Pelizaeus-Merzbacher DiseaseNCT01391637
Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)
An observational study of Pelizaeus-Merzbacher Disease and PMD, run by StemCells, Inc. at 1 site in United States.
Trial summary
The purpose of this study is to determine the long term safety and preliminary effect of HuCNS-SC cells transplanted in subjects with Connatal Pelizaeus-Merzbacher Disease (PMD). Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Pelizaeus-Merzbacher Disease, PMD
- What is being tested?
- HuCNS-SC transplant in the lead-in phase
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2011to Mar 2016
- Phase
- Observational
- 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 Any age
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: PMD Subjects who underwent transplantation of HuCNS-SC cells under CL-N01-PMD study
Who may be able to join
- Subjects who received HuCNS-SC cells under Protocol CL-N01-PMD
Who cannot join
- Subjects who received off-protocol immunosuppressive medications.
- Subjects who are concurrently enrolled in another investigational study.
Trial locations and contacts
1 site in 1 country.
United States1 site
UCSF Medical Center
San Francisco, California, 94143
About this study
Official title: Long-Term Follow-Up Safety and Preliminary Efficacy Study of Human Central Nervous System Stem Cell (HuCNS-SC®) Transplantation in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)
The purpose of this study is to determine the long term safety and preliminary effect of HuCNS-SC cells transplanted in subjects with Connatal Pelizaeus-Merzbacher Disease (PMD).
Read the detailed description
Only subjects who underwent HuCNS-SC transplantation under Protocol CL-N01-PMD will be enrolled in this long term follow-up study.
Subjects will return to the site six months and one year after completion of the Phase I study and then annually for a total study duration of four years. Phone calls will also be made by the Investigator to the subject's parent/legal guardian bi-annually to conduct a phone visit through the four-year duration of the study.
- Conditions
- Pelizaeus-Merzbacher Disease, PMD
- Being tested
- HuCNS-SC transplant in the lead-in phase
- Keywords
- Long term follow-up, HuCNS-SC cells, human 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 transplant in the lead-in phaseBiological
Long-term safety follow-up study
| Study group | Role | What they receive |
|---|---|---|
| HuCNS-SC transplanted subjects in the lead-in phase | Subjects who had HuCNS-SC transplant in the lead-in phase study CL-N01-PMD Receives: Biological: HuCNS-SC transplant in the lead-in phase |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of serious adverse events (SAEs), results of physical and neurological examination, laboratory tests and vital signs.
Measured: 4 years
Secondary outcomes (2)
Preliminary efficacy using Bayley-III and Callier-Azusa Scale.
Changes compared to baseline
Measured: 4 years
Changes in brain magnetic resonance imaging (MRI), electroencephalogram (EEG), seizure frequency and somato-sensory evoked potentials (SSEP).
Changes compared to baseline
Measured: 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jul 2011 (estimated)
- Study startJun 2011
- Primary completionMar 2016
- Study completionMar 2016
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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- 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
- NCT01391637
- Sponsor's protocol ID
- CL-N02-PMD
- Status last verified
- May 2016
- First submitted
- 6 Jul 2011
- Last updated
- 13 May 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 May 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-Term Follow-Up Safety and Preliminary Efficacy Study of Human Central Nervous System Stem Cell (HuCNS-SC®) Transplantation in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)
- ClinicalTrials.gov ID
- NCT01391637
- Acronym / sponsor ID
- CL-N02-PMD
- Conditions
- Pelizaeus-Merzbacher Disease, PMD
- Keywords
- Long term follow-up, HuCNS-SC cells, human central nervous system stem cells
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- StemCells, Inc.Industry (pharma, biotech, devices)
- Study start
- Jun 2011
- Primary completion
- Mar 2016 (actual)
- Study completion
- Mar 2016 (actual)
- First posted
- 12 Jul 2011 (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 long term safety and preliminary effect of HuCNS-SC cells transplanted in subjects with Connatal Pelizaeus-Merzbacher Disease (PMD).
Detailed description
Only subjects who underwent HuCNS-SC transplantation under Protocol CL-N01-PMD will be enrolled in this long term follow-up study.
Subjects will return to the site six months and one year after completion of the Phase I study and then annually for a total study duration of four years. Phone calls will also be made by the Investigator to the subject's parent/legal guardian bi-annually to conduct a phone visit through the four-year duration of the study.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HuCNS-SC transplanted subjects in the lead-in phase | – | Biological: HuCNS-SC transplant in the lead-in phaseSubjects who had HuCNS-SC transplant in the lead-in phase study CL-N01-PMD |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- PMD Subjects who underwent transplantation of HuCNS-SC cells under CL-N01-PMD study
Key inclusion criteria (1 listed)
- Subjects who received HuCNS-SC cells under Protocol CL-N01-PMD
Key exclusion criteria (2 listed)
- Subjects who received off-protocol immunosuppressive medications.
- Subjects who are concurrently enrolled in another investigational study.
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 transplant in the lead-in phaseLong-term safety follow-up study | Biological | HuCNS-SC transplanted subjects in the lead-in phase |
Primary outcome
Incidence of serious adverse events (SAEs), results of physical and neurological examination, laboratory tests and vital signs.
Time frame: 4 years
2 secondary outcomes
Preliminary efficacy using Bayley-III and Callier-Azusa Scale.
Time frame: 4 years
Changes in brain magnetic resonance imaging (MRI), electroencephalogram (EEG), seizure frequency and somato-sensory evoked potentials (SSEP).
Time frame: 4 years
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 13 May 2016.
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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.
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?
An observational study of Pelizaeus-Merzbacher Disease and PMD, 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 1 inclusion and 2 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 13 May 2016. 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.
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?
Incidence of serious adverse events (SAEs), results of physical and neurological examination, laboratory tests and vital signs. (time frame: 4 years). 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 13 May 2016.