Piloting Treatment With Intranasal Oxytocin in Phelan-McDermid Syndrome

A

Alexander Kolevzon

Status and phase

Completed
Phase 2

Conditions

Phelan-McDermid Syndrome

Treatments

Drug: Saline
Drug: Oxytocin

Study type

Interventional

Funder types

Other

Identifiers

NCT02710084
GCO 15-1223

Details and patient eligibility

About

This is a pilot study examining the efficacy, safety and tolerability of intranasal oxytocin as a novel treatment in Phelan-McDermid syndrome (PMS). This study will utilize a randomized, placebo-controlled design for 12 weeks (phase 1), followed by an open-label extension for 12 weeks (phase 2). The purpose is to evaluate the effect of intranasal oxytocin on impairments in attention, social memory, socialization, language, and repetitive behaviors.

Enrollment

18 patients

Sex

All

Ages

5 to 17 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Have a diagnosis of Phelan-McDermid syndrome defined by pathogenic deletion or mutation of the SHANK3 gene and confirmed by chromosomal microarray or sequencing. Must be between the ages of 5 and 17 Must be on stable medication and psychosocial therapy regimens for at least three months prior to enrollment

Exclusion criteria

Allergy to oxytocin Active cardiovascular disease, epilepsy, or renal disease that is not controlled by medication Pregnant, lactating or refuse to practice contraception if sexually active Have caretakers who are unable to speak English, be consistently present at visits to report on symptoms, or are otherwise judged unable to comply with the protocol by the study team Comorbid conditions such that the patient is too medically compromised to participate

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Parallel Assignment

Masking

Quadruple Blind

18 participants in 2 patient groups, including a placebo group

Oxytocin
Experimental group
Description:
The first phase of the study will follow a double-blind, placebo-controlled design. Participants randomized to the experimental group will receive intranasal oxytocin in doses of 24 IU, two times daily, for a total of 48 IU. Doses may be reduced by 8 IU/day if safety concerns emerge. During the second phase of the study, all participants will receive oxytocin, in identical doses.
Treatment:
Drug: Oxytocin
Saline
Placebo Comparator group
Description:
During the first phase, patients randomized to the placebo group will receive intranasal saline solution in doses of 24 IU two times daily, for a total of 48 IU. During the second phase of the study, all participants will receive oxytocin, in identical doses.
Treatment:
Drug: Saline

Trial documents
1

Trial contacts and locations

1

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Data sourced from clinicaltrials.gov

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