Inherited ErythromelalgiaPhase 2IEMNCT01769274

Evaluation Of The Efficacy And Safety Of Single Doses Of PF-05089771 In Patients With Primary (Inherited) Erythromelalgia

A Phase 2 study testing PF-05089771 in people with Inherited Erythromelalgia, run by Pfizer at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial 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. Read more about this study

Who is it for?: Ages 18 to 78 years
Conditions the study is for: Inherited Erythromelalgia
What is being tested?: PF-05089771
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 5 participants
Where: 1 siteUnited States
Study dates: 22 Oct 2012to 12 Jul 2013
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 78 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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

Who cannot join

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.

Trial locations and contacts

1 site in 1 country.

United States1 site

New Haven Clinical Research Unit
New Haven, Connecticut, 06511

Other trials in United States

About this study

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

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.

Conditions: Inherited Erythromelalgia
Being tested: PF-05089771, Placebo

What participants receive

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

PF-05089771Drug
A single oral dose of PF-05089771 1600 mg solution to be administered on Day 1 of each treatment session. There are 2 treatment sessions, therefore 2 single oral doses of PF-05089771 will be adminstered.
PlaceboDrug
Placebo for PF-05089771 1600 mg solution administered in each treatment session. There are 2 treatment sessions, therefore 2 single oral doses of placebo will be administered.

Study groups in NCT01769274
Study group	Role	What they receive
PF-05089771 1600 mg	Experimental	Not described Receives: Drug: PF-05089771
Placebo comparator: matching placebo	Placebo comparator	Single oral dose of placebo for PF-05089771 1600 mg Receives: Drug: Placebo

What is being measured

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

Average Pain Intensity Numerical Rating Scale (PI-NRS) Score - From 0 to 4 Hours Post-dose
Participants rated severity of their pain at 11 point PI-NRS, by choosing a number between 0 and 10, where 0= no pain and 10= worst possible pain; higher scores signify more pain. The average pain score was calculated as the mean of the pain scores recorded every 15 minutes from 0 to 4 hours post-dose.
Measured: Every 15 minutes from 0 to 4 hours post-dose

Secondary outcomes (22)

