Duchenne's Muscular DystrophyPhase 1NCT02235844
Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for a Single Male Patient With Duchenne Muscular Dystrophy (DMD)
A Phase 1 study testing Umbilical Cord Mesenchymal Stem Cells in people with Duchenne's Muscular Dystrophy, run by Allergy and Asthma Consultants, Wichita, Kansas at 1 site in United States.
Trial summary
This research study is designed to evaluate the effects of human umbilical cord mesenchymal stem cells (UC-MSCs), on Duchenne's muscular dystrophy (DMD). The potential muscle regenerative and anti-inflammatory properties of UC MSCs position them as a possible treatment option for DMD. Read more about this study
- Who is it for?
- Ages 28 to 31 years, men only
- Conditions the study is for
- Duchenne's Muscular Dystrophy
- What is being tested?
- Umbilical Cord Mesenchymal Stem Cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2014to 30 Sep 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 28 to 31 years
- Men only
- No healthy volunteers
- Adult
Who may be able to join
- Duchenne's Muscular Dystrophy
Who cannot join
- None
Trial locations and contacts
1 site in 1 country.
United States1 site
Asthma and Allergy Consultants
Wichita, Kansas, 67205
About this study
Official title: Allogeneic Transplantation of Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) for a Single Male Patient With Duchenne Muscular Dystrophy (DMD)
This research study is designed to evaluate the effects of human umbilical cord mesenchymal stem cells (UC-MSCs), on Duchenne's muscular dystrophy (DMD). The potential muscle regenerative and anti-inflammatory properties of UC MSCs position them as a possible treatment option for DMD. Both of these properties could lead to potential benefits for a DMD patient.
- Conditions
- Duchenne's Muscular Dystrophy
- Being tested
- Umbilical Cord Mesenchymal Stem Cells
- Keywords
- Mesenchymal, Stem Cells, Umbilical Cord, Duchenne's, Muscular Dystrophy
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Umbilical Cord Mesenchymal Stem CellsBiological
| Study group | Role | What they receive |
|---|---|---|
| Mesenchymal Stem Cells | Experimental | Umbilical Cord Mesenchymal Stem Cells Receives: Biological: Umbilical Cord Mesenchymal Stem Cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events
No occurrence of adverse events
Measured: 3 months after final treatment
Secondary outcomes (7)
Change from baseline of weight
Measured: 3 months after final treatment
Change of muscle diameter (circumferential measurements) from baseline
Measured: 3 months after final treatment
Change from baseline of Pulmonary Maximum Expiratory Pressure
Measured: 3 months after final treatment
Change from baseline of Pulmonary Forced Vital Capacity
Measured: 3 months after final treatment
Maximum Change from baseline of Predicted Inspiratory Pressure %
Measured: 3 months after final treatment
Change from baseline of Predicted Maximum Expiratory Pressure %
Measured: 3 months after final treatment
Change from baseline of Predicted Forced Vital Capacity %
Measured: 3 months after final treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Sep 2014 (estimated)
- Study startSep 2014
- Primary completion30 Sep 2017
- Study completion30 Sep 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne's 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
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Allergy and Asthma Consultants, Wichita, Kansas
- Sponsor type
- University, hospital or other
- Collaborators
- Aidan Foundation, Neil H. Riordan PhD
- Responsible party
- Sponsor
- Investigators
- Maurice HV Strickland, MD, Principal investigator, Allergy and Asthma Consultants of Wichita, KS
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02235844
- Sponsor's protocol ID
- IND 16026 DMD Single Patient
- Status last verified
- Sep 2019
- First submitted
- 8 Sep 2014
- Last updated
- 16 Sep 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allogeneic Transplantation of Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) for a Single Male Patient With Duchenne Muscular Dystrophy (DMD)
- ClinicalTrials.gov ID
- NCT02235844
- Acronym / sponsor ID
- IND 16026 DMD Single Patient
- Conditions
- Duchenne's Muscular Dystrophy
- Keywords
- Mesenchymal, Stem Cells, Umbilical Cord, Duchenne's, Muscular Dystrophy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Allergy and Asthma Consultants, Wichita, KansasUniversity, hospital or other
- Collaborators
- Aidan Foundation, Neil H. Riordan PhD
- Study start
- Sep 2014 (actual)
- Primary completion
- 30 Sep 2017 (actual)
- Study completion
- 30 Sep 2017 (actual)
- First posted
- 10 Sep 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.
This research study is designed to evaluate the effects of human umbilical cord mesenchymal stem cells (UC-MSCs), on Duchenne's muscular dystrophy (DMD). The potential muscle regenerative and anti-inflammatory properties of UC MSCs position them as a possible treatment option for DMD. Both of these properties could lead to potential benefits for a DMD patient.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Mesenchymal Stem Cells | Experimental | Biological: Umbilical Cord Mesenchymal Stem CellsUmbilical Cord Mesenchymal Stem Cells |
Who may qualify
- Ages
- 28 to 31 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (1 listed)
- Duchenne's Muscular Dystrophy
Key exclusion criteria (1 listed)
- None
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 |
|---|---|---|
| Umbilical Cord Mesenchymal Stem Cells | Biological | Mesenchymal Stem Cells |
Primary outcome
Adverse Events
Time frame: 3 months after final treatment
No occurrence of adverse events
7 secondary outcomes
Change from baseline of weight
Time frame: 3 months after final treatment
Change of muscle diameter (circumferential measurements) from baseline
Time frame: 3 months after final treatment
Change from baseline of Pulmonary Maximum Expiratory Pressure
Time frame: 3 months after final treatment
Change from baseline of Pulmonary Forced Vital Capacity
Time frame: 3 months after final treatment
Maximum Change from baseline of Predicted Inspiratory Pressure %
Time frame: 3 months after final treatment
Change from baseline of Predicted Maximum Expiratory Pressure %
Time frame: 3 months after final treatment
Change from baseline of Predicted Forced Vital Capacity %
Time frame: 3 months after final treatment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2019.
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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.
Maurice HV Strickland, MD principal investigator
Allergy and Asthma Consultants of Wichita, KS
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 Umbilical Cord Mesenchymal Stem Cells in people with Duchenne's Muscular Dystrophy, run by Allergy and Asthma Consultants, Wichita, Kansas at 1 site in United States.
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 1 inclusion and 1 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: finished, as of the sponsor's last update on 16 Sep 2019. 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 United States. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Adverse Events (time frame: 3 months after final 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 16 Sep 2019; AzTrials last refreshed its copy on 1 Nov 2026.