NCT01734941

TSO in Pediatric Autistic Spectrum Disorders

A Phase 2 Randomized Three-Arm Double-Blind Placebo-Controlled Study to Evaluate the Safety and Efficacy of 16 Weeks of Treatment With Trichuris Suis Ova (TSO) Therapy in Pediatric Patients Ages 6 to 17 With Autism

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: Hadassah Medical Organization · Academic / Other
• Sex: All
• Age: 6 Years – 17 Years
• Healthy volunteers: Not accepted

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)

Detailed description

This is a randomized, three-arm double-blind, placebo-controlled, single-center study to evaluate the effects of oral administration of Trichuris suis ova (as compared to placebo) in the treatment of pediatric patients diagnosed with Autism. The target sample size to be randomized into the study will be approximately 60, randomly assigned in a 1:1:1 ratio to one of three treatment groups: 1. Placebo (n= 20 patients). These patients will receive a blinded dose of placebo every other week. 2. 2500 TSO every other week (n= 20 patients). These patients will receive a blinded dose of TSO every other week 3. 7500 TSO every other week (n= 20 patients). These patients will receive a blinded dose of TSO every other week Double-blind treatment will be given for a total of 16 weeks. This study will have 3 phases: * Screening period, comprising up to 5 weeks prior to Baseline (Day 1) * Double-blind treatment period for 16 weeks * An untreated follow-up period for 26 weeks. Following informed consent, patients will be screened on the basis of diagnosis of autism, vital signs, clinical laboratories medical history and a physical examination. Eligible patients will be randomized to double-blind treatment with TSO 2500 every other week, TSO 7500 every other week, or placebo every other week, in a ratio of 1:1:1. During the double-blind study phase, study drug will be provided in the clinic in a liquid form and will be administered every other week, starting with the Baseline visit, through Week 14. Week 14 is the last double-blind treatment administration of the study, while Week 16 is the primary time point for assessment of efficacy. Patients will return to the clinic every other week during the double blind treatment period. After completion of the double-blind phase, patients will then return to the clinic 26 weeks following the last dose of study medication for a safety assessment and stool sample culture.

Conditions

• Autistic Spectrum Disorders

Interventions

Type	Name	Description
DRUG	TSO	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
DRUG	Placebo

Timeline

• Start date: 2014-04-01
• Primary completion: 2015-05-01
• Completion: 2015-05-01
• First posted: 2012-11-28
• Last updated: 2016-02-02

Locations

1 site across 1 country: Israel