Prader-Willi SyndromePhase 3OTBB3NCT04283578
Oxytocin Treatment in Neonates and Infants With Prader-Willi Syndrome
A Phase 3 study testing OT in people with Prader-Willi Syndrome, run by University Hospital, Toulouse at 7 sites in 3 countries.
Trial summary
Prospective, randomized, placebo-controlled, double-blind part of the phase III trial to assess the safety and efficacy of 4 weeks oxytocin (OT) administration on oral and social skills in neonates/infants with Prader-Willi Syndrome (PWS) aged less than 3 months at inclusion. Phase III clinical trial. Read more about this study
- Who is it for?
- Ages 1 day to 92 days
- Conditions the study is for
- Prader-Willi Syndrome
- What is being tested?
- OT
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 52 participants
- Where
- 7 sitesin 3 countries
- Study dates
- 10 Mar 2020to 14 Mar 2022
- 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 1 day to 92 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male or female neonate or infant, with PWS genetically confirmed.
- Age <92 days (plus a tolerance of up to 8 days maximum) (for preterm infants, born before 37 weeks amenorrhea, corrected age will be applied).
- Signed informed consent obtained from the parents/holders of parental authority.
- Parents willing and able to comply with all study procedures.
Who cannot join
- 1. Neonate or infant currently admitted to the emergency care unit for ongoing life-threatening comorbidities like severe respiratory, cardiovascular or neurological abnormalities.
- 2. Neonate or infant with prolongation of the QT interval. 3. Neonate or infant without medical insurance. 4. Neonate or infant with hypersensitivity to oxytocin or excipients of the product.
- 5. Neonate or infant with concomitant treatment prolonging QT interval 6. Neonate or infant with family history of genetic pathology causing QT interval prolongation.
- 7. Neonate or infant with hypokalemia (clinically relevant at the discretion of the doctor).
- 8. Neonate or infant participating simultaneously in another interventional study.
- 9. Neonates or infants whose parents' situations may jeopardize the interpretation of the results.
- 10. Neonates or infants whose parents' refuse video recording, required to respond to the primary objective of the study.
Trial locations and contacts
7 sites in 3 countries.
7 sites in 3 countries
France5 sites
Hôpital Femme Mère Enfant
Bron
Hôpital Jeanne de Flandre
Lille
Hôpital de la Timone Enfant
Marseille
Groupe Hospitalier Necker - Enfants Malades
Paris
Centre de réfrence Prader-Willi, Hospital of infants
Toulouse, 31059
About this study
Official title: Oxytocin Treatment in Neonates and Infants Aged From 0 to 3 Months With Prader-Willi Syndrome : a Study of Safety and Efficacy on Oral and Social Skills and Feeding Behavior of Intranasal Administration of Oxytocin vs Placebo
Prospective, randomized, placebo-controlled, double-blind part of the phase III trial to assess the safety and efficacy of 4 weeks oxytocin (OT) administration on oral and social skills in neonates/infants with Prader-Willi Syndrome (PWS) aged less than 3 months at inclusion. Phase III clinical trial.
- Conditions
- Prader-Willi Syndrome
- Being tested
- OT, Placebo comparator
- 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.
OTDrug
Also called oxytocin
One group will receive OT for 4 weeks, then placebo or OT for 8 weeks
Placebo comparatorDrug
One group will receive Placebo for 4 weeks, then Placebo or OT for 8 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Oxytocin | Experimental | intranasal administration of OT Receives: Drug: OT; Drug: Placebo comparator |
| Placebo | Placebo comparator | intranasal administration of placebo Receives: Drug: OT; Drug: Placebo comparator |
What is being measured
The primary outcome is the main question the study is designed to answer.
Neonatal Oral-Motor Assessment Scale (NOMAS) scale
the score goes from 8 to 28, the higher the score meaning a worse outcome
Measured: 4 weeks
Secondary outcomes (3)
Ghrelin dosage
Concentration of ghrelin (unacylated/UAG and acylated/AG)
Measured: Day 0, Week 1 and week 4
Oxytocin dosage
Plasma Oxytocin concentration
Measured: Day 0, week 4
Proficiency score
The volume of milk taken in the first five minutes of feeding
Measured: Day 0, Week and week 4
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2020
- Study start10 Mar 2020
- Primary completion16 Oct 2021
- Study completion14 Mar 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 52 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Toulouse
- Sponsor type
- University, hospital or other
- Collaborators
- International Clinical Trials Association, Epidemiological and Clinical Research Information Network
- Responsible party
- Sponsor
- Investigators
- Maithé TAUBER, MD, Principal investigator, University Hospital, Toulouse
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04283578
- Sponsor's protocol ID
- RC31/15/7825
- Acronym
- OTBB3
- Status last verified
- May 2023
- First submitted
- 19 Feb 2020
- Last updated
- 1 Jun 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Borie AM, Dromard Y, Guillon G, Olma A, Manning M, Muscatelli F, Desarmenien MG, Jeanneteau F. Correction of vasopressin deficit in the lateral septum ameliorates social deficits of mouse autism model. J Clin Invest. 2021 Jan 19;131(2):e144450. doi: 10.1172/JCI144450.
