Clinical trial NCT01769274: Evaluation Of The Efficacy And Safety Of Single Doses Of PF-05089771 In Patients With Primary (Inherited) Erythromelalgia
Official title: A RANDOMIZED, DOUBLE BLIND THIRD PARTY OPEN PLACEBO-CONTROLLED EXPLORATORY STUDY TO EVALUATE THE EFFICACY AND SAFETY OF SINGLE DOSES OF PF-05089771 IN PATIENTS WITH PRIMARY (INHERITED) ERYTHROMELALGIA
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Inherited Erythromelalgia
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2013-01-16
Enrollment: 5
Sex: ALL
Minimum age (years): 18
Maximum age (years): 78
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the efficacy and safety of single doses of PF-05089771 against placebo in treatment of pain in patients with primary, inherited erythromelalgia.


Eligibility criteria: Inclusion Criteria:

* Male and or female subjects between the ages of 18-78 years
* Subject has clinical signs of IEM
* Minimum BMI 17.5kg/m2 and total body weight \>50kg

Exclusion Criteria:

* Other severe pain conditions, e.g. rheumatologic, that may impair subject's self-assessment of pain due to IEM.
* Evidence of clinically significant hypertension, clinically significant hematological, dermatological, renal, endocrine (except diabetes mellitus), pulmonary, gastrointestinal, cardiovascular, hepatic, neurological (other than IEM), or allergic disease (including drug allergies but excluding untreated asymptomatic seasonal allergies).
* Subjects with severe obesity.

Primary outcomes:
measure: Average Pain Intensity Numerical Rating Scale (PI-NRS) Score - From 0 to 4 Hours Post-dose
timeFrame: Every 15 minutes from 0 to 4 hours post-dose
Secondary outcomes:
measure: Average PI-NRS Score - From Post Evoked Pain Time Point 2 (EP2) to 8 Hours Post-dose
timeFrame: Post EP2, every 5 minutes for the first hour, then every 15 minutes up to 8 hours post-dose
measure: Average PI-NRS Scores - From Post Evoked Pain Time Point 3 (EP3) to 10 Hours Post-dose
timeFrame: Post EP3, every 5 minutes for the first hour, then every 15 minutes up to 10 hours post-dose
measure: Average PI-NRS Scores - From Post Evoked Pain Time Point 4 (EP4) to 28 Hours Post-dose
timeFrame: Post EP4, every 5 minutes for the first hour, then every 15 minutes up to 28 hours post-dose
measure: Maximum PI-NRS Scores - From 0 Hour to 4 Hours Post-dose
timeFrame: From 0 hour to 4 hours post-dose
measure: Maximum PI-NRS Scores - From Post EP2 to 8 Hours Post-dose
timeFrame: From post EP2 to 8 hours post-dose
measure: Maximum PI-NRS Scores - From Post EP3 to 10 Hours Post-dose
timeFrame: From the end of EP3 to 10 hours post dose
measure: Maximum PI-NRS Scores - From Post EP4 to 28 Hours Post-dose
timeFrame: Post EP4, every 5 minutes for the first hour, then every 15 minutes up to 28 hours post-dose
measure: Duration When PI-NRS Scores Were Greater Than (>) 5 - From 0 Hour to 4 Hours Post-dose
timeFrame: From 0 hour to 4 hours post-dose
measure: Duration When PI-NRS Scores Were >5 - From Post EP2 to 8 Hours Post-dose
timeFrame: Post EP2, every 5 minutes for the first hour, then every 15 minutes up to 8 hours post-dose
measure: Duration When PI-NRS Scores Were >5 - From Post EP3 to 10 Hours Post-dose
timeFrame: Post EP3, every 5 minutes for the first hour, then every 15 minutes up to 10 hours post-dose
measure: Duration When PI-NRS Scores Were >5 - From Post EP4 to 28 Hours Post-dose
timeFrame: After EP4, every 5 minutes for the first hour, then every 15 minutes up to 28 hours post-dose
measure: Number of Participants With Participant's Global Satisfaction Score
timeFrame: At 4 hour post-dose or at time of first rescue therapy, whichever occurred first
measure: Time to First Use of Rescue Therapy or Medication
timeFrame: Up to maximum of 24 hours post-dose
measure: Area Under the Plasma Concentration-Time Curve From Time Zero to the 24 Hour Post-dose (AUC24) of PF-05089771
timeFrame: Predose, 0.5, 2, 4, 6, and 24 hours post-dose
measure: Area Under the Curve From Time Zero to Last Quantifiable Concentration (AUClast) of PF-05089771
timeFrame: Predose and 0.5, 2, 4, 6, and 24 hours post dose
measure: Maximum Observed Plasma Concentration (Cmax) of PF-05089771
timeFrame: Predose and 0.5, 2, 4, 6, and 24 hours post dose
measure: Time to Reach Maximum Observed Plasma Concentration (Tmax) of PF-05089771
timeFrame: Predose and 0.5, 2, 4, 6, and 24 hours post dose
measure: Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Baseline up to a maximum of Day 83
measure: Number of Participants With Laboratory Abnormalities
timeFrame: Baseline up to a maximum of Day 73
measure: Number of Participants With Clinically Significant Changes From Baseline in Core Body Temperature
timeFrame: Baseline up to a maximum of Day 83
measure: Number of Participants With Clinically Significant Changes From Baseline in Blood Pressure
timeFrame: Baseline up to a maximum of Day 83
measure: Number of Participants With Clinically Significant Changes From Baseline in Electrocardiogram (ECG)
timeFrame: Baseline up to a maximum of Day 83
Other outcomes:
Locations:
city: New Haven
country: United States
facility: New Haven Clinical Research Unit
state: Connecticut
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References:
citation: Cao L, McDonnell A, Nitzsche A, Alexandrou A, Saintot PP, Loucif AJ, Brown AR, Young G, Mis M, Randall A, Waxman SG, Stanley P, Kirby S, Tarabar S, Gutteridge A, Butt R, McKernan RM, Whiting P, Ali Z, Bilsland J, Stevens EB. Pharmacological reversal of a pain phenotype in iPSC-derived sensory neurons and patients with inherited erythromelalgia. Sci Transl Med. 2016 Apr 20;8(335):335ra56. doi: 10.1126/scitranslmed.aad7653.