Duchenne Muscular DystrophyPhase 1/2NCT01610440
Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy
A Phase 1/2 study testing human umbilical cord mesenchymal stem cells in people with Duchenne Muscular Dystrophy, run by Shenzhen Beike Bio-Technology Co., Ltd. at 1 site in China.
Trial summary
Duchenne muscular dystrophy (DMD), an X-linked recessive genetic disease always progressed slowly，tends to leading proximal skeletal muscle atrophy and weakness of limbs, as well as impaired respiratory muscle and cardiac muscle. Read more about this study
- Who is it for?
- Ages 5 to 12 years
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- human umbilical cord mesenchymal stem cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteChina
- Study dates
- Oct 2011to Oct 2013
- 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 5 to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Aged 5-12 years
- Clinical manifestation, enzymology, electromyogram, gene type confirmed the diagnose of Duchenne muscular dystrophy
- Sign the consent form and follow the clinic trail procedure
Who cannot join
- Not Duchenne muscular dystrophy
- Any history of hypersensitivity to serum products,or other know drug and food allergy
- Combined Pneumonia or other Severe systemic bacteria infection
- HIV+, TPPA +， patients diagnosed as HBV or HCV
- Tumor Markers +
- Severe psychotic patients, cognitive dysfunction
Show all 12Show fewer
- Coagulation disorders
- Uncontrolled hypertension after treatment，blood pressure≥180mmHg/110 mmHg
- Other severe systemic or organic disease
- Enrollment in other trials in the last 3 months
- Received any stem cell therapy in past 6 months
- Other criteria that investigator consider improper for inclusion
Trial locations and contacts
1 site in 1 country.
Liqing Yao contact
China1 site, 1 recruiting
- Recruiting
The Second Affiliated Hospital of Kunming Medical College
Kunming, Yunnan, 650031
Liqing Yao, yaoliqing98731@yahoo.com.cn
Liqing Yao
About this study
Official title: Phase I/II Study of Stem Cell Therapy in Patients With Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD), an X-linked recessive genetic disease always progressed slowly，tends to leading proximal skeletal muscle atrophy and weakness of limbs, as well as impaired respiratory muscle and cardiac muscle. To a large extent, patients always lose motor function gradually and die for heart failure or severe infection at the end stage of DMD. At present, the treatment strategy relies on heteropathy accompanied with rehabilitation training. However, the therapeutic effect remains extremely limited.
Human umbilical cord mesenchymal stem cells (hUC-MSCs) have been evidenced to improve motor function, increase muscle strength and reduce abnormal levels of related enzymes, such as creatine kinase (CK), lactate dehydrogenase (LDH), alanine aminotransferase (ALT) and aspartate aminotransferase (AST). This study is aimed to explore the safety and efficacy of hUC-MSCs transplantation for DMD.
Read the detailed description
This study is designed to investigate the safety and efficacy of human umbilical cord mesenchymal stem cells transplantation in patients with progressive muscular dystrophy.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- human umbilical cord mesenchymal stem cells
- Keywords
- Duchenne muscular dystrophy, Human Umbilical Cord Mesenchymal 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.
human umbilical cord mesenchymal stem cellsBiological
rehabilitation therapy plus human umbilical cord mesenchymal stem cells
| Study group | Role | What they receive |
|---|---|---|
| Intervention Group | Experimental | Participants will be given rehabilitation therapy plus human umbilical cord mesenchymal stem cells transplantation with one year follow-up Receives: Biological: human umbilical cord mesenchymal stem cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Activities of Daily Living(ADL)scale
Measured: 1 year after treatment
Secondary outcomes (7)
Incidences of Adverse Event and Serious Adverse Event
Measured: 1 year after treatment
Change from baseline in CK
Measured: 1 year after treatment
Change from baseline in LDH
Measured: 1 year after treatment
Change from baseline in ALT
Measured: 1 year after treatment
Change from baseline in AST
Measured: 1 year after treatment
Change from baseline to manual muscle test(MMT)
Measured: 1 year after treatment
Change from baseline in electromyography(EMG)
Measured: 1 year after treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jun 2012 (estimated)
- Study startOct 2011
- Primary completionMar 2013 (estimated)
- Study completionOct 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne 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
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shenzhen Beike Bio-Technology Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- The Second Affiliated Hospital of Kunming Medical University
- Responsible party
- Sponsor
- Investigators
- Liqing Yao, Principal investigator, The Second Affiliated Hospital of Kunming Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01610440
- Sponsor's protocol ID
- BKCR-DMD-1(Ⅰ)
- Status last verified
- Nov 2012
- First submitted
- 21 May 2012
- Last updated
- 30 Nov 2012
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Nov 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I/II Study of Stem Cell Therapy in Patients With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT01610440
- Acronym / sponsor ID
- BKCR-DMD-1(Ⅰ)
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne muscular dystrophy, Human Umbilical Cord Mesenchymal Stem Cells
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Shenzhen Beike Bio-Technology Co., Ltd.Industry (pharma, biotech, devices)
- Collaborators
- The Second Affiliated Hospital of Kunming Medical University
- Study start
- Oct 2011
- Primary completion
- Mar 2013 (estimated)
- Study completion
- Oct 2013 (estimated)
- First posted
- 4 Jun 2012 (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.
