Romiplostim in Inherited marrow failure syndromes - Safety and Efficacy
Phase 2
- Conditions
- Health Condition 1: D610- Constitutional aplastic anemia
- Registration Number
- CTRI/2024/03/063966
- Lead Sponsor
- INDIAN COUNCIL OF MEDICAL RESEARCH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Open to Recruitment
- Sex
- Not specified
- Target Recruitment
- 0
Inclusion Criteria
Patients with IBMFS including Fanconi anemia, dyskeratosis congenita causing cytopenias Hb less than 8 gram per decilitre, ANC less than 500 cells or Platelet count less than 30,000 per microlitre or bone marrow cellularity less than 25 percent and with danazol resistance or intolerance
Exclusion Criteria
Known hypersensitivity or past use of Romiplostim, history of thrombosis, patients with IBMF with co-existing myelodysplasia or malignancies and organ dysfunction; presence of FANCD mutation
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method A. Twenty five percent reduction in transfusion requirement compared to baseline <br/ ><br>B. Achieving platelet count more than 30,000 per microlitre, Hb more than 8gram per decilitre or ANC more than 500 per microlitre compared to baseline <br/ ><br> C. Transfusion independence for 4 consecutive weeks <br/ ><br> D. Bone marrow response, defined as an increase in bone marrow cellularity by at least two-fold from baseline. <br/ ><br>Timepoint: 12 and 24 weeks
- Secondary Outcome Measures
Name Time Method The following known drug related adverse reactions will be monitored <br/ ><br>1. Occurrence of clonal evolution <br/ ><br> 2. Thrombotic events <br/ ><br>3. Allergy/ hypersensitivity <br/ ><br>4. Pain at injection site <br/ ><br>5. Headache <br/ ><br> 6. Myalgia <br/ ><br>7. Fatigue <br/ ><br>8. Dizziness <br/ ><br>9. Transaminitis <br/ ><br>10. Any other symptom reported by the patient up to 4 weeks after study completion. <br/ ><br> <br/ ><br> Along with disease related adverse events like <br/ ><br> 1. Infection <br/ ><br> 2. Anemia <br/ ><br>3. Bleeding <br/ ><br>Timepoint: weekly basis from week 1 to week 36