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CTRI Number  CTRI/2023/09/057791 [Registered on: 18/09/2023] Trial Registered Prospectively
Last Modified On: 15/09/2023
Post Graduate Thesis  Yes 
Type of Trial  Interventional 
Type of Study   Drug 
Study Design  Randomized, Parallel Group Trial 
Public Title of Study   To find whether Romiplostim is useful in Aplastic anemia treatment 
Scientific Title of Study   Comparative study of treatment outcomes of patients of aplastic anemia with combination therapy cyclosporine A + danazol + eltrombopag vs cyclosporine A + danazol + high dose Romiplostim 
Trial Acronym   
Secondary IDs if Any  
Secondary ID  Identifier 
NIL  NIL 
 
Details of Principal Investigator or overall Trial Coordinator (multi-center study)  
Name  Mritunjay Kumar 
Designation  Postgraduate Trainee 
Affiliation  IMS, BHU 
Address  Department of Medicine Institute of Medical Sciences, Banaras Hindu University, Varanasi

Varanasi
UTTAR PRADESH
221005
India 
Phone  9625016364  
Fax    
Email  mritunjay9625@gmail.com  
 
Details of Contact Person
Scientific Query  
Name  Shruti Mishra 
Designation  Assistant Professor 
Affiliation  IMS, BHU 
Address  Bone Marrow Transplantation and Stem Cell Research Centre [BMT-SRC], Institute of Medical Sciences, Banaras Hindu University, Varanasi

Varanasi
UTTAR PRADESH
221005
India 
Phone  8017377253  
Fax    
Email  drshruti.mishra@bhu.ac.in  
 
Details of Contact Person
Public Query  
Name  Mritunjay Kumar 
Designation  Postgraduate Trainee 
Affiliation  IMS, BHU 
Address  Department of Medicine Institute of Medical Sciences, Banaras Hindu University, Varanasi

Varanasi
UTTAR PRADESH
221005
India 
Phone  9625016364  
Fax    
Email  mritunjay9625@gmail.com  
 
Source of Monetary or Material Support  
MLA/MP Fund/ CM Fund The study will be conducted in Dept. of Medicine, SS Hospital, IMS, BHU 
 
Primary Sponsor  
Name  Government of Uttar Pradesh 
Address  Chief Minister office, Lucknow, Uttar Pradesh 
Type of Sponsor  Government funding agency 
 
Details of Secondary Sponsor  
Name  Address 
NIL  NIL 
 
Countries of Recruitment     India  
Sites of Study  
No of Sites = 1  
Name of Principal Investigator  Name of Site  Site Address  Phone/Fax/Email 
Dr Shruti Mishra  Sir Sunderlal Hospital, IMS, BHU  5th floor, Div of Hematology, Dept of Medicine, SS Hospital, Institute of Medical Sciences, BHU
Varanasi
UTTAR PRADESH 		 8017377253

drshruti.mishra@bhu.ac.in 
 
Details of Ethics Committee  
No of Ethics Committees= 1  
Name of Committee  Approval Status 
Institutional Ethics Committee, ECR/526/Inst/UP/2014/RR-20dt.19.5.2020, e  Approved 
 
Regulatory Clearance Status from DCGI  
Status 
Not Applicable 
 
Health Condition / Problems Studied  
Health Type  Condition 
Patients  (1) ICD-10 Condition: D619||Aplastic anemia, unspecified,  
 
Intervention / Comparator Agent  
Type  Name  Details 
Comparator Agent  Eltrombopag  Tab Cyclosporin A 100mg BD Tab Mennabol 2mg TDS Tab Eltrombopag 150mg daily For 6months 
Intervention  Romiplostim  Tab Cyclosporin A 100mg BD Tab Mennabol 2mg TDS Inj Rompilostim 600mcg s/c weekly For 6months 
 
Inclusion Criteria  
Age From  15.00 Year(s)
Age To  80.00 Year(s)
Gender  Both 
Details  1. Confirmed cases of Aplastic anemia
2. Patient age >15years
3. Not willing for ASCT and ATG.
4. Non- pregnant women.
5. Those who consent to take part in the study.
6. No hepatic or renal disease.
7. No drug h/o that causes Myelo-suppression. 
 
