Phase 2 Completed N=272 Randomized Quadruple-blind Treatment

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

Huntington's Disease

Source: ClinicalTrials.gov NCT02197130 ↗

Enrolled (actual)

272

Serious AEs

6.3%

Results posted

Nov 2017

Primary outcomePrimary: Change From Baseline in the Total Motor Score (TMS) Assessment of the Unified Huntington Disease Rating Scale (UHDRS) After 26 Weeks of Treatment. — 0.4; -0.8; -1.4 units on a scale — p=0.2033

Summary

This study is a 26 week, randomized, parallel group, double blind comparison of PF-02545920 5 mg, PF-02545920 20 mg, and placebo dosed BID in the treatment of motor impairment of subjects with Huntington's Disease. A total of approximately 260 subjects are planned to be randomized in the study. Primary endpoint is the change from baseline in the Total Motor Score (TMS) assessment of the Unified Huntington Disease Rating Scale (UHDRS) after 26 weeks of treatment. secondary endpoints will include change from baseline in the Total Maximum Chorea (TMC) score of the UHDRS after 13 and 26 weeks of treatment and Clinical Global Impression-Improvement score after 13 and 26 weeks of treatment.

Outcome Measures

Outcome	Result	p-value
PRIMARY Change From Baseline in the Total Motor Score (TMS) Assessment of the Unified Huntington Disease Rating Scale (UHDRS) After 26 Weeks of Treatment.	0.4; -0.8; -1.4	0.2033
SECONDARY Number of Participants That Met White Blood Count (WBC) and Absolute Neutrophil Count (ANC) Stopping Criteria	0; 1; 0; 0; 0; 0	—
SECONDARY Number of Participants With Adverse Events	76; 82; 63	—
SECONDARY Number of Participants With Serious Adverse Events	8; 2; 7	—
SECONDARY Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormalities)	47; 46; 48	—
SECONDARY Number of Participants With Laboratory Test Abnormalities (With Normal Baseline)	40; 36; 41	—
SECONDARY Number of Participants With Vital Sign Data That Met Criteria for Potential Clinical Concern (Absolute Values)	1; 0; 0; 3; 2; 3	—
SECONDARY Number of Participants With Vital Sign Data That Met Criteria for Potential Clinical Concern (Increase From Baseline)	4; 1; 4; 7; 8; 6	—
SECONDARY Number of Participants With Vital Sign Data That Met Criteria for Potential Clinical Concern (Decrease From Baseline)	1; 7; 3; 9; 8; 9	—
SECONDARY Number of Participants With Electrocardiogram (ECG) Data That Met Criteria for Potential Clinical Concern(Absolute Values)	0; 0; 0; 0; 0; 0	—
SECONDARY Number of Participants With Electrocardiogram (ECG) Data That Met Criteria for Potential Clinical Concern(Increase From Baseline)	0; 0; 0; 0; 0; 0	—
SECONDARY Severity of Adverse Events Related to Extrapyramidal Symptoms (EPS) Including Dystonia and Akathisia	0; 0; 0; 0; 0; 0	—
SECONDARY Change From Baseline in the Total Maximum Chorea (TMC) Score of the UHDRS After 13 and 26 Weeks of Treatment.	1.1; -0.2; -0.9; 0.7; -0.4; -0.8	0.0030 sig
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	0; 0; 0; 1; 0; 0	—
SECONDARY Clinical Global Impression of Improvement (CGI-I) Scale Score After 13 and 26 Weeks of Treatment.	4.0; 3.7; 3.6; 3.9; 3.8; 3.8	0.0181 sig

Eligibility Criteria

Inclusion Criteria

CAG repeat equal or greater than 36;
Total motor score equal or greater than 10;
Total functional capacity equal or greater than 7.

Exclusion Criteria

Clinically significant neurologic disorder other than Huntington's disease;
Other severe acute psychiatric conditions, mania and/or psychosis;
History of neutropenia, and myeloproliferative disorders;

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02197130). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.