Moroctocog Alfa (AF-CC) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Hemophilia A Patients

NCT04396639 | Completed Phase 4 Results

• 2 other identifiers: B18310972020-004570-21
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 5

Brief Summary

Moroctocog-alfa (AF-CC) is indicated for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in patients with hemophilia A (congenital factor VIII deficiency or classic hemophilia). The current single country, multi-centric, open label, non-randomized pragmatic 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 moroctocog-alfa (AF-CC) in Indian subjects with hemophilia A. The primary objective of study is to study the safety of moroctocog alfa (AF-CC) when administered for prophylaxis with respect to incidence of FVIII inhibitor development. The secondary objectives are to evaluate the incidence of adverse events (AEs) and serious adverse events (SAEs) in subjects receiving moroctocog alfa (AF-CC) prophylaxis, to evaluate the efficacy of moroctocog alfa (AF-CC) during a prophylaxis regimen, to evaluate the total annualized consumption of moroctocog alfa (AF-CC) by subjects following a prophylaxis regimen, to evaluate the efficacy of moroctocog alfa (AF-CC) for the treatment of breakthrough bleeding episodes (on-demand treatment) while following a prophylaxis regimen. Fifty male subjects aged \>/= 12 years to ≤65 years with moderate or severe hemophilia A will be enrolled in the study. The subjects will be selected based on protocol specified eligibility criteria. The overall treatment duration for each subject will be up to 8 weeks, with up to a 4-week screening period and a subsequent post-treatment 28-day safety observation period. Subjects are requested to continue in the study until 24 exposure days (EDs) or a period of up to 8 weeks on moroctocog alfa (AF-CC) treatment had occurred (whichever occurs first). Efficacy and safety assessments will be performed as specified in the protocol.

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants Who Developed Factor VIII (FVIII) Inhibitors

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

  24 exposure days or 8 weeks of treatment during the study (whichever occurred first)

Secondary Outcomes (6)

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

  Day 1 up to 28 days after last dose of study drug (approximately maximum up to 12 Weeks)

• Number of Participants With Treatment Emergent Serious Adverse Events (SAEs)

  Day 1 up to 28 days after last dose of study drug (approximately maximum up to 12 Weeks)

• Mean Annualized Bleeding Rate (ABR) During Prophylaxis

  24 exposure days or 8 weeks of treatment during the study (whichever occurred first)

• Mean Annualized Total Factor Consumption (TFC)

  24 exposure days or 8 weeks of treatment during the study (whichever occurred first)

• Mean Annualized Total Factor Consumption (TFC) by Weight

  24 exposure days or 8 weeks of treatment during the study (whichever occurred first)

Study Arms (1)

Treatment Arm

EXPERIMENTAL

Fifty eligible male subjects will be enrolled in the treatment arm to receive Moroctocog alfa (AF-CC) until 24 exposure days (EDs) or a period of up to 8 weeks on treatment had occurred (whichever occurs first).

Biological: Moroctocog-alfa (AF-CC)

Interventions

Moroctocog-alfa (AF-CC) BIOLOGICAL

Moroctocog-alfa (AF-CC) is indicated for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in patients with hemophilia A (congenital factor VIII deficiency or classic hemophilia).

Also known as: Xyntha

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 moderate or severe hemophilia A (FVIII:C ≤5%).
• Documented history of at least 50 exposure days (EDs) to FVIII-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 FVIII or positive inhibitor testing (≥0.6 BU/mL) during Screening. Clinical signs or symptoms of decreased response to FVIII.
• 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 A.
• 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 A, 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

Surat, Gujarat, 395002, India

Location

K.J.Somaiya Hospital and Research Centre

Mumbai, Maharashtra, 400022, India

Location

Sahyadri Clinical Research and Development Centre

Pune, Maharashtra, 411004, India

Location

Sahyadri Super Specialty Hospital

Pune, Maharashtra, 411004, India

Location

Christian Medical College and Hospital

Ludhiana, Punjab, 141008, India

Location

Related Links

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

MeSH Terms

Conditions

Hemophilia A

Interventions

Factor VIII; F8 protein, human

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

Intervention Hierarchy (Ancestors)

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: November 26, 2019
• First Posted: May 20, 2020
• Study Start: January 25, 2020
• Primary Completion: September 24, 2020
• Study Completion: September 24, 2020
• Last Updated: May 3, 2021
• Results First Posted: May 3, 2021

Record last verified: 2021-04

Data Sharing

• IPD Sharing: Will share

Locations

IN (5)