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

Primary Purpose

Cerebral Palsy

Status

Completed

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

sibling umbilical cord blood

About this trial

This is an interventional treatment trial for Cerebral Palsy

Eligibility Criteria

1 Year - 6 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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.

Exclusion Criteria:

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.

Sites / Locations

Duke University Medical Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

Matched related umbilical cord blood

Mismatched related umbilical cord blood

Arm Description

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

Nine subjects will receive an infusion of HLA-mismatched (≥3/6 match) or matched sibling umbilical cord blood cells.

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Assessment for improvement in gross motor function

Assessment of improvement in gross motor function using validated tools.

Assessment for improvement in fine motor function

Assessment of fine motor function using validated tools.

Full Information

NCT ID

NCT02599207

First Posted

November 2, 2015

Last Updated

December 2, 2019

Sponsor

Joanne Kurtzberg, MD

1. Study Identification

Unique Protocol Identification Number

NCT02599207

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

December 2019

Overall Recruitment Status

Completed

Study Start Date

November 2015 (undefined)

Primary Completion Date

October 2016 (Actual)

Study Completion Date

July 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor-Investigator

Name of the Sponsor

Joanne Kurtzberg, MD

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Cerebral Palsy

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

15 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Matched related umbilical cord blood

Arm Type

Experimental

Arm Description

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

Arm Title

Mismatched related umbilical cord blood

Arm Type

Experimental

Arm Description

Nine subjects will receive an infusion of HLA-mismatched (≥3/6 match) or matched sibling umbilical cord blood cells.

Intervention Type

Biological

Intervention Name(s)

sibling umbilical cord blood

Intervention Description

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.

Primary Outcome Measure Information:

Title

Assessment for infusion reactions, infections, graft versus host disease or any other adverse events

Description

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.

Time Frame

6 months

Secondary Outcome Measure Information:

Title

Assessment for improvement in gross motor function

Description

Assessment of improvement in gross motor function using validated tools.

Time Frame

6 months

Title

Assessment for improvement in fine motor function

Description

Assessment of fine motor function using validated tools.

Time Frame

6 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

1 Year

Maximum Age & Unit of Time

6 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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. Exclusion Criteria: 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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Joanne Kurtzberg, MD

Organizational Affiliation

Duke University

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Jessica Sun, MD

Organizational Affiliation

Duke University

Official's Role

Principal Investigator

Facility Information:

Facility Name

Duke University Medical Center

City

Durham

State/Province

North Carolina

ZIP/Postal Code

27705

Country

United States

12. IPD Sharing Statement

Learn more about this trial

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

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