PubMed 33232306
Study at a glance
From the registry record, last updated by the sponsor on 1 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Oxytocin Treatment in Neonates and Infants Aged From 0 to 3 Months With Prader-Willi Syndrome : a Study of Safety and Efficacy on Oral and Social Skills and Feeding Behavior of Intranasal Administration of Oxytocin vs Placebo
- ClinicalTrials.gov ID
- NCT04283578
- Acronym / sponsor ID
- OTBB3 / RC31/15/7825
- Conditions
- Prader-Willi Syndrome
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 52 participants (actual)
- Lead sponsor
- University Hospital, ToulouseUniversity, hospital or other
- Collaborators
- International Clinical Trials Association, Epidemiological and Clinical Research Information Network
- Study start
- 10 Mar 2020 (actual)
- Primary completion
- 16 Oct 2021 (actual)
- Study completion
- 14 Mar 2022 (actual)
- First posted
- 25 Feb 2020 (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.
Prospective, randomized, placebo-controlled, double-blind part of the phase III trial to assess the safety and efficacy of 4 weeks oxytocin (OT) administration on oral and social skills in neonates/infants with Prader-Willi Syndrome (PWS) aged less than 3 months at inclusion. Phase III clinical trial.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Oxytocin | Experimental | Drug: OT, Drug: Placebo comparatorintranasal administration of OT |
| Placebo | Placebo comparator | Drug: OT, Drug: Placebo comparatorintranasal administration of placebo |
Who may qualify
- Ages
- 1 day to 92 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Male or female neonate or infant, with PWS genetically confirmed.
- Age <92 days (plus a tolerance of up to 8 days maximum) (for preterm infants, born before 37 weeks amenorrhea, corrected age will be applied).
- Signed informed consent obtained from the parents/holders of parental authority.
- Parents willing and able to comply with all study procedures.
Key exclusion criteria (7 listed)
- 1. Neonate or infant currently admitted to the emergency care unit for ongoing life-threatening comorbidities like severe respiratory, cardiovascular or neurological abnormalities.
- 2. Neonate or infant with prolongation of the QT interval. 3. Neonate or infant without medical insurance. 4. Neonate or infant with hypersensitivity to oxytocin or excipients of the product.
- 5. Neonate or infant with concomitant treatment prolonging QT interval 6. Neonate or infant with family history of genetic pathology causing QT interval prolongation.
- 7. Neonate or infant with hypokalemia (clinically relevant at the discretion of the doctor).
- 8. Neonate or infant participating simultaneously in another interventional study.
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 |
|---|---|---|
| OTAlso: oxytocinOne group will receive OT for 4 weeks, then placebo or OT for 8 weeks | Drug | Oxytocin, Placebo |
| Placebo comparatorOne group will receive Placebo for 4 weeks, then Placebo or OT for 8 weeks. | Drug | Oxytocin, Placebo |
Primary outcome
Neonatal Oral-Motor Assessment Scale (NOMAS) scale
Time frame: 4 weeks
the score goes from 8 to 28, the higher the score meaning a worse outcome
3 secondary outcomes
Ghrelin dosage
Time frame: Day 0, Week 1 and week 4
Oxytocin dosage
Time frame: Day 0, week 4
Proficiency score
Time frame: Day 0, Week and week 4
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jun 2023.
- Derived from this study: PubMed 33232306Borie AM, Dromard Y, Guillon G, Olma A, Manning M, Muscatelli F, Desarmenien MG, Jeanneteau F. Correction of vasopressin deficit in the lateral septum ameliorates social deficits of mouse autism model. J Clin Invest. 2021 Jan 19;131(2):e144450. doi: 10.1172/JCI144450.
- Individual participant dataSharing planned: No
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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
7 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing OT in people with Prader-Willi Syndrome, run by University Hospital, Toulouse at 7 sites in 3 countries.
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 7 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: finished, as of the sponsor's last update on 1 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in France, Belgium, Germany. 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 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Neonatal Oral-Motor Assessment Scale (NOMAS) scale (time frame: 4 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 NCT04283578, which also lists 1 linked publication.
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 1 Jun 2023.