Nonacog Alfa Prophylaxis And Treatment Of Bleeding Episodes In Previously Treated Patients With Hemophilia B

NCT04286412 | Completed Phase 4 Results

• 2 other identifiers: B18210592020-004430-38
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 5

Brief Summary

Nonacog alfa is indicated for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in patients with hemophilia B. The current single country, multi-centric, open label, non-randomized clinical trial is a post-approval study to fulfill the Central Drugs Standard Control Organization (CDSCO) request for supplementary information relating to the use of nonacog alfa in Indian subjects with hemophilia B.

Conditions

Hemophilia B

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants Who Developed Factor IX (FIX) Inhibitors

  FIX inhibitor development was defined as an inhibitor titer \>= 0.6 Bethesda units per milliliter (BU/mL) confirmed by central laboratory testing during the course of the study.

  At Visit 4 (any 1 day between Day 52 to Day 60)

Secondary Outcomes (6)

• Number of Participants With Serious Adverse Events (SAEs) and Medically Important Events (MIEs)

  Baseline up to 28 days after last dose of study drug (i.e, up to 116 days)

• Number of Participants With Treatment Emergent Adverse Events (TEAEs)

  Baseline up to 28 days after last dose of study drug (i.e, up to 116 days)

• Mean Annualized Bleeding Rate (ABR)

  Up to 88 Days

• Mean Annualized Total Factor Consumption (TFC) Per Participant

  Up to 88 Days

• Mean Annualized Total Factor Consumption (TFC) by Weight Per Participant

  Up to 88 days

Study Arms (1)

Treatment Arm

EXPERIMENTAL

At least twenty five eligible male subjects will be enrolled in the treatment arm to receive Nonacog alfa until 16 exposure days (EDs) or a period of up to 8 weeks on treatment had occurred (whichever occurs first).

Biological: Nonacog alfa

Interventions

Nonacog alfa BIOLOGICAL

Nonacog alfa is indicated in India for the treatment and prophylaxis of bleeding in patients with hemophilia B (congenital factor IX deficiency).

Also known as: BeneFIX

Treatment Arm

Eligibility Criteria

• Age: 12 Years - 65 Years
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Male subjects ≥12 years to ≤65 years with a diagnosis of congenital moderately-severe to severe hemophilia B (FIX activity ≤2%).
• Documented history of at least 50 exposure days (EDs) to FIX-containing products.
• Evidence of a personally signed and dated informed consent document indicating that the subject (or a legally acceptable representative, parent(s)/legal guardian) has been informed of all pertinent aspects of the study. For minors under the age of legal consent in India, assent of the participating child needs to be documented for the age range 12 to 18 in addition to the parental informed consent.

You may not qualify if:

• Prior history of inhibitor to FIX or positive inhibitor testing (≥0.6 BU/mL) during Screening. Clinical signs or symptoms of decreased response to FIX.
• Known hypersensitivity to the active substance or any of the excipients.
• Known allergic reaction to hamster proteins.
• Presence of any bleeding disorder in addition to hemophilia B.
• Participation in other studies involving investigational drug(s) (Phases 1-4) within 30 days before the current study begins and/or during study participation.
• Planned surgery within 6 months from the start of the study.
• Unsuitable to participate in study for any other reason as assessed by the investigator; including any disorder, except for conditions associated with hemophilia B, which in the investigator's opinion might jeopardize subject's safety or compliance with the protocol.
• Subjects (or a legally acceptable representative) is not able to understand study documents and study procedure.
• Immunocompromised subjects due to human immunodeficiency virus (HIV) infection (defined as viral load above or equal to 100,000 copies/mL; and for HIV+ subjects: cluster of differentiation 4 positive (CD4+) lymphocyte count below or equal to 200/μL). HIV status and CD4+ lymphocyte count results may be obtained at screening or from available medical records; results must be not older than 6 months prior to screening.
• Subjects who are investigational site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the Investigator, subjects who have been previously enrolled into the study, or subjects who are Pfizer employees directly involved in the conduct of the study.
• Planned use of any non-study medication for treatment of hemophilia (eg, other factor replacement agents, bypassing agents, or non-factor treatments \[such as anti-tissue factor pathway inhibitors\]).

Sponsors & Collaborators

• Pfizer: lead

Study Sites (5)

Nirmal Hospital Pvt Ltd.

Surat, Gujarat, 395002, India

Location

K.J. Somaiya Hospital and Research Centre

Mumbai, Maharashtra, 400022, India

Location

Sahyadri Clinical Research & Development Centre

Pune, Maharashtra, 411004, India

Location

Christian Medical College and Hospital

Ludhiana, Punjab, 141008, India

Location

Sahyadri Super Specialty Hospital

Pune, 411004, India

Location

Related Links

• To obtain contact information for a study center near you, click here.

MeSH Terms

Conditions

Hemophilia B

Interventions

Factor IX

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Intervention Hierarchy (Ancestors)

Enzyme Precursors; Enzymes and Coenzymes; Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 12, 2020
• First Posted: February 27, 2020
• Study Start: February 10, 2020
• Primary Completion: September 24, 2020
• Study Completion: September 24, 2020
• Last Updated: July 1, 2022
• Results First Posted: July 30, 2021

Record last verified: 2022-06

Data Sharing

• IPD Sharing: Will share

Locations

IN (5)