Pelizaeus-Merzbacher DiseasePhase 1NCT01005004
Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects
A Phase 1 study testing HuCNS-SC cells in people with Pelizaeus-Merzbacher Disease, run by StemCells, Inc. at 1 site in United States.
Trial summary
The purpose of this study is to determine the safety and preliminary effectiveness of human central nervous system stem cells (HuCNS-SC®) transplantation in patients with Connatal Pelizaeus-Merzbacher Disease (PMD). Read more about this study
- Who is it for?
- Ages 6 months to 5 years, men only
- Conditions the study is for
- Pelizaeus-Merzbacher Disease
- What is being tested?
- HuCNS-SC cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2009to Dec 2012
- 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 6 months to 5 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Confirmed clinical diagnosis of connatal PMD
- Molecular genetic confirmation of mutation in the proteolipid protein 1 (PLP1) gene
- MRI consistent with PMD as interpreted by a qualified neuroradiologist
Who cannot join
- Other significant congenital brain abnormality not related to PMD
- Previous participation in gene transfer or cell transplant trial
- Presence of neurological signs and symptoms not consistent with PMD
- Current or prior malignancy
- Prior organ, tissue or bone marrow transplant
Trial locations and contacts
1 site in 1 country.
United States1 site
University of California, San Francisco
San Francisco, California, 94143
About this study
Official title: Phase I Study of the Safety and Preliminary Efficacy of Intracerebral Transplantation of HuCNS-SC® Cells for Connatal Pelizaeus-Merzbacher Disease (PMD)
The purpose of this study is to determine the safety and preliminary effectiveness of human central nervous system stem cells (HuCNS-SC®) transplantation in patients with Connatal Pelizaeus-Merzbacher Disease (PMD).
Read the detailed description
Enrolled subjects will be transplanted with HuCNS-SC cells into the brain and will receive immunosuppression for nine months. The study observation period is for one year after transplant surgery. Thereafter, subjects will be enrolled in a long-term observational follow-up study for four years.
- Conditions
- Pelizaeus-Merzbacher Disease
- Being tested
- HuCNS-SC cells
- Keywords
- PMD, stem cells, Pelizaeus-Merzbacher Disease, human stem cells, central nervous system stem cells
- Medical subject headings
- Pelizaeus-Merzbacher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HuCNS-SC cellsBiological
Also called Human central nervous system stem cells
intracerebral transplantation
| Study group | Role | What they receive |
|---|---|---|
| HuCNS-SC cells | Experimental | Intracerebral implantation of HuCNS-SC via direct injection during surgery Receives: Biological: HuCNS-SC cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety assessment through clinical neurological and MRI evaluation.
Measured: one year post transplant
Secondary outcomes (1)
MRI examination for post-transplant myelination
Measured: one year post transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Oct 2009 (estimated)
- Study startNov 2009
- Primary completionOct 2012
- Study completionDec 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pelizaeus-Merzbacher DiseaseStudies 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
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- StemCells, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Stephen Huhn, MD, Study director, StemCells, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01005004
- Sponsor's protocol ID
- CL-N01-PMD
- Status last verified
- Jan 2015
- First submitted
- 28 Oct 2009
- Last updated
- 15 Jan 2015
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 15 Jan 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Study of the Safety and Preliminary Efficacy of Intracerebral Transplantation of HuCNS-SC® Cells for Connatal Pelizaeus-Merzbacher Disease (PMD)
- ClinicalTrials.gov ID
- NCT01005004
- Acronym / sponsor ID
- CL-N01-PMD
- Conditions
- Pelizaeus-Merzbacher Disease
- Keywords
- PMD, stem cells, Pelizaeus-Merzbacher Disease, human stem cells, central nervous system stem cells
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- StemCells, Inc.Industry (pharma, biotech, devices)
- Study start
- Nov 2009
- Primary completion
- Oct 2012 (actual)
- Study completion
- Dec 2012 (actual)
- First posted
- 30 Oct 2009 (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 is to determine the safety and preliminary effectiveness of human central nervous system stem cells (HuCNS-SC®) transplantation in patients with Connatal Pelizaeus-Merzbacher Disease (PMD).
Detailed description
Enrolled subjects will be transplanted with HuCNS-SC cells into the brain and will receive immunosuppression for nine months. The study observation period is for one year after transplant surgery. Thereafter, subjects will be enrolled in a long-term observational follow-up study for four years.
Study design
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HuCNS-SC cells | Experimental | Biological: HuCNS-SC cellsIntracerebral implantation of HuCNS-SC via direct injection during surgery |
Who may qualify
- Ages
- 6 months to 5 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Confirmed clinical diagnosis of connatal PMD
- Molecular genetic confirmation of mutation in the proteolipid protein 1 (PLP1) gene
- MRI consistent with PMD as interpreted by a qualified neuroradiologist
Key exclusion criteria (5 listed)
- Other significant congenital brain abnormality not related to PMD
- Previous participation in gene transfer or cell transplant trial
- Presence of neurological signs and symptoms not consistent with PMD
- Current or prior malignancy
- Prior organ, tissue or bone marrow transplant
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 |
|---|---|---|
| HuCNS-SC cellsAlso: Human central nervous system stem cellsintracerebral transplantation | Biological | HuCNS-SC cells |
Primary outcome
Safety assessment through clinical neurological and MRI evaluation.
Time frame: one year post transplant
1 secondary outcome
MRI examination for post-transplant myelination
Time frame: one year post transplant
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 15 Jan 2015.
Related trials for Pelizaeus-Merzbacher Disease
RecruitingObservational study
Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.
Inherited Disease, Rare Diseases
RecruitingObservational study
Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease
Pelizaeus-Merzbacher Disease
RecruitingPhase 1
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Adrenoleukodystrophy, Batten Disease
RecruitingObservational study
Longitudinal Study of Neurodegenerative Disorders
MLD, Krabbe Disease
Recruiting
Mesh-Free Versus Mesh-Based Surgery for Female Stress Urinary Incontinence: A Prospective Comparison of Pubo-Urethral Ligament Plication and Transobturator Tape
Stress Urinary Incontinence (SUI)
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.
Stephen Huhn, MD study director
StemCells, Inc.
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 HuCNS-SC cells in people with Pelizaeus-Merzbacher Disease, run by StemCells, Inc. 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 3 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: finished, as of the sponsor's last update on 15 Jan 2015. 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?
Safety assessment through clinical neurological and MRI evaluation. (time frame: one year post transplant). 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 15 Jan 2015.