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TSO in Pediatric Autistic Spectrum Disorders (TSO)

Primary Purpose

Autistic Spectrum Disorders

Status

Terminated

Phase

Phase 2

Locations

Israel

Study Type

Interventional

Intervention

TSO

Placebo

About this trial

This is an interventional treatment trial for Autistic Spectrum Disorders focused on measuring Autism, Trichuris Suis Ova, Behavior, Irritability

Eligibility Criteria

6 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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

Exclusion Criteria:

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:
1. White blood cell count ≤ 3,000/mm3 (≤ 3.0 x 109/L) or ≥ 14,000/mm3 (≥14 x 109/L)
2. Platelet count ≤ 100,000/μL (≤100 x 109/L)
3. Serum creatinine ≥ 1.5 mg/dL (≥ 132.6 μmol/L) or >2 x upper limit of normal (ULN)
4. AST (SGOT) or ALT (SGPT) > 2 x ULN
5. Total bilirubin >2 mg/dL (34 μmol/L)
6. Hemoglobin < 9 g/dL

Sites / Locations

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

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Active Comparator

Placebo Comparator

Arm Label

TSO 2500

7500 TSO

Placebo

Arm Description

2500 TSO every other week

7500 TSO every other week

placebo every other week.

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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

Secondary assessments of efficacy will be assessed via: • The change from baseline in the Clinical Global Impression - Improvement scale (CGI-I)

Full Information

NCT ID

NCT01734941

First Posted

November 19, 2012

Last Updated

January 31, 2016

Sponsor

Hadassah Medical Organization

1. Study Identification

Unique Protocol Identification Number

NCT01734941

Brief Title

TSO in Pediatric Autistic Spectrum Disorders

Acronym

TSO

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

April 2014

Overall Recruitment Status

Terminated

Why Stopped

Well tolerated but failed to demonstrate significant activity. So it did not meet its primary endpoint or key secondary endpoints.

Study Start Date

April 2014 (undefined)

Primary Completion Date

May 2015 (Actual)

Study Completion Date

May 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Hadassah Medical Organization

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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)

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: Placebo (n= 20 patients). These patients will receive a blinded dose of placebo every other week. 2500 TSO every other week (n= 20 patients). These patients will receive a blinded dose of TSO every other week 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Autistic Spectrum Disorders

Keywords

Autism, Trichuris Suis Ova, Behavior, Irritability

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigator

Allocation

Randomized

Enrollment

9 (Actual)

8. Arms, Groups, and Interventions

Arm Title

TSO 2500

Arm Type

Active Comparator

Arm Description

2500 TSO every other week

Arm Title

7500 TSO

Arm Type

Active Comparator

Arm Description

7500 TSO every other week

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

placebo every other week.

Intervention Type

Drug

Intervention Name(s)

TSO

Other Intervention Name(s)

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

Intervention Description

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

Intervention Type

Drug

Intervention Name(s)

Placebo

Primary Outcome Measure Information:

Title

Aberrant Behavior Checklist (ABC) subscale scores

Description

Time Frame

16 weeks

Secondary Outcome Measure Information:

Title

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

Description

Secondary assessments of efficacy will be assessed via: • The change from baseline in the Clinical Global Impression - Improvement scale (CGI-I)

Time Frame

49 weeks

Other Pre-specified Outcome Measures:

Title

Clinical Laboratory

Description

Clinical laboratories will include hematology and serum chemistry panels, as well as C-reactive protein

Time Frame

49 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

6 Years

Maximum Age & Unit of Time

17 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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 Exclusion Criteria: 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) AST (SGOT) or ALT (SGPT) > 2 x ULN Total bilirubin >2 mg/dL (34 μmol/L) Hemoglobin < 9 g/dL

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Itai Berger, MD

Organizational Affiliation

Hadassah Medical Organization

Official's Role

Principal Investigator

Facility Information:

Facility Name

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

City

Jerusalem

State/Province

Mount Scopus

ZIP/Postal Code

91240

Country

Israel

12. IPD Sharing Statement

Citations:

PubMed Identifier

15591509

Citation

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.

Results Reference

background

PubMed Identifier

21372112

Citation

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.

Results Reference

background

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TSO in Pediatric Autistic Spectrum Disorders

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