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A Study of UCB and MSCs in Children With CP: ACCeNT-CP (ACCeNT-CP)

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ClinicalTrials.gov Identifier: NCT03473301
Recruitment Status : Recruiting
First Posted : March 22, 2018
Last Update Posted : April 6, 2018
Sponsor:
Collaborator:
The Marcus Foundation
Information provided by (Responsible Party):
Joanne Kurtzberg, MD, Duke University Medical Center

Brief Summary:
The main purpose of this study is to estimate change in motor function 12 months after treatment with a single dose of allogeneic umbilical cord blood (AlloCB) or repeated doses of umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in children with cerebral palsy. In addition, this study will contribute much needed data to the clinical trials community on the natural history of the motor function in CP over short-term (less than 1 year) time periods relevant to the conduct of clinical trials and assess the safety of AlloCB and hCT-MSC infusion in children with cerebral palsy.

Condition or disease Intervention/treatment Phase
Cerebral Palsy Biological: Infusion of allogeneic umbilical cord blood Biological: Infusion of MSCs Phase 1 Phase 2

Detailed Description:
This study is a phase I/II, prospective, randomized, open-label trial designed to determine the effect size of change in GMFM-66 score in subjects treated with hCT-MSC or allogeneic CB and assess the safety of repeated doses of hCT-MSC in children with cerebral palsy. Children ages 2-5 years with cerebral palsy due to hypoxic ischemic encephalopathy, stroke, or periventricular leukomalacia may be eligible to participate. All participants will ultimately be treated with an allogeneic cell product at some point during the study. Participants will be randomized to one of three arms: (1) the "AlloCB" arm will receive one allogeneic CB infusion at the baseline visit; (2) the "MSC" arm will receive three hCT-MSC infusions, one each at baseline, three months, and six months; (3) the "natural history" arm will not receive an infusion at baseline but will receive an allogeneic CB infusion at 12 months. Motor outcome measures will be assessed at baseline, six-months, and one-year time points. Safety will be evaluated at each infusion visit and remotely for an additional 12 months after the final visit. Duration of study participation will be 24 months from the time of baseline visit. Randomization to treatment arms will be stratified by GMFCS level at study entry and etiology of CP (Stroke vs. Other).

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 90 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Masking Description: Every attempt will be made to blind the outcomes assessor.
Primary Purpose: Treatment
Official Title: A Phase I/II Study of Allogeneic Umbilical Cord Blood and Umbilical Cord Tissue-Derived Mesenchymal Stromal Cell Infusions in Children With Cerebral Palsy
Actual Study Start Date : April 4, 2018
Estimated Primary Completion Date : August 2019
Estimated Study Completion Date : August 2020

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arm Intervention/treatment
Experimental: Allogeneic Umbilical Cord Blood
Subjects will receive a single intravenous infusion of a maximum of 10x107/kg allogeneic umbilical cord blood (CB) cells
Biological: Infusion of allogeneic umbilical cord blood
Subjects will receive a single infusion of allogeneic umbilical cord blood at the baseline visit.
Experimental: Cord Tissue Mesenchymal Stromal Cells
Subjects will receive three intravenous infusions of 2x106/kg human umbilical cord tissue cells (hCT-MSC), manufactured from allogeneic umbilical cord donors
Biological: Infusion of MSCs
Subjects will receive 3 infusions of MSCs (baseline, 3 months and 6 months).
Active Comparator: Natural History
Subjects will not receive any study product infusion until after the 12 month assessment. At the 12 month visit, they will receive an infusion of allogeneic umbilical cord blood cells so that all study participants will receive some type of cellular therapy.
Biological: Infusion of allogeneic umbilical cord blood
Subjects will receive a single infusion of allogeneic umbilical cord blood at the baseline visit.



Primary Outcome Measures :
  1. GMFM-66 score 12 months after study infusion [ Time Frame: 12 months from baseline visit ]
    The primary endpoint of this study is the difference between a child's observed and expected changes in GMFM-66 score 12 months after the initial study infusion.


Secondary Outcome Measures :
  1. Adverse events [ Time Frame: 12 months ]
    The secondary endpoint of this study is the number of adverse events occurring over a 12-month period post-treatment with hCT-MSC or AlloCB.



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Ages Eligible for Study:   24 Months to 60 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Age ≥24 months and ≤60 months adjusted age at the time of enrollment.
  2. Diagnosis: Unilateral or bilateral hypertonic cerebral palsy secondary to in utero or perinatal stroke/hemorrhage, hypoxic ischemic encephalopathy (including, but not limited to, birth asphyxia), and/or periventricular leukomalacia.
  3. Performance status: Gross Motor Function Classification Score levels I - IV
  4. Review of brain imaging (obtained as standard of care prior to study entry) does not suggest a genetic condition or brain malformation.
  5. Legal authorized representative consent.

Exclusion Criteria:

  1. Available qualified autologous cord blood unit.
  2. Hypotonic or ataxic cerebral palsy without spasticity.
  3. Autism and autistic spectrum disorders.
  4. Hypsarrhythmia.
  5. Legally blind
  6. Intractable seizures causing epileptic encephalopathy.
  7. Evidence of a progressive neurologic disease.
  8. Has an active, uncontrolled systemic infection or documentation of HIV+ status.
  9. Known genetic disease or phenotypic evidence of a genetic disease on physical exam.
  10. Concurrent genetic or acquired disease or comorbidity(ies) that could require a future allogeneic stem cell transplant.
  11. Requires ventilatory support, including home ventilator, CPAP, BiPAP, or supplemental oxygen.
  12. 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.
  13. Possible immunosuppression, defined as WBC <3,000 cells/mL or absolute lymphocyte count (ALC) <1500 with abnormal T-cell subsets.
  14. Patient's medical condition does not permit safe travel.
  15. Previously received any form of cellular therapy.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03473301


Contacts
Contact: Duke Pediatric Cell Therapy Team 8448002673 cordbloodtherapyinfo@dm.duke.edu

Locations
United States, North Carolina
Duke University Medical Center Recruiting
Durham, North Carolina, United States, 27705
Contact: Jessica Sun, MD       cordbloodtherapyinfo@dm.duke.edu   
Sponsors and Collaborators
Joanne Kurtzberg, MD
The Marcus Foundation
Investigators
Principal Investigator: Joanne Kurtzberg, MD Duke University

Responsible Party: Joanne Kurtzberg, MD, MD, Duke University Medical Center
ClinicalTrials.gov Identifier: NCT03473301     History of Changes
Other Study ID Numbers: Pro00089362
First Posted: March 22, 2018    Key Record Dates
Last Update Posted: April 6, 2018
Last Verified: April 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Joanne Kurtzberg, MD, Duke University Medical Center:
CP
Cerebral Palsy
Stem Cell
Cord Blood
MSCs

Additional relevant MeSH terms:
Cerebral Palsy
Brain Damage, Chronic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases