Safety and Effectiveness of Banked Cord Blood or Bone Marrow Stem Cells in Children With Cerebral Palsy (CP).

NCT01988584 | Completed Phase 2 Results DMC

• 1 other identifier: HSC-MS-12-0876
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to compare the safety and effectiveness of two types of stem cells,(either banked cord blood or bone marrow), in children between the ages of 2 to 10 years with CP. 15 children with banked cord blood at CBR and 15 children without banked cord blood will be enrolled into the study. The study involves one baseline/treatment visit and 3 follow-up visits at 6 months, 12 months, and 2 years. Five children in each group will be randomized to a placebo control group at the baseline/treatment visit. Parents will not be told if their child received stem cells or a placebo until the 12 month follow-up visit. At that time parents may elect to have their child receive the stem cell treatment; either bone marrow harvest or umbilical cord blood if banked with CBR. All study visits will be conducted at the UTHealth Medical School and Children's Memorial Hermann Hospital in Houston, Texas. As of 1/21/2014 we have met our enrollment limit for children without banked cord blood undergoing bone marrow harvest for stem cells.

Conditions

Cerebral Palsy

Keywords

Cerebral Palsy; Brain Injury; Stem Cells; Mononuclear Cells; Bone Marrow; Umbilical Cord Blood

Outcome Measures

Primary Outcomes (2)

• Safety as Assessed by Number of Participants With In-hospital Infusion Toxicity

  In-hospital infusion toxicity includes hemodynamic, pulmonary, hepatic, renal, or neurologic complications.

  24 hours after infusion

• Long-term Safety

  Long-term safety as assessed by number of participants who developed new mass lesions or other pathological structural changes or had worsening neurological status

  from the time of infusion to 1 year after infusion

Secondary Outcomes (41)

• Number of Participants With an Improvement in White Matter Integrity.

  from baseline to 1 year after infusion

• Gross Motor Function Classification Score (GMFM-66)

  baseline before infusion

• Gross Motor Function Classification Score (GMFM-66)

  1 year after infusion

• Gross Motor Function Classification Score (GMFM-88)

  baseline before infusion

• Gross Motor Function Classification Score (GMFM-88)

  1 year after infusion

Study Arms (4)

umbilical cord blood (UCB) cells

EXPERIMENTAL

Children who have banked UCB with CBR will receive an umbilical cord blood stem cell infusion at the baseline/treatment visit.

Biological: umbilical cord blood (hUCB) cells

bone marrow-derived mononuclear cells (BMMNCs)

EXPERIMENTAL

Children in the BMMNC group will undergo bone marrow harvest and stem cell infusion at the baseline/treatment visit.

Biological: bone marrow derived mononuclear cells (BMMNCs)

saline infusion (placebo), then umbilical cord blood (UCB) cells

EXPERIMENTAL

Five children in each group will be randomly assigned to receive an inactive substance (placebo) at the baseline/treatment visit. Parents will be given the opportunity to cross-over to either the umbilical cord blood or bone marrow harvest group at the one year visit.

Biological: umbilical cord blood (hUCB) cells; Drug: Saline Infusion (Placebo)

saline infusion (placebo), then bone marrow-derived mononuclear cells (BMMNCs)

EXPERIMENTAL

Drug: Saline Infusion (Placebo); Biological: bone marrow derived mononuclear cells (BMMNCs)

Interventions

umbilical cord blood (hUCB) cells BIOLOGICAL

Autologous umbilical cord blood banked with the Cord Blood Registry.

Also known as: Autolgous Stem Cells

saline infusion (placebo), then umbilical cord blood (UCB) cells; umbilical cord blood (UCB) cells

Saline Infusion (Placebo) DRUG

A total of 10 children (5 from each cohort) will be randomized to a placebo infusion at the baseline visit and then have the opportunity to cross-over to stem cell treatment at the 1yr. visit.

saline infusion (placebo), then bone marrow-derived mononuclear cells (BMMNCs); saline infusion (placebo), then umbilical cord blood (UCB) cells

bone marrow derived mononuclear cells (BMMNCs) BIOLOGICAL

Autologous stem cells from bone marrow harvest.

Also known as: Autologous Stem Cells

bone marrow-derived mononuclear cells (BMMNCs); saline infusion (placebo), then bone marrow-derived mononuclear cells (BMMNCs)

Eligibility Criteria

• Age: 2 Years - 10 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Children with diagnosis of Cerebral Palsy (spastic CP due to periventricular white matter damage or neonatal brain injury from perinatal stroke or intra-ventricular hemorrhage)
• Gross Motor Function Classification Score level II-V
• Ages 24 months to 10 years
• English speaking, if verbal
• Ability to travel to Houston for treatment and follow-up -

You may not qualify if:

• Known history of:
• Intractable seizures
• Traumatic brain injury
• Genetic disorder (as demonstrated by newborn screening or genetic diagnostic testing)
• Recently treated or current infection
• Renal insufficiency or altered renal function (as defined by serum creatinine \> 1.5 mg/dl at screening)
• Hepatic disease or altered liver function (as defined by SGPT \> 150 U/L \[non-contusion related\], and/or T. Bilirubin \>1.3 mg/dL at screening)
• HIV+ (as demonstrated by positive blood test)
• Immunosuppression (as defined by WBC \<3,000 cells/ml at screening)
• Infectious related neurological injury
• Sensitivity to Ethylene Oxide (EtO) \[found in fumigants and disinfectants\]
• If Athetoid CP diagnosis, other etiologies such as degenerative, mitochondrial, and metabolic disorders must be excluded, and the outcome assessments must be able to be conducted to assess for potential treatment effects
• Normal brain MRI
• Evidence of acute illness at the time of infusion, such as, but not limited to, fever (temperature \> 37.5 C), vomiting, diarrhea, wheezing or crackles
• Progressing neurological disease (as defined by Batten Disease, Leukodystrophies, Metabolic disorders, Mitochondrial disorders, Neurotransmitter disorders)

Sponsors & Collaborators

• The University of Texas Health Science Center, Houston: lead
• Cord Blood Registry, Inc.: collaborator
• Let's Cure CP Foundation: collaborator
• Mission Connect, a program of TIRR Foundation: collaborator

Study Sites (1)

UTHealth, Medical School, Dept. of Pediatric Surgery

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Cerebral Palsy; Brain Injuries

Interventions

Cell Count

Condition Hierarchy (Ancestors)

Brain Damage, Chronic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Craniocerebral Trauma; Trauma, Nervous System; Wounds and Injuries

Intervention Hierarchy (Ancestors)

Cytological Techniques; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Investigative Techniques; Cell Physiological Phenomena

Limitations and Caveats

Limitations common to treating heterogeneous conditions and to cell based therapies. First, patients enrolled had wide range of clinical and imaging findings, which made pooling small data sample problematic. Imaging studies are problematic with hemispheric volume loss secondary to in utero stroke, etc. Second, variability in available cord blood doses. Due to a competing trial, few US families with banked autologous units and not already treated. Unable to reach target enrollment.

Results Point of Contact

• Title: Charles S. Cox, MD, Professor
• Organization: The University of Texas Health Science Center at Houston

charles.s.cox@uth.tmc.edu

713-500-7300

Study Officials

• Charles S Cox, MD

  UTHealth, Medical School, Dept. of Pediatric Surgery

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: The Children's Fund Distinguished Professor, Department of Pediatric Surgery

Study Record Dates

• First Submitted: November 4, 2013
• First Posted: November 20, 2013
• Study Start: November 1, 2013
• Primary Completion: February 21, 2018
• Study Completion: February 21, 2018
• Last Updated: November 21, 2022
• Results First Posted: November 21, 2022

Record last verified: 2022-10

Locations

US (1)