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| ID | Type | Description | Link |
|---|---|---|---|
| 2014-004900-31 | EudraCT Number | ||
| OPEN LABEL TO A8241021 | Other Identifier | Alias Study Number |
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Study terminated on 15DEC2016 due to study A8241021 showing no significant difference on primary endpoint between PF-02545920 & placebo. No safety concerns.
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This study is a 12 month open label extension study of PF-02545920 20 mg dosed BID following study A8241021 in subjects with HD. Primary endpoints will be to assess long-term safety and tolerability of 20 mg BID of PF-02545920. Secondary endpoints will be the change from baseline in the Total Motor Score (TMS)assessment, and/ior the Total maximum Chorea (TMC) assessment of the Unified Huntington Disease Rating Scale (UHDRS) after 6 and 12 months of treatment, and Clinical Global Impression-Improvement score after 6 and 12 months of treatment. Subjects, who were assigned to the 20 mg PF-02545920 dose group in the preceding A8241021 study, will receive 20 mg PF-02545920 without any titration. All other subjects will be titrated to the 20 mg BID dose 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. Up to 260 subjects may take part in this open label extension
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 20 mg BID PF-02545920 non-titrated | Experimental | Subjects who received 20 mg BID in completed study A8241021 will continue to receive 20 mg BID PF-02545920 |
|
| 20mg BID PF-02545920 titrated | Experimental | Subjects who received either Placebo or 5mg BID of PF-02545920 in completed study A8241021 will be titrated up to 20 mg with 5mg increment per week, over 4 weeks (5mg increment/wk) |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 20 mg BID of PF-02545920 | Drug | All subject who completed A8241021 will receive 20 mg BID (with or without titration) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events and Serious Adverse Events | An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a product or medical device, regardless of its causal relationship with study treatment. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; was life-threatening (immediate risk of death); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent AEs are events between first dose of study treatment and up to 28 calendar days after the last administration of study treatment that were absent before treatment or that worsened after treatment. AEs included both serious and non-serious AEs. | 1 year |
| Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) | The laboratory test included: hematology (hemoglobin, hematocrit, red blood cell count, platelet count, white blood cell count, absolute total neutrophils, eosinophils, monocytes, basophils, and lymphocytes), chemistry (blood urea nitrogen/urea, creatinine, glucose, glycosylated hemoglobin [diabetics only], calcium, phosphorus, magnesium, creatine kinase, sodium, potassium, chloride, total carbon dioxide, aspartate aminotransferase, alanine aminotransferase, lactate dehydrogenase, total bilirubin, alkaline phosphatase, uric acid, albumin, total protein), urinalysis (color, appearance, specific gravity, pH, qualitative glucose, qualitative protein, qualitative blood, ketones, nitrites, leukocyte esterase, and microscopy), and other tests (follicle stimulating hormone, urine drug screen, urine pregnancy [human chorionic gonadotropin, hCG], serum beta hCG). Abnormality was determined by the investigator. | 1 year |
| Number of Participants With Vital Signs Data Meeting Categorical Summarization Criteria | Number of participants with vital signs data meeting the following criteria is presented: (1) supine systolic blood pressure (SBP) <90 millimeters of mercury (mmHg); (2) standing SBP <90 mmHg; (3) supine diastolic blood pressure (DBP) <50 mmHg; (4) standing DBP <50 mmHg; (5) supine pulse rate <40 beats per minute (bpm); (6) supine pulse rate >120 bpm; (7) standing pulse rate <40 bpm; (8) standing pulse rate >140 bpm; (9) maximum increase from baseline in supine SBP >= 30 mmHg; (10) maximum increase from baseline in standing SBP >= 30 mmHg; (11) maximum increase from baseline in supine DBP >= 20 mmHg; (12) maximum increase from baseline in standing DBP >= 20 mmHg; (13) maximum decrease from baseline in supine SBP >=30 mmHg; (14) maximum decrease from baseline in standing SBP >=30 mmHg; (15) maximum decrease from baseline in supine DBP >=20 mmHg; (16) maximum decrease from baseline in standing DBP >=20 mmHg. |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Unified Huntington's Disease Rating Scale (UHDRS) Total Motor Score | The UHDRS is a clinical rating scale developed to provide a uniform assessment of the clinical features and course of Huntington's disease (HD). The components of the full UHDRS assess motor function, cognition, behavior and functional abilities. Total Motor Score (TMS) assesses motor features of HD with standardized ratings of oculomotor function, dysarthria, chorea, dystonia, gait, and postural ability. The total motor impairment scores was the sum of all the individual 31 motor sub-items (each rated from 0 to 4), with higher scores indicating more severe motor impairment than lower scores. The range of TMS is 0-124. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The Kirkland Clinic of UAB Hospital | Birmingham | Alabama | 35233 | United States | ||
| University of Alabama at Birmingham |
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| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
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This was an open-label extension study conducted in participants who had completed study A8241021 (NCT02197130). Treatment assignment was double-blinded from Day 1 to Day 21, and became open-label from Day 22, since all participants began receiving the same dose level from Day 22.
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| ID | Title | Description |
|---|---|---|
| FG000 | 20 mg PF-02545920 | Participants who received PF-02545920 20 mg twice daily (BID) in Study A8241021 continued to receive PF-02545920 20 mg BID for 12 months in this study. Four 5-mg tablets were administered orally each time. |
| FG001 | 5 mg PF-02545920 Titration up to 20 mg |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 29, 2017 | Feb 2, 2018 |
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| 1 year |
| Number of Participants With Electrocardiogram (ECG) Data Meeting Categorical Summarization Criteria | Maximum absolute values and increases from baseline were summarized for PR interval (interval between the start of the ECG P wave and the start of the QRS complex corresponding to the time between the onset of the atrial depolarization and onset of ventricular depolarization), QRS complex (time from Q wave to the end of the S wave corresponding to ventricular depolarization), and QTcF interval (time from ECG Q wave to the end of T wave corresponding to electrical systole, corrected for heart rate using Fridericia's formula). Number of participants with ECG data meeting the following criteria is presented: (1) PR interval >=300 msec; (2) QRS complex >=140 msec; (3) QTcF interval: 450 to <480 msec; (4) QTcF interval: 480 to <500 msec; (5) QTcF interval >=500 msec; (6) PR interval increase from baseline >=25/50 percent; (7) QRS complex increase from baseline >=50 percent; (8) QTcF interval increase from baseline: 30 to <60 msec; (9) QTcF interval increase from baseline >=60 msec. | 1 year |
| Number of Participants With Abnormal White Blood Cell Count and Absolute Neutrophil Count (Without Regard to Baseline Abnormality) | Number of participants with white blood cell (WBC) count and absolute neutrophil count (ANC) meeting the following criteria is presented: (1) WBC count <0.6 *the lower limit of normal (LLN); (2) WBC count >1.5 times the upper limit of normal (ULN); (3) ANC <0.8*LLN; and (4) ANC >1.2*ULN. | 1 year |
| Number of Participants With Laboratory Test Abnormalities (Normal Baseline) | The laboratory test included: hematology (hemoglobin, hematocrit, red blood cell count, platelet count, white blood cell count, absolute total neutrophils, eosinophils, monocytes, basophils, and lymphocytes), chemistry (blood urea nitrogen/urea, creatinine, glucose, glycosylated hemoglobin [diabetics only], calcium, phosphorus, magnesium, creatine kinase, sodium, potassium, chloride, total carbon dioxide, aspartate aminotransferase, alanine aminotransferase, lactate dehydrogenase, total bilirubin, alkaline phosphatase, uric acid, albumin, total protein), urinalysis (color, appearance, specific gravity, pH, qualitative glucose, qualitative protein, qualitative blood, ketones, nitrites, leukocyte esterase, and microscopy), and other tests (follicle stimulating hormone, urine drug screen, urine pregnancy [human chorionic gonadotropin, hCG], serum beta hCG). Abnormality was determined by the investigator. | 1 year |
| Number of Participants With Adverse Events Related to Extrapyramidal Symptoms by Severity | Adverse events related to extrapyramidal symptoms included dystonia, akathisia, tardive dyskinesia). Severity was assessed by the investigator. Mild means the AE didn't interfere with the participant's usual function. Moderate means the AE interfered to some extent the participant's usual function. Severe means the AE interfered significantly with the participant's usual function. | 1 year |
| Number of Participants in Each Columbia Classification Algorithm of Suicide Assessment (C-CASA) Category | Columbia Suicide Severity Rating Scale (C-SSRS) was an interview-based rating scale to systematically assess suicidal ideation and suicidal behavior, and was used in this study. C-SSRS responses were mapped onto the Columbia Classification Algorithm of Suicide Assessment (C-CASA). Number of participants with any of the following behaviors occurring since last visit was summarized: completed suicide; suicide attempt; preparatory acts towards suicide; suicidal ideation; self-injurious behavior (no suicidal intent). | Baseline (Day 1), Weeks 2 and 4, Months 3, 6, 9 and 12, follow-up visit (7-14 days after the last dose of Month 12) |
| Baseline (Day 1), Month 6, and Month 12 |
| Change From Baseline in Unified Huntington's Disease Rating Scale (UHDRS) Total Maximum Chorea (TMC) Score | The UHDRS is a clinical rating scale developed to provide a uniform assessment of the clinical features and course of Huntington's disease (HD). The components of the full UHDRS assess motor function, cognition, behavior and functional abilities. Total Maximum Chorea (TMC) is a subset of the TMS assessment, and composed of the scoring of 7 chorea assessments (face, orobuccolingual, trunk, right and left upper extremities, right and left lower extremities). Each assessment is rated from 0 to 4 (absent to prolonged). TMC is obtained by adding up each of the separate scores, leading to max score of 28. The minimum score is 0. The higher the score, the worse the symptoms. | Baseline (Day 1), Month 6, and Month 12 |
