Muscular DystrophyPhase 1/2BMMNCNCT01834040
Study Safety and Efficacy of BMMNC for the Patient With Duchenne Muscular Dystrophy
A Phase 1/2 study testing Intralesional/ Intravenous of Autologous Stem cells. 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 center trial to check the safety and efficacy of BMMNC (100 million per dose) for the patient with Duchenne Muscular Dystrophy, Read more about this study
- Who is it for?
- Ages 4 to 20 yearsHealthy volunteers can take part
- Conditions the study is for
- Muscular Dystrophy, Duchenne Muscular Dystrophy
- What is being tested?
- Intralesional/ Intravenous of Autologous Stem cells.
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteIndia
- Study dates
- Sep 2014to Oct 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 4 to 20 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- Patient with Diagnose of Duchenne Muscular Dystrophy.
- Aged in between 4 to 20 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 with History of Immunodeficiency HIV+,Hepatitis B ,HBV and TPPA+, Tumor Markers+
- Patients with History of Hypertension and Hypersensitive.
- Patient who is not Diagnose of Duchenne Muscular Dystrophy.
- Alcohol and drug abuse / dependence.
Trial locations and contacts
1 site in 1 country.
India1 site, 1 recruiting
- Recruiting
Chaitanya Hospital
Pune, Maharashtra, 411009
Sachin S Jamadar, D ORTHO, +918888788880, sac2751982@gmail.com
About this study
Official title: Study Safety and Efficacy of of AUTOLOGOUS Bone Marrow Derived Mono Nuclear Stem Cell (BMMNCs) for the Patient With Duchenne Muscular Dystrophy. It is Self Funded (Patients' Own Funding) Clinical Trial
This Study is single arm, single center trial to check the safety and efficacy of BMMNC (100 million per dose) for the patient with Duchenne Muscular Dystrophy,
Read the detailed description
Muscular dystrophies, or MD, are a group of inherited conditions, which means they are passed down through families. They may occur in childhood or adulthood. There are many different types of muscular dystrophy. They include:
Duchenne muscular dystrophy is a form of muscular dystrophy that worsens quickly. Other muscular dystrophy (including Becker's muscular dystrophy) get worse much more slowly.
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.
Symptoms usually appear before age 6 and may appear as early as infancy. They may include:
Fatigue
Learning difficulties (the IQ (intelligence quotient )can be below 75)
Intellectual disability (possible, but does not get worse over time)
Muscle weakness
Begins in the legs and pelvis, but also occurs less severely in the arms, neck, and other areas of the body
Difficulty with motor skills (running, hopping, jumping)
Frequent falls
Trouble getting up from a lying position or climbing stairs
Weakness quickly gets worse
Progressive difficulty walking
Ability to walk may be lost by age 12, and the child will have to use a wheelchair
Breathing difficulties and heart disease usually start by age 20
- Conditions
- Muscular Dystrophy, Duchenne Muscular Dystrophy
- Being tested
- Intralesional/ Intravenous of Autologous Stem cells.
- Keywords
- Duchenne Muscular Dystrophy, Muscular Dystrophy, Myopathy
- Medical subject headings
- Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Intralesional/ Intravenous of Autologous Stem cells.Other
Also called Intralesional/ Intravenous of Autologous MNCs., Intralesional/ Intravenous of Autologous MNCs
Intralesional/ Intravenous of Autologous MNCs per dose
| Study group | Role | What they receive |
|---|---|---|
| intralesional and Intravenous | Other | Intralesional/ Intravenous of Autologous Stem cells. Receives: Other: Intralesional/ Intravenous of Autologous Stem cells. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Improvement of daily living scale.
Measured: 6 MONTH
Secondary outcomes (1)
Improvement of Muscular dystrophy specific functional Rating scale
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 completionSep 2016 (estimated)
- Study completionOct 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
- 30 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, M.S, Principal investigator, CHAITANYA HOSPITAL
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01834040
- Sponsor's protocol ID
- 00102
- Acronym
- BMMNC
- 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
- Study Safety and Efficacy of of AUTOLOGOUS Bone Marrow Derived Mono Nuclear Stem Cell (BMMNCs) for the Patient With Duchenne Muscular Dystrophy. It is Self Funded (Patients' Own Funding) Clinical Trial
- ClinicalTrials.gov ID
- NCT01834040
- Acronym / sponsor ID
- BMMNC / 00102
- Conditions
- Muscular Dystrophy, Duchenne Muscular Dystrophy
- Keywords
- Duchenne Muscular Dystrophy, Muscular Dystrophy, Myopathy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Chaitanya Hospital, PuneUniversity, hospital or other
- Study start
- Sep 2014
- Primary completion
- Sep 2016 (estimated)
- Study completion
- Oct 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 center trial to check the safety and efficacy of BMMNC (100 million per dose) for the patient with Duchenne Muscular Dystrophy,
Detailed description
Muscular dystrophies, or MD, are a group of inherited conditions, which means they are passed down through families. They may occur in childhood or adulthood. There are many different types of muscular dystrophy. They include:
Duchenne muscular dystrophy is a form of muscular dystrophy that worsens quickly. Other muscular dystrophy (including Becker's muscular dystrophy) get worse much more slowly.
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.
Symptoms usually appear before age 6 and may appear as early as infancy. They may include:
Fatigue
Learning difficulties (the IQ (intelligence quotient )can be below 75)
Intellectual disability (possible, but does not get worse over time)
Muscle weakness
Begins in the legs and pelvis, but also occurs less severely in the arms, neck, and other areas of the body
Difficulty with motor skills (running, hopping, jumping)
Frequent falls
Trouble getting up from a lying position or climbing stairs
Weakness quickly gets worse
Progressive difficulty walking
Ability to walk may be lost by age 12, and the child will have to use a wheelchair
Breathing difficulties and heart disease usually start by age 20
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| intralesional and Intravenous | Other | Other: Intralesional/ Intravenous of Autologous Stem cells.Intralesional/ Intravenous of Autologous Stem cells. |
Who may qualify
- Ages
- 4 to 20 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 4 to 20 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 (4 listed)
- Patient with History of Immunodeficiency HIV+,Hepatitis B ,HBV and TPPA+, Tumor Markers+
- Patients with History of Hypertension and Hypersensitive.
- Patient who is not Diagnose of Duchenne Muscular Dystrophy.
- 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 |
|---|---|---|
| Intralesional/ Intravenous of Autologous Stem cells.Also: Intralesional/ Intravenous of Autologous MNCs., Intralesional/ Intravenous of Autologous MNCsIntralesional/ Intravenous of Autologous MNCs per dose | Other | intralesional and Intravenous |
Primary outcome
Improvement of daily living scale.
Time frame: 6 MONTH
1 secondary outcome
Improvement of Muscular dystrophy specific functional Rating scale
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.
Related trials for Muscular Dystrophy
RecruitingPhase 3
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
Myotonic Dystrophy Type 1 (DM1), DM1
Active, not recruiting
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingObservational study
Preclinical Assessment of an Engineered HMGB1 Protein as a Novel Companion Therapy for Muscular Dystrophies
LAMA2-MD (Merosin Deficient Congenital Muscular Dystrophy, MDC1A)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
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, M.S 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 Intralesional/ Intravenous of Autologous Stem cells. 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 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 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 S Jamadar, Dortho (+918888788880, sac2751982@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Improvement of daily living scale. (time frame: 6 MONTH). 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.