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TSO in Pediatric Autistic Spectrum Disorders

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 2014 by Hadassah Medical Organization
Sponsor:
Information provided by (Responsible Party):
Hadassah Medical Organization
ClinicalTrials.gov Identifier:
NCT01734941
First received: November 19, 2012
Last updated: April 16, 2014
Last verified: April 2014
  Purpose

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)


Condition Intervention Phase
Autistic Spectrum Disorders
Drug: TSO
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Hadassah Medical Organization:

Primary Outcome Measures:
  • Aberrant Behavior Checklist (ABC) subscale scores [ Time Frame: 16 weeks ] [ Designated as safety issue: No ]
    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. [ Time Frame: 49 weeks ] [ Designated as safety issue: No ]

    Secondary assessments of efficacy will be assessed via:

    • The change from baseline in the Clinical Global Impression - Improvement scale (CGI-I)



Other Outcome Measures:
  • Clinical Laboratory [ Time Frame: 49 weeks ] [ Designated as safety issue: Yes ]
    Clinical laboratories will include hematology and serum chemistry panels, as well as C-reactive protein


Estimated Enrollment: 60
Study Start Date: April 2014
Estimated Study Completion Date: December 2015
Estimated Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: TSO 2500
2500 TSO every other week
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
Other Names:
  • 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
Active Comparator: 7500 TSO
7500 TSO every other week
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
Other Names:
  • 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
Placebo Comparator: Placebo
placebo every other week.
Drug: Placebo

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.

  Eligibility

Ages Eligible for Study:   6 Years to 17 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Males or females, ages 6 to 17 years, inclusive
  2. Diagnosis via Diagnostic and Statistical Manual for Mental Disorders- Forth Edition (DSM-IV)-and confirmed by Autism Diagnostic Observation

    Schedule (ADOSI):

  3. CGI-Severity score > 4 and ABC irritability score > 18
  4. Mental age of > 18 months
  5. Weight of at least ** kg
  6. Currently psychotropic medication free or on stable dose of psychotropic medication for at least 3 months prior to the study.
  7. Willing to comply with the schedule of study visits and protocol requirements
  8. Patient and/or guardian have the ability to provide informed consent

Exclusion Criteria:

  1. Previous diagnosis of Rett's Disorder, Aspergers Disorder, Childhood Disintegrative Disorder, Fragile X Syndrome, or other disorders on the autism spectrum
  2. History of Bipolar Disorder, Psychotic Disorders, or major Depression
  3. Seizure within the previous 6 months
  4. 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
  5. Patient with history of drug or alcohol abuse within 6 months prior to Screening
  6. Patient with evidence of poor compliance with medical advice and instruction including diet or medication
  7. Patient is unable or unwilling to swallow study medication suspension
  8. 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
  9. Patient who has participated in another clinical trial within 30 days of Screening for this trial and/or any experimental treatment for this population
  10. Females of childbearing potential who are unwilling or unable to use an acceptable method to avoid pregnancy for the entire study period
  11. Females who are pregnant or breastfeeding at the time of enrollment
  12. 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01734941

Contacts
Contact: Itai Berger, MD +972-2-584-4751 itberg@hadassah.org.il
Contact: Ofra Haviv, MA +972-2-584-4903 neuro.cog.c@gmail.com

Locations
Israel
The Neuro-Cognitive Center, Pediatric Division, Hadassah-Hebrew University Medical Center Recruiting
Jerusalem, Mount Scopus, Israel, 91240
Contact: Ofra Haviv, MA    +972-2-584-4903    neuro.cog.c@gmail.com   
Principal Investigator: Itai Berger, MD         
Sponsors and Collaborators
Hadassah Medical Organization
Investigators
Principal Investigator: Itai Berger, MD Hadassah Medical Organization
  More Information

Publications:
Responsible Party: Hadassah Medical Organization
ClinicalTrials.gov Identifier: NCT01734941     History of Changes
Other Study ID Numbers: 0522-12-HMO
Study First Received: November 19, 2012
Last Updated: April 16, 2014
Health Authority: Israel: Ministry of Health

Keywords provided by Hadassah Medical Organization:
Autism
Trichuris Suis Ova
Behavior
Irritability

Additional relevant MeSH terms:
Autistic Disorder
Child Development Disorders, Pervasive
Disease
Mental Disorders
Mental Disorders Diagnosed in Childhood
Pathologic Processes

ClinicalTrials.gov processed this record on November 24, 2014