| Absolute Value in Clinical Global Impression of Improvement (CGI-I) Score | The Clinical Global Impression of Improvement (CGI-I) is a global measure of improvement or change based on the clinician's assessment of all available clinical data obtained from interviewing the participant. The CGI-I consists of a single 7-point rating of total improvement or change from baseline severity, regardless of whether or not the change is due entirely to drug treatment. Raters select 1 response based on the following question, "Compared to your participant's condition at the beginning of treatment, how much has he/she changed?" Scores are: 1: Very much improved; 2: Much improved; 3: Minimally improved; 4: No change; 5: Minimally worse; 6: Much worse; or 7: Very much worse. | Month 6 and Month 12 |
| Birmingham |
| Alabama |
| 35233 |
| United States |
| Mayo Clinic Arizona | Scottsdale | Arizona | 85259 | United States |
| University of California, Irvine | Irvine | California | 92697 | United States |
| Ronald Reagan UCLA Medical Center Drug Information Center | Los Angeles | California | 90095 | United States |
| UCLA Neurology Clinic | Los Angeles | California | 90095 | United States |
| UCLA Radiology | Los Angeles | California | 90095 | United States |
| Rocky Mountain Movement Disorders Center | Englewood | Colorado | 80113 | United States |
| University of Florida Center for Movement Disorders & Neurorestoration | Gainesville | Florida | 32607 | United States |
| Indiana University Health Neuroscience Center | Indianapolis | Indiana | 46202 | United States |
| Washington University School of Medicine | St Louis | Missouri | 63110 | United States |
| Albany Medical College | Albany | New York | 12208 | United States |
| Wake Forest Baptist Medical Center | Winston-Salem | North Carolina | 27157 | United States |
| The Ohio State University | Columbus | Ohio | 43221 | United States |
| The Wexner Medical Center at the Ohio State University | Columbus | Ohio | 43221 | United States |
| The Wright Center of Innovation- The Ohio State University | Columbus | Ohio | 43221 | United States |
| Baylor College of Medicine | Houston | Texas | 77030 | United States |
| The Centre For Huntington Disease, The University of British Columbia | Vancouver | British Columbia | V6T 2B5 | Canada |
| Center For Movement Disorders | Toronto | Ontario | M3B 2S7 | Canada |
| CHUM-Notre-Dame Hospital | Montreal | Quebec | H2L 4M1 | Canada |
| CHUM-Notre-Dame, Pharmacie | Montreal | Quebec | H4L 4M1 | Canada |
| Uniklinik RWTH Aachen | Aachen | 52074 | Germany |
| Charité - Universitätsmedizin Berlin | Berlin | 10117 | Germany |
| St. Josef Hospital | Bochum | 44791 | Germany |
| Friedrich-Alexander-Universität | Erlangen | 91054 | Germany |
| Universität zu Lübeck | Lübeck | 23562 | Germany |
| Philipps Universitat Marburg | Marburg | 35043 | Germany |
| Technische Universität München | München | 81675 | Germany |
| George-Huntington-Institut | Münster | 48149 | Germany |
| Kbo-Isar-Amper-Klinikum gGmbH | Taufkirchen | 84416 | Germany |
| Universitätsklinikum Ulm | Ulm | 89081 | Germany |
| Universitaetsklinikum Wuerzburg | Würzburg | 97080 | Germany |
| Copernicus Podmiot Leczniczy sp.zo.o | Gdansk | 80462 | Poland |
| Krakowska Akademia Neurologii Sp. zo.o | Krakow | 31505 | Poland |
| Solumed Centrum Medyczne | Poznan | 60-529 | Poland |
| Instytut Psychiatrii i Neurologii, I Klinika Neurologiczna | Warsaw | 02957 | Poland |
| Central Manchester University Hospitals NHS Foundation Trust | Oxford Road | Manchester | M13 9WL | United Kingdom |
| St Nicholas Hospital | Gosforth | Newcastle UPON TYNE | NE3 3XT | United Kingdom |
| Bimingham & Solihull Mental Health NHS Foundation Trust Department of Neuropsychiatry | Birmingham | WEST Midlands | B15 2FG | United Kingdom |
| NHS Grampian, Aberdeen Royal Infirmary, Clinical Genetics Centre | Aberdeen | AB25 2ZA | United Kingdom |
| Institute of Psychological Medicine and Clinical Neurosciences | Cardiff | CF14 4XW | United Kingdom |
| NHS Greater Glasgow and Clyde | Glasgow | G51 4TF | United Kingdom |
| Guy's and St. Thomas' NHS Foundation Trust | London | SE19RT | United Kingdom |
| University College London Hospitals Huntington's Diesease | London | wc1b 5eh | United Kingdom |
| University College London Hospitals NHS Foundation Trust | London | WC1N 3BG | United Kingdom |
| The National Institute for Health Research / Wellcome Trust Clinical Research Facility | Manchester | M13 9WL | United Kingdom |
| Newcastle Magnetic Resonance Centre | Newcastle upon Tyne | NE4 5PL | United Kingdom |
| Oxford University Hospitals NHS Trust | Oxford | OX3 9DU | United Kingdom |
| Sheffield Teaching Hospital NHS Foundation Trust | Sheffield | S10 2JF | United Kingdom |
| University Hospital Southampton NHS Foundation Trust, Wessex Neurological Centre | Southampton | SO16 6YD | United Kingdom |