Average PI-NRS Score - From Post Evoked Pain Time Point 2 (EP2) to 8 Hours Post-dose
Participants rated severity of their pain at 11 point PI-NRS, by choosing a number between 0 and 10. Where 0= no pain and 10= worst possible pain; higher scores signify more pain. Evoked pain time point 2= first time point post-dose when pain was evoked using the participant specific heat pain stimulus. EP2 is approximately a time point 4-5 hours post-dose. The average pain score was calculated as the mean of the pain scores recorded from EP2 to 8 hours post-dose.
Measured: Post EP2, every 5 minutes for the first hour, then every 15 minutes up to 8 hours post-dose
Average PI-NRS Scores - From Post Evoked Pain Time Point 3 (EP3) to 10 Hours Post-dose
Participants rated severity of their pain at 11 point PI-NRS, by choosing a number between 0 and 10. Where 0= no pain and 10= worst possible pain; higher scores signify more pain. Evoked pain time point 3= second time point post-dose when pain was evoked using the participant specific heat pain stimulus. EP3 is approximately a time point 8-9 hours post-dose. The average pain score was calculated as the mean of the pain scores recorded from EP3 to 10 hours post-dose.
Measured: Post EP3, every 5 minutes for the first hour, then every 15 minutes up to 10 hours post-dose
Average PI-NRS Scores - From Post Evoked Pain Time Point 4 (EP4) to 28 Hours Post-dose
Participants rated severity of their pain at 11 point PI-NRS, by choosing a number between 0 and 10. Where 0= no pain and 10= worst possible pain; higher scores signify more pain. Evoked pain time point 4= third time point post-dose when pain was evoked using the participant specific heat pain stimulus. EP4 is approximately a time point 24-25 hours post-dose. The average pain score was calculated as the mean of the pain scores recorded from EP4 to 28 hours post-dose.
Measured: Post EP4, every 5 minutes for the first hour, then every 15 minutes up to 28 hours post-dose
Maximum PI-NRS Scores - From 0 Hour to 4 Hours Post-dose
Participants rated severity of their pain at 11 point PI-NRS, by choosing a number between 0 and 10. Where 0= no pain and 10= worst possible pain; higher scores signify more pain. Maximum pain score in 4 hours period post-dosing is reported.
Measured: From 0 hour to 4 hours post-dose
Maximum PI-NRS Scores - From Post EP2 to 8 Hours Post-dose
Participants rated severity of their pain at 11 point PI-NRS, by choosing a number between 0 and 10. Where 0= no pain and 10= worst possible pain; higher scores signify more pain. Evoked pain time point 2= first time point post-dose when pain was evoked using the participant specific heat pain stimulus. EP2 is approximately a time point 4-5 hours post-dose. Maximum pain score in period from post EP2 to 8 hours post-dose is reported.
Measured: From post EP2 to 8 hours post-dose
Maximum PI-NRS Scores - From Post EP3 to 10 Hours Post-dose
Participants rated severity of their pain at 11 point PI-NRS, by choosing a number between 0 and 10. Where 0= no pain and 10= worst possible pain; higher scores signify more pain. Evoked pain time point 3= second time point post-dose when pain was evoked using the participant specific heat pain stimulus. EP3 is approximately a time point 8-9 hours post-dose. Maximum pain score in period from post EP3 to 10 hours post-dose is reported.
Measured: From the end of EP3 to 10 hours post dose
Maximum PI-NRS Scores - From Post EP4 to 28 Hours Post-dose
Participants rated severity of their pain at 11 point PI-NRS, by choosing a number between 0 and 10. Where 0= no pain and 10= worst possible pain; higher scores signify more pain. Evoked pain time point 4= third time point post-dose when pain was evoked using the participant specific heat pain stimulus. EP4 is approximately a time point 24-25 hours post-dose. Maximum pain score in period from post EP4 to 28 hours post-dose is reported.
Measured: Post EP4, every 5 minutes for the first hour, then every 15 minutes up to 28 hours post-dose
Duration When PI-NRS Scores Were Greater Than (>) 5 - From 0 Hour to 4 Hours Post-dose
Participants rated severity of their pain at 11 point PI-NRS, by choosing a number between 0 and 10. Where 0= no pain and 10= worst possible pain; higher scores signify more pain. The duration of time that participants experienced PI-NRS score greater than 5 from the 0 hours to 4 hours post-dose.
Measured: From 0 hour to 4 hours post-dose
Duration When PI-NRS Scores Were >5 - From Post EP2 to 8 Hours Post-dose
Participants rated severity of their pain at 11 point PI-NRS, by choosing a number between 0 and 10. Where 0= no pain and 10= worst possible pain; higher scores signify more pain. Evoked pain time point 2= first time point post-dose when pain was evoked using the participant specific heat pain stimulus. EP2 is approximately a time point 4-5 hours post-dose. The duration of time that participants experienced PI-NRS score >5 from in period from post EP2 to 8 hours post-dose is reported.
Measured: Post EP2, every 5 minutes for the first hour, then every 15 minutes up to 8 hours post-dose
Duration When PI-NRS Scores Were >5 - From Post EP3 to 10 Hours Post-dose
