Phase 2 N=30 Randomized Triple-blind Treatment

Autologous Cord Blood Stem Cells for Autism

Autism

Bottom Line

View on ClinicalTrials.gov: NCT01638819 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Aug 2018

Primary outcome: Primary: Change in Language (Total Standard Score, Range 40 - 160) — 79.62; 79.66; 80.45; 80.59 units on a scale

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Autologous Cord Blood Stem Cells (Biological); Placebo (Biological)
Age: Pediatric · 2+ yrs
Sex: All
Sponsor: Sutter Health
Primary completion: Dec 2015

Outcome Measures

Outcome	Result	p-value
PRIMARY Change in Language (Total Standard Score, Range 40 - 160)	79.62; 79.66; 80.45; 80.59; 74.55; 73.10	—
SECONDARY Change in Behavior/Learning (Standard Score, Range 40 - 160 for Each Subtest)	66.00; 74.27; 67.57; 75.00; 66.00; 79.60	—
SECONDARY Change in Knowledge and Fluid Reasoning (Scaled Score, Range 1-19 for Each Subtest)	5.86; 5.97; 6.79; 6.41; 3.97; 4.45	—

Summary

Evaluate the efficacy of one infusion of stem cells from autologous umbilical cord blood in patients with autism over six months after infusion as measured by changes in expressive and receptive language. Also demonstrate improved behavior, learning, and changes in Serum tumor necrosis factor alpha (TNF-α), tumor necrosis factor beta (TNF-β), interleukin 1-alpha (IL-1α), interleukin 1-beta (IL-1β), interleukin 6 (IL-6), interleukin 10 (IL-10), and interleukin 13 (IL-13).

Eligibility Criteria

Inclusion Criteria

Age 2 to 7 years of age
Diagnosis of Autistic Disorder as diagnosed by the Diagnostic and Statistical Manual, 4th Edition, Text Revision (DSM-IV-TR) developmental delays, and ADOS
A sufficient quantity of autologous cord blood stored at Cord Blood Registry that was stored and processed using the Thermogenesis AutoXpress Platform
Stable on any current medications for at least 2 months prior to infusion of cord blood
Medical records indicating that patient does not have genetic conditions such as cerebral palsy, cystic fibrosis, muscular dystrophy, crohns disease, rheumatoid disease, fragile X, Retts Syndrome, Angelman Syndrome, tuberous sclerosis, epilepsy, or known genetic defects that overlap autism spectrum.
Results of an EEG within 12-months of baseline
English speaking

Exclusion Criteria

CNS infection
Extreme prematurity ( 1.5 mg/dl and total Bilirubin > 1.5 mg/dl
Allergic to diphenhydramine (Benadryl)
Treatment with chelation therapy, hyperbaric oxygen therapy, pig worm therapy, or other alternative therapies the investigator deems clinically relevant

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01638819). 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.