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PF-06741086 Long-term Treatment in Severe Hemophilia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03363321
Recruitment Status : Completed
First Posted : December 6, 2017
Results First Posted : July 27, 2021
Last Update Posted : July 27, 2021
Sponsor:
Information provided by (Responsible Party):
Pfizer

Brief Summary:
This study is designed to evaluate the safety, tolerability and efficacy of long-term treatment with PF-06741086 in subjects with severe hemophilia who participated in the 3-month Phase 1b/2 B7841002 study. Additionally, de novo subjects will be recruited into this study.

Condition or disease Intervention/treatment Phase
Hemophilia A or B Biological: PF-06741086 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 20 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: A MULTICENTER, OPEN-LABEL STUDY TO EVALUATE THE LONG-TERM SAFETY, TOLERABILITY AND EFFICACY OF SUBCUTANEOUS PF-06741086 IN SUBJECTS WITH SEVERE HEMOPHILIA
Actual Study Start Date : May 30, 2018
Actual Primary Completion Date : August 5, 2020
Actual Study Completion Date : August 5, 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: PF-06741086 (Cohort 1) Biological: PF-06741086
PF-06741086 subcutaneous injection

Experimental: PF-06741086 (Cohort 2) Biological: PF-06741086
PF-06741086 subcutaneous injection

Experimental: PF-06741086 (Cohort 3) Biological: PF-06741086
PF-06741086 subcutaneous injection

Experimental: PF-06741086 (Cohort 4) Biological: PF-06741086
PF-06741086 subcutaneous injection

Experimental: PF-06741086 (Cohort 5) Biological: PF-06741086
PF-06741086 subcutaneous injection

Experimental: PF-06741086 (Cohort 6) Biological: PF-06741086
PF-06741086 subcutaneous injection




Primary Outcome Measures :
  1. Number of Participants With Treatment-Emergent Adverse Events (TEAEs), TEAEs by Severity, and Serious Adverse Events (SAEs) (All Causality and Treatment-Related) [ Time Frame: Day 1 up to Day 393 ]
    An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a product; the event did not need to have a causal relationship with the treatment. A serious adverse event (SAE) was any untoward medical occurrence at any dose that resulted in death; was life threatening; required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability/incapacity; resulted in congenital anomaly/birth defect. AEs included both SAEs and non-serious AEs. TEAEs were AEs occurred following the start of treatment or AEs increasing in severity during treatment. Treatment-related TEAEs were determined by the investigator. Grades 3 AEs were severe and undesirable adverse events. Grades 4 AEs were life threatening or disabling adverse events.

  2. Number of Participants With Abnormal Laboratory Findings Without Regard to Baseline Abnormality (Including Hematology, Serum Chemistry, and Urinalysis) [ Time Frame: Hematology and serum chemistry: Baseline, Days 1, 29, 57, 85, 169, 253, and 365 visits. Urinalysis: Baseline, Days 1, 85, 169, 253, and 365 visits. ]
    Following parameters were analyzed for laboratory examination: hematology, clinical chemistry, and urinalysis. The hematology parameters and pre-defined criteria included: neutrophils (10^3/millimeter[mm]^3) <0.8*lower limit of normal (LLN), and basophils (10^3/mm^3) >1.2*upper limit of normal (ULN). The clinical chemistry parameter and pre-defined criteria included: bilirubin (milligrams [mg]/decilitre [dL]) >1.5 ULN, aspartate aminotransferase (units [U]/liter [L]) >3.0 ULN, glucose (mg/dL) >1.5*ULN. The urinalysis parameter and pre-defined criteria included: urine glucose ≥1, ketones (scalar) ≥1, urine protein ≥1, urine hemoglobin (scalar) ≥1, and hyaline casts per low power field (/LPF). Participants met criteria at any time point were included.

  3. Number of Participants With Changes From Baseline in Vital Signs Measurements Meeting the Pre-Defined Categorical Summarization Criteria [ Time Frame: Baseline, Days 1, 29, 57, 85, 113, 141, 169, 197, 225, 253, 281, 309, 337, 365 and 393 visits. ]
    Following parameters were analyzed for vital sign examination: blood pressure (BP), pulse rate (PR), temperature, respiration rate. Categorical vital signs: Temperature >38.5 degree(s) Celsius (℃), Supine PR: <40 or >120 beats per minute (BPM), Systolic BP: <90 mm Hg, >=30 mm Hg change from baseline, Diastolic BP: <50 mm Hg, >=20 mm Hg change from baseline.

  4. Number of Participants With Change From Baseline in Electrocardiogram (ECG) Parameters Meeting the Pre-defined Categorical Summarization Criteria [ Time Frame: Baseline, Days 1 and 29 visits ]
    Baseline was defined as the average of triplicate ECG measurements collected prior to dosing on Day 1 in B7841003. Criteria for potentially clinically important changes in ECG were defined as: PR interval value >=300 millisecond (msec); PR interval baseline >200 msec and change >=25%; PR interval baseline <=200 msec and change >=50%; QRS complex value >=140 msec and change >=50%; QTcF value >=450 msec and change >=30 msec. Only the number of participants meeting pre-defined criteria was reported below.

  5. Number of Participants With Abnormalities in Physical Examination Findings [ Time Frame: Day 1 to Day 393 ]
    Physical examinations were conducted by a physician, trained physician's assistant, or nurse practitioner as acceptable according to local regulation. A full physical examination included head, ears, eyes, nose, mouth, skin, heart and lung examinations, lymph nodes, gastrointestinal, musculoskeletal, and neurological systems. The limited or abbreviated physical examination was focused on general appearance, the respiratory and cardiovascular systems, as well as towards participant reported symptoms. For measuring weight, a scale with appropriate range and resolution was used and must have been placed on a stable, flat surface. Participants removed shoes, bulky layers of clothing, and jackets so that only light clothing remains. They also removed the contents of their pockets and remain still during measurement of weight.

  6. Number of Participants With Injection Site Reactions [ Time Frame: Day 1 to Day 365, and Day 393 visit. ]
    Injection site reactions included but were not limited to: erythema, induration, ecchymosis, pain and pruritus. Grade of severity was defined as follows: Mild: Transient or mild discomfort (< 48 hours); no medical intervention/therapy required. Moderate: Mild to moderate limitation in activity - some assistance may be needed; no or minimal medical intervention/therapy required. Severe: Marked limitation in activity, some assistance usually required; medical intervention/therapy required, hospitalizations possible.


Secondary Outcome Measures :
  1. Annualized Bleeding Rate (ABR) [ Time Frame: Day 1 to Day 365, and Day 393 visit. Pre-Treatment summarized the data up to 6 months prior to participation in B7841003 for de novo participants and up to 6 months prior to participation in B7841002 for roll over participants. ]
    The ABR was calculated as ([number of bleeding events × 365.25] / observed treatment period in days)



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   12 Years to 74 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Severe hemophilia A or B (Factor VIII or Factor IX activity ≤ 1%)
  • Subjects enrolled as Factor VIII or Factor IX inhibitor patients must have a positive inhibitor test result (above the upper limit of normal) at the local laboratory and must receive a bypass agent as primary treatment for bleeding episodes.
  • Episodic (on-demand) treatment regimen prior to screening
  • At least 6 acute bleeding episodes during the 6-month period prior to screening

Exclusion Criteria:

  • Known coronary artery, thrombotic, or ischemic disease
  • Concomitant treatment with activated prothrombin complex concentrate

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03363321


Locations
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United States, Colorado
UC Denver Hemophilia and Thrombosis Center
Aurora, Colorado, United States, 80045
Brazil
Centro de Hematologia e Hemoterapia de Campinas- Hemocentro de Campinas
Campinas, SAO Paulo, Brazil, 13083-878
Chile
Hospital Dr. Sotero del Rio
Santiago, Puente ALTO, Chile, 8207257
Croatia
Klinicki bolnicki centar Zagreb
Zagreb, Croatia, 10000
Poland
Klinika Hematologii i Transplantologii Uniwersyteckie Centrum Kliniczne
Gdansk, Poland, 80-214
South Africa
Phoenix Pharma (Pty) Ltd
Port Elizabeth, Eastern CAPE, South Africa, 6001
Charlotte Maxeke Johannesburg Academic Hospital
Johannesburg, Gauteng, South Africa, 2193
Switzerland
UniversitatsSpital Zurich
Zurich, Switzerland, 8091
Sponsors and Collaborators
Pfizer
Investigators
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Study Director: Pfizer CT.gov Call Center Pfizer
  Study Documents (Full-Text)

Documents provided by Pfizer:
Study Protocol  [PDF] November 8, 2018
Statistical Analysis Plan  [PDF] March 8, 2018

Additional Information:
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Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT03363321    
Other Study ID Numbers: B7841003
2017-001255-31 ( EudraCT Number )
First Posted: December 6, 2017    Key Record Dates
Results First Posted: July 27, 2021
Last Update Posted: July 27, 2021
Last Verified: July 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: 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.
URL: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Pfizer:
hemophilia
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn