Study Evaluating The Safety, Tolerability And Brain Function Of 2 Doses Of PF-0254920 In Subjects With Early Huntington's Disease

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

• Ages 18 to 70 years
• All sexes
• No healthy volunteers
• Adult, Older adult

Trial locations and contacts

1 site in 1 country.

About this study

Official title: A Phase 2, Double-blind Randomized, Sequential Treatment Group, Placebo-controlled Study To Evaluate The Safety, Tolerability And Brain Cortico-striatal Function Of 2 Doses Of Pf-02545920 In Subjects With Early Huntington's Disease

Conditions

Huntington's Disease

Being tested

PF-02545920, Placebo

Medical subject headings

Huntington Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

• PF-02545920Drug

  • Dose will be titrated up every 2 days by 5mg increments: 5mg Days 1-2, 10mg days 3-4, 15mg days 5-6, and reach 20 mg from Days 7 to Day28.
  • Orally, approx. Q12H (range 10-14 hours), administered at least one hour prior to, or two hours after meals.
  • Treatment for 28 days.

• PlaceboDrug

  • Orally, approx. Q12H (range 10-14 hours), administered at least one hour prior to, or two hours after meals.

  Dosing for 28 days.

• PF-02545920Drug

  • 5mg dose
  • Orally, approx. Q12H (range 10-14 hours), administered at least one hour prior to, or two hours after meals.
  • Dosing for 28 days.

• PlaceboDrug

  • Orally, approx. Q12H (range 10-14 hours), administered at least one hour prior to, or two hours after meals.
  • Dosing for 28 days.

What is being measured

The primary outcome is the main question the study is designed to answer.

1. Number of Participants With Treatment-Emergent Adverse Events (AEs) or Serious Adverse Events (SAEs)

   An AE was any untoward medical occurrence without regard to causality in a participant who received study drug. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent are events between first dose of treatment and up to the follow-up period that were absent before treatment or that worsened relative to pretreatment state. AEs included both SAEs and non-serious AEs.

   Measured: Baseline up to Day 38

2. Number of Participants With Laboratory Abnormalities Meeting the Criteria for Potential Clinical Concern (Without Regard to Baseline Abnormality)

   The following laboratory parameters were analyzed: hematology (hemoglobin, hematocrit, red blood cell [RBC] count, platelet count, white blood cell [WBC] count, total neutrophils, eosinophils, monocytes, basophils, lymphocytes); blood chemistry (blood urea nitrogen [BUN], creatinine, glucose, calcium, sodium, potassium, chloride, total bicarbonate, aspartate aminotransferase [AST], alanine aminotransferase [ALT], total and direct bilirubin, alkaline phosphatase, uric acid, albumin, and total protein; urinalysis (pH, glucose, protein/albumin, hemoglobin/blood, ketones/acetone, nitrites, leukocyte esterase, microscopy [if urine dipstick was positive for blood, protein, nitrites or leukocyte esterase]); others (urine/serum pregnancy test, glycosylated hemoglobin [HbA1c, if diabetic]).

   Measured: Baseline up to Day 38

3. Number of Participants With Potentially Clinically Significant Vital Signs Findings

   Vital signs assessment included pulse rate and blood pressure. Criteria for vital sign values meeting potential clinical concern included: supine/sitting pulse rate less than (<)40 or greater than (>)120 beats per minute (bpm), standing pulse rate <40 or >140 bpm; systolic blood pressure (SBP) of greater than or equal to (>=)30 millimeters of mercury (mmHg) change from baseline or SBP <90 mmHg, diastolic blood pressure (DBP) >=20 mmHg change from baseline or DBP <50 mmHg.

   Measured: Baseline up to Day 38

4. Number of Participants With Potentially Clinically Significant Electrocardiogram (ECG) Findings

   ECG parameters included PR interval, QRS complex, and corrected QT interval using Fridericia's formula (QTcF). Criteria for ECG changes meeting potential clinical concern included: PR interval ≥200 milliseconds (msec) or ≥25% increase when baseline is >100 msec; QRS interval ≥50% increase from baseline when baseline is less than or equal to (<=)200 msec; and QTcF ≥450 msec or ≥30 msec increase from baseline.

   Measured: Baseline up to Day 38

5. Number of Participants With Change From Baseline in Body Weight of >=7%

   Weight assessment was performed by a study physician or a trained study nurse and was included in the physical examination.

   Measured: Baseline up to Day 38

6. Categorical Summary of Participants Meeting Stopping Criteria

   Absolute neutrophil count (ANC) and WBC were monitored for safety. Participants with WBC <3000 but >=2000 cells/mm^3 or ANC <1500 but >=1000 cells/mm^3 were to have study treatment suspended. Participants with WBC <2000 or ANC <1000 cells/mm^3 were to be discontinued from study participation.

   Measured: Baseline up to Day 38

7. Change From Baseline in Unified Huntington Disease Rating Scale (UHDRS) Total Motor Score at Day 28

   The UHDRS is a clinical rating scale to provide a uniform assessment of the clinical features and course of Huntington Disease. The Total Motor Score (TMS) is 1 of the 6 components of UHDRS, includes 31 items, and ranges from a scale of 0 to 124 (higher scores indicate more severe disease).

   Measured: Baseline, Day 28

8. Number of Participants With Suicidal Tendencies (C-SSRS Mapped to C-CASA) at Baseline

   C-SSRS is a participant rated questionnaire to assess suicidal ideation, suicidal behavior, actual attempts (yes or no responses), and intensity of ideation (rated 1=low severity to 5=high severity). Yes/No responses are mapped to Columbia Classification Algorithm of Suicide Assessment (C-CASA) categories: completed suicide, suicide attempt, preparatory acts towards imminent suicidal behavior, suicidal ideation, and self-injurious behavior, or no suicidal intent. A participant could have a yes or no response in more than 1 category.

   Measured: Baseline (Day 1)

9. Number of Participants With Suicidal Tendencies (C-SSRS Mapped to C-CASA) at Day 7

   C-SSRS is a participant rated questionnaire to assess suicidal ideation, suicidal behavior, actual attempts (yes or no responses), and intensity of ideation (rated 1=low severity to 5=high severity). Yes/No responses are mapped to Columbia Classification Algorithm of Suicide Assessment (C-CASA) categories: completed suicide, suicide attempt, preparatory acts toward imminent suicidal behavior, suicidal ideation, and self-injurious behavior, or no suicidal intent. A participant could have a yes or no response in more than 1 category.

   Measured: Day 7

10. Number of Participants With Suicidal Tendencies (C-SSRS Mapped to C-CASA) at Day 28

    C-SSRS is a participant rated questionnaire to assess suicidal ideation, suicidal behavior, actual attempts (yes or no responses), and intensity of ideation (rated 1=low severity to 5=high severity). Yes/No responses are mapped to Columbia Classification Algorithm of Suicide Assessment (C-CASA) categories: completed suicide, suicide attempt, preparatory acts toward imminent suicidal behavior, suicidal ideation, and self-injurious behavior, or no suicidal intent. A participant could have a yes or no response in more than 1 category.

    Measured: Day 28

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1. First posted7 Mar 2013 (estimated)
2. Study startSep 2013
3. Primary completionJan 2015
4. Study completionJan 2015

Resources

• Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
• Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
• Other trials for Huntington's DiseaseStudies of the same condition, open and under way.
• More trials run by PfizerThe sponsor's other registered studies.
• What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.