Participants who received PF-02545920 5 mg twice daily (BID) in Study A8241021 were administered PF-02545920 orally according to a double-blind titration schedule in this study: 5 mg BID for 7 days (one 5-mg tablet and 3 placebo tablets); 10 mg BID for 7 days (two 5-mg tablets and 2 placebo tablets); 15 mg BID for 7 days (three 5-mg tablets and 1 placebo tablet); 20 mg BID to Month 12 (four 5-mg tablets). |
| FG002 | Placebo and Titration up to 20 mg PF-02545920 | Participants who received placebo twice daily (BID) in Study A8241021 were administered PF-02545920 orally according to a double-blind titration schedule in this study: 5 mg BID for 7 days (one 5-mg tablet and 3 placebo tablets); 10 mg BID for 7 days (two 5-mg tablets and 2 placebo tablets); 15 mg BID for 7 days (three 5-mg tablets and 1 placebo tablet); 20 mg BID to Month 12 (four 5-mg tablets). |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | 20 mg PF-02545920 | Participants who received PF-02545920 20 mg twice daily (BID) in Study A8241021 continued to receive PF-02545920 20 mg BID for 12 months in this study. Four 5-mg tablets were administered orally each time. |
| BG001 | 5 mg PF-02545920 Titration up to 20 mg | Participants who received PF-02545920 5 mg twice daily (BID) in Study A8241021 were administered PF-02545920 orally according to a double-blind titration schedule in this study: 5 mg BID for 7 days (one 5-mg tablet and 3 placebo tablets); 10 mg BID for 7 days (two 5-mg tablets and 2 placebo tablets); 15 mg BID for 7 days (three 5-mg tablets and 1 placebo tablet); 20 mg BID to Month 12 (four 5-mg tablets). |
| BG002 | Placebo and Titration up to 20 mg PF-02545920 | Participants who received placebo twice daily (BID) in Study A8241021 were administered PF-02545920 orally according to a double-blind titration schedule in this study: 5 mg BID for 7 days (one 5-mg tablet and 3 placebo tablets); 10 mg BID for 7 days (two 5-mg tablets and 2 placebo tablets); 15 mg BID for 7 days (three 5-mg tablets and 1 placebo tablet); 20 mg BID to Month 12 (four 5-mg tablets). |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
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| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Number | participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||
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| Primary | Number of Participants With Treatment-Emergent Adverse Events and Serious Adverse Events | An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a product or medical device, regardless of its causal relationship with study treatment. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; was life-threatening (immediate risk of death); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent AEs are events between first dose of study treatment and up to 28 calendar days after the last administration of study treatment that were absent before treatment or that worsened after treatment. AEs included both serious and non-serious AEs. | Safety analysis population included all participants who entered the extension study with at least 1 dose of study medication. | Posted | Count of Participants | Participants | 1 year |
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| Primary | Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) | The laboratory test included: hematology (hemoglobin, hematocrit, red blood cell count, platelet count, white blood cell count, absolute total neutrophils, eosinophils, monocytes, basophils, and lymphocytes), chemistry (blood urea nitrogen/urea, creatinine, glucose, glycosylated hemoglobin [diabetics only], calcium, phosphorus, magnesium, creatine kinase, sodium, potassium, chloride, total carbon dioxide, aspartate aminotransferase, alanine aminotransferase, lactate dehydrogenase, total bilirubin, alkaline phosphatase, uric acid, albumin, total protein), urinalysis (color, appearance, specific gravity, pH, qualitative glucose, qualitative protein, qualitative blood, ketones, nitrites, leukocyte esterase, and microscopy), and other tests (follicle stimulating hormone, urine drug screen, urine pregnancy [human chorionic gonadotropin, hCG], serum beta hCG). Abnormality was determined by the investigator. | Safety analysis population included all participants who entered the extension study with at least 1 dose of study medication. | Posted | Count of Participants | Participants | 1 year |
