Study Evaluating The Safety And Efficacy Of PF-03654746 In Adult Subjects With Tourette's Syndrome

A Phase 2 Multicenter, Randomized, Double-Blind, Placebo-Controlled, Cross-Over Study Of The Safety And Efficacy Of PF-03654746 In Adults With Tourette's Syndrome

The purpose of this study is to evaluate the safety and efficacy of an investigational compound designated PF-03654746 compared to placebo in the treatment of adults with Tourette's Syndrome. The study will also explore the pharmacokinetics of PF-03654746 in adults with Tourette's Syndrome.

Study Overview

• Status: Withdrawn
• Conditions: Tourette's Syndrome
• Intervention / Treatment: Drug: PF-03654746; Drug: Placebo; Drug: Placebo

Detailed Description

The study was terminated 11-Apr-2012 due to an internal reassessment of priorities by the sponsor. The decision to terminate was not based on any safety or efficacy concerns.

• Study Type: Interventional
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • New York
    • Manhasset, New York, United States, 11030
      • Pfizer Investigational Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 55 years (ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Primary diagnosis of Tourette's Syndrome in English-speaking male or female adults 18 to 55 years of age who are in generally good health.
• Free of medications to treat tics for at least 6 weeks prior to randomization.
• Females of childbearing potential must use medically acceptable birth control for the duration of the study and for 28 days after study participation.

Exclusion Criteria:

• Tic treatment including protocol-specified drugs, training in tic-suppressing behavioral techniques, habit reversal training or use of Onabotulinum toxin A injection.
• History or neurologic evidence of a secondary tic disorder, psychosis, bipolar disorder, tardive dyskinesia, untreated or unstable DSM-IV Axis I disorder requiring treatment.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: CROSSOVER
• Masking: DOUBLE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
ACTIVE_COMPARATOR: PF-03654746; Subjects are randomized to either active drug or placebo in Period 1; in Period 2 the sequence is reversed.	Drug: PF-03654746; 20-day dose titration phase: all dosages in capsules starting at 0.25 mg qd x 5 d, then 0.5 mg qd x 5 d, then 1.0 mg qd x 5 d, then 2.0 mg qd x 5 d. If a subject has intolerable, severe, or serious AEs after taking 2 mg qd for 1 to 5 days of dosing, the dose will be decreased by the investigator to 1 mg qd. If, in the investigator's opinion, the subject is determined to be unlikely to tolerate continued dosing at a dose of 1 mg qd, the subject should be discontinued from the study. Subjects remaining in the study will proceed to the 3-week Stable Dosing Phase; doses will be 2 mg daily x 21 days or 1 mg daily x 21 days.; Drug: Placebo; once daily dosing of placebo capsules following the dosing scheme described in 1.1.; Drug: Placebo; once daily dosing of placebo capsules following the dosing scheme described in 1.1
PLACEBO_COMPARATOR: Placebo; Subjects are randomized to either active drug or placebo in Period 1; in Period 2 the sequence is reversed.	Drug: PF-03654746; 20-day dose titration phase: all dosages in capsules starting at 0.25 mg qd x 5 d, then 0.5 mg qd x 5 d, then 1.0 mg qd x 5 d, then 2.0 mg qd x 5 d. If a subject has intolerable, severe, or serious AEs after taking 2 mg qd for 1 to 5 days of dosing, the dose will be decreased by the investigator to 1 mg qd. If, in the investigator's opinion, the subject is determined to be unlikely to tolerate continued dosing at a dose of 1 mg qd, the subject should be discontinued from the study. Subjects remaining in the study will proceed to the 3-week Stable Dosing Phase; doses will be 2 mg daily x 21 days or 1 mg daily x 21 days.; Drug: Placebo; once daily dosing of placebo capsules following the dosing scheme described in 1.1.; Drug: Placebo; once daily dosing of placebo capsules following the dosing scheme described in 1.1

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Change in Total Tic Score (Yale Global Tic Severity Scale) from baseline (D0) to end of the 3 wk stable dosing phase (D41)(primary). Average of the 2 assessments of Total Tic Score in 3 wk stable dosing phase is secondary. Score 0-50 (50 = severe)	Period 1: Days 0, 10, 20, 34, 41; Period 2: Days 0, 10, 20, 34, 41

Secondary Outcome Measures

Outcome Measure	Time Frame
Change in Tic Symptom Self Report from baseline to end of 3-wk stable dosing phase (primary); average of 2 assessments of TSSR during 3-wk stable dosing phase is 2ndary. Each symptom is scored 0-3; higher score is worse.	Period 1: Days 0, 10, 20, 34, 41; Period 2: Days 0, 10, 20, 34, 41
Change in Premonitory Urge for Tic Scale from baseline to end of 3-wk stable dosing phase (primary); average of 2 assessments of PUTS during 3-wk stable dosing phase is 2ndary. Score 9-36; higher score is worse.	Period 1: Days 0, 10, 20, 34, 41; Period 2: Days 0, 10, 20, 34, 41
Change in Clinical Global Impression of Severity from baseline to end of 3-wk stable dosing phase. Score 1-7; higher scores indicate more severity.	Period 1, Days 0, 41; Period 2: Days 0, 41
Change in Clinical Global Impression of Improvement from baseline to end of 3-wk stable dosing phase (primary); average of 2 assessments during 3-wk stable dosing phase is 2ndary. Score 1-7; higher score is worse.	Period 1: Days 10, 20, 34, 41; Period 2: Days 10, 20, 34, 41
Change in Conners' Continuous Performance Test II from baseline to end of 3-wk stable dosing phase. Calculated T-scores (under 40 to 65 and over); higher score is worse.	Period 1: Days 0, 20, 41; Period 2: Days 0, 20, 41
Change in Medical Outcomes Study--Sleep Scale from baseline to end of 3-wk stable dosing phase. Score 0-100; a higher score reflect greater amount of quality implied by subscale name.	Period 1: Days 0, 10, 20, 34, 41; Period 2: Days 0, 10, 20, 34, 41
Change in Columbia Suicide Severity Rating Scale from baseline to end of 3-wk stable dosing phase.	Screening; Period 1: Days 0, 10, 20, 34, 41; Period 2: Days 0, 10, 20, 34, 41
Suicide Behaviors Questionnaire-Revised. Total score greater than 8 require assessment by clinician or mental health professional skilled in evaluation of suicidality.	Up to 21 days prior to Baseline (Day 0)
Change in Yale-Brown Obsessive-Compulsive Scale from baseline to end of 3-wk stable dosing phase. Items 1-10 have score range of 0-40; higher score is worse.	Period 1: Days 0, 41; Period 2: Days 0, 41

Collaborators and Investigators

• Sponsor: Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start: April 1, 2012
• Primary Completion (ACTUAL): April 1, 2012
• Study Completion (ACTUAL): April 1, 2012

Study Registration Dates

• First Submitted: October 25, 2011
• First Submitted That Met QC Criteria: November 16, 2011
• First Posted (ESTIMATE): November 21, 2011

Study Record Updates

• Last Update Posted (ACTUAL): April 1, 2019
• Last Update Submitted That Met QC Criteria: March 28, 2019
• Last Verified: March 1, 2019

More Information

Terms related to this study

• Keywords: Safety and Efficacy; Randomized; Placebo Control; Cross-over; Tourette's Syndrome in adults
• Additional Relevant MeSH Terms: Mental Disorders; Pathologic Processes; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Disease; Genetic Diseases, Inborn; Basal Ganglia Diseases; Movement Disorders; Neurodegenerative Diseases; Heredodegenerative Disorders, Nervous System; Neurodevelopmental Disorders; Tic Disorders; Syndrome; Tourette Syndrome
• Other Study ID Numbers: A8801035