHealthy volunteers can take part
- Conditions the study is for
- Muscular Dystrophy, Duchenne Muscular Dystrophy,
- What is being tested?
- Stem Cell
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 1 siteIndia
- Study dates
- Sep 2014to Dec 2016
- 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 6 to 25 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- Patient with Diagnose of Duchenne Muscular Dystrophy.
- Aged in between 6 to 25 Years.
- Willingness to undergo Bone Marrow derived Autologous cell Therapy.
- Ability to comprehend the explained protocol and thereafter give an informed consent as well as sign the required Informed Consent form(ICF) for the study.
- Ability and willingness to regular visit to hospital for protocol procedures and follow up
Who cannot join
- Patient who is not Diagnose of Duchenne Muscular Dystrophy.
- Patient with History of Immunodeficiency HIV+,Hepatitis B ,HBV and TPPA+,Tumor Markers+
- History of Life threatening allergic or immune -Mediated Reaction.
- the site of bone marrow aspiration potentially limiting Procedure.
- Alcohol and drug abuse / dependence.
- Patients with History of Hypertension and Hypersensitive.
Trial locations and contacts
1 site in 1 country.
India1 site, 1 recruiting
- Recruiting
Chaitnany Hospital
Pune, Maharashtra, 411009
Sachin P Jamadar, D ORTHO, +918888788880, sac2751982@gmail.com
ANANT E BAGUL, MS ORTHO
About this study
Official title: Safety and Efficacy of Bone Marrow Autologous Cells in Muscular Dystrophy. It is Self Funded (Patients' Own Funding) Clinical Trial
This Study is single arm, single centre trial to check the safety and efficacy of Bone Marrow derived autologous cell(100 million per dose) for the patient with Duchenne Muscular Dystrophy.
Read the detailed description
Muscular dystrophy is a group of inherited disorders that involve muscle weakness and loss of muscle tissue, which get worse over time. Duchenne muscular dystrophy is caused by a defective gene for dystrophin (a protein in the muscles). However, it often occurs in people without a known family history of the condition. slowly progress diseases.it causes Muscle weaknesses, Difficulty with motor skills ,Progressive difficulty walking.Breathing difficulties and heart disease,Frequent falls,weak limbs,lose motor Function.Begins in the legs and pelvis, but also occurs less severely in the arms, neck, and other areas of the body.Trouble getting up from a lying position or climbing stairs.
- Conditions
- Muscular Dystrophy, Duchenne Muscular Dystrophy,
- Being tested
- Stem Cell
- Keywords
- muscular Dystrophy stem cell
- Medical subject headings
- Muscular Dystrophies, Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Stem CellBiological
Also called Intravenous transfer of Autologous Stem Cell ( MNCs )
Intralesional transfer of Autologous Stem cell (MNCs) per dose. 6 doses in 3 months
| Study group | Role | What they receive |
|---|---|---|
| STEM CELL | Other | Intra thecal transplantation of autologous Stem Cell MNCs Receives: Biological: Stem Cell |
What is being measured
The primary outcome is the main question the study is designed to answer.
Significant Improvement in Muscle strength by using Kinetics Muscle testing or by using MMT( manual muscle test }score
Measured: 6 Months
Secondary outcomes (1)
-Improvement of daily living scale and baseline in EMG(electromyography)
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 posted17 Apr 2013 (estimated)
- Study startSep 2014
- Primary completionNov 2016 (estimated)
- Study completionDec 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chaitanya Hospital, Pune
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Dr. Sachin Jamadar, Chaitanya Hospital, Pune
- Investigators
- ANANT E BAGUL, MS ORTHO, Principal investigator, CHAITANYA HOSPITAL
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01834066
- Sponsor's protocol ID
- 00101
- Acronym
- mdp
- Status last verified
- Sep 2014
- First submitted
- 26 Feb 2013
- Last updated
- 17 Sep 2014
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of Bone Marrow Autologous Cells in Muscular Dystrophy. It is Self Funded (Patients' Own Funding) Clinical Trial
- ClinicalTrials.gov ID
- NCT01834066
- Acronym / sponsor ID
- mdp / 00101
- Conditions
- Muscular Dystrophy, Duchenne Muscular Dystrophy,
- Keywords
- muscular Dystrophy stem cell
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- Chaitanya Hospital, PuneUniversity, hospital or other
- Study start
- Sep 2014
- Primary completion
- Nov 2016 (estimated)
- Study completion
- Dec 2016 (estimated)
- First posted
- 17 Apr 2013 (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.
This Study is single arm, single centre trial to check the safety and efficacy of Bone Marrow derived autologous cell(100 million per dose) for the patient with Duchenne Muscular Dystrophy.
Detailed description
Muscular dystrophy is a group of inherited disorders that involve muscle weakness and loss of muscle tissue, which get worse over time. Duchenne muscular dystrophy is caused by a defective gene for dystrophin (a protein in the muscles). However, it often occurs in people without a known family history of the condition. slowly progress diseases.it causes Muscle weaknesses, Difficulty with motor skills ,Progressive difficulty walking.Breathing difficulties and heart disease,Frequent falls,weak limbs,lose motor Function.Begins in the legs and pelvis, but also occurs less severely in the arms, neck, and other areas of the body.Trouble getting up from a lying position or climbing stairs.
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 | Other | Biological: Stem CellIntra thecal transplantation of autologous Stem Cell MNCs |
Who may qualify
- Ages
- 6 to 25 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Patient with Diagnose of Duchenne Muscular Dystrophy.
- Aged in between 6 to 25 Years.
- Willingness to undergo Bone Marrow derived Autologous cell Therapy.
- Ability to comprehend the explained protocol and thereafter give an informed consent as well as sign the required Informed Consent form(ICF) for the study.
- Ability and willingness to regular visit to hospital for protocol procedures and follow up
Key exclusion criteria (6 listed)
- Patient who is not Diagnose of Duchenne Muscular Dystrophy.
- Patient with History of Immunodeficiency HIV+,Hepatitis B ,HBV and TPPA+,Tumor Markers+
- History of Life threatening allergic or immune -Mediated Reaction.
- the site of bone marrow aspiration potentially limiting Procedure.
- Alcohol and drug abuse / dependence.
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 |
|---|---|---|
| Stem CellAlso: Intravenous transfer of Autologous Stem Cell ( MNCs )Intralesional transfer of Autologous Stem cell (MNCs) per dose. 6 doses in 3 months | Biological | STEM CELL |
Primary outcome
Significant Improvement in Muscle strength by using Kinetics Muscle testing or by using MMT( manual muscle test }score
Time frame: 6 Months
1 secondary outcome
-Improvement of daily living scale and baseline in EMG(electromyography)
Time frame: 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Sep 2014.
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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.
ANANT E BAGUL, MS ORTHO principal investigator
CHAITANYA HOSPITAL
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Stem Cell in people with Muscular Dystrophy and Duchenne Muscular Dystrophy,, run by Chaitanya Hospital, Pune 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 5 inclusion and 6 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 Sep 2014. 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 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 Sachin P Jamadar, D.Ortho (+918888788880, sac2751982@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Significant Improvement in Muscle strength by using Kinetics Muscle testing or by using MMT( manual muscle test }score (time frame: 6 Months). 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 17 Sep 2014.