Long-term Interventional Follow-up Study of Children With Prader-Willi Syndrome Included in the OTBB3 Clinical Trial (OTBB3-FU)

April 29, 2024 updated by: University Hospital, Toulouse

Long-term Interventional Follow-up Study up to 4 Years of Age of Children With Prader-Willi Syndrome Included in the OTBB3 Clinical Trial and Comparison With an Untreated Cohort of Children With Prader-Willi Syndrome

This study is a prospective, multicentre, interventional cohort study in children with Prader-Willi Syndrome (PWS) over 4 years (no treatment administered). The duration of the preceding OTTB3 study is 26 weeks. An untreated cohort of children with PWS will be included at an age of 2 years and followed up until an age of 4 years.

Regarding the untreated cohort, children with PWS born in France and too old to be recruited in OTBB3 trial, principally those who were born within one year before the start of OTBB3 trial, will be offered to participate in this study. Infants born later who couldn't be included in OTBB3 study will be also offered to participate.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

80

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 3 years (Child)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Male or female child with a genetically confirmed diagnosis of PWS (patients can be enrolled if the genetic subtype is not available at inclusion, but the genetic subtype needs to be confirmed during the study);
  2. The parents (or legal representative) must have signed the consent form;
  3. Treated cohort: the child participated in the OTBB3 study and is aged 16±4 months at inclusion,
  4. Untreated cohort: the child has never received OT, is aged 30±6 months at inclusion (in order to maximise the number of children in the untreated cohort) and is followed in France.

Exclusion Criteria:

  1. Administrative problems:

    1. Inability for the parents (or legal representative) to understand/fulfil study requirements;
    2. No coverage by a social security regime;
  2. Refusal of parents (or legal representative) to sign the consent form;

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Other
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: Untreated cohort
babies not included in the OTBB3 study and therefore never treated with Oxytocin
Other: Follow-up study of the untreated cohort
follow-up study of the patients in the untreated cohort: that have NOT been included in the otbb3 study
Other: OXYTOCIN (OT) Treated cohort
babies treated with Oxytocin during the OTBB3 study
Drug: Follow-up study of the treated cohort
follow-up study of the patients in the treated cohort: that have been included in the otbb3 study

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Confirmation of the long term safety profile (1)
Time Frame: 4 years
The number of patients with adverse events (AEs)
4 years
Confirmation of the long term safety profile (2)
Time Frame: 4 years
The percentage of patients with adverse events (AEs)
4 years
Confirmation of the long term safety profile (3)
Time Frame: 4 years
Assessment in the treated cohort of the occurrence of the main comorbidities in Prader Willi Syndrome
4 years
Confirmation of the long term safety profile (4)
Time Frame: 4 years

Assessment in the treated cohort of:

The occurrence of medications, surgery and rehabilitations by collecting type, age (years) at start and stop, dosing or frequency
4 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Complete the safety assessment by the description of the development of the child (1.1)
Time Frame: 4 years
Assessment in the treated cohort of: weight (kilograms)
4 years
Complete the safety assessment by the description of the development of the child (1.2)
Time Frame: 4 years
Assessment in the treated cohort of: height (meters)
4 years
Complete the safety assessment by the description of the development of the child (1.3)
Time Frame: 4 years
Assessment in the treated cohort of: BMI (kg/m^2)
4 years
Complete the safety assessment by the description of the development of the child (2.1)
Time Frame: 4 years
Assessment in the treated cohort of Child development: age at which sitting has been reached
4 years
Complete the safety assessment by the description of the development of the child (2.2)
Time Frame: 4 years
Assessment in the treated cohort of Child development: age at which crawling has been reached
4 years
Complete the safety assessment by the description of the development of the child (2.3)
Time Frame: 4 years
Assessment in the treated cohort of Child development: age at which walking has been reached
4 years
Complete the safety assessment by the description of the development of the child (2.4)
Time Frame: 4 years
Assessment in the treated cohort of Child development: age at which running has been reached
4 years
Complete the safety assessment by the description of the severity of the disease
Time Frame: 4 years

Severity of the disease for:

Eating disorders by using Hyperphagia Questionnaire for Clinical Trials (HQCT);
4 years
Complete the safety assessment by the description of the severity of the disease (2)
Time Frame: 4 years

Severity of the disease for:

Psychiatric disorders by using the Child Behaviour Checklist (CBCL);
4 years
Assessment of endocrine disorders by IGF1
Time Frame: 4 years
Analysis of plasma Insulin-like growth factor 1 (IGF1, ng/mL)
4 years
Assessment of endocrine disorders by TSH
Time Frame: 4 years
Analysis of plasma thyroid stimulating hormone (TSH, µUI/mL)
4 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University Hospital, Toulouse

Investigators

  • Principal Investigator: Maithe TAUBER, MD, University Hospital, Toulouse

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 7, 2021

Primary Completion (Estimated)

April 1, 2025

Study Completion (Estimated)

April 1, 2025

Study Registration Dates

First Submitted

June 24, 2021

First Submitted That Met QC Criteria

August 26, 2021

First Posted (Actual)

September 2, 2021

Study Record Updates

Last Update Posted (Actual)

April 30, 2024

Last Update Submitted That Met QC Criteria

April 29, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RC31/20/0421

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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