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Randomized, Placebo Controlled Study Of The Efficacy And Safety Of PF-02545920 In Subjects With Huntington's Disease

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ClinicalTrials.gov Identifier: NCT02197130
Recruitment Status : Completed
First Posted : July 22, 2014
Results First Posted : November 17, 2017
Last Update Posted : November 17, 2017
Sponsor:
Information provided by (Responsible Party):
Pfizer

Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Huntington's Disease
Interventions: Drug: PF-02545920
Other: Placebo

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
A total of 272 subjects (133 males and 139 females) were randomized and assigned study treatment.

Reporting Groups
  Description
PF-02545920 5 mg BID Participants took 4 tablets packaged in blister packs (3 placebo tablets and one 5 mg PF-02545920) twice a day approximately every 12 hours from Baseline Day 1 (V2) to Week 26 (V9), at approximately the same time of day throughout the study. Study medication was swallowed whole, and was not manipulated or chewed prior to swallowing.
Placebo Participants took 4 tablets of placebo packaged in blister packs twice a day approximately every 12 hours, at approximately the same time of day throughout the study. Study medication was swallowed whole, and was not manipulated or chewed prior to swallowing.
PF-02545920 20 mg BID The 20 mg BID dose of PF-02545920 was titrated as follows: 5 mg BID for 7 days, 10 mg BID for 7 days, 15 mg BID for 7 days, then 20 mg BID for the remainder of the treatment phase. Participants took 4 tablets packaged in blister packs twice a day approximately every 12 hours, at approximately the same time of day throughout the study. The blister packs contained 3 placebo tablets and one 5 mg PF-02545920 tablet for Days 1-7, 2 placebo tablets and two 5 mg PF-02545920 tablets for Days 8-14, 1 placebo tablet and three 5 mg PF-02545920 tablets for Days 15-21, and four 5 mg PF-02545920 tablets from Day 22 through Day 182. Study medication was swallowed whole, and was not manipulated or chewed prior to swallowing.

Participant Flow:   Overall Study
    PF-02545920 5 mg BID   Placebo   PF-02545920 20 mg BID
STARTED   95   89   88 
Received Treatment   95   88   87 
COMPLETED   79   81   56 
NOT COMPLETED   16   8   32 
Adverse Event                13                4                23 
Death                0                1                0 
Lost to Follow-up                2                0                1 
Does not meet entrance criteria                0                0                1 
Withdrawal by Subject                0                3                4 
Other                1                0                3 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
PF-02545920 5 mg BID Participants took 4 tablets packaged in blister packs (3 placebo tablets and one 5 mg PF-02545920) twice a day approximately every 12 hours from Baseline Day 1 (V2) to Week 26 (V9), at approximately the same time of day throughout the study. Study medication was swallowed whole, and was not manipulated or chewed prior to swallowing.
Placebo Participants took 4 tablets of placebo packaged in blister packs twice a day approximately every 12 hours, at approximately the same time of day throughout the study. Study medication was swallowed whole, and was not manipulated or chewed prior to swallowing.
PF-02545920 20 mg BID The 20 mg BID dose of PF-02545920 was titrated as follows: 5 mg BID for 7 days, 10 mg BID for 7 days, 15 mg BID for 7 days, then 20 mg BID for the remainder of the treatment phase. Participants took 4 tablets packaged in blister packs twice a day approximately every 12 hours, at approximately the same time of day throughout the study. The blister packs contained 3 placebo tablets and one 5 mg PF-02545920 tablet for Days 1-7, 2 placebo tablets and two 5 mg PF-02545920 tablets for Days 8-14, 1 placebo tablet and three 5 mg PF-02545920 tablets for Days 15-21, and four 5 mg PF-02545920 tablets from Day 22 through Day 182. Study medication was swallowed whole, and was not manipulated or chewed prior to swallowing.
Total Total of all reporting groups

Baseline Measures
   PF-02545920 5 mg BID   Placebo   PF-02545920 20 mg BID   Total 
Overall Participants Analyzed 
[Units: Participants]
 95   88   87   270 
Age 
[Units: Years]
Mean (Standard Deviation)
 48.3  (8.6)   50.2  (9.4)   48.4  (9.2)   48.9  (9.1) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
       
Female      42  44.2%      54  61.4%      43  49.4%      139  51.5% 
Male      53  55.8%      34  38.6%      44  50.6%      131  48.5% 


  Outcome Measures

1.  Primary:   Change From Baseline in the Total Motor Score (TMS) Assessment of the Unified Huntington Disease Rating Scale (UHDRS) After 26 Weeks of Treatment.   [ Time Frame: Baseline, Week 26 ]

2.  Secondary:   Number of Participants That Met White Blood Count (WBC) and Absolute Neutrophil Count (ANC) Stopping Criteria   [ Time Frame: Screening, Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192) ]

3.  Secondary:   Number of Participants With Adverse Events   [ Time Frame: Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192) ]

4.  Secondary:   Number of Participants With Serious Adverse Events   [ Time Frame: Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192) ]

5.  Secondary:   Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormalities)   [ Time Frame: Screening, Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192) ]

6.  Secondary:   Number of Participants With Laboratory Test Abnormalities (With Normal Baseline)   [ Time Frame: Screening, Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192) ]

7.  Secondary:   Number of Participants With Vital Sign Data That Met Criteria for Potential Clinical Concern (Absolute Values)   [ Time Frame: Screening, Day 1, 28, 91, and 182 ]

8.  Secondary:   Number of Participants With Vital Sign Data That Met Criteria for Potential Clinical Concern (Increase From Baseline)   [ Time Frame: Screening, Day 1, 28, 91, and 182 ]

9.  Secondary:   Number of Participants With Vital Sign Data That Met Criteria for Potential Clinical Concern (Decrease From Baseline)   [ Time Frame: Screening, Day 1, 28, 91, and 182 ]

10.  Secondary:   Number of Participants With Electrocardiogram (ECG) Data That Met Criteria for Potential Clinical Concern(Absolute Values)   [ Time Frame: Screening, Day 1, 28, 91, and 182 ]

11.  Secondary:   Number of Participants With Electrocardiogram (ECG) Data That Met Criteria for Potential Clinical Concern(Increase From Baseline)   [ Time Frame: Screening, Day 1, 28, 91, and 182 ]

12.  Secondary:   Severity of Adverse Events Related to Extrapyramidal Symptoms (EPS) Including Dystonia and Akathisia   [ Time Frame: Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192) ]

13.  Secondary:   Change From Baseline in the Total Maximum Chorea (TMC) Score of the UHDRS After 13 and 26 Weeks of Treatment.   [ Time Frame: Baseline, Week 13, Week 26 ]

14.  Secondary:   Number of Participants With Suicidal Ideation or Suicidal Behavior as Assessed by the Columbia Suicide Severity Rating Scale (C-SSRS) From Baseline to Follow-up Visit   [ Time Frame: Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192) ]

15.  Secondary:   Clinical Global Impression of Improvement (CGI-I) Scale Score After 13 and 26 Weeks of Treatment.   [ Time Frame: Week 13 & Week 26 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Pfizer ClinicalTrials.gov Call Center
Organization: Pfizer, Inc.
phone: 18007181021
e-mail: ClinicalTrials.gov_Inquiries@pfizer.com



Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT02197130     History of Changes
Other Study ID Numbers: A8241021
2014-001291-56 ( EudraCT Number )
First Submitted: July 16, 2014
First Posted: July 22, 2014
Results First Submitted: September 7, 2017
Results First Posted: November 17, 2017
Last Update Posted: November 17, 2017