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

NCT02599207 | Completed Phase 1 DMC

• 1 other identifier: Pro00065043
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Cerebral Palsy

Outcome Measures

Primary Outcomes (1)

• 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.

  6 months

Secondary Outcomes (2)

• Assessment for improvement in gross motor function

  6 months

• Assessment for improvement in fine motor function

  6 months

Study Arms (2)

Matched related umbilical cord blood

EXPERIMENTAL

Six subjects will receive an infusion of HLA matched sibling umbilical cord blood cells.

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.

Biological: sibling umbilical cord blood

Interventions

sibling umbilical cord blood BIOLOGICAL

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.

Matched related umbilical cord blood; Mismatched related umbilical cord blood

Eligibility Criteria

• Age: 1 Year - 6 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Joanne Kurtzberg, MD: lead

Study Sites (1)

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

MeSH Terms

Conditions

Cerebral Palsy

Condition Hierarchy (Ancestors)

Brain Damage, Chronic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Study Officials

• Joanne Kurtzberg, MD

  Duke University

  PRINCIPAL INVESTIGATOR

• Jessica Sun, MD

  Duke University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of Pediatrics

Study Record Dates

• First Submitted: November 2, 2015
• First Posted: November 6, 2015
• Study Start: November 1, 2015
• Primary Completion: October 1, 2016
• Study Completion: July 1, 2018
• Last Updated: December 4, 2019

Record last verified: 2019-12

Locations

US (1)