Neuronal Ceroid LipofuscinosisPhase 1NCT01238315
Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis
A Phase 1 study testing HuCNS-SC in people with Neuronal Ceroid Lipofuscinosis, run by StemCells, Inc. at 1 site in United States.
Trial summary
The purpose of this Phase Ib study is to determine if "Human Central Nervous System Stem Cells"(HuCNS-SC) is safe to be transplanted in subjects with infantile and late infantile neuronal ceroid lipofuscinosis. The study will also measure post-transplantation disease progression. Read more about this study
- Who is it for?
- Ages 6 months to 6 years
- Conditions the study is for
- Neuronal Ceroid Lipofuscinosis
- What is being tested?
- HuCNS-SC
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2010to Apr 2011
- 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 6 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age 6 months to 6 years
- Male or female
- Clinical diagnosis of Infantile neuronal ceroid lipofuscinosis or late infantile neuronal ceroid lipofuscinosis
- CLN1 or CLN2 mutation
Who cannot join
- Previously received an organ, tissue or bone marrow transplantation
- Previously participated in any gene or cell therapy study
- Infection with hepatitis virus, cytomegalovirus, Epstein-Barr virus, or Human Immunodeficiency Virus (HIV)
- Current or prior cancer
- Bleeding disorder
- Unable to have an MRI scan
Trial locations and contacts
1 site in 1 country.
United States1 site
Oregon Health and Science University
Portland, Oregon, 97239
About this study
Official title: A Phase Ib Study of the Safety and Preliminary Efficacy of Allogeneic Intracerebral Human Central Nervous System Stem Cell Transplantation in Subjects With Non-Refractory Infantile and Late Infantile Neuronal Ceroid Lipofuscinosis
The purpose of this Phase Ib study is to determine if "Human Central Nervous System Stem Cells"(HuCNS-SC) is safe to be transplanted in subjects with infantile and late infantile neuronal ceroid lipofuscinosis. The study will also measure post-transplantation disease progression.
Read the detailed description
Participation in this study will involve screening assessments, surgery to implant HuCNS-SC, medication to suppress immune system and a series of follow-up assessments.
- Conditions
- Neuronal Ceroid Lipofuscinosis
- Being tested
- HuCNS-SC
- Keywords
- Batten's disease, INCL, LINCL, Neuronal Ceroid Lipofuscinosis, NCL
- Medical subject headings
- Neuronal Ceroid-Lipofuscinoses
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HuCNS-SCBiological
Also called human neural stem cells, stem cells, neural stem cells
Surgery to implant human CNS stem cells
| Study group | Role | What they receive |
|---|---|---|
| HuCNS-SC | Other | Not described Receives: Biological: HuCNS-SC |
What is being measured
The primary outcome is the main question the study is designed to answer.
number of participants with adverse events.
Safety will be measured by number of adverse events and changes in neurological status.
Measured: one year following transplantation
Secondary outcomes (1)
Number of subjects with improvement in neurological, neuropsychological development, MRI and MRS
Measured: one year following transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Nov 2010 (estimated)
- Study startNov 2010
- Primary completionApr 2011
- Study completionApr 2011
Why it stopped: Lack of timely patient accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuronal Ceroid LipofuscinosisStudies 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
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- StemCells, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Nathan Selden, MD, PhD, Principal investigator, Oregon Health and Science University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01238315
- Sponsor's protocol ID
- CL-N03-NCL
- Status last verified
- Jan 2015
- First submitted
- 5 Nov 2010
- 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
- A Phase Ib Study of the Safety and Preliminary Efficacy of Allogeneic Intracerebral Human Central Nervous System Stem Cell Transplantation in Subjects With Non-Refractory Infantile and Late Infantile Neuronal Ceroid Lipofuscinosis
- ClinicalTrials.gov ID
- NCT01238315
- Acronym / sponsor ID
- CL-N03-NCL
- Conditions
- Neuronal Ceroid Lipofuscinosis
- Keywords
- Batten's disease, INCL, LINCL, Neuronal Ceroid Lipofuscinosis, NCL
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Lack of timely patient accrual
- Enrolment
- 0 participants (actual)
- Lead sponsor
- StemCells, Inc.Industry (pharma, biotech, devices)
- Study start
- Nov 2010
- Primary completion
- Apr 2011 (actual)
- Study completion
- Apr 2011 (actual)
- First posted
- 10 Nov 2010 (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 Phase Ib study is to determine if "Human Central Nervous System Stem Cells"(HuCNS-SC) is safe to be transplanted in subjects with infantile and late infantile neuronal ceroid lipofuscinosis. The study will also measure post-transplantation disease progression.
Detailed description
Participation in this study will involve screening assessments, surgery to implant HuCNS-SC, medication to suppress immune system and a series of follow-up assessments.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HuCNS-SC | Other | Biological: HuCNS-SC |
Who may qualify
- Ages
- 6 months to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Age 6 months to 6 years
- Male or female
- Clinical diagnosis of Infantile neuronal ceroid lipofuscinosis or late infantile neuronal ceroid lipofuscinosis
- CLN1 or CLN2 mutation
Key exclusion criteria (6 listed)
- Previously received an organ, tissue or bone marrow transplantation
- Previously participated in any gene or cell therapy study
- Infection with hepatitis virus, cytomegalovirus, Epstein-Barr virus, or Human Immunodeficiency Virus (HIV)
- Current or prior cancer
- Bleeding disorder
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-SCAlso: human neural stem cells, stem cells, neural stem cellsSurgery to implant human CNS stem cells | Biological | HuCNS-SC |
Primary outcome
number of participants with adverse events.
Time frame: one year following transplantation
Safety will be measured by number of adverse events and changes in neurological status.
1 secondary outcome
Number of subjects with improvement in neurological, neuropsychological development, MRI and MRS
Time frame: one year following transplantation
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jan 2015.
- Related InfoLink provided by the sponsor.
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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.
Nathan Selden, MD, PhD principal investigator
Oregon Health and Science 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 study testing HuCNS-SC in people with Neuronal Ceroid Lipofuscinosis, 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 4 inclusion and 6 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: withdrawn, 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 was withdrawn before enrolling participants, so there are no results.
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?
number of participants with adverse events. (time frame: one year following transplantation). 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.