Phase 2
N=91
A Study of UCB and MSCs in Children With CP: ACCeNT-CP
Cerebral Palsy
Bottom Line
View on ClinicalTrials.gov: NCT03473301 ↗Enrolled (actual)
91
Serious AEs
20.3%
Results posted
Feb 2021
Primary outcome: Primary: Change in Gross Motor Function Measure (GMFM-66) in Excess of Expected Change — 5.83; 4.27; 3.15 score on a scale
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Infusion of allogeneic umbilical cord blood (Biological); Infusion of MSCs (Biological)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Joanne Kurtzberg, MD
- Primary completion
- Feb 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Gross Motor Function Measure (GMFM-66) in Excess of Expected Change |
5.83; 4.27; 3.15 | — |
| SECONDARY Number of Adverse Events |
30; 48; 16; 9 | — |
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.
Eligibility Criteria
Inclusion Criteria
- Age ≥24 months and ≤60 months adjusted age at the time of enrollment.
- 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.
- Performance status: Gross Motor Function Classification Score levels I - IV
- Review of brain imaging (obtained as standard of care prior to study entry) does not suggest a genetic condition or brain malformation.
- Legal authorized representative consent.
Exclusion Criteria
- Available qualified autologous cord blood unit.
- Hypotonic or ataxic cerebral palsy without spasticity.
- Autism and autistic spectrum disorders.
- Hypsarrhythmia.
- Legally blind
- 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) <1500 with abnormal T-cell subsets.
- Patient's medical condition does not permit safe travel.
- Previously received any form of cellular therapy.
Data sourced from ClinicalTrials.gov (NCT03473301). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.