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| Primary | Number of Participants With Vital Signs Data Meeting Categorical Summarization Criteria | Number of participants with vital signs data meeting the following criteria is presented: (1) supine systolic blood pressure (SBP) <90 millimeters of mercury (mmHg); (2) standing SBP <90 mmHg; (3) supine diastolic blood pressure (DBP) <50 mmHg; (4) standing DBP <50 mmHg; (5) supine pulse rate <40 beats per minute (bpm); (6) supine pulse rate >120 bpm; (7) standing pulse rate <40 bpm; (8) standing pulse rate >140 bpm; (9) maximum increase from baseline in supine SBP >= 30 mmHg; (10) maximum increase from baseline in standing SBP >= 30 mmHg; (11) maximum increase from baseline in supine DBP >= 20 mmHg; (12) maximum increase from baseline in standing DBP >= 20 mmHg; (13) maximum decrease from baseline in supine SBP >=30 mmHg; (14) maximum decrease from baseline in standing SBP >=30 mmHg; (15) maximum decrease from baseline in supine DBP >=20 mmHg; (16) maximum decrease from baseline in standing DBP >=20 mmHg. | The analysis population included all participants who entered the extension study with at least 1 dose of study medication, and with available data for at least 1 category. | Posted | Count of Participants | Participants | 1 year |
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| Primary | Number of Participants With Electrocardiogram (ECG) Data Meeting Categorical Summarization Criteria | Maximum absolute values and increases from baseline were summarized for PR interval (interval between the start of the ECG P wave and the start of the QRS complex corresponding to the time between the onset of the atrial depolarization and onset of ventricular depolarization), QRS complex (time from Q wave to the end of the S wave corresponding to ventricular depolarization), and QTcF interval (time from ECG Q wave to the end of T wave corresponding to electrical systole, corrected for heart rate using Fridericia's formula). Number of participants with ECG data meeting the following criteria is presented: (1) PR interval >=300 msec; (2) QRS complex >=140 msec; (3) QTcF interval: 450 to <480 msec; (4) QTcF interval: 480 to <500 msec; (5) QTcF interval >=500 msec; (6) PR interval increase from baseline >=25/50 percent; (7) QRS complex increase from baseline >=50 percent; (8) QTcF interval increase from baseline: 30 to <60 msec; (9) QTcF interval increase from baseline >=60 msec. | The analysis population included all participants who entered the extension study with at least 1 dose of study medication, and with available data for at least 1 category. | Posted | Count of Participants | Participants | 1 year |
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| Primary | Number of Participants With Abnormal White Blood Cell Count and Absolute Neutrophil Count (Without Regard to Baseline Abnormality) | Number of participants with white blood cell (WBC) count and absolute neutrophil count (ANC) meeting the following criteria is presented: (1) WBC count <0.6 *the lower limit of normal (LLN); (2) WBC count >1.5 times the upper limit of normal (ULN); (3) ANC <0.8*LLN; and (4) ANC >1.2*ULN. | The analysis population included all participants who entered the extension study with at least 1 dose of study medication, and with available data for at least 1 category. | Posted | Count of Participants | Participants | 1 year |
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| Primary | Number of Participants With Laboratory Test Abnormalities (Normal Baseline) | The laboratory test included: hematology (hemoglobin, hematocrit, red blood cell count, platelet count, white blood cell count, absolute total neutrophils, eosinophils, monocytes, basophils, and lymphocytes), chemistry (blood urea nitrogen/urea, creatinine, glucose, glycosylated hemoglobin [diabetics only], calcium, phosphorus, magnesium, creatine kinase, sodium, potassium, chloride, total carbon dioxide, aspartate aminotransferase, alanine aminotransferase, lactate dehydrogenase, total bilirubin, alkaline phosphatase, uric acid, albumin, total protein), urinalysis (color, appearance, specific gravity, pH, qualitative glucose, qualitative protein, qualitative blood, ketones, nitrites, leukocyte esterase, and microscopy), and other tests (follicle stimulating hormone, urine drug screen, urine pregnancy [human chorionic gonadotropin, hCG], serum beta hCG). Abnormality was determined by the investigator. | Safety analysis population included all participants who entered the extension study with at least 1 dose of study medication. | Posted | Count of Participants | Participants | 1 year |
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| Primary | Number of Participants With Adverse Events Related to Extrapyramidal Symptoms by Severity | Adverse events related to extrapyramidal symptoms included dystonia, akathisia, tardive dyskinesia). Severity was assessed by the investigator. Mild means the AE didn't interfere with the participant's usual function. Moderate means the AE interfered to some extent the participant's usual function. Severe means the AE interfered significantly with the participant's usual function. | Safety analysis population included all participants who entered the extension study with at least 1 dose of study medication. | Posted | Count of Participants | Participants | 1 year |
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| Primary | Number of Participants in Each Columbia Classification Algorithm of Suicide Assessment (C-CASA) Category | Columbia Suicide Severity Rating Scale (C-SSRS) was an interview-based rating scale to systematically assess suicidal ideation and suicidal behavior, and was used in this study. C-SSRS responses were mapped onto the Columbia Classification Algorithm of Suicide Assessment (C-CASA). Number of participants with any of the following behaviors occurring since last visit was summarized: completed suicide; suicide attempt; preparatory acts towards suicide; suicidal ideation; self-injurious behavior (no suicidal intent). | The analysis population included all participants who entered the extension study with at least 1 dose of study medication, and with available data for at least 1 time point. | Posted | Count of Participants | Participants | Baseline (Day 1), Weeks 2 and 4, Months 3, 6, 9 and 12, follow-up visit (7-14 days after the last dose of Month 12) |
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| Secondary | Change From Baseline in Unified Huntington's Disease Rating Scale (UHDRS) Total Motor Score | The UHDRS is a clinical rating scale developed to provide a uniform assessment of the clinical features and course of Huntington's disease (HD). The components of the full UHDRS assess motor function, cognition, behavior and functional abilities. Total Motor Score (TMS) assesses motor features of HD with standardized ratings of oculomotor function, dysarthria, chorea, dystonia, gait, and postural ability. The total motor impairment scores was the sum of all the individual 31 motor sub-items (each rated from 0 to 4), with higher scores indicating more severe motor impairment than lower scores. The range of TMS is 0-124. | Full analysis set (FAS) included all participants who had an open-label extension study baseline efficacy evaluation and completed at least Week 2 visit with a valid UHDRS TMS score, and took >=1 dose of open-label study medication. | Posted | Mean | Standard Deviation | units on a scale | Baseline (Day 1), Month 6, and Month 12 |
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| Secondary | Change From Baseline in Unified Huntington's Disease Rating Scale (UHDRS) Total Maximum Chorea (TMC) Score | The UHDRS is a clinical rating scale developed to provide a uniform assessment of the clinical features and course of Huntington's disease (HD). The components of the full UHDRS assess motor function, cognition, behavior and functional abilities. Total Maximum Chorea (TMC) is a subset of the TMS assessment, and composed of the scoring of 7 chorea assessments (face, orobuccolingual, trunk, right and left upper extremities, right and left lower extremities). Each assessment is rated from 0 to 4 (absent to prolonged). TMC is obtained by adding up each of the separate scores, leading to max score of 28. The minimum score is 0. The higher the score, the worse the symptoms. | Full analysis set (FAS) included all participants who had an open-label extension study baseline efficacy evaluation and completed at least Week 2 visit with a valid UHDRS TMS score, and took >=1 dose of open-label study medication. | Posted | Mean | Standard Deviation | Units on a scale | Baseline (Day 1), Month 6, and Month 12 |
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| Secondary | Absolute Value in Clinical Global Impression of Improvement (CGI-I) Score | The Clinical Global Impression of Improvement (CGI-I) is a global measure of improvement or change based on the clinician's assessment of all available clinical data obtained from interviewing the participant. The CGI-I consists of a single 7-point rating of total improvement or change from baseline severity, regardless of whether or not the change is due entirely to drug treatment. Raters select 1 response based on the following question, "Compared to your participant's condition at the beginning of treatment, how much has he/she changed?" Scores are: 1: Very much improved; 2: Much improved; 3: Minimally improved; 4: No change; 5: Minimally worse; 6: Much worse; or 7: Very much worse. | Full analysis set (FAS) included all participants who had an open-label extension study baseline efficacy evaluation and completed at least Week 2 visit with a valid UHDRS TMS score, and took >=1 dose of open-label study medication. | Posted | Mean | Standard Deviation | units on a scale | Month 6 and Month 12 |
|
1 year
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 20 mg PF-02545920 | Participants who received PF-02545920 20 mg twice daily (BID) in Study A8241021 continued to receive PF-02545920 20 mg BID for 12 months in this study. Four 5-mg tablets were administered orally each time. | 0 | 51 | 7 | 51 | 32 | 51 |
| EG001 | 5 mg PF-02545920 Titration up to 20 mg | Participants who received PF-02545920 5 mg twice daily (BID) in Study A8241021 were administered PF-02545920 orally according to a double-blind titration schedule in this study: 5 mg BID for 7 days (one 5-mg tablet and 3 placebo tablets); 10 mg BID for 7 days (two 5-mg tablets and 2 placebo tablets); 15 mg BID for 7 days (three 5-mg tablets and 1 placebo tablet); 20 mg BID to Month 12 (four 5-mg tablets). | 0 | 71 | 9 | 71 | 53 | 71 |
