Cerebral PalsyPhase 1NCT02599207
Assessment of the Safety of Allogeneic Umbilical Cord Blood Infusions in Children With Cerebral Palsy
A Phase 1 study testing sibling umbilical cord blood in people with Cerebral Palsy, run by Joanne Kurtzberg, MD at 1 site in United States.
Trial summary
This study is a single site, phase I, prospective study of the safety of intravenous sibling cord blood infusion in 15 children ages 1-6 years with Cerebral Palsy (CP). All subjects will be treated with sibling cord blood cells. The first six will receive cord blood cells from an HLA-matched sibling. Read more about this study
- Who is it for?
- Ages 1 year to 6 years
- Conditions the study is for
- Cerebral Palsy
- What is being tested?
- sibling umbilical cord blood
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2015to Jul 2018
- 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 1 year to 6 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age ≥12 months and ≤ 6 years at the time of CB infusion.
- Diagnosis: Cerebral palsy with diplegia, hemiplegia, or quadriplegia.
- Performance status:
- Bilateral cerebral palsy (diplegia or quadraplegia):
- Gross Motor Function Classification Score levels II - IV, or Gross Motor Function Classification Score level I, age ≥ 2 years
- Hemiplegia: Gross Motor Function Classification Score levels II - IV or minimal functional capabilities in the affected upper extremity. A subject classified as GMFCS level I with significant upper extremity impairment will be eligible if the affected upper extremity is used as an assist only.
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- Review of brain imaging (obtained as standard of care prior to study entry) does not suggest a genetic condition or brain malformation.
- Suitably matched sibling donor CB unit (see section 6.2 for matching details) available at a private or public cord blood bank with a minimum total nucleated cell dose of ≥ 2.5 x 107 cells/kilogram.
- Legal authorized representative consent.
Who cannot join
- Available qualified autologous cord blood unit
- Autism and autistic spectrum disorders without motor disability.
- Hypsarrhythmia.
- Intractable seizures causing epileptic encephalopathy.
- Evidence of a progressive neurologic disease.
- Has an active, uncontrolled systemic infection or documentation of HIV+ status.
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- Known genetic disease or phenotypic evidence of a genetic disease on physical exam.
- Concurrent genetic or acquired disease or comorbidity(ies) that could require a future allogeneic stem cell transplant.
- Requires ventilatory support, including home ventilator, CPAP, BiPAP, or supplemental oxygen.
- Impaired renal or liver function as determined by serum creatinine >1.5mg/dL and/or total bilirubin >1.3mg/dL except in patients with known Gilbert's disease.
- Possible immunosuppression, defined as WBC <3,000 cells/mL or absolute lymphocyte count (ALC) below normal for age with abnormal T-cell subsets.
- Patient's medical condition does not permit safe travel.
- Previously received any form of cellular therapy.
Trial locations and contacts
1 site in 1 country.
United States1 site
Duke University Medical Center
Durham, North Carolina, 27705
About this study
This study is a single site, phase I, prospective study of the safety of intravenous sibling cord blood infusion in 15 children ages 1-6 years with Cerebral Palsy (CP). All subjects will be treated with sibling cord blood cells. The first six will receive cord blood cells from an HLA-matched sibling. The following nine subjects will receive cord blood cells from an HLA-mismatched (≥3/6 match) or matched sibling. The duration of study participation will be six months from the time of the cord blood infusion.
Read the detailed description
This study is a phase I, prospective, open-label trial designed to assess the safety of a single allogeneic sibling cord blood infusion in young children with cerebral palsy. Children ages one to six years with uncomplicated cerebral palsy and an available HLA matched or haploidentical, qualified, sibling cord blood unit will be eligible to participate. All participants will receive a single intravenous infusion of allogeneic sibling cord blood. All participants will have an initial clinical evaluation to verify the diagnosis of cerebral palsy and determine eligibility. The main endpoint is safety, for which acute infusion reactions as well as incidence of infections and graft versus host disease will be assessed. Functional outcome measures, described below, will be assessed at baseline and six months post sibling cord blood infusion and described.
- Conditions
- Cerebral Palsy
- Being tested
- sibling umbilical cord blood
- Medical subject headings
- Cerebral Palsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
sibling umbilical cord bloodBiological
All subjects will receive a single infusion of allogeneic sibling cord blood. The first six subjects will receive cord blood cells from an HLA-matched sibling. The following 9 subjects will receive cord blood cells from an HLA-mismatched (≥3/6 match) or matched sibling. Six of the 15 subjects must be treated with haplo-identical sibling CB. Duration of study participation will be six months from the time of CB infusion.
| Study group | Role | What they receive |
|---|---|---|
| Matched related umbilical cord blood | Experimental | Six subjects will receive an infusion of HLA matched sibling umbilical cord blood cells. Receives: Biological: sibling umbilical cord blood |
| Mismatched related umbilical cord blood | Experimental | Nine subjects will receive an infusion of HLA-mismatched (≥3/6 match) or matched sibling umbilical cord blood cells. Receives: Biological: sibling umbilical cord blood |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assessment for infusion reactions, infections, graft versus host disease or any other adverse events
The primary endpoint of this study is safety which will be evaluated by assessing the incidence of acute infusion reactions, infections, graft versus host disease.
Measured: 6 months
Secondary outcomes (2)
Assessment for improvement in gross motor function
Assessment of improvement in gross motor function using validated tools.
Measured: 6 months
Assessment for improvement in fine motor function
Assessment of fine motor function using validated tools.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Nov 2015 (estimated)
- Study startNov 2015
- Primary completionOct 2016
- Study completionJul 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cerebral PalsyStudies 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Joanne Kurtzberg, MD
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Joanne Kurtzberg, MD, Duke University
- Investigators
- Joanne Kurtzberg, MD, Principal investigator, Duke UniversityJessica Sun, MD, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02599207
- Sponsor's protocol ID
- Pro00065043
- Status last verified
- Dec 2019
- First submitted
- 2 Nov 2015
- Last updated
- 4 Dec 2019
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 4 Dec 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessment of the Safety of Allogeneic Umbilical Cord Blood Infusions in Children With Cerebral Palsy
- ClinicalTrials.gov ID
- NCT02599207
- Acronym / sponsor ID
- Pro00065043
- Conditions
- Cerebral Palsy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Joanne Kurtzberg, MDUniversity, hospital or other
- Study start
- Nov 2015
- Primary completion
- Oct 2016 (actual)
- Study completion
- Jul 2018 (actual)
- First posted
- 6 Nov 2015 (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 study is a single site, phase I, prospective study of the safety of intravenous sibling cord blood infusion in 15 children ages 1-6 years with Cerebral Palsy (CP). All subjects will be treated with sibling cord blood cells. The first six will receive cord blood cells from an HLA-matched sibling. The following nine subjects will receive cord blood cells from an HLA-mismatched (≥3/6 match) or matched sibling. The duration of study participation will be six months from the time of the cord blood infusion.
Detailed description
This study is a phase I, prospective, open-label trial designed to assess the safety of a single allogeneic sibling cord blood infusion in young children with cerebral palsy. Children ages one to six years with uncomplicated cerebral palsy and an available HLA matched or haploidentical, qualified, sibling cord blood unit will be eligible to participate. All participants will receive a single intravenous infusion of allogeneic sibling cord blood. All participants will have an initial clinical evaluation to verify the diagnosis of cerebral palsy and determine eligibility. The main endpoint is safety, for which acute infusion reactions as well as incidence of infections and graft versus host disease will be assessed. Functional outcome measures, described below, will be assessed at baseline and six months post sibling cord blood infusion and described.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Matched related umbilical cord blood | Experimental | Biological: sibling umbilical cord bloodSix subjects will receive an infusion of HLA matched sibling umbilical cord blood cells. |
| Mismatched related umbilical cord blood | Experimental | Biological: sibling umbilical cord bloodNine subjects will receive an infusion of HLA-mismatched (≥3/6 match) or matched sibling umbilical cord blood cells. |
Who may qualify
- Ages
- 1 year to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- Age ≥12 months and ≤ 6 years at the time of CB infusion.
- Diagnosis: Cerebral palsy with diplegia, hemiplegia, or quadriplegia.
- Performance status:
- Gross Motor Function Classification Score levels II - IV, or Gross Motor Function Classification Score level I, age ≥ 2 years
- Review of brain imaging (obtained as standard of care prior to study entry) does not suggest a genetic condition or brain malformation.
Key exclusion criteria (13 listed)
- Available qualified autologous cord blood unit
- Autism and autistic spectrum disorders without motor disability.
- Hypsarrhythmia.
- Intractable seizures causing epileptic encephalopathy.
- Evidence of a progressive neurologic disease.
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 |
|---|---|---|
| sibling umbilical cord bloodAll subjects will receive a single infusion of allogeneic sibling cord blood. The first six subjects will receive cord blood cells from an HLA-matched sibling. The following 9 subjects will receive cord blood cells from an HLA-mismatched (≥3/6 match) or matched sibling. Six of the 15 subjects must be treated with haplo-identical sibling CB. Duration of study participation will be six months from the time of CB infusion. | Biological | Matched related umbilical cord blood, Mismatched related umbilical cord blood |
Primary outcome
Assessment for infusion reactions, infections, graft versus host disease or any other adverse events
Time frame: 6 months
The primary endpoint of this study is safety which will be evaluated by assessing the incidence of acute infusion reactions, infections, graft versus host disease.
2 secondary outcomes
Assessment for improvement in gross motor function
Time frame: 6 months
Assessment for improvement in fine motor function
Time frame: 6 months
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 4 Dec 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.
Joanne Kurtzberg, MD principal investigator
Duke University
Jessica Sun, MD principal investigator
Duke 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 sibling umbilical cord blood in people with Cerebral Palsy, run by Joanne Kurtzberg, MD 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 9 inclusion and 13 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 4 Dec 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?
Assessment for infusion reactions, infections, graft versus host disease or any other adverse events (time frame: 6 months). 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 4 Dec 2019.