Low Dose Emicizumab vs Low Dose Factor VIII in Prophylaxis in Hemophilia A Patients
NCT ID: NCT06938659
Last Updated: 2025-04-22
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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NOT_YET_RECRUITING
PHASE2/PHASE3
20 participants
INTERVENTIONAL
2025-06-01
2026-07-31
Brief Summary
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Detailed Description
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Sample Size: Sample size is determined by availability of drugs rather than any scientific formula. The investigators can provide Emicizumab to 10 patients for 28 weeks as prophylaxis. So, the investigators intend to include 10 patients in each group.
Sampling technique: The investigators will select 20 severe hemophilia A patients randomly from interested patients. The investigators will randomize 6 patients with inhibitor 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 Variables to be studied- A. Independent variable
1. Socio-demographic variables- Age, Educational status, Weight, Occupation
2. Variables related to Hemophilia- Presence of Inhibitor B. Dependent variable
1\. Main outcome variables- Annualized bleeding rate (ABR), Annualized joint bleeding rate (AJBR), Annualized spontaneous bleeding rate (ASBR), APTT 2. Confounding variables- Development of new inhibitor to factor VIII Patients and/or parents will be thoroughly informed about the study, drugs to be used, risk \& benefits and follow up plan. Their consent for this study will be taken and they will be enrolled in the study. For Participants under 18 years, consent will be obtained from parents or legal guardian. Participants ≥18 years will give his/her own consent. Randomization will be generated by computer.
Dose- Emicizumab 0.8-1.5mg/kg weekly for 4 weeks as loading dose. Then 0.8-1.5mg/kg 4-weekly for 24 weeks. Inj. Emicizumab has 30mg in vail. Dose will be rounded to 30mg, 60 mg or any dose nearest to 1 mg/kg when fractionation of vail is possible.
Factor VIII- 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.
Follow Up- Participants in Emicizumab group will visit weekly for first 4 week than once in every 4 week for 24 weeks (Total 28 weeks).
Participants in Factor VIII group will visit twice weekly for 28 weeks. Information of data collection sheet will be collected once in a week.
Patient must attend physically for every dose administration. He will be asked for number of bleeding- traumatic, spontaneous bleeding, joint bleeding, frequency of hospital visit due to hemophilia related problems in between doses and investigate for APTT.
Primary End Point- This study will include 28 weeks follow up of the patients. Each patient will receive 28 weeks prophylaxis. After that period patients will continue prophylaxis if drugs are available but will not be considered for this study. Otherwise, they will receive on demand treatment in our HTC (hemophilia treatment center).
Supportive care and rescue- A Data and Safety Monitoring Board (DSMB) will be formed which will include members suggested by IRB. Any adverse event or serious adverse event (will be delt with proper medical care.
If any patient developed spontaneous or traumatic bleeding episode during prophylaxis, they will be treated with standard dose on demand treatment on priority basis.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
patients with inhibitor (Emicizumab Group-I):
Patients without inhibitor (Emicizumab Group-WI):
Patients without inhibitor (Factor VIII Group).
TREATMENT
NONE
Study Groups
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Emicizumab Group- I
Severe hemophilia A patients with inhibitor
Emicizumab Injection
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
Emicizumab Group- WI
Severe hemophilia A Patients without inhibitor (Emicizumab Group):
Emicizumab Injection
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
Factor VIII Group
Severe hemophilia A Patients without inhibitor.
Factor VIII (FVIII)
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.
Interventions
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Emicizumab Injection
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
Factor VIII (FVIII)
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.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
Exclusion Criteria
ALL
No
Sponsors
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Dhaka Medical College
OTHER
Responsible Party
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Akhil Ranjon Biswas
Professor and Head of the Department of Hematology.
Principal Investigators
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Akhil R. Biswas, MBBS, FCPS
Role: PRINCIPAL_INVESTIGATOR
Dhaka Medical College
Locations
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Dhaka Medical College Hospital
Dhaka, , Bangladesh
Countries
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Central Contacts
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References
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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.
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.
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.
Provided Documents
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Document Type: Study Protocol, Statistical Analysis Plan, and Informed Consent Form
Other Identifiers
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ERC-DMC/ECC/2024/215
Identifier Type: -
Identifier Source: org_study_id
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