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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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03363321
Recruitment Status : Recruiting
First Posted : December 6, 2017
Last Update Posted : August 29, 2019
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)
Estimated Enrollment : 36 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
Estimated Primary Completion Date : August 11, 2020
Estimated Study Completion Date : August 11, 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. Frequency, severity and causal relationship of treatment emergent adverse events (TEAEs) and withdrawals due to TEAEs [ Time Frame: Day 1 up to Day 393 ]
    Treatment-related AE was any untoward medical occurrence attributed to study drug in a participant who received study drug. 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; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.

  2. Percentage of subjects with laboratory abnormalities [ Time Frame: Day 1 to Day 393 ]
    Following parameters were analyzed for laboratory examination: hematology, serum chemistry, and urinalysis.

  3. Number of subjects with changes from baseline in vital signs [ Time Frame: Day 1 to Day 393 ]
    Following parameters were analyzed for vital sign examination: blood pressure, pulse rate, temperature, respiration rate.

  4. Number of subjects with change from baseline in electrocardiogram (ECG) parameters [ Time Frame: Day 1 to Day 29 ]
    Following parameters were analyzed for ECG examinations: QT interval (QTc), QTcF interval (Fridericia's Correction), QRS complex, PR interval, and RR interval.

  5. Percentage of subjects with injection site reactions [ Time Frame: Day 1 to Day 393 ]
    Following parameters were analyzed for injection site reaction examinations: induration, erythema, edema, rash, pruritus.

  6. Percentage of subejct with infusion site reactions [ Time Frame: Day 1 to Day 393 ]
    Following parameters were analyzed for infusion site reactions: induration, erythema, edema, rash, pruritus.


Secondary Outcome Measures :
  1. Number of bleeding episodes [ Time Frame: Day 1 to Day 393 ]
    Bleeding episodes requiring treatment with factor replacement.

  2. Number of treatments for breakthrough bleeding episodes [ Time Frame: Day 1 to Day 393 ]
    Treatments with factor replacement.



Information from the National Library of Medicine

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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


Contacts
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Contact: Pfizer CT.gov Call Center 1-800-718-1021 ClinicalTrials.gov_Inquiries@pfizer.com

Locations
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United States, Colorado
UC Denver Hemophilia and Thrombosis Center Recruiting
Aurora, Colorado, United States, 80045
University of Colorado Denver Hemophilia and Thrombosis Center Recruiting
Aurora, Colorado, United States, 80045
Brazil
Centro de Hematologia e Hemoterapia de Campinas- Hemocentro de Campinas Recruiting
Campinas, SAO Paulo, Brazil, 13083-878
Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti- HEMORIO Not yet recruiting
Rio de Janeiro, Brazil, 20211-030
Chile
Hospital Dr. Sotero del Rio Recruiting
Santiago, Puente ALTO, Chile, 8207257
Croatia
Klinicki bolnicki centar Zagreb Recruiting
Zagreb, Croatia, 10000
France
Hôpital Louis Pradel Not yet recruiting
Bron cedex, France, 69677
Centre de Reference pour le Traitement des maladies Hemorragiques (CRTH), Hopital Bicetre AP-HP Not yet recruiting
Le Kremlin Bicetre, France, 94270
Poland
Apteka Szpitalna UCK Recruiting
Gdansk, Poland, 80-214
Klinika Hematologii i Transplantologii Recruiting
Gdansk, Poland, 80-952
South Africa
Phoenix Pharma (Pty) Ltd Recruiting
Port Elizabeth, Eastern CAPE, South Africa, 6001
Charlotte Maxeke Johannesburg Academic Hospital Recruiting
Johannesburg, Gauteng, South Africa, 2193
Spain
Hospital Universitario La Paz: Servicio de Farmacia Not yet recruiting
Madrid, Spain, 28046
Hospital Universitario La Paz Not yet recruiting
Madrid, Spain, 28046
Switzerland
UniversitatsSpital Zurich Recruiting
Zurich, Switzerland, 8091
Sponsors and Collaborators
Pfizer
Investigators
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Study Director: Pfizer CT.gov Call Center Pfizer

Additional Information:
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Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT03363321     History of Changes
Other Study ID Numbers: B7841003
2017-001255-31 ( EudraCT Number )
First Posted: December 6, 2017    Key Record Dates
Last Update Posted: August 29, 2019
Last Verified: August 2019
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