Overview
Sponsor-declared trial summary
prader-willi syndrome
The primary objective of this study will be to confirm the long-term safety profile, including the associated main comorbidities, in all children with PWS who have been treated in the OTBB3 study.
Key facts
- Sponsor
- Centre Hospitalier Universitaire De Toulouse
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Trial duration
- completed 29 Apr 2025
- Decision date (initial)
- 2024-12-17
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- OT4B start-up
External identifiers
- EU CT number
- 2024-517925-25-00
- EudraCT number
- 2020-006161-11
- ClinicalTrials.gov
- NCT05032326
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety, Efficacy
The primary objective of this study will be to confirm the long-term safety profile, including the associated main comorbidities, in all children with PWS who have been treated in the OTBB3 study.
Conditions and MedDRA coding
prader-willi syndrome
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 3
- 1. Male or female child with a genetically confirmed diagnosis of PWS ; 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. Inclusion visit will be performed considering the next routine consultation. 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. Inclusion visit will be performed considering the next routine consultation.
- 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. Inclusion visit will be performed considering the next routine consultation.
Exclusion criteria 2
- 1. Administrative problems: a. Inability for the parents (or legal representative) to understand/fulfil study requirements; b. No coverage by a social security regime;
- 2. Refusal of parents (or legal representative) to sign the consent form;
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 3
- The number and percentage of patients with adverse events (AEs) and serious adverse events (SAEs);
- The occurrence of the main comorbidities
- The occurrence of medications, surgery and rehabilitations by collecting type, age at start and stop, dosing or frequency.
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
Otwillo 44.44 IU/mL, nasal spray solution
PRD11709894 · Product
- Active substance
- Oxytocin
- Substance synonyms
- GR121619
- Pharmaceutical form
- NASAL SPRAY, SOLUTION
- Route of administration
- INTRANASAL USE
- Max daily dose
- 4 U unit(s)
- Max total dose
- 4 U unit(s)
- Max treatment duration
- 8 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- OT4B
- Paediatric formulation
- No
- Orphan designation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Centre Hospitalier Universitaire De Toulouse
- Sponsor organisation
- Centre Hospitalier Universitaire De Toulouse
- Address
- 2 Rue Viguerie
- City
- Toulouse
- Postcode
- 31300
- Country
- France
Scientific contact point
- Organisation
- Centre Hospitalier Universitaire De Toulouse
- Contact name
- TAUBER
Public contact point
- Organisation
- Centre Hospitalier Universitaire De Toulouse
- Contact name
- Nadege ALGANS
Locations
1 EU/EEA country · 11 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| France | Ended | 65 | 11 |
| Rest of world | — | 0 | — |
Investigational sites
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 5 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | OTBB3 FU PROTOCOL | 6 |
| Recruitment arrangements (for publication) | Liste investigateurs | 5 |
| Subject information and informed consent form (for publication) | Consent treated cohort | 5 |
| Subject information and informed consent form (for publication) | Consent untreated cohort | 6 |
| Synopsis of the protocol (for publication) | OTBB3 FU RESUME | 4 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-11-04 | France | Acceptable 2024-12-03
|
2024-12-17 |