ExclusionCriteria 
Details  1. Previous history of HSCT,
2. Patients eligible for HSCT
3. Patients with HIV/ HCV/ HBV infections.
4. Significant PNH clone and Myelodysplasia. 
 
Method of Generating Random Sequence   Random Number Table 
Method of Concealment   Case Record Numbers 
Blinding/Masking   Open Label 
Primary Outcome  
Outcome  TimePoints 
1. Complete response (CR) defined as—transfusion independent,
2. Non-responders will be transfusion-dependent
 		 3months and 6 months 
 
Secondary Outcome  
Outcome  TimePoints 
1. Hemoglobin ≥11 g/dL, Absolute neutrophil count (ANC) more than 1.5 x 10 9 /L and platelet ≥150 x 10 9 /L;
2. Partial response (PR) will be those who are transfusion independent, hemoglobin ≥8 g/dL, ANC more than 0.5 x 10 9 /L and platelet ≥20 x 10 9 /L
 		 3months and 6months 
 
Target Sample Size   Total Sample Size="20"
Sample Size from India="20" 
Final Enrollment numbers achieved (Total)= "Applicable only for Completed/Terminated trials"
Final Enrollment numbers achieved (India)="Applicable only for Completed/Terminated trials" 
Phase of Trial   Phase 3/ Phase 4 
Date of First Enrollment (India)   25/09/2023 
Date of Study Completion (India) Applicable only for Completed/Terminated trials 
Date of First Enrollment (Global)  Date Missing 
Date of Study Completion (Global) Applicable only for Completed/Terminated trials 
Estimated Duration of Trial   Years="2"
Months="0"
Days="0" 
Recruitment Status of Trial (Global)   Not Applicable 
Recruitment Status of Trial (India)  Not Yet Recruiting 
Publication Details   None yet 
Individual Participant Data (IPD) Sharing Statement

Will individual participant data (IPD) be shared publicly (including data dictionaries)?  

Response - NO
Brief Summary  

Background

 

Aplastic anemia (AA) was first described in a pregnant woman in 1888. The term now refers to a clinical syndrome defined as pancytopenia with hypocellular bone marrow without abnormal infiltration or increased reticulin. AA can be inherited or acquired. Acquired AA is usually the result of an autoimmune attack that appears to be directed at hematopoietic stem/progenitor cells.

Immunosuppressive therapies (IST) are effective, but reduced numbers of residual stem cells may limit their efficacy. In patients with AA, that was refractory to standard immunosuppressive therapy. Eltrombopag, a synthetic thrombopoietin-receptor agonist, has been tried alone or added to standard immunosuppressive therapy as routine care and has recently been approved by the FDA for treating aplastic anemia. High-dose Romiplostim is effective and well tolerated in treating patients with AA refractory to IST. Although ATG plus Cyclosporine A is the therapy for AA patients without a donor when neither HSCT nor IST is available, Danazol remains an acceptable therapeutic option. The tendency to achieve remission was similar among severity groups in a previous comparative study. We will compare the efficacy of standard immunosuppressive therapy combined with Eltrombopag and Danazol v/s high dose Romiplostim and Danazol in newly diagnosed cases of Aplastic anemia.

 

Purpose of the study:

 

ATG has been used to treat patients ineligible for hematopoietic stem cell transplantation. However, the number of hematopoietic stem cells is a limiting factor for its response. Eltrombopag and Romiplostim are Thrombopoietic Receptor Agonists (TPO-RAs). Their role in managing Immune Thrombocytopenia (ITP) has been well established. It has been seen that TPO-RAs are also helpful in the management of Aplastic Anemia. However, there needs to be more literature on using these drugs, especially Romiplostim, in the first-line therapy of Aplastic anemia cases. Our study aims to compare the clinical outcomes of adding Eltrombopag and Danazol v/s High-dose Romiplostim and Danazol to standard immunosuppressive therapy. 


References:

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17.   Jang JH, Tomiyama Y, Miyazaki K, Nagafuji K, Usuki K, et al. Efficacy and safety of romiplostim in refractory aplastic anaemia: a Phase II/III, multicenter, open-label study. Br J Haematol. 2021 Jan; 192(1):190-199.

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