Clinical trial NCT04768816: Clinical Effect and Safety of Autologous Umbilical Cord Blood Transfusion in the Treatment of Autism Spectrum Disorder
Official title: Clinical Effect and Safety of Autologous Umbilical Cord Blood Transfusion in the Treatment of Autism Spectrum Disorder
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
Conditions: Safety Issues;Effect of Drugs
Keywords: Autism Spectrum Disorder;Autologous Umbilical Cord Blood;safety;effect
Lead sponsor: Guangdong Women and Children Hospital
Sponsor class: OTHER
First posted: 2021-02-24
Enrollment: 25
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Parallel Assignment
Primary purpose: TREATMENT

Brief summary: To study the clinical efficacy and safety of autologous umbilical cord blood transfusion in the treatment of autism spectrum disorder.

Detailed description: This is a Phase 1 clinical trial that constitutes two time points cohorts with participants who will receive intravenous autologous umbilical cord blood and 0.9% sodium chloride respectively. The timing of treatment is any time after the diagnosis of autism spectrum disorder. The investigator will proceed the groups during the same period.

1. Demographic Data and Baseline Characteristics of the Studied Group were collected:

   * Basic patient's information survey
   * Medical history
   * Physical examination
   * Basic blood test result
   * Autism Behavior Checklist,CARS before the treatment
   * Brain Magnetic Resonance Imaging-Diffusion Tensor Imaging (MRIDTI) before the treatment
   * Neurocognitive function test before the treatment
2. Assessment of clinical condition in the course by measurement of blood pressure, heart and respiratory rates, temperature and adverse events was recorded.
3. Autologous cord blood doses is 20-30ml (total Mononuclear cells# 1\*10\^7/kg)#the infusion speed is 1ml/min, and with same volume of 0.9% sodium chloride as placebo.
4. The follow-up: clinical test until 24th month in 3 month gaps.

Eligibility criteria: Inclusion Criteria:

* • A patient who was diagnosed with ASD.

Exclusion Criteria:

* • In case a patient underwent a surgical procedure, or was administered autologous cord blood within one year before participating in a clinical trial.

  * Accompanied by a serious disease, such as chromosome abnormality, etc.
  * In case where a patient's medical condition is judged to be maladapted by a researcher.
  * In case a patient or his or her legal representative doesn't agree to participation in a clinical trial.
  * A patient having a predisposition to allergies.
  * A patient having serious disorders in the liver, kidney, and cardiopulmonary function.

Primary outcomes:
measure: ABC
timeFrame: up to 24 months after therapy at a 3-month interval
measure: CARS
timeFrame: up to 24 months after therapy at a 3-month interval
Secondary outcomes:
Other outcomes:
Locations:
city: Guangzhou
country: China
facility: Guangdong Women and Children's Hospital and Health Institute
state: Guangdong
Study officials:
References:
citation: Dawson G, Sun JM, Davlantis KS, Murias M, Franz L, Troy J, Simmons R, Sabatos-DeVito M, Durham R, Kurtzberg J. Autologous Cord Blood Infusions Are Safe and Feasible in Young Children with Autism Spectrum Disorder: Results of a Single-Center Phase I Open-Label Trial. Stem Cells Transl Med. 2017 May;6(5):1332-1339. doi: 10.1002/sctm.16-0474. Epub 2017 Apr 5.
citation: Dawson G, Sun JM, Baker J, Carpenter K, Compton S, Deaver M, Franz L, Heilbron N, Herold B, Horrigan J, Howard J, Kosinski A, Major S, Murias M, Page K, Prasad VK, Sabatos-DeVito M, Sanfilippo F, Sikich L, Simmons R, Song A, Vermeer S, Waters-Pick B, Troy J, Kurtzberg J. A Phase II Randomized Clinical Trial of the Safety and Efficacy of Intravenous Umbilical Cord Blood Infusion for Treatment of Children with Autism Spectrum Disorder. J Pediatr. 2020 Jul;222:164-173.e5. doi: 10.1016/j.jpeds.2020.03.011. Epub 2020 May 19.