Autistic Spectrum DisordersPhase 2TSONCT01734941
TSO in Pediatric Autistic Spectrum Disorders
A Phase 2 study testing TSO in people with Autistic Spectrum Disorders, run by Hadassah Medical Organization at 1 site in Israel.
Trial 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… Read more about this study
- Who is it for?
- Ages 6 to 17 years
- Conditions the study is for
- Autistic Spectrum Disorders
- What is being tested?
- TSO
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 9 participants
- Where
- 1 siteIsrael
- Study dates
- Apr 2014to May 2015
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
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- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country.
Israel1 site
The Neuro-Cognitive Center, Pediatric Division, Hadassah-Hebrew University Medical Center
Jerusalem, Mount Scopus, 91240
About this study
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
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)
Read the 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.
- Conditions
- Autistic Spectrum Disorders
- Being tested
- TSO, Placebo
- Keywords
- Autism, Trichuris Suis Ova, Behavior, Irritability
- Medical subject headings
- Autism Spectrum Disorder, Autistic Disorder, Behavior
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TSODrug
Also called 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
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
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| TSO 2500 | Active comparator | 2500 TSO every other week Receives: Drug: TSO |
| 7500 TSO | Active comparator | 7500 TSO every other week Receives: Drug: TSO |
| Placebo | Placebo comparator | placebo every other week. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Secondary assessments of efficacy will be assessed via:
- The change from baseline in the Clinical Global Impression - Improvement scale (CGI-I)
Measured: 49 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Nov 2012 (estimated)
- Study startApr 2014
- Primary completionMay 2015
- Study completionMay 2015
Why it stopped: Well tolerated but failed to demonstrate significant activity. So it did not meet its primary endpoint or key secondary endpoints.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Autistic Spectrum DisordersStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hadassah Medical Organization
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Itai Berger, MD, Principal investigator, Hadassah Medical Organization
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01734941
- Sponsor's protocol ID
- 0522-12-HMO
- Acronym
- TSO
- Status last verified
- Apr 2014
- First submitted
- 19 Nov 2012
- Last updated
- 2 Feb 2016
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 15591509Fleming 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.
PubMed 21372112
Study at a glance
From the registry record, last updated by the sponsor on 2 Feb 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01734941
- Acronym / sponsor ID
- TSO / 0522-12-HMO
- Conditions
- Autistic Spectrum Disorders
- Keywords
- Autism, Trichuris Suis Ova, Behavior, Irritability
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Well tolerated but failed to demonstrate significant activity. So it did not meet its primary endpoint or key secondary endpoints.
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Hadassah Medical OrganizationUniversity, hospital or other
- Study start
- Apr 2014
- Primary completion
- May 2015 (actual)
- Study completion
- May 2015 (actual)
- First posted
- 28 Nov 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| TSO 2500 | Active comparator | Drug: TSO2500 TSO every other week |
| 7500 TSO | Active comparator | Drug: TSO7500 TSO every other week |
| Placebo | Placebo comparator | Drug: Placeboplacebo every other week. |
Who may qualify
- Ages
- 6 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- 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
- CGI-Severity score > 4 and ABC irritability score > 18
- Mental age of > 18 months
- Weight of at least ** kg
Key exclusion criteria (18 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TSOAlso: 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 centerThere 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 | 7500 TSO, TSO 2500 |
| Placebo | Drug | Placebo |
Primary outcome
Aberrant Behavior Checklist (ABC) subscale scores
Time frame: 16 weeks
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.
1 secondary outcome
Secondary assessments of efficacy will be assessed via: The change from baseline in the CY-BOCS, CGI-I, SRS, SCQ.
Time frame: 49 weeks
Results and safety
The study was stopped early (reason given: Well tolerated but failed to demonstrate significant activity. So it did not meet its primary endpoint or key secondary endpoints.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Feb 2016.
- Background: PubMed 15591509Summers 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.
- Background: PubMed 21372112Fleming 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Itai Berger, MD principal investigator
Hadassah Medical Organization
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing TSO in people with Autistic Spectrum Disorders, run by Hadassah Medical Organization at 1 site in Israel.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 2 Feb 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Israel. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Well tolerated but failed to demonstrate significant activity. So it did not meet its primary endpoint or key secondary endpoints.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Aberrant Behavior Checklist (ABC) subscale scores (time frame: 16 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01734941, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Feb 2016.