Participants rated severity of their pain at 11 point PI-NRS, by choosing a number between 0 and 10. Where 0= no pain and 10= worst possible pain; higher scores signify more pain. Evoked pain time point 3= second time point post-dose when pain was evoked using the participant specific heat pain stimulus. EP3 is approximately a time point 8-9 hours post-dose. The duration of time that participants experienced PI-NRS score >5 from the post EP3 to 10 hours post dose.
Measured: Post EP3, every 5 minutes for the first hour, then every 15 minutes up to 10 hours post-dose
Duration When PI-NRS Scores Were >5 - From Post EP4 to 28 Hours Post-dose
Participants rated severity of their pain at 11 point PI-NRS, by choosing a number between 0 and 10. Where 0= no pain and 10= worst possible pain; higher scores signify more pain. Evoked pain time point 4= third time point post-dose when pain was evoked using the participant specific heat pain stimulus. EP4 is approximately a time point 24-25 hours post-dose. The duration of time that participants experienced PI-NRS score >5 from post EP4 to 28 hours post-dose.
Measured: After EP4, every 5 minutes for the first hour, then every 15 minutes up to 28 hours post-dose
Number of Participants With Participant's Global Satisfaction Score
Participant was asked "How would you rate the study medication you received for pain?". The participant was provided the following choices as an answer: excellent=4; good=3; fair=2; poor=1. Response to this question, was participant's overall impression (global evaluation) of the study medication at 4 hour post-dose or at time of first rescue treatment or medication, which ever occurred first.
Measured: At 4 hour post-dose or at time of first rescue therapy, whichever occurred first
Time to First Use of Rescue Therapy or Medication
Time to rescue medication (hour) was calculated as: date/time of rescue medication minus date/time of first dose for each period. If participant who did not receive rescue medication, the time of censoring was cut off at 24 hours or the time of withdrawal, whichever was earlier. Kaplan-Meier method was used for estimation.
Measured: Up to maximum of 24 hours post-dose
Area Under the Plasma Concentration-Time Curve From Time Zero to the 24 Hour Post-dose (AUC24) of PF-05089771
Measured: Predose, 0.5, 2, 4, 6, and 24 hours post-dose
Area Under the Curve From Time Zero to Last Quantifiable Concentration (AUClast) of PF-05089771
Measured: Predose and 0.5, 2, 4, 6, and 24 hours post dose
Maximum Observed Plasma Concentration (Cmax) of PF-05089771
Measured: Predose and 0.5, 2, 4, 6, and 24 hours post dose
Time to Reach Maximum Observed Plasma Concentration (Tmax) of PF-05089771
Measured: Predose and 0.5, 2, 4, 6, and 24 hours post dose
Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. 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. A treatment emergent AE was defined as an event that emerged during the treatment period that was absent before treatment, or worsened during the treatment period relative to the pretreatment state.
Measured: Baseline up to a maximum of Day 83
Number of Participants With Laboratory Abnormalities
Laboratory abnormalities: Hemoglobin (Hgb), hematocrit, red blood cell count: less than(<)0.8*lower limit of normal(LLN), platelet: <0.5*LLN/greater than (>)1.75*upper limit of normal (ULN), white blood cell: <0.6*LLN/>1.5*ULN, lymphocyte, neutrophil (absolute, %):<0.8*LLN/>1.2*ULN, total neutrophil <0.8*LLN;basophil, eosinophil, monocyte (absolute, %):>1.2*ULN; mean corpuscular (MC) volume, mean cell hemoglobin, MC hemoglobin concentration, mean platelet volume: <0.9*LLN/>1.1*ULN; total bilirubin >1.5*ULN, aspartate aminotransferase (AT), alanine AT, gammaglutamyl transferase, alkaline phosphatase:> 3.0*ULN, total protein, albumin: <0.8*LLN/>1.2*ULN; blood urea nitrogen, creatinine:>1.3*ULN, uric acid >1.2*ULN; sodium <0.95*LLN/>1.05*ULN, potassium, chloride, calcium, magnesium, bicarbonate: <0.9*LLN/>1.1*ULN; glucose <0.6*LLN/>1.5*ULN; urine (specific gravity <1.003/>1.030, pH <4.5/>8, glucose, ketone, protein, blood/Hgb, urobilinogen, bilirubin, nitrite, leukocyte esterase >=1).
Measured: Baseline up to a maximum of Day 73
Number of Participants With Clinically Significant Changes From Baseline in Core Body Temperature
The minimum starting temperature to measure core body temperature used was 33 degree Celsius. Clinically significant changes from baseline in core body temperature was judged by investigator.
Measured: Baseline up to a maximum of Day 83
Number of Participants With Clinically Significant Changes From Baseline in Blood Pressure
Criteria for clinically significant blood pressure abnormalities: systolic blood pressure >=30 millimetre of Mercury (mmHg) change from baseline in same posture, systolic blood pressure <90 mmHg, diastolic blood pressure >=20 mmHg change from baseline in same posture, diastolic blood pressure <50 mmHg.
Measured: Baseline up to a maximum of Day 83
Number of Participants With Clinically Significant Changes From Baseline in Electrocardiogram (ECG)
Criteria for clinically significant abnormalities in ECG : PR interval >=300 millisecond (msec) and 25 percent (%) increase when baseline >200 msec, 50% increase when baseline less than or equal to (<=) 200 msec; QRS interval >=140 msec, >=50% increase from baseline; QT interval >=500 msec; QT interval corrected using the Fridericia formula (QTcF) 450 msec to <480 msec, >=480 msec, 30 to <60 msec increase from baseline, >=60 msec increase from baseline.
Measured: Baseline up to a maximum of Day 83

Timeline

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

22 Oct 201212 Jul 2013

First posted16 Jan 2013 (estimated)
Study start22 Oct 2012
Primary completion12 Jul 2013
Study completion12 Jul 2013

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.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Inherited ErythromelalgiaStudies 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.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Crossover
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 5 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01769274
Sponsor's protocol ID: B3291006
Acronym: IEM
Status last verified: Nov 2019
First submitted: 18 Oct 2012
Last updated: 19 Nov 2019
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
Individual data sharing: Yes: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.

Publications

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.
PubMed 27099175

Study at a glance

From the registry record, last updated by the sponsor on 19 Nov 2019. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT01769274
Acronym / sponsor ID: IEM / B3291006
Conditions: Inherited Erythromelalgia
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 5 participants (actual)
Lead sponsor: PfizerIndustry (pharma, biotech, devices)
Study start: 22 Oct 2012 (actual)
Primary completion: 12 Jul 2013 (actual)
Study completion: 12 Jul 2013 (actual)
First posted: 16 Jan 2013 (estimated)
Results: Posted 19 Nov 2019

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Crossover
Masking: Double blind (participant, investigator)
Number of arms: 2

Arm	Type	Receives
PF-05089771 1600 mg	Experimental	Drug: PF-05089771
Placebo comparator: matching placebo	Placebo comparator	Drug: PlaceboSingle oral dose of placebo for PF-05089771 1600 mg

Who may qualify

Ages: 18 to 78 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (3 listed)

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

Key exclusion criteria (3 listed)

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
PF-05089771A single oral dose of PF-05089771 1600 mg solution to be administered on Day 1 of each treatment session. There are 2 treatment sessions, therefore 2 single oral doses of PF-05089771 will be adminstered.	Drug	PF-05089771 1600 mg
PlaceboPlacebo for PF-05089771 1600 mg solution administered in each treatment session. There are 2 treatment sessions, therefore 2 single oral doses of placebo will be administered.	Drug	Placebo comparator: matching placebo

Primary outcome

Average Pain Intensity Numerical Rating Scale (PI-NRS) Score - From 0 to 4 Hours Post-dose
Time frame: Every 15 minutes from 0 to 4 hours post-dose
Participants rated severity of their pain at 11 point PI-NRS, by choosing a number between 0 and 10, where 0= no pain and 10= worst possible pain; higher scores signify more pain. The average pain score was calculated as the mean of the pain scores recorded every 15 minutes from 0 to 4 hours post-dose.

22 secondary outcomes

Average PI-NRS Score - From Post Evoked Pain Time Point 2 (EP2) to 8 Hours Post-dose
Time frame: Post EP2, every 5 minutes for the first hour, then every 15 minutes up to 8 hours post-dose
Average PI-NRS Scores - From Post Evoked Pain Time Point 3 (EP3) to 10 Hours Post-dose
Time frame: Post EP3, every 5 minutes for the first hour, then every 15 minutes up to 10 hours post-dose
Average PI-NRS Scores - From Post Evoked Pain Time Point 4 (EP4) to 28 Hours Post-dose
Time frame: Post EP4, every 5 minutes for the first hour, then every 15 minutes up to 28 hours post-dose
Maximum PI-NRS Scores - From 0 Hour to 4 Hours Post-dose
Time frame: From 0 hour to 4 hours post-dose
Maximum PI-NRS Scores - From Post EP2 to 8 Hours Post-dose
Time frame: From post EP2 to 8 hours post-dose
Maximum PI-NRS Scores - From Post EP3 to 10 Hours Post-dose
Time frame: From the end of EP3 to 10 hours post dose
Maximum PI-NRS Scores - From Post EP4 to 28 Hours Post-dose
Time frame: Post EP4, every 5 minutes for the first hour, then every 15 minutes up to 28 hours post-dose
Duration When PI-NRS Scores Were Greater Than (>) 5 - From 0 Hour to 4 Hours Post-dose
Time frame: From 0 hour to 4 hours post-dose
Duration When PI-NRS Scores Were >5 - From Post EP2 to 8 Hours Post-dose
Time frame: Post EP2, every 5 minutes for the first hour, then every 15 minutes up to 8 hours post-dose
Duration When PI-NRS Scores Were >5 - From Post EP3 to 10 Hours Post-dose
Time frame: Post EP3, every 5 minutes for the first hour, then every 15 minutes up to 10 hours post-dose
Duration When PI-NRS Scores Were >5 - From Post EP4 to 28 Hours Post-dose
Time frame: After EP4, every 5 minutes for the first hour, then every 15 minutes up to 28 hours post-dose
Number of Participants With Participant's Global Satisfaction Score
Time frame: At 4 hour post-dose or at time of first rescue therapy, whichever occurred first
Time to First Use of Rescue Therapy or Medication
Time frame: Up to maximum of 24 hours post-dose
Area Under the Plasma Concentration-Time Curve From Time Zero to the 24 Hour Post-dose (AUC24) of PF-05089771
Time frame: Predose, 0.5, 2, 4, 6, and 24 hours post-dose
Area Under the Curve From Time Zero to Last Quantifiable Concentration (AUClast) of PF-05089771
Time frame: Predose and 0.5, 2, 4, 6, and 24 hours post dose
Maximum Observed Plasma Concentration (Cmax) of PF-05089771
Time frame: Predose and 0.5, 2, 4, 6, and 24 hours post dose
Time to Reach Maximum Observed Plasma Concentration (Tmax) of PF-05089771
Time frame: Predose and 0.5, 2, 4, 6, and 24 hours post dose
Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Baseline up to a maximum of Day 83
Number of Participants With Laboratory Abnormalities
Time frame: Baseline up to a maximum of Day 73
Number of Participants With Clinically Significant Changes From Baseline in Core Body Temperature
Time frame: Baseline up to a maximum of Day 83
Number of Participants With Clinically Significant Changes From Baseline in Blood Pressure
Time frame: Baseline up to a maximum of Day 83
Number of Participants With Clinically Significant Changes From Baseline in Electrocardiogram (ECG)
Time frame: Baseline up to a maximum of Day 83

