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Piloting Treatment With Intranasal Oxytocin in Phelan-McDermid Syndrome

NCT ID: NCT02710084

Last Updated: 2021-12-06

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

18 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-04-29

Study Completion Date

2020-07-29

Brief Summary

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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.

Detailed Description

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Conditions

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Phelan-McDermid Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Oxytocin

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.

Group Type EXPERIMENTAL

Oxytocin

Intervention Type DRUG

Intranasal oxytocin

Saline

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.

Group Type PLACEBO_COMPARATOR

Saline

Intervention Type DRUG

Placebo

Interventions

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Oxytocin

Intranasal oxytocin

Intervention Type DRUG

Saline

Placebo

Intervention Type DRUG

Other Intervention Names

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Syntocinon

Eligibility Criteria

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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
Minimum Eligible Age

5 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Alexander Kolevzon

OTHER

Sponsor Role lead

Responsible Party

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Alexander Kolevzon

Alexander Kolevzon, Clinical Director

Responsibility Role SPONSOR_INVESTIGATOR

Principal Investigators

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Alexander Kolevzon, MD

Role: PRINCIPAL_INVESTIGATOR

Icahn School of Medicine at Mount Sinai

Locations

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Icahn School of Medicine at Mount Sinai

New York, New York, United States

Site Status

Countries

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United States

References

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Fastman J, Foss-Feig J, Frank Y, Halpern D, Harony-Nicolas H, Layton C, Sandin S, Siper P, Tang L, Trelles P, Zweifach J, Buxbaum JD, Kolevzon A. A randomized controlled trial of intranasal oxytocin in Phelan-McDermid syndrome. Mol Autism. 2021 Sep 30;12(1):62. doi: 10.1186/s13229-021-00459-1.

Reference Type RESULT
PMID: 34593045 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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GCO 15-1223

Identifier Type: -

Identifier Source: org_study_id