Autologous Umbilical Cord Blood Infusion for Children With Autism Spectrum Disorder (ASD)

Autism Spectrum Disorder Clinical Trial

Official title:

Autologous Umbilical Cord Blood Infusion for Children With Autism Spectrum Disorder (ASD)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02176317
• Other study ID #: Pro00052449
• Status: Completed
• Phase: Phase 1
• Start date: June 2014
• Est. completion date: December 2015

Study information

• Verified date: December 2019
• Source: Duke University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This study is a prospective phase 1 single-center trial designed to determine the safety of a single intravenous infusion of autologous umbilical cord blood in children with Autism Spectrum Disorder (ASD) and assess the feasibility of various outcome measures to determine which measure(s) can be used as primary and secondary endpoints for a future randomized phase 2 clinical trial. All subjects will receive infusion of cord blood cells at baseline with follow up assessments at 6 and 12 months.

Description:

Autism Spectrum Disorder (ASD) is a neurodevelopment disorder with early onset in life. Currently, available treatments for patients with ASD are supportive, but not curative. Umbilical cord blood (UCB) has been shown to lessen the clinical and radiographic impact of hypoxic brain injury and stroke in animal models and in infants with hypoxic ischemic encephalopathy. UCB also engrafts and differentiates in the brain, facilitating neural cell repair in animal models and human patients with inborn errors of metabolism undergoing allogeneic, unrelated donor UCB transplantation. Infusion of autologous UCB does not require immunosuppression and has been shown to be safe in young children with brain injuries such as cerebral palsy and stroke. In this study, the investigators hypothesize that infusion of a patient's own umbilical cord blood cells (UCB) can offer neural protection/repair in the brain and reduction of inflammation associated with this disorder.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 25
• Est. completion date: December 2015
• Est. primary completion date: December 2015
• Accepts healthy volunteers: No
• Gender: All
• Age group: 24 Months to 72 Months

Eligibility

Inclusion Criteria:

1. Age = 24 months to =72months at the time of visit 1

2. Confirmed clinical DSM-5 diagnosis of Autism Spectrum Disorder using all three of the following measures:

• Autism Diagnostic Observation Schedule - Toddler or Generic (ADOS)

• Autism Diagnostic Interview-Revised (ADI-R)

• DSM-5 checklist

3. IQ = 35 on Stanford Binet Intelligence Scale or similar standardized test

4. Autologous umbilical cord blood available from a cord blood bank with a minimum total nucleated cell dose of = 1 x 107 cells/kilogram of subject weight that meets acceptance criteria outlined in section 6.0 with confirmed HLA matching

5. Stable on current medications for at least 2 months prior to infusion of cord blood

6. Ability to travel to Duke University three times (0, 6, 12 mo.), parent/guardian able to participate in electronic communication tracking two times in the study and interim phone surveys every 3 months

7. Parental consent

8. Subject and parent/guardian must be English speaking

Exclusion Criteria:

1. Unwilling to commit to follow up for a year

2. History of prior cell therapy

3. Use of IVIG or other anti-inflammatory medications with the exception of NSAIDs

4. Medical records indicate that child has genetic or other syndromes such as fragile X, neurofibromatosis, Rett syndrome, tuberous sclerosis, PTEN mutation, cerebral palsy, cystic fibrosis, muscular dystrophy, Crohn's disease, or rheumatoid disease

5. Co-morbid condition that would influence child's performance on assessments.

6. Central Nervous System (CNS) infection

7. History of unstable epilepsy or uncontrolled seizure disorder, infantile spasms, Lennox Gastaut syndrome, Dravet syndrome

8. Known pathogenic copy number variation (CNV) (e.g. 16p11.2, 15q13.2, 2q13.3)

9. Significant sensory (i.e., deafness, blind) or motor impairment (CP) (if using Language Environment Analysis (LENA), no uncorrected hearing impairment)

10. Presence of obvious physical dysmorphology

11. Review of medical records indicates ASD diagnosis not likely or other serious complicating genetic or medical condition present

12. Impaired renal or liver function as determined by serum creatinine >1.5mg/dL and/or total bilirubin>1.3mg/dL

13. Clinically significant abnormalities in Complete Blood Count (CBC): Hemoglobin < 10.0 g/dL, White Blood Count (WBC) < 3.8 x 10e9, Platelets < 150x 10e9.

14. Known metabolic disorder, mitochondrial dysfunction

15. Uncontrolled infection, presence of or infection with HIV

16. Active malignancy

17. Macroencephaly or microencephaly ( >2 standard deviations in the relevant direction between head circumference and height)

18. Change in current stable use of psychoactive medications; as per parent report.

Related Conditions & MeSH terms

• Autism Spectrum Disorder
• Autistic Disorder
• Child Development Disorders, Pervasive
• Disease

Intervention

Biological:
• Autologous Umbilical Cord Blood
All participants will receive autologous umbilical cord blood cells with a pre-cryopreservation cell dose of 1-5 x 10^7 Total Nucleated Cells (TNC)/kilogram of subject body weight. The cells will be administered as a single intravenous (into the vein) infusion over 2 to 25 minutes

Locations

Country	Name	City	State
United States	Duke University Medical Center	Durham	North Carolina

Sponsors (3)

Lead Sponsor	Collaborator
Duke University	PerkinElmer, Inc., The Marcus Foundation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Change in Pervasive Developmental Disorder- Behavior Inventory		up to 12 months
Other	Change in Repetitive Behavior Scal		up to 12 months
Other	Change in Sensory Experience Questionnaire		up to 12 months
Other	Change in Behavior Assessment of Children		up to 12 months
Other	Change in Autism Diagnostic Observation Scale		up to 12 months
Other	Change in Clinical Global Impression- Severity and Improvement Scales		up to 12 months
Other	Change in Stanford Binet Intelligence Scale or other standardized IQ test		up to 12 months
Other	Change in frequency of child vocalization/ conversational turns		up to 12 months
Other	Change in Expressive One-Word Picture Vocabulary Test		up to 12 months
Other	Change in Preschool Age Psychiatric Assessment		up to 12 months
Other	Change in Aberrant Behavior Checklist		up to 12 months
Other	Prevalent and incident of GI symptoms		up to 12 months
Other	Change in Parenting Stress Index		up to 12 months
Other	Change in attention to social stimuli assessed via eye-tracking and electroencephalography (EEG)		up to 12 months
Primary	Number of participants with non-serious and serious adverse events.		up to 12 months
Secondary	The primary efficacy measure will be change in the Vineland Adaptive Behavior Scale- II		up to 12 months