Dose Clinical Trial of Guanfacine Extended Release for the Reduction of Aggression and Self-injuries Behavior Associated With Prader-Willi Syndrome

NCT04066088 | Withdrawn Phase 4 FDA Drug

• 1 other identifier: 19-00936
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

This is a placebo-controlled clinical trial to assess the utility of Guanfacine Extended Release (GXR) in the management of patients with Prader Willi Syndrome (PWS) who have significant aggression or self-injury. The purpose of this trial is to establish the safety of GXR with a specific focus on metabolic effects.

Conditions

Prader-Willi Syndrome

Outcome Measures

Primary Outcomes (1)

• CGI-I scale rating

  A positive clinical response will be determined by a rating of 1 or 2 (Very much/Much improved) on the CGI-I scale at the end of the blinded trial.

  19 Weeks

Secondary Outcomes (3)

• Aberrant Behavior Checklist

  19 Weeks

• Self-Injury Trauma scale

  19 Weeks

• Modified Overt Aggression Scale

  19 Weeks

Study Arms (2)

Placebo

SHAM COMPARATOR

Other: Placebo

GXR

EXPERIMENTAL

Immediately following the 8-week blinded randomized trial, an 8-week open-label continuation phase will be pursued to further define efficacy and tolerability of GXR, and to establish its safety with specific focus on metabolic profile.

Drug: Guanfacine extended release (GXR)

Interventions

Placebo OTHER

Placebo will be administered same times as GXR

Placebo

Guanfacine extended release (GXR) DRUG

The starting dose for all subjects will be 1 mg per day. If the medication is well-tolerated, the dose can be raised to 2 mg until day 28 and increased to 3mg for the remaining 4 weeks in the trial. The dose schedule will not be fixed; the treating clinician can delay a planned increase or lower the dose to manage adverse effects. At week 8, the study will be unblinded and subjects will continue treatment for 8 more weeks.

GXR

Eligibility Criteria

• Age: 6 Years - 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• and 35 years of age
• diagnosis of PWS confirmed by genetic testing.
• rating of moderate or above on the Clinical Global Impression- Severity Scale will be required for entry.

You may not qualify if:

• Subjects with a positive pregnancy test, swallowing difficulty, and/or presenting with active psychosis or mania will be excluded.
• Individuals with pre-existing, clinically significant bradycardia (\< 8 years: \<64 bpm; 8 to 12 years: \<59 bpm; 12 to 16 years: \<53 bpm) or hypotension, defined as 5th percentile for height and gender,26 will be excluded from the study.
• Subjects receiving antipsychotic medications due to a documented history of psychosis or bipolar disorder will be allowed to continue taking the medication without dosage modification.
• Growth hormone, thyroid hormone replacement treatment, and non-psychiatric medicines will be allowed to continue.
• N-Acetyl Cysteine and anticonvulsant medication (only if prescribed for seizures) will be allowed to continue, with specific instructions to not make any dosage changes during the clinical trial.

Sponsors & Collaborators

• NYU Langone Health: lead
• Winthrop University Hospital: collaborator

Study Sites (1)

NYU Langone Health

New York, New York, 10016, United States

Location

MeSH Terms

Conditions

Prader-Willi Syndrome

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases

Study Officials

• Deepan Singh, MD

  New York Langone Health

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 21, 2019
• First Posted: August 26, 2019
• Study Start: December 1, 2019
• Primary Completion: August 21, 2020
• Study Completion: August 21, 2020
• Last Updated: August 26, 2020

Record last verified: 2020-08

Locations

US (1)