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Cerebral Palsy

Assessment of the Safety of Allogeneic Umbilical Cord Blood Infusions in Children With Cerebral Palsy

Sponsored by Joanne Kurtzberg, MD

About this trial

Last updated 6 years ago

Study ID

Pro00065043

Status

Completed

Type

Interventional

Phase

Phase 1

Placebo

No

Accepting

18-75 Years
1 to 6 Years
All
All

Not accepting

Not accepting
Healthy Volunteers

Trial Timing

Ended 7 years ago

What is this trial about?

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.

What are the participation requirements?

Yes

Inclusion Criteria

1. Age ≥12 months and ≤ 6 years at the time of CB infusion.

2. Diagnosis: Cerebral palsy with diplegia, hemiplegia, or quadriplegia.

3. 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.

4. Review of brain imaging (obtained as standard of care prior to study entry) does not suggest a genetic condition or brain malformation.

5. 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.

6. Legal authorized representative consent.

No

Exclusion Criteria

1. Available qualified autologous cord blood unit

2. Autism and autistic spectrum disorders without motor disability.

3. Hypsarrhythmia.

4. Intractable seizures causing epileptic encephalopathy.

5. Evidence of a progressive neurologic disease.

6. Has an active, uncontrolled systemic infection or documentation of HIV+ status.

7. Known genetic disease or phenotypic evidence of a genetic disease on physical exam.

8. Concurrent genetic or acquired disease or comorbidity(ies) that could require a future allogeneic stem cell transplant.

9. Requires ventilatory support, including home ventilator, CPAP, BiPAP, or supplemental oxygen.

10. 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.

11. Possible immunosuppression, defined as WBC <3,000 cells/mL or absolute lymphocyte count (ALC) below normal for age with abnormal T-cell subsets.

12. Patient's medical condition does not permit safe travel.

13. Previously received any form of cellular therapy.