Results and safety

Results posted to the registry on 19 Nov 2019, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Part A	5	5
Part B: PF-05089771 Then Placebo Then Placebo Then PF-05089771	0	0
Part B: Placebo Then PF-05089771 Then PF-05089771 Then Placebo	0	0

Primary outcome: Average Pain Intensity Numerical Rating Scale (PI-NRS) Score - From 0 to 4 Hours Post-dose

Time frame: Every 15 minutes from 0 to 4 hours post-dose. Mean (Standard Deviation). Unit: Units on a scale

Measure	Treatment Session 1: PF-05089771 1600 mg n = 5	Treatment Session 1: Placebo n = 5	Treatment Session 2: PF-05089771 1600 mg n = 5	Treatment Session 2: Placebo n = 5
Value	2.08 (2.20)	2.73 (2.68)	1.95 (2.29)	2.04 (1.81)

Population analysed: Full analysis set included all participants who successfully completed Part A, were randomized in Part B, and who received at least 1 dose of randomized study medication.

22 secondary outcomes are reported in the full results.

Adverse events

Other adverse events reported at 1% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Treatment Session 1: PF-05089771 1600 mg	0 of 5 (0%)	5 of 5 (100%)	Not reported
Treatment Session 1: Placebo	0 of 5 (0%)	5 of 5 (100%)	Not reported
Treatment Session 2: PF-05089771 1600 mg	0 of 5 (0%)	5 of 5 (100%)	Not reported
Treatment Session 2: Placebo	0 of 5 (0%)	4 of 5 (80%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 19 Nov 2019.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 27099175Cao 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.
To obtain contact information for a study center near you, click here.Link provided by the sponsor.
Individual participant dataSharing planned: Yes. Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Related trials for Inherited Erythromelalgia

Active, not recruitingPhase 1
A Phase 1b Study of BHV-7000 in Participants With Inherited Erythromelalgia
Familial Erythromelalgia

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Pfizer CT.gov Call Center study director

Pfizer

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing PF-05089771 in people with Inherited Erythromelalgia, run by Pfizer at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 19 Nov 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 19 Nov 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Average Pain Intensity Numerical Rating Scale (PI-NRS) Score - From 0 to 4 Hours Post-dose (time frame: Every 15 minutes from 0 to 4 hours post-dose). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01769274, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Nov 2019.

Trial summary

Key requirements

Who may be able to join

Who cannot join

Trial locations and contacts

About this study

What participants receive

What is being measured

Timeline

Resources

More details

Publications

Study at a glance

Clinical summary

Study design

Who may qualify

Key inclusion criteria (3 listed)

Key exclusion criteria (3 listed)

Interventions

Primary outcome

Results and safety

Participant flow

Primary outcome: Average Pain Intensity Numerical Rating Scale (PI-NRS) Score - From 0 to 4 Hours Post-dose

Adverse events

Research links

Related trials for Inherited Erythromelalgia

A Phase 1b Study of BHV-7000 in Participants With Inherited Erythromelalgia

Referral and contact

Questions about this trial

Participant flow

Primary outcome: Average Pain Intensity Numerical Rating Scale (PI-NRS) Score - From 0 to 4 Hours Post-dose

Adverse events