Cerebral PalsyPhase 2NCT04243408
Treatment of Children With Cerebral Palsy With Autologous Umbilical Cord Blood, a Pilot Study
A Phase 2 study testing Autologous umbilical cord blood transfusion in people with Cerebral Palsy, run by Sheba Medical Center at 1 site in Israel.
Trial summary
This study is a phase II, prospective, double blind, placebo-controlled study of the efficacy of autologous umbilical cord blood infusion. The study population will consist of 72 children ages 2 months to 12 years with cerebral palsy. The population will be randomly assigned to 2 groups, 36 children in each group. Read more about this study
- Who is it for?
- Ages 2 months to 12 years
- Conditions the study is for
- Cerebral Palsy
- What is being tested?
- Autologous umbilical cord blood transfusion
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 72 participants
- Where
- 1 siteIsrael
- Study dates
- 8 Jul 2020to Dec 2024
- Phase
- Phase 2
- 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 2 months to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age ≥ 2 month and ≤ 12 years.
- Performance status for children older than 2 months and younger than 12 months including all the following (Gross Motor Function Classification Score is less indicative before 12 months):
- Abnormal General Movements (in infants 2-6 months)
- Abnormal Hammersmith Infant Neurological Examination
- Performance status for children older than 12 months
- Bilateral spastic cerebral palsy (diplegia or quadraplegia): Gross Motor Function Classification Score levels I - IV
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- Spastic hemiplegia: Gross Motor Function Classification Score levels I - IV. 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.
- Bilateral hypotonic cerebral palsy (diplegia or quadraplegia): Gross Motor Function Classification Score levels I - IV.
- An abnormal brain MRI suggestive of an acquired etiology (and not genetic etiology or brain malformation).
- 4. Autologous umbilical cord blood available at a private or public cord blood bank with a minimum total nucleated cell dose of ≥ 2 x 10e7 cells/kilogram.
- 5. Parental consent.
Who cannot join
- Autism and autistic spectrum disorders without motor disability.
- Hypsarrhythmia.
- Intractable seizures causing epileptic encephalopathy.
- Evidence of a progressive neurologic disease.
- Known HIV or uncontrolled bacterial, fungal, or viral infections.
- Impaired renal or liver function as determined by serum creatinine >1.5mg/dL and/or total bilirubin >1.3mg/dL.
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- Head circumference >3 standard deviations below the mean for age.
- Known genetic disease or phenotypic evidence of a genetic disease on physical examination.
- Requires ventilatory support, including home ventilator
- Surgical procedure or botulinum toxin injection from 6 months prior to the study and during the time of the study
- Patient's medical condition does not permit safe travel.
- Previously received any form of cellular therapy.
- Autologous umbilical cord blood unit has any of the following:
- Total nuclear cell dose < 2 x 10e7 cells/kilogram
- Positive maternal infectious disease markers (except CMV)
- Evidence of infectious contamination of the cord blood unit
- Lack of a test sample to confirm identity
- Evidence of a genetic disease
- Unable to obtain parental consent.
Trial locations and contacts
1 site in 1 country.
Israel1 site, 1 recruiting
- Recruiting
Chaim Seba Medical Center
Ramat Gan
Omer Bar-Yosef
About this study
This study is a phase II, prospective, double blind, placebo-controlled study of the efficacy of autologous umbilical cord blood infusion.
The study population will consist of 72 children ages 2 months to 12 years with cerebral palsy. The population will be randomly assigned to 2 groups, 36 children in each group. The study group be treated by cord blood in the beginning of the study and the control group by placebo product. The study population will be stratified to reduce variance 3 groups by age: 2-12 months / 1-6 years / 6-12 years The study will consist of 4 stages Stage 1: initial assessment by physiotherapist and occupational therapist / treatment by cord blood or placebo / blood work before and after treatment Stage 2: at stage 1 + 3 months assessment by physiotherapist and occupational therapist Stage 3: at stage 1 + 6 months assessment by physiotherapist and occupational therapist / cross-over treatment by cord blood or placebo / blood work before and after treatment Stage 4: at stage 1 + 12 months assessment by physiotherapist and occupational therapist The primary outcome is improvement motor skills six months after treatment at stage 3
- Conditions
- Cerebral Palsy
- Being tested
- Autologous umbilical cord blood transfusion, placebo
- Keywords
- cerebral palsy, autologous 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.
Autologous umbilical cord blood transfusionOther
single dose of an autologous umbilical cord blood transfusion
placeboOther
The placebo product will consist of the standard ingredients of the acellular content of the UCB unit. It will consist of 20 ml Dextran (Plander 40.000 - 50g/500ml, solution for infusion) and 20 ml of human Albumin 5% (solution for infusion). The volume of placebo product will be 40 ml
| Study group | Role | What they receive |
|---|---|---|
| group 1 | Experimental | Not described Receives: Other: Autologous umbilical cord blood transfusion |
| group 2 | Placebo comparator | Not described Receives: Other: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Motor developmental improvement
Gross Motor Function Measurement - 66 (GMFM-66). All score are normalized with mean 100 and STD of 15. High score is better than low score .