Duchenne muscular dystrophy (DMD), an X-linked recessive genetic disease always progressed slowly，tends to leading proximal skeletal muscle atrophy and weakness of limbs, as well as impaired respiratory muscle and cardiac muscle. To a large extent, patients always lose motor function gradually and die for heart failure or severe infection at the end stage of DMD. At present, the treatment strategy relies on heteropathy accompanied with rehabilitation training. However, the therapeutic effect remains extremely limited.
Human umbilical cord mesenchymal stem cells (hUC-MSCs) have been evidenced to improve motor function, increase muscle strength and reduce abnormal levels of related enzymes, such as creatine kinase (CK), lactate dehydrogenase (LDH), alanine aminotransferase (ALT) and aspartate aminotransferase (AST). This study is aimed to explore the safety and efficacy of hUC-MSCs transplantation for DMD.
Detailed description
This study is designed to investigate the safety and efficacy of human umbilical cord mesenchymal stem cells transplantation in patients with progressive 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 |
|---|---|---|
| Intervention Group | Experimental | Biological: human umbilical cord mesenchymal stem cellsParticipants will be given rehabilitation therapy plus human umbilical cord mesenchymal stem cells transplantation with one year follow-up |
Who may qualify
- Ages
- 5 to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Aged 5-12 years
- Clinical manifestation, enzymology, electromyogram, gene type confirmed the diagnose of Duchenne muscular dystrophy
- Sign the consent form and follow the clinic trail procedure
Key exclusion criteria (12 listed)
- Not Duchenne muscular dystrophy
- Any history of hypersensitivity to serum products,or other know drug and food allergy
- Combined Pneumonia or other Severe systemic bacteria infection
- HIV+, TPPA +， patients diagnosed as HBV or HCV
- Tumor Markers +
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 |
|---|---|---|
| human umbilical cord mesenchymal stem cellsrehabilitation therapy plus human umbilical cord mesenchymal stem cells | Biological | Intervention Group |
Primary outcome
Activities of Daily Living(ADL)scale
Time frame: 1 year after treatment
7 secondary outcomes
Incidences of Adverse Event and Serious Adverse Event
Time frame: 1 year after treatment
Change from baseline in CK
Time frame: 1 year after treatment
Change from baseline in LDH
Time frame: 1 year after treatment
Change from baseline in ALT
Time frame: 1 year after treatment
Change from baseline in AST
Time frame: 1 year after treatment
Change from baseline to manual muscle test(MMT)
Time frame: 1 year after treatment
Change from baseline in electromyography(EMG)
Time frame: 1 year after treatment
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Nov 2012.
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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.
Liqing Yao contact
Liqing Yao principal investigator
The Second Affiliated Hospital of Kunming Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing human umbilical cord mesenchymal stem cells in people with Duchenne Muscular Dystrophy, run by Shenzhen Beike Bio-Technology Co., Ltd. at 1 site in China.
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 3 inclusion and 12 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 30 Nov 2012. 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 China. 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 Liqing Yao (yaoliqing98731@yahoo.com.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Activities of Daily Living(ADL)scale (time frame: 1 year after treatment). 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 30 Nov 2012.