Clinical trial NCT01502488: Adipose Derived Stem Cell Therapy for Autism
Official title: An Open-label, Non-Randomized, Multi-Center Study to Assess the Safety and Effects of Autologous Adipose-Derived Stromal Cells Delivered Intravenously in Patients With Autism
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE1
PHASE2
Conditions: Autism
Keywords: Autistic
ASC therapy
ADSC
Lead sponsor: Ageless Regenerative Institute
Sponsor class: INDUSTRY
Collaborators: name: Instituto de Medicina Regenerativa
First posted: 2011-12-30
Enrollment: 0
Sex: ALL
Minimum age (years): 3
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The intent of this clinical study is to answer the questions:

1. Is the proposed treatment safe
2. Is treatment effective in improving the disease pathology of patients with Autism.

Detailed description: This will be an open-label, non-randomized multi-center patient sponsored study of ASC implantation after liposuction using an IV delivery system. ASCs will be derived from the patient's adipose-derived tissue. Liposuction using local anesthesia and syringe collection will be performed to collect the adipose tissue specimen for subsequent processing to isolate the stem cells. The cells will be delivered intravenously.

Eligibility criteria: Inclusion Criteria:

* Children between the ages of 3 and 12 years.
* DSM-IV diagnosis of Autistic Disorder.
* Total score of CARS ≥ 30.
* Parents or legal guardian willing to sign the ICF.

Exclusion Criteria:

* History of prior or current DSM-IV psychotic disorder (e.g., schizophrenia, bipolar disorder, other psychosis), Pervasive Developmental Disorder not otherwise specified (PDD NOS), Asperger's, or Rett's.
* History of Epileptic seizure activity in the past 6 months.
* Autism caused by seizure disorders (active), cerebrovascular disease or brain trauma.
* The global autism ratings are assessed as being absent, minimal or mild.
* Existing moderate or severe extrapyramidal symptoms (EPS) or history of tardive dyskinesia.
* Active infectious disease and/or known to have tested positive for HIV, HTLV, HBV, HCV, CMV (IgM \> IgG) and/or syphilis will have expert consultation to determine eligibility based on the patient's infectious status
* Enrollment in other trials in the last 3 months without agreement to discontinue them.
* Life expectancy \< 6 months due to concomitant illnesses.
* Any illness which, in the Investigator's judgment, will interfere with the patient's ability to comply with the protocol, compromise patient safety, or interfere with the interpretation of the study results
* Patients on chronic immunosuppressive transplant therapy
* Active clinical infection within one week of enrollment.
* Known drug or alcohol dependence or any other factors which will interfere with the study conduct or interpretation of the results or who in the opinion of the investigator are not suitable to participate.
* History of cancer
* Parental unwillingness and/or not able to give written informed consent.

Primary outcomes:
measure: Improvement in the Childhood Autism Rating Scale，CARS
timeFrame: 3 months
measure: Improvement in the Clinical Global Impression Scale, CGI
timeFrame: 3 months
measure: Improvement in the Childhood Autism Rating Scale，CARS
timeFrame: 6 months
measure: Improvement in the Clinical Global Impression Scale, CGI
timeFrame: 6 months
Secondary outcomes:
measure: Improvement in the Aberrant Behavior Checklist, ABC
timeFrame: 3 months
measure: Improvement in the Aberrant Behavior Checklist, ABC
timeFrame: 6 months
Other outcomes:
Locations:
city: Aventura
country: United States
facility: Ageless Regenerative Institute LLC
state: Florida
Study officials:
affiliation: Ageless Regenerative Institute
name: Sharon McQuillan, MD
role: PRINCIPAL_INVESTIGATOR
References: