A pilot Randomised Control Trial comparing the efficacy and safety of Standard Dose and Low dose Emicizumab prophylaxis with Extended Half Life Factor VIII concentrate in children with severe Hemophilia A without Inhibitors
Overview
- Phase
- Phase 2
- Status
- Not yet recruiting
- Enrollment
- 30
- Locations
- 1
- Primary Endpoint
- To assess and compare Annualized Bleeding Rate ( spontaneous, Traumatic, Joint bleeds) between various treatment groups
Overview
Brief Summary
This is a pilot randomized controlled open label trial comparing the efficacy and safety of prophylaxis with extended half life recombinant factor VIII and two dosing regimens of emicizumab in patients with hemophilia A without inhibitors. A total of 30 participants will be enrolled, with 10 participants in each group. Randomization will be done using random number tables, and allocation concealment will be maintained using sequentially numbered sealed envelopes. Each participant will receive the assigned prophylactic regimen for six months. The primary outcome will be the annualized bleeding rate. Secondary outcomes will include Hemophilia Joint Health Score, Haemo QoL score assessed at end of 6 months and adverse events recorded at any time during the study period.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Masking
- None
Eligibility Criteria
- Ages
- 2.00 Year(s) to 18.00 Year(s) (—)
- Sex
- Male
Inclusion Criteria
- •Severe Hemophilia A without Inhibitors and has not been on prophylactic therapy with either Extended Half Life Factor VIII or Emicizumab.
Exclusion Criteria
- •Presence of FVIII inhibitors Known case of coagulopathies and bleeding disorders other than Hemophilia A.
Outcomes
Primary Outcomes
To assess and compare Annualized Bleeding Rate ( spontaneous, Traumatic, Joint bleeds) between various treatment groups
Time Frame: At baseline and end of 6 months of prophylaxis
Secondary Outcomes
- Hemophilia Joint Health Score (HJHS)(At baseline and and of 6 months)
- Haemo-QoL score(At baseline and end of 6 months)
- Adverse events monitoring(Any time during the study)
Investigators
Aswathy V Nair
Shyam Shah Medical College and Associated SGMH, GMH