Clinical trial NCT04286412: Nonacog Alfa Prophylaxis And Treatment Of Bleeding Episodes In Previously Treated Patients With Hemophilia B
Official title: A SINGLE COUNTRY, MULTICENTER, OPEN-LABEL AND NON-RANDOMIZED CLINICAL TRIAL WITH NONACOG ALFA PROPHYLAXIS AND TREATMENT OF BLEEDING EPISODES IN PREVIOUSLY TREATED PATIENTS WITH MODERATELY-SEVERE TO SEVERE HEMOPHILIA B FOR A DURATION OF 8 WEEKS.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Hemophilia B
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2020-02-27
Enrollment: 25
Sex: MALE
Minimum age (years): 12
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

1. Male subjects ≥12 years to ≤65 years with a diagnosis of congenital moderately-severe to severe hemophilia B (FIX activity ≤2%).
2. Documented history of at least 50 exposure days (EDs) to FIX-containing products.
3. 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.

Exclusion Criteria:

1. 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.
2. Known hypersensitivity to the active substance or any of the excipients.
3. Known allergic reaction to hamster proteins.
4. Presence of any bleeding disorder in addition to hemophilia B.
5. Participation in other studies involving investigational drug(s) (Phases 1-4) within 30 days before the current study begins and/or during study participation.
6. Planned surgery within 6 months from the start of the study.
7. 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.
8. Subjects (or a legally acceptable representative) is not able to understand study documents and study procedure.
9. 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.
10. 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.
11. Planned use of any non-study medication for treatment of hemophilia (eg, other factor replacement agents, bypassing agents, or non-factor treatments \[such as anti-tissue factor pathway inhibitors\]).

Primary outcomes:
measure: Percentage of Participants Who Developed Factor IX (FIX) Inhibitors
timeFrame: At Visit 4 (any 1 day between Day 52 to Day 60)
Secondary outcomes:
measure: Number of Participants With Serious Adverse Events (SAEs) and Medically Important Events (MIEs)
timeFrame: Baseline up to 28 days after last dose of study drug (i.e, up to 116 days)
measure: Number of Participants With Treatment Emergent Adverse Events (TEAEs)
timeFrame: Baseline up to 28 days after last dose of study drug (i.e, up to 116 days)
measure: Mean Annualized Bleeding Rate (ABR)
timeFrame: Up to 88 Days
measure: Mean Annualized Total Factor Consumption (TFC) Per Participant
timeFrame: Up to 88 Days
measure: Mean Annualized Total Factor Consumption (TFC) by Weight Per Participant
timeFrame: Up to 88 days
measure: Mean Number of Nonacog Alfa Infusions Used to Treat Each Bleed
timeFrame: Up to 88 days
Other outcomes:
Locations:
city: Surat
country: India
facility: Nirmal Hospital Pvt Ltd.
state: Gujarat
city: Mumbai
country: India
facility: K.J. Somaiya Hospital and Research Centre
state: Maharashtra
city: Pune
country: India
facility: Sahyadri Clinical Research & Development Centre
state: Maharashtra
city: Ludhiana
country: India
facility: Christian Medical College and Hospital
state: Punjab
city: Pune
country: India
facility: Sahyadri Super Specialty Hospital
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: