Oxytocin Treatment in Neonates and Infants With Prader-Willi Syndrome (OTBB3)

Toulouse University Hospital

Status and phase: Completed; Phase 3

Conditions: Prader-Willi Syndrome

Treatments: Drug: OT; Drug: Placebo comparator

Study type: Interventional

Funder types: Other

Identifiers

NCT04283578

RC31/15/7825

Details and patient eligibility

About

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.

Enrollment: 52 patients

Sex: All

Ages: 1 to 92 days old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Male or female neonate or infant, with PWS genetically confirmed.
2. 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).
3. Signed informed consent obtained from the parents/holders of parental authority.
4. Parents willing and able to comply with all study procedures.

Exclusion criteria

• 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.

  3. Neonate or infant with concomitant treatment prolonging QT interval 6. Neonate or infant with family history of genetic pathology causing QT interval prolongation.

  4. Neonate or infant with hypokalemia (clinically relevant at the discretion of the doctor).

  5. Neonate or infant participating simultaneously in another interventional study.

  6. Neonates or infants whose parents' situations may jeopardize the interpretation of the results.

  7. Neonates or infants whose parents' refuse video recording, required to respond to the primary objective of the study.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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52 participants in 2 patient groups, including a placebo group

Oxytocin

Experimental group

Description:

intranasal administration of OT

Treatment:

Drug: OT

Drug: Placebo comparator

Placebo

Placebo Comparator group

Description:

intranasal administration of placebo

Treatment:

Drug: OT

Drug: Placebo comparator