Prader-Willi SyndromePhase 3OTBB3-FUNCT05032326
Long-term Interventional Follow-up Study of Children With Prader-Willi Syndrome Included in the OTBB3 Clinical Trial
A Phase 3 study testing Follow-up study of the treated cohort and Follow-up study of the untreated cohort in people with Prader-Willi Syndrome, run by University Hospital, Toulouse at 12 sites in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 to 36 months
- Conditions the study is for
- Prader-Willi Syndrome
- What is being tested?
- Follow-up study of the treated cohort and Follow-up study of the untreated cohort
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 80 participants
- Where
- 12 sitesFrance
- Study dates
- 7 Sep 2021to 1 Apr 2025
- Phase
- Phase 3
- 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 12 to 36 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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;
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
France12 sites, 12 recruiting
- Recruiting
Hôpital Femme Mère Enfant
Bron
Marc Nicolino, MD, marc.nicolino@chu-lyon.fr
- Recruiting
CHU Dijon Hôpital des Enfants
Dijon
Marie Bournez, MD, marie.bournez@chu-dijon.fr
- Recruiting
CHU de Grenoble
Grenoble
Anne Spiteri, MD, aspiteri@chu-grenoble.fr
- Recruiting
Hôpital Jeanne de Flandre
Lille
Iva Gueorguieva, MD, iva.gueorguieva@chru-lille.fr
- Recruiting
Hôpital de la Timone Enfant
Marseille
Rachel Reynaud, MD, rachel.reynaud@ap-hm.fr
- Recruiting
CHU Nancy
Nancy
Carole Legagneur, MD, c.legagneur@chru-nancy.fr
- Recruiting
CHU Nantes
Nantes
Sabine Baron, MD, sabine.baron@chu-nantes.fr
- Recruiting
Hôpital CHU-Lenval
Nice
Elsa Haine, MD, haine.e@pediatrie-chulenval-nice.fr
- Recruiting
Groupe Hospitalier Necker - Enfants Malades
Paris
Graziella Pinto, MD, graziella.pinto@aphp.fr
- Recruiting
CHU Rennes
Rennes
Marie-Béatrice Saade, Marie-beatrice.SAADE@chu-rennes.fr
- Recruiting
CHU Rouen
Rouen
Mireille Castanet, MD, mireille.castanet@chu-rouen.fr
- Recruiting
Centre de réfrence Prader-Willi, Hospital of infants
Toulouse, 31059
Catherine MOLINAS, 053458698 ext. +33, molinas.c@chu-toulouse.fr
Maïthé TAUBER
About this study
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
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.
- Conditions
- Prader-Willi Syndrome
- Being tested
- Follow-up study of the treated cohort, Follow-up study of the untreated cohort
- Keywords
- Prader-Willi syndrome, Oxytocin
- Medical subject headings
- Prader-Willi Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Follow-up study of the treated cohortDrug
follow-up study of the patients in the treated cohort: that have been included in the otbb3 study
Follow-up study of the untreated cohortOther
follow-up study of the patients in the untreated cohort: that have NOT been included in the otbb3 study
| Study group | Role | What they receive |
|---|---|---|
| OXYTOCIN (OT) Treated cohort | Other | babies treated with Oxytocin during the OTBB3 study Receives: Drug: Follow-up study of the treated cohort |
| Untreated cohort | Other | babies not included in the OTBB3 study and therefore never treated with Oxytocin Receives: Other: Follow-up study of the untreated cohort |
What is being measured
The primary outcome is the main question the study is designed to answer.
Confirmation of the long term safety profile (1)
The number of patients with adverse events (AEs)
Measured: 4 years
Confirmation of the long term safety profile (2)
The percentage of patients with adverse events (AEs)
Measured: 4 years
Confirmation of the long term safety profile (3)
Assessment in the treated cohort of the occurrence of the main comorbidities in Prader Willi Syndrome
Measured: 4 years
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
Measured: 4 years
Secondary outcomes (11)
Complete the safety assessment by the description of the development of the child (1.1)
Assessment in the treated cohort of: weight (kilograms)
Measured: 4 years
Complete the safety assessment by the description of the development of the child (1.2)
Assessment in the treated cohort of: height (meters)
Measured: 4 years
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)
Measured: 4 years
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
Measured: 4 years
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
Measured: 4 years
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
Measured: 4 years
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
Measured: 4 years
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);
Measured: 4 years
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);
Measured: 4 years
Assessment of endocrine disorders by IGF1
Analysis of plasma Insulin-like growth factor 1 (IGF1, ng/mL)
Measured: 4 years
Assessment of endocrine disorders by TSH
Analysis of plasma thyroid stimulating hormone (TSH, µUI/mL)
Measured: 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Sep 2021
- Study start7 Sep 2021
- Primary completion1 Apr 2025 (estimated)
- Study completion1 Apr 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Prader-Willi SyndromeStudies of the same condition, open and under way.
- More trials run by University Hospital, ToulouseThe sponsor's other registered studies.
- 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 3
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Toulouse
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Maithé TAUBER, MD, Principal investigator, University Hospital, Toulouse
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05032326
- Sponsor's protocol ID
- RC31/20/0421
- Acronym
- OTBB3-FU
- Status last verified
- Apr 2024
- First submitted
- 24 Jun 2021
- Last updated
- 30 Apr 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 30 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05032326
- Acronym / sponsor ID
- OTBB3-FU / RC31/20/0421
- Conditions
- Prader-Willi Syndrome
- Keywords
- Prader-Willi syndrome, Oxytocin
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- University Hospital, ToulouseUniversity, hospital or other
- Study start
- 7 Sep 2021 (actual)
- Primary completion
- 1 Apr 2025 (estimated)
- Study completion
- 1 Apr 2025 (estimated)
- First posted
- 2 Sep 2021 (actual)
- 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.
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 design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| OXYTOCIN (OT) Treated cohort | Other | Drug: Follow-up study of the treated cohortbabies treated with Oxytocin during the OTBB3 study |
| Untreated cohort | Other | Other: Follow-up study of the untreated cohortbabies not included in the OTBB3 study and therefore never treated with Oxytocin |
Who may qualify
- Ages
- 12 to 36 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (4 listed)
- Administrative problems:
- Refusal of parents (or legal representative) to sign the consent form;
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 |
|---|---|---|
| Follow-up study of the treated cohortfollow-up study of the patients in the treated cohort: that have been included in the otbb3 study | Drug | OXYTOCIN (OT) Treated cohort |
| Follow-up study of the untreated cohortfollow-up study of the patients in the untreated cohort: that have NOT been included in the otbb3 study | Other | Untreated cohort |
Primary outcome
Confirmation of the long term safety profile (1)
Time frame: 4 years
The number of patients with adverse events (AEs)
Confirmation of the long term safety profile (2)
Time frame: 4 years
The percentage of patients with adverse events (AEs)
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
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
11 secondary outcomes
Complete the safety assessment by the description of the development of the child (1.1)
Time frame: 4 years
Complete the safety assessment by the description of the development of the child (1.2)
Time frame: 4 years
Complete the safety assessment by the description of the development of the child (1.3)
Time frame: 4 years
Complete the safety assessment by the description of the development of the child (2.1)
Time frame: 4 years
Complete the safety assessment by the description of the development of the child (2.2)
Time frame: 4 years
Complete the safety assessment by the description of the development of the child (2.3)
Time frame: 4 years
Complete the safety assessment by the description of the development of the child (2.4)
Time frame: 4 years
Complete the safety assessment by the description of the severity of the disease
Time frame: 4 years
Complete the safety assessment by the description of the severity of the disease (2)
Time frame: 4 years
Assessment of endocrine disorders by IGF1
Time frame: 4 years
Assessment of endocrine disorders by TSH
Time frame: 4 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Apr 2024.
- Individual participant dataSharing planned: Undecided
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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.
Maithé TAUBER, MD principal investigator
University Hospital, Toulouse
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Follow-up study of the treated cohort and Follow-up study of the untreated cohort in people with Prader-Willi Syndrome, run by University Hospital, Toulouse at 12 sites in France.
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 4 inclusion and 4 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: status not confirmed, as of the sponsor's last update on 30 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in France. 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. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Maithé TAUBER, MD (534 55 85 51 ext. +33, tauber.mt@chu-toulouse.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Confirmation of the long term safety profile (1) (time frame: 4 years). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 30 Apr 2024.