TSO in Pediatric Autistic Spectrum Disorders

NCT01734941 | Terminated Phase 2 DMC

• 1 other identifier: 0522-12-HMO
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

The primary objective of this study is to evaluate and compare the safety and efficacy of Trichuris suis ova (TSO) therapy (versus placebo) in pediatric patients with autism. Evaluation of the safety and tolerability of treatment with TSO in the target population across the dose range being tested is considered a primary objective, while the primary efficacy objective will be assessed via the change from baseline in the Aberrant Behavior Checklist (ABC) subscale scores. Dose response will be considered a primary objective as well. Secondary assessments of efficacy will be assessed via: • The change from baseline in the Clinical Global Impression scale (CGI-I)

Conditions

Autistic Spectrum Disorders

Keywords

Autism; Trichuris Suis Ova; Behavior; Irritability

Outcome Measures

Primary Outcomes (1)

• Aberrant Behavior Checklist (ABC) subscale scores

  The ABC consists of 58 questions and the five subscales as described above. Each question on the ABC is rated on a 4-point scale: 0 = 'not a problem,' 1 ='the behavior is a problem but slight in degree,' 2 = 'the problem is moderately serious,' and 3 = 'the problem is severe in degree.' The subscale score is the sum of the responses to the questions that make up the subscale.

  16 weeks

Secondary Outcomes (1)

• Secondary assessments of efficacy will be assessed via: The change from baseline in the CY-BOCS, CGI-I, SRS, SCQ.

  49 weeks

Other Outcomes (1)

• Clinical Laboratory

  49 weeks

Study Arms (3)

TSO 2500

ACTIVE COMPARATOR

2500 TSO every other week

Drug: TSO

7500 TSO

ACTIVE COMPARATOR

7500 TSO every other week

Drug: TSO

Placebo

PLACEBO COMPARATOR

placebo every other week.

Drug: Placebo

Interventions

TSO DRUG

There is evidence of a relationship between ASD symptomatology and immune dysfunction suggests that immunomodulatory treatments effective in other autoimmune disorders might be investigated in ASD, including Trichuris Ova Suis (TSO), a helminth porcine whipworm

Also known as: Each dose of 2500 and 7500 active Trichuris suis ova will be provided, in 15 mL of aqueous suspension (supplied in 30 mL glass container), or matching placebo administered orally at the investigational center

7500 TSO; TSO 2500

Placebo DRUG

Placebo

Eligibility Criteria

• Age: 6 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Males or females, ages 6 to 17 years, inclusive
• Diagnosis via Diagnostic and Statistical Manual for Mental Disorders- Forth Edition (DSM-IV)-and confirmed by Autism Diagnostic Observation
• Schedule (ADOSI):
• CGI-Severity score \> 4 and ABC irritability score \> 18
• Mental age of \> 18 months
• Weight of at least \*\* kg
• Currently psychotropic medication free or on stable dose of psychotropic medication for at least 3 months prior to the study.
• Willing to comply with the schedule of study visits and protocol requirements
• Patient and/or guardian have the ability to provide informed consent

You may not qualify if:

• Previous diagnosis of Rett's Disorder, Aspergers Disorder, Childhood Disintegrative Disorder, Fragile X Syndrome, or other disorders on the autism spectrum
• History of Bipolar Disorder, Psychotic Disorders, or major Depression
• Seizure within the previous 6 months
• Patient received antibiotic, antifungal or antiparasitic medication in the last 2 weeks prior to Screening and/or would potentially require this during the study treatment period
• Patient with history of drug or alcohol abuse within 6 months prior to Screening
• Patient with evidence of poor compliance with medical advice and instruction including diet or medication
• Patient is unable or unwilling to swallow study medication suspension
• Patient with a significant medical condition which puts the patient at risk for study participation and/or for any reason is considered by the Investigator to be an unsuitable candidate to receive TSO or is potentially put at risk by study procedures
• Patient who has participated in another clinical trial within 30 days of Screening for this trial and/or any experimental treatment for this population
• Females of childbearing potential who are unwilling or unable to use an acceptable method to avoid pregnancy for the entire study period
• Females who are pregnant or breastfeeding at the time of enrollment
• Patients with any of the following laboratory values:
• White blood cell count ≤ 3,000/mm3 (≤ 3.0 x 109/L) or ≥ 14,000/mm3 (≥14 x 109/L)
• Platelet count ≤ 100,000/μL (≤100 x 109/L)
• Serum creatinine ≥ 1.5 mg/dL (≥ 132.6 μmol/L) or \>2 x upper limit of normal (ULN)

Sponsors & Collaborators

• Hadassah Medical Organization: lead

Study Sites (1)

The Neuro-Cognitive Center, Pediatric Division, Hadassah-Hebrew University Medical Center

Jerusalem, Mount Scopus, 91240, Israel

Location

Related Publications (2)

• Summers RW, Elliott DE, Urban JF Jr, Thompson R, Weinstock JV. Trichuris suis therapy in Crohn's disease. Gut. 2005 Jan;54(1):87-90. doi: 10.1136/gut.2004.041749.

  PMID: 15591509 BACKGROUND

• Fleming JO, Isaak A, Lee JE, Luzzio CC, Carrithers MD, Cook TD, Field AS, Boland J, Fabry Z. Probiotic helminth administration in relapsing-remitting multiple sclerosis: a phase 1 study. Mult Scler. 2011 Jun;17(6):743-54. doi: 10.1177/1352458511398054. Epub 2011 Mar 3.

  PMID: 21372112 BACKGROUND

MeSH Terms

Conditions

Autism Spectrum Disorder; Autistic Disorder; Behavior

Interventions

Equipment and Supplies

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Study Officials

• Itai Berger, MD

  Hadassah Medical Organization

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 19, 2012
• First Posted: November 28, 2012
• Study Start: April 1, 2014
• Primary Completion: May 1, 2015
• Study Completion: May 1, 2015
• Last Updated: February 2, 2016

Record last verified: 2014-04

Locations

IL (1)