| EG002 | Placebo and Titration up to 20 mg PF-02545920 | Participants who received placebo twice daily (BID) in Study A8241021 were administered PF-02545920 orally according to a double-blind titration schedule in this study: 5 mg BID for 7 days (one 5-mg tablet and 3 placebo tablets); 10 mg BID for 7 days (two 5-mg tablets and 2 placebo tablets); 15 mg BID for 7 days (three 5-mg tablets and 1 placebo tablet); 20 mg BID to Month 12 (four 5-mg tablets). | 0 | 66 | 5 | 66 | 54 | 66 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Huntington's disease | Congenital, familial and genetic disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Complication associated with device | General disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Cervical vertebral fracture | Injury, poisoning and procedural complications | MedDRA 20.0 | Non-systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 20.0 | Non-systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA 20.0 | Non-systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA 20.0 | Non-systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Intervertebral disc protrusion | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Chorea | Nervous system disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Hyperkinesia | Nervous system disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Transient ischaemic attack | Nervous system disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Agitation | Psychiatric disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Panic attack | Psychiatric disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Suicide attempt | Psychiatric disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Urinary tract disorder | Renal and urinary disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Prostatomegaly | Reproductive system and breast disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 20.0 | Non-systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 20.0 | Non-systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA 20.0 | Non-systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Akathisia | Nervous system disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Chorea | Nervous system disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Dyskinesia | Nervous system disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Hyperkinesia | Nervous system disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Sedation | Nervous system disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Restlessness | Psychiatric disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Sleep disorder | Psychiatric disorders | MedDRA 20.0 | Non-systematic Assessment |
|
This study was terminated on 15 December 2016 due to study A8241021 showing no significant difference on primary endpoint between PF-02545920 and placebo. No safety concerns were associated with this termination.
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer, Inc. | 1-800-718-1021 | ClinicalTrials.gov_Inquiries@pfizer.com |
| SAP_000.pdf |
| Prot | Yes | No | No | Study Protocol | Oct 23, 2014 | Feb 2, 2018 | Prot_001.pdf |
| ID | Term |
|---|---|
| D006816 | Huntington Disease |
| D002819 | Chorea |
| D001526 | Behavioral Symptoms |
| ID | Term |
|---|---|
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D003704 | Dementia |
| D020820 | Dyskinesias |
| D009069 | Movement Disorders |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D003072 | Cognition Disorders |
| D019965 | Neurocognitive Disorders |
| D001523 | Mental Disorders |
| D009461 | Neurologic Manifestations |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D001519 | Behavior |
Not provided
Not provided
| Male |
|
| Other |
|
| Title | Measurements |
|---|---|
|
Participants who received PF-02545920 5 mg twice daily (BID) in Study A8241021 were administered PF-02545920 orally according to a double-blind titration schedule in this study: 5 mg BID for 7 days (one 5-mg tablet and 3 placebo tablets); 10 mg BID for 7 days (two 5-mg tablets and 2 placebo tablets); 15 mg BID for 7 days (three 5-mg tablets and 1 placebo tablet); 20 mg BID to Month 12 (four 5-mg tablets).
| OG002 | Placebo and Titration up to 20 mg PF-02545920 | Participants who received placebo twice daily (BID) in Study A8241021 were administered PF-02545920 orally according to a double-blind titration schedule in this study: 5 mg BID for 7 days (one 5-mg tablet and 3 placebo tablets); 10 mg BID for 7 days (two 5-mg tablets and 2 placebo tablets); 15 mg BID for 7 days (three 5-mg tablets and 1 placebo tablet); 20 mg BID to Month 12 (four 5-mg tablets). |
|
|
Participants who received PF-02545920 5 mg twice daily (BID) in Study A8241021 were administered PF-02545920 orally according to a double-blind titration schedule in this study: 5 mg BID for 7 days (one 5-mg tablet and 3 placebo tablets); 10 mg BID for 7 days (two 5-mg tablets and 2 placebo tablets); 15 mg BID for 7 days (three 5-mg tablets and 1 placebo tablet); 20 mg BID to Month 12 (four 5-mg tablets). |
| OG002 | Placebo and Titration up to 20 mg PF-02545920 | Participants who received placebo twice daily (BID) in Study A8241021 were administered PF-02545920 orally according to a double-blind titration schedule in this study: 5 mg BID for 7 days (one 5-mg tablet and 3 placebo tablets); 10 mg BID for 7 days (two 5-mg tablets and 2 placebo tablets); 15 mg BID for 7 days (three 5-mg tablets and 1 placebo tablet); 20 mg BID to Month 12 (four 5-mg tablets). |
|
|
| 5 mg PF-02545920 Titration up to 20 mg |
Participants who received PF-02545920 5 mg twice daily (BID) in Study A8241021 were administered PF-02545920 orally according to a double-blind titration schedule in this study: 5 mg BID for 7 days (one 5-mg tablet and 3 placebo tablets); 10 mg BID for 7 days (two 5-mg tablets and 2 placebo tablets); 15 mg BID for 7 days (three 5-mg tablets and 1 placebo tablet); 20 mg BID to Month 12 (four 5-mg tablets). |
| OG002 | Placebo and Titration up to 20 mg PF-02545920 | Participants who received placebo twice daily (BID) in Study A8241021 were administered PF-02545920 orally according to a double-blind titration schedule in this study: 5 mg BID for 7 days (one 5-mg tablet and 3 placebo tablets); 10 mg BID for 7 days (two 5-mg tablets and 2 placebo tablets); 15 mg BID for 7 days (three 5-mg tablets and 1 placebo tablet); 20 mg BID to Month 12 (four 5-mg tablets). |
|
|
| OG002 |
| Placebo and Titration up to 20 mg PF-02545920 |
Participants who received placebo twice daily (BID) in Study A8241021 were administered PF-02545920 orally according to a double-blind titration schedule in this study: 5 mg BID for 7 days (one 5-mg tablet and 3 placebo tablets); 10 mg BID for 7 days (two 5-mg tablets and 2 placebo tablets); 15 mg BID for 7 days (three 5-mg tablets and 1 placebo tablet); 20 mg BID to Month 12 (four 5-mg tablets). |
|
|
Participants who received PF-02545920 5 mg twice daily (BID) in Study A8241021 were administered PF-02545920 orally according to a double-blind titration schedule in this study: 5 mg BID for 7 days (one 5-mg tablet and 3 placebo tablets); 10 mg BID for 7 days (two 5-mg tablets and 2 placebo tablets); 15 mg BID for 7 days (three 5-mg tablets and 1 placebo tablet); 20 mg BID to Month 12 (four 5-mg tablets).
| OG002 | Placebo and Titration up to 20 mg PF-02545920 | Participants who received placebo twice daily (BID) in Study A8241021 were administered PF-02545920 orally according to a double-blind titration schedule in this study: 5 mg BID for 7 days (one 5-mg tablet and 3 placebo tablets); 10 mg BID for 7 days (two 5-mg tablets and 2 placebo tablets); 15 mg BID for 7 days (three 5-mg tablets and 1 placebo tablet); 20 mg BID to Month 12 (four 5-mg tablets). |
|
|
| OG002 |
| Placebo and Titration up to 20 mg PF-02545920 |
Participants who received placebo twice daily (BID) in Study A8241021 were administered PF-02545920 orally according to a double-blind titration schedule in this study: 5 mg BID for 7 days (one 5-mg tablet and 3 placebo tablets); 10 mg BID for 7 days (two 5-mg tablets and 2 placebo tablets); 15 mg BID for 7 days (three 5-mg tablets and 1 placebo tablet); 20 mg BID to Month 12 (four 5-mg tablets). |
|
|
| OG002 | Placebo and Titration up to 20 mg PF-02545920 | Participants who received placebo twice daily (BID) in Study A8241021 were administered PF-02545920 orally according to a double-blind titration schedule in this study: 5 mg BID for 7 days (one 5-mg tablet and 3 placebo tablets); 10 mg BID for 7 days (two 5-mg tablets and 2 placebo tablets); 15 mg BID for 7 days (three 5-mg tablets and 1 placebo tablet); 20 mg BID to Month 12 (four 5-mg tablets). |
|
|
| OG002 | Placebo and Titration up to 20 mg PF-02545920 | Participants who received placebo twice daily (BID) in Study A8241021 were administered PF-02545920 orally according to a double-blind titration schedule in this study: 5 mg BID for 7 days (one 5-mg tablet and 3 placebo tablets); 10 mg BID for 7 days (two 5-mg tablets and 2 placebo tablets); 15 mg BID for 7 days (three 5-mg tablets and 1 placebo tablet); 20 mg BID to Month 12 (four 5-mg tablets). |
|
|
| OG002 | Placebo and Titration up to 20 mg PF-02545920 | Participants who received placebo twice daily (BID) in Study A8241021 were administered PF-02545920 orally according to a double-blind titration schedule in this study: 5 mg BID for 7 days (one 5-mg tablet and 3 placebo tablets); 10 mg BID for 7 days (two 5-mg tablets and 2 placebo tablets); 15 mg BID for 7 days (three 5-mg tablets and 1 placebo tablet); 20 mg BID to Month 12 (four 5-mg tablets). |
|
|
| OG002 | Placebo and Titration up to 20 mg PF-02545920 | Participants who received placebo twice daily (BID) in Study A8241021 were administered PF-02545920 orally according to a double-blind titration schedule in this study: 5 mg BID for 7 days (one 5-mg tablet and 3 placebo tablets); 10 mg BID for 7 days (two 5-mg tablets and 2 placebo tablets); 15 mg BID for 7 days (three 5-mg tablets and 1 placebo tablet); 20 mg BID to Month 12 (four 5-mg tablets). |
|
|