Measured: 6 months
Motor developmental improvement
Peabody developmental motor scales-second edition (PDMS-2). All score are normalized with mean 100 and STD 10. High score is better
Measured: 6 months
Functional assessment
Pediatric Evaluation of Disability Evaluation . Score 0-100 higher score better functionality Pediatric Evaluation of Disability Inventory-Computer Adaptive Test (PEDI-CAT)
Measured: 6 months
Secondary outcomes (2)
Functional assessment
Adaptive Behavior Assessment System, Third Edition (ABAS-3). All score are normalized with mean 100 and STD of 10. High score is better than low score .
Measured: 6 months
Functional assessment
Vineland Adaptive Behavior Scales-Second Edition (VINELAND-II). All score are normalized with mean 100 and STD of 15. High score is better than low score .
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 posted28 Jan 2020
- Study start8 Jul 2020
- Primary completionDec 2024 (estimated)
- Study completionDec 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Cerebral PalsyStudies of the same condition, open and under way.
- More trials run by Sheba Medical CenterThe sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- This study is a phase II, prospective, double blind, placebo-control study of the efficacy of autologous umbilical cord blood infusion. The study population will consist of 72 children ages 2 months to 12 years with cerebral palsy. The population will be randomly assigned to 2 groups, the study group be treated by cord blood in the beginning of the study and the control group by placebo product. The study population will be stratified to reduce variance 3 groups by age: 2-12 months / 1-6 years / 6-12 years The study will consist of 4 stages Stage 1: initial assessment by physiotherapist and occupational therapist / treatment by cord blood or placebo / blood work before and after treatment Stage 2: at stage 1 + 3 months assessment by physiotherapist and occupational therapist Stage 3: at stage 1 + 6 months assessment by physiotherapist and occupational therapist
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 72 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sheba Medical Center
- Sponsor type
- Other government
- Responsible party
- Principal investigator, Dr. Omer Bar-Yosef, Sheba Medical Center
- Investigators
- Omer Bar-Yosef, MD.PHD, Study director, Sheba Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04243408
- Sponsor's protocol ID
- SHEBA-18-5102-OBY-CTIL
- Status last verified
- Jan 2021
- First submitted
- 15 Jan 2020
- Last updated
- 20 Jan 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: data set of the baseline of the children, the quantity of cord blood transfusion they received and the clinical followup information.
Publications
Bax M, Goldstein M, Rosenbaum P, Leviton A, Paneth N, Dan B, Jacobsson B, Damiano D; Executive Committee for the Definition of Cerebral Palsy. Proposed definition and classification of cerebral palsy, April 2005. Dev Med Child Neurol. 2005 Aug;47(8):571-6. doi: 10.1017/s001216220500112x.
PubMed 16108461Colver A, Fairhurst C, Pharoah PO. Cerebral palsy. Lancet. 2014 Apr 5;383(9924):1240-9. doi: 10.1016/S0140-6736(13)61835-8. Epub 2013 Nov 20.
PubMed 24268104Clark SL, Hankins GD. Temporal and demographic trends in cerebral palsy--fact and fiction. Am J Obstet Gynecol. 2003 Mar;188(3):628-33. doi: 10.1067/mob.2003.204.
PubMed 12634632
Study at a glance
From the registry record, last updated by the sponsor on 20 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of Children With Cerebral Palsy With Autologous Umbilical Cord Blood, a Pilot Study
- ClinicalTrials.gov ID
- NCT04243408
- Acronym / sponsor ID
- SHEBA-18-5102-OBY-CTIL
- Conditions
- Cerebral Palsy
- Keywords
- cerebral palsy, autologous umbilical cord blood
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 72 participants (estimated)
- Lead sponsor
- Sheba Medical CenterOther government
- Study start
- 8 Jul 2020 (actual)
- Primary completion
- Dec 2024 (estimated)
- Study completion
- Dec 2024 (estimated)
- First posted
- 28 Jan 2020 (actual)
- 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 phase II, prospective, double blind, placebo-controlled study of the efficacy of autologous umbilical cord blood infusion.
The study population will consist of 72 children ages 2 months to 12 years with cerebral palsy. The population will be randomly assigned to 2 groups, 36 children in each group. The study group be treated by cord blood in the beginning of the study and the control group by placebo product. The study population will be stratified to reduce variance 3 groups by age: 2-12 months / 1-6 years / 6-12 years The study will consist of 4 stages Stage 1: initial assessment by physiotherapist and occupational therapist / treatment by cord blood or placebo / blood work before and after treatment Stage 2: at stage 1 + 3 months assessment by physiotherapist and occupational therapist Stage 3: at stage 1 + 6 months assessment by physiotherapist and occupational therapist / cross-over treatment by cord blood or placebo / blood work before and after treatment Stage 4: at stage 1 + 12 months assessment by physiotherapist and occupational therapist The primary outcome is improvement motor skills six months after treatment at stage 3
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- This study is a phase II, prospective, double blind, placebo-control study of the efficacy of autologous umbilical cord blood infusion. The study population will consist of 72 children ages 2 months to 12 years with cerebral palsy. The population will be randomly assigned to 2 groups, the study group be treated by cord blood in the beginning of the study and the control group by placebo product. The study population will be stratified to reduce variance 3 groups by age: 2-12 months / 1-6 years / 6-12 years The study will consist of 4 stages Stage 1: initial assessment by physiotherapist and occupational therapist / treatment by cord blood or placebo / blood work before and after treatment Stage 2: at stage 1 + 3 months assessment by physiotherapist and occupational therapist Stage 3: at stage 1 + 6 months assessment by physiotherapist and occupational therapist
| Arm | Type | Receives |
|---|---|---|
| group 1 | Experimental | Other: Autologous umbilical cord blood transfusion |
| group 2 | Placebo comparator | Other: placebo |
Who may qualify
- Ages
- 2 months to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (11 listed)
- Age ≥ 2 month and ≤ 12 years.
- Performance status for children older than 2 months and younger than 12 months including all the following (Gross Motor Function Classification Score is less indicative before 12 months):
- Performance status for children older than 12 months
- An abnormal brain MRI suggestive of an acquired etiology (and not genetic etiology or brain malformation).
- 4. Autologous umbilical cord blood available at a private or public cord blood bank with a minimum total nucleated cell dose of ≥ 2 x 10e7 cells/kilogram.
Key exclusion criteria (19 listed)
- Autism and autistic spectrum disorders without motor disability.
- Hypsarrhythmia.
- Intractable seizures causing epileptic encephalopathy.
- Evidence of a progressive neurologic disease.
- Known HIV or uncontrolled bacterial, fungal, or viral infections.
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 |
|---|---|---|
| Autologous umbilical cord blood transfusionsingle dose of an autologous umbilical cord blood transfusion | Other | group 1 |
| placeboThe placebo product will consist of the standard ingredients of the acellular content of the UCB unit. It will consist of 20 ml Dextran (Plander 40.000 - 50g/500ml, solution for infusion) and 20 ml of human Albumin 5% (solution for infusion). The volume of placebo product will be 40 ml | Other | group 2 |
Primary outcome
Motor developmental improvement
Time frame: 6 months
Gross Motor Function Measurement - 66 (GMFM-66). All score are normalized with mean 100 and STD of 15. High score is better than low score .
Motor developmental improvement
Time frame: 6 months
Peabody developmental motor scales-second edition (PDMS-2). All score are normalized with mean 100 and STD 10. High score is better
Functional assessment
Time frame: 6 months
Pediatric Evaluation of Disability Evaluation . Score 0-100 higher score better functionality Pediatric Evaluation of Disability Inventory-Computer Adaptive Test (PEDI-CAT)
2 secondary outcomes
Functional assessment
Time frame: 6 months
Functional assessment
Time frame: 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jan 2021.
- Background: PubMed 16108461Bax M, Goldstein M, Rosenbaum P, Leviton A, Paneth N, Dan B, Jacobsson B, Damiano D; Executive Committee for the Definition of Cerebral Palsy. Proposed definition and classification of cerebral palsy, April 2005. Dev Med Child Neurol. 2005 Aug;47(8):571-6. doi: 10.1017/s001216220500112x.
- Background: PubMed 24268104Colver A, Fairhurst C, Pharoah PO. Cerebral palsy. Lancet. 2014 Apr 5;383(9924):1240-9. doi: 10.1016/S0140-6736(13)61835-8. Epub 2013 Nov 20.
- Background: PubMed 12634632Clark SL, Hankins GD. Temporal and demographic trends in cerebral palsy--fact and fiction. Am J Obstet Gynecol. 2003 Mar;188(3):628-33. doi: 10.1067/mob.2003.204.
- Individual participant dataSharing planned: Yes. data set of the baseline of the children, the quantity of cord blood transfusion they received and the clinical followup information.
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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.
Omer Bar-Yosef, MD.PHD study director
Sheba Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Autologous umbilical cord blood transfusion in people with Cerebral Palsy, run by Sheba Medical Center at 1 site in Israel.
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 11 inclusion and 19 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: status not confirmed, as of the sponsor's last update on 20 Jan 2021. 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 Israel. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Omer Bar-Yosef, MD.PHD (972-35302895, Omer.BarYosef@sheba.health.gov.il), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Motor developmental improvement (time frame: 6 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04243408, which also lists 3 linked publications.
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 20 Jan 2021.