Back to resultsLong-term Interventional Follow-up Study of Children With Prader-Willi Syndrome Included in the OTBB3 Clinical Trial (OTBB3-FU)
Primary Purpose
Prader-Willi Syndrome
Status
Recruiting
Phase
Phase 3
Locations
France
Study Type
Interventional
Intervention
Follow-up study of the treated cohort
Follow-up study of the untreated cohort
Sponsored by
About this trial
This is an interventional other trial for Prader-Willi Syndrome focused on measuring Prader-Willi syndrome, Oxytocin
Eligibility Criteria
Inclusion Criteria:
- 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);
- The parents (or legal representative) must have signed the consent form;
- Treated cohort: the child participated in the OTBB3 study and is aged 16±4 months at inclusion,
- 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:
Administrative problems:
- Inability for the parents (or legal representative) to understand/fulfil study requirements;
- No coverage by a social security regime;
- Refusal of parents (or legal representative) to sign the consent form;
Sites / Locations
- Hôpital Femme Mère EnfantRecruiting
- CHU Dijon Hôpital des EnfantsRecruiting
- CHU de GrenobleRecruiting
- Hôpital Jeanne de FlandreRecruiting
- Hôpital de la Timone EnfantRecruiting
- CHU NancyRecruiting
- CHU NantesRecruiting
- Hôpital CHU-LenvalRecruiting
- Groupe Hospitalier Necker - Enfants MaladesRecruiting
- CHU RennesRecruiting
- CHU RouenRecruiting
- Centre de réfrence Prader-Willi, Hospital of infantsRecruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Other
Other
Arm Label
OXYTOCIN (OT) Treated cohort
Untreated cohort
Arm Description
babies treated with Oxytocin during the OTBB3 study
babies not included in the OTBB3 study and therefore never treated with Oxytocin
Outcomes
Primary Outcome Measures
Confirmation of the long term safety profile (1)
The number of patients with adverse events (AEs)
Confirmation of the long term safety profile (2)
The percentage of patients with adverse events (AEs)
Confirmation of the long term safety profile (3)
Assessment in the treated cohort of the occurrence of the main comorbidities in Prader Willi Syndrome
Confirmation of the long term safety profile (4)
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
Secondary Outcome Measures
Complete the safety assessment by the description of the development of the child (1.1)
Assessment in the treated cohort of: weight (kilograms)
Complete the safety assessment by the description of the development of the child (1.2)
Assessment in the treated cohort of: height (meters)
Complete the safety assessment by the description of the development of the child (1.3)
Assessment in the treated cohort of: BMI (kg/m^2)
Complete the safety assessment by the description of the development of the child (2.1)
Assessment in the treated cohort of Child development: age at which sitting has been reached
Complete the safety assessment by the description of the development of the child (2.2)
Assessment in the treated cohort of Child development: age at which crawling has been reached
Complete the safety assessment by the description of the development of the child (2.3)
Assessment in the treated cohort of Child development: age at which walking has been reached
Complete the safety assessment by the description of the development of the child (2.4)
Assessment in the treated cohort of Child development: age at which running has been reached
Complete the safety assessment by the description of the severity of the disease
Severity of the disease for:
Eating disorders by using Hyperphagia Questionnaire for Clinical Trials (HQCT);
Complete the safety assessment by the description of the severity of the disease (2)
Severity of the disease for:
Psychiatric disorders by using the Child Behaviour Checklist (CBCL);
Assessment of endocrine disorders by IGF1
Analysis of plasma Insulin-like growth factor 1 (IGF1, ng/mL)
Assessment of endocrine disorders by TSH
Analysis of plasma thyroid stimulating hormone (TSH, µUI/mL)
Full Information
NCT ID
NCT05032326
First Posted
June 24, 2021
Last Updated
March 29, 2023
Sponsor
University Hospital, Toulouse
1. Study Identification
Unique Protocol Identification Number
NCT05032326
Brief Title
Long-term Interventional Follow-up Study of Children With Prader-Willi Syndrome Included in the OTBB3 Clinical Trial
Acronym
OTBB3-FU
Official Title
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
Study Type
Interventional
2. Study Status
Record Verification Date
March 2023
Overall Recruitment Status
Recruiting
Study Start Date
September 7, 2021 (Actual)
Primary Completion Date
April 1, 2025 (Anticipated)
Study Completion Date
April 1, 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University Hospital, Toulouse
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Prader-Willi Syndrome
Keywords
Prader-Willi syndrome, Oxytocin
7. Study Design
Primary Purpose
Other
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
80 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
OXYTOCIN (OT) Treated cohort
Arm Type
Other
Arm Description
babies treated with Oxytocin during the OTBB3 study
Arm Title
Untreated cohort
Arm Type
Other
Arm Description
babies not included in the OTBB3 study and therefore never treated with Oxytocin
Intervention Type
Drug
Intervention Name(s)
Follow-up study of the treated cohort
Intervention Description
follow-up study of the patients in the treated cohort: that have been included in the otbb3 study
Intervention Type
Other
Intervention Name(s)
Follow-up study of the untreated cohort
Intervention Description
follow-up study of the patients in the untreated cohort: that have NOT been included in the otbb3 study
Primary Outcome Measure Information:
Title
Confirmation of the long term safety profile (1)
Description
The number of patients with adverse events (AEs)
Time Frame
4 years
Title
Confirmation of the long term safety profile (2)
Description
The percentage of patients with adverse events (AEs)
Time Frame
4 years
Title
Confirmation of the long term safety profile (3)
Description
Assessment in the treated cohort of the occurrence of the main comorbidities in Prader Willi Syndrome
Time Frame
4 years
Title
Confirmation of the long term safety profile (4)
Description
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
Time Frame
4 years
Secondary Outcome Measure Information:
Title
Complete the safety assessment by the description of the development of the child (1.1)
Description
Assessment in the treated cohort of: weight (kilograms)
Time Frame
4 years
Title
Complete the safety assessment by the description of the development of the child (1.2)
Description
Assessment in the treated cohort of: height (meters)
Time Frame
4 years
Title
Complete the safety assessment by the description of the development of the child (1.3)
Description
Assessment in the treated cohort of: BMI (kg/m^2)
Time Frame
4 years
Title
Complete the safety assessment by the description of the development of the child (2.1)
Description
Assessment in the treated cohort of Child development: age at which sitting has been reached
Time Frame
4 years
Title
Complete the safety assessment by the description of the development of the child (2.2)
Description
Assessment in the treated cohort of Child development: age at which crawling has been reached
Time Frame
4 years
Title
Complete the safety assessment by the description of the development of the child (2.3)
Description
Assessment in the treated cohort of Child development: age at which walking has been reached
Time Frame
4 years
Title
Complete the safety assessment by the description of the development of the child (2.4)
Description
Assessment in the treated cohort of Child development: age at which running has been reached
Time Frame
4 years
Title
Complete the safety assessment by the description of the severity of the disease
Description
Severity of the disease for:
Eating disorders by using Hyperphagia Questionnaire for Clinical Trials (HQCT);
Time Frame
4 years
Title
Complete the safety assessment by the description of the severity of the disease (2)
Description
Severity of the disease for:
Psychiatric disorders by using the Child Behaviour Checklist (CBCL);
Time Frame
4 years
Title
Assessment of endocrine disorders by IGF1
Description
Analysis of plasma Insulin-like growth factor 1 (IGF1, ng/mL)
Time Frame
4 years
Title
Assessment of endocrine disorders by TSH
Description
Analysis of plasma thyroid stimulating hormone (TSH, µUI/mL)
Time Frame
4 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
12 Months
Maximum Age & Unit of Time
36 Months
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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);
The parents (or legal representative) must have signed the consent form;
Treated cohort: the child participated in the OTBB3 study and is aged 16±4 months at inclusion,
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:
Administrative problems:
Inability for the parents (or legal representative) to understand/fulfil study requirements;
No coverage by a social security regime;
Refusal of parents (or legal representative) to sign the consent form;
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Maithé TAUBER, MD
Phone
534 55 85 51
Ext
+33
Email
tauber.mt@chu-toulouse.fr
First Name & Middle Initial & Last Name or Official Title & Degree
Julie CORTADELLAS
Phone
534 55 85 51
Ext
+33
Email
cortadellas.j@chu-toulouse.fr
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Maithé TAUBER, MD
Organizational Affiliation
University Hospital, Toulouse
Official's Role
Principal Investigator
Facility Information:
Facility Name
Hôpital Femme Mère Enfant
City
Bron
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Marc Nicolino, MD
Email
marc.nicolino@chu-lyon.fr
Facility Name
CHU Dijon Hôpital des Enfants
City
Dijon
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Marie Bournez, MD
Email
marie.bournez@chu-dijon.fr
Facility Name
CHU de Grenoble
City
Grenoble
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Anne Spiteri, MD
Email
aspiteri@chu-grenoble.fr
Facility Name
Hôpital Jeanne de Flandre
City
Lille
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Iva Gueorguieva, MD
Email
iva.gueorguieva@chru-lille.fr
Facility Name
Hôpital de la Timone Enfant
City
Marseille
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Rachel Reynaud, MD
Email
rachel.reynaud@ap-hm.fr
Facility Name
CHU Nancy
City
Nancy
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Carole Legagneur, MD
Email
c.legagneur@chru-nancy.fr
Facility Name
CHU Nantes
City
Nantes
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sabine Baron, MD
Email
sabine.baron@chu-nantes.fr
Facility Name
Hôpital CHU-Lenval
City
Nice
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Elsa Haine, MD
Email
haine.e@pediatrie-chulenval-nice.fr
Facility Name
Groupe Hospitalier Necker - Enfants Malades
City
Paris
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Graziella Pinto, MD
Email
graziella.pinto@aphp.fr
Facility Name
CHU Rennes
City
Rennes
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Marie-Béatrice Saade
Email
Marie-beatrice.SAADE@chu-rennes.fr
Facility Name
CHU Rouen
City
Rouen
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Mireille Castanet, MD
Email
mireille.castanet@chu-rouen.fr
Facility Name
Centre de réfrence Prader-Willi, Hospital of infants
City
Toulouse
ZIP/Postal Code
31059
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Catherine MOLINAS
Phone
053458698
Ext
+33
Email
molinas.c@chu-toulouse.fr
First Name & Middle Initial & Last Name & Degree
Maïthé TAUBER
First Name & Middle Initial & Last Name & Degree
Gwennaelle DIENE
12. IPD Sharing Statement
Plan to Share IPD
Undecided
Learn more about this trial
Long-term Interventional Follow-up Study of Children With Prader-Willi Syndrome Included in the OTBB3 Clinical Trial
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