Low Dose Emicizumab vs Low Dose Factor VIII in Prophylaxis in Hemophilia A Patients

NCT06938659 | Not Yet Recruiting Phase 2 DMC

• 1 other identifier: ERC-DMC/ECC/2024/215
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

Low dose factor VIII prophylaxis is practiced around the world. Role of standard dose Emicizumab prophylaxis is well established. Emicizumab is an expensive drug. Standard dose Emicizumab prophylaxis is very expensive for hemophilia A patients and troublesome for government to ensure continuous supply. This study intends to compare low dose Factor VIII prophylaxis with low dose Emicizumab prophylaxis and compare whether low dose Emicizumab is as effective as low dose Factor VIII prophylaxis. So, it is possible to continue prophylaxis program in hemophilia A patients with a cost-effective way in our country without risking the patient health. Moreover, Emicizumab prophylaxis reduces the chance of developing inhibitor to Factor VIII and it is convenient for administration due to less frequent and subcutaneous administration. 20 severe hemophilia A patients will be selected randomly from interested patients for this study. 6 patients with inhibitor will be randomized in Low dose Emicizumab with Inhibitor group (I) and rest 14 will be randomized by block randomization in Low dose Emicizumab (without inhibitor-WI) and low dose Factor VIII group at 4:10 ratio. Initial loading dose will be given and the participants will be followed up for 6 months. At the end of the study Annualized bleeding rate (ABR), Annualized joint bleeding rate (AJBR), Annualized spontaneous bleeding rate (ASBR), APTT will be compared among the groups. Inhibitor to factor VIII will also be evaluated after six months in Factor VIII group.

Conditions

Hemophilia A

Keywords

Low dose Factor VIII; Low dose Emicizumab; Prophylaxis; Hemophilia A with inhibitor

Outcome Measures

Primary Outcomes (4)

• Mean of Annualized bleeding rate (ABR) among the study groups will be compared.

  ABR will be calculated as the number of reported bleeding events divided by 06 (the number of months in the reporting time window) and multiplied by 12. e.g. if a participant total bleeding event are 12 in 6 months, ABR of this patients is 12/6\*12=24

  06 months

• Mean of Annualized joint bleeding rate (AJBR) among the study groups will be compared.

  AJBR will be calculated as the number of reported joint bleeding events divided by 06 (the number of months in the reporting time window) and multiplied by 12. e.g. if a participant Joint Bleeding event is 6 in 6 months than AJBR will be 6/6\*12=12.

  06 months

• Mean of Annualized spontaneous bleeding rate (ASBR) among the study groups will be compared.

  ASBR will be calculated as the number of reported spontaneous bleeding events divided by 06 (the number of months in the reporting time window) and multiplied by 12. e.g. if a participant has total 4 spontaneous bleeding event in six months than ASBR will be 4/6\*12= 7.99

  06 months

• Mean of APTT among the groups will be compared.

  APTT

  06 months

Secondary Outcomes (1)

• Inhibitor to Factor VIII-

  06 months

Study Arms (3)

Emicizumab Group- I

EXPERIMENTAL

Severe hemophilia A patients with inhibitor

Drug: Emicizumab Injection

Emicizumab Group- WI

EXPERIMENTAL

Severe hemophilia A Patients without inhibitor (Emicizumab Group):

Drug: Emicizumab Injection

Factor VIII Group

ACTIVE COMPARATOR

Severe hemophilia A Patients without inhibitor.

Drug: Factor VIII (FVIII)

Interventions

Emicizumab Injection DRUG

Emicizumab 0.8-1.5mg/kg weekly for 4 weeks as loading dose. Then 0.8-1.5mg/kg 4-weekly for 24 weeks will be used in Emicizumab group-I and Emicizumab Group- WI

Also known as: Hemlibra

Emicizumab Group- I; Emicizumab Group- WI

Factor VIII (FVIII) DRUG

10-15 unit/Kg thrice weekly (for conventional half-life products) or twice weekly (for extended half-life products) for 28 weeks. Inj. Factor VIII is available in 250U, 500U, 750U \& 1,000 IU vail sizes. Dose will be round up to full nearest full vail strength.

Also known as: Afstyla, Kovaltry

Factor VIII Group

Eligibility Criteria

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

You may qualify if:

• Severe hemophilia A patient with or without inhibitor to factor VIII.

You may not qualify if:

• \. Severe hemophilia A patient not willing to participate.

Sponsors & Collaborators

• Dhaka Medical College: lead

Study Sites (1)

Dhaka Medical College Hospital

Dhaka, 1000, Bangladesh

Location

Related Publications (3)

• Shima M, Hanabusa H, Taki M, Matsushita T, Sato T, Fukutake K, Kasai R, Yoneyama K, Yoshida H, Nogami K. Long-term safety and efficacy of emicizumab in a phase 1/2 study in patients with hemophilia A with or without inhibitors. Blood Adv. 2017 Sep 27;1(22):1891-1899. doi: 10.1182/bloodadvances.2017006684. eCollection 2017 Oct 10.

  PMID: 29296836 RESULT

• Young G, Liesner R, Chang T, Sidonio R, Oldenburg J, Jimenez-Yuste V, Mahlangu J, Kruse-Jarres R, Wang M, Uguen M, Doral MY, Wright LY, Schmitt C, Levy GG, Shima M, Mancuso ME. A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors. Blood. 2019 Dec 12;134(24):2127-2138. doi: 10.1182/blood.2019001869.

  PMID: 31697801 RESULT

• Xu Y, Wang Y, Wu R, Zheng C, Zhang L, Xu W, Feng X, Wang H, Cao X, He L, Xue T, Jin M, Xie B, Ling J, Sun L, Su R, Cheng H, Fang Y, Poon MC, Liu W, Zhang L, Xue F, Yang R. Reduced doses of emicizumab achieve good efficacy: Results from a national-wide multicentre real-world study in China. Haemophilia. 2024 Jul;30(4):959-969. doi: 10.1111/hae.15062. Epub 2024 Jun 9.

  PMID: 38853005 RESULT

MeSH Terms

Conditions

Hemophilia A

Interventions

emicizumab; 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

Study Officials

• Akhil R. Biswas, MBBS, FCPS

  Dhaka Medical College

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Akhil R. Biswas, MBBS,FCPS

CONTACT

+8801712290706; akhil.biswas@yahoo.com

Gazi Y. Islam, MBBS, FCPS

CONTACT

+8801716715995; yeasin100heam@gmail.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor and Head of the Department of Hematology.

Model Details: we will select 20 severe hemophilia A patients randomly from interested patients. we will randomize 6 patients with inhibitor to factor VIII in Low dose Emicizumab with Inhibitor group (I) and rest 14 will be randomized by block randomization in Low dose Emicizumab (without inhibitor-WI) and low dose Factor VIII group. patients with inhibitor (Emicizumab Group-I): Patients without inhibitor (Emicizumab Group-WI): Patients without inhibitor (Factor VIII Group).

Study Record Dates

• First Submitted: March 12, 2025
• First Posted: April 22, 2025
• Study Start: June 1, 2025
• Primary Completion (Estimated): May 31, 2026
• Study Completion (Estimated): July 31, 2026
• Last Updated: April 22, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE
• Time Frame: 01.06.2025 to 30.05.2030
• Access Criteria: We can share information to doctors by e-mail.

Locations

BA (1)