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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03473301
Recruitment Status : Active, not recruiting
First Posted : March 22, 2018
Last Update Posted : June 25, 2020
Sponsor:
Collaborator:
The Marcus Foundation
Information provided by (Responsible Party):
Joanne Kurtzberg, MD, Duke University

Tracking Information
First Submitted Date  ICMJE March 15, 2018
First Posted Date  ICMJE March 22, 2018
Last Update Posted Date June 25, 2020
Actual Study Start Date  ICMJE April 9, 2018
Estimated Primary Completion Date September 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 15, 2018)
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 15, 2018)
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.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study of UCB and MSCs in Children With CP: ACCeNT-CP
Official Title  ICMJE A Phase I/II Study of Allogeneic Umbilical Cord Blood and Umbilical Cord Tissue-Derived Mesenchymal Stromal Cell Infusions in Children With Cerebral Palsy
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.
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  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE 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
Condition  ICMJE Cerebral Palsy
Intervention  ICMJE
  • Biological: Infusion of allogeneic umbilical cord blood
    Subjects will receive a single infusion of allogeneic umbilical cord blood at the baseline visit.
  • Biological: Infusion of MSCs
    Subjects will receive 3 infusions of MSCs (baseline, 3 months and 6 months).
Study Arms  ICMJE
  • Experimental: Allogeneic Umbilical Cord Blood
    Subjects will receive a single intravenous infusion of a maximum of 10x107/kg allogeneic umbilical cord blood (CB) cells
    Intervention: Biological: Infusion of allogeneic umbilical cord blood
  • 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
    Intervention: Biological: Infusion of MSCs
  • 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.
    Intervention: Biological: Infusion of allogeneic umbilical cord blood
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: March 15, 2018)
90
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE May 2021
Estimated Primary Completion Date September 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 24 Months to 60 Months   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03473301
Other Study ID Numbers  ICMJE Pro00089362
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Joanne Kurtzberg, MD, Duke University
Study Sponsor  ICMJE Joanne Kurtzberg, MD
Collaborators  ICMJE The Marcus Foundation
Investigators  ICMJE
Principal Investigator: Joanne Kurtzberg, MD Duke University
PRS Account Duke University
Verification Date June 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP