| CTRI Number |
CTRI/2025/08/093855 [Registered on: 28/08/2025] Trial Registered Prospectively |
| Last Modified On: |
27/08/2025 |
| Post Graduate Thesis |
No |
| Type of Trial |
Interventional |
|
Type of Study
|
Drug |
| Study Design |
Non-randomized, Active Controlled Trial |
|
Public Title of Study
|
To study the effect of drug Desidustat in patient with blood disorders |
|
Scientific Title of Study
|
To study the safety and efficacy of oral Desidustat for treatment of anemia in patients with Lower risk Myelodysplastic syndrome [MDS] and Myeloproliferative neoplasms [MPN] |
| Trial Acronym |
Not applicable |
|
Secondary IDs if Any
|
| Secondary ID |
Identifier |
| NOT APPLICABLE |
NIL |
|
|
Details of Principal Investigator or overall Trial Coordinator (multi-center study)
|
| Name |
Archita R |
| Designation |
Assistant Professor |
| Affiliation |
Christian Medical College Vellore Ranipet campus |
| Address |
Department of Haematology,
Room no 25, A Block Fifth floor
Vellore
TAMIL NADU
632517
India |
| Phone |
04172224553 |
| Fax |
|
| Email |
archita.r@cmcvellore.ac.in |
|
Details of Contact Person
Scientific Query
|
| Name |
Archita R |
| Designation |
Assistant Professor |
| Affiliation |
Christian Medical College Vellore Ranipet campus |
| Address |
Department of Haematology,
Room no 25, A Block Fifth floor
TAMIL NADU
632517
India |
| Phone |
04172224553 |
| Fax |
|
| Email |
archita.r@cmcvellore.ac.in |
|
Details of Contact Person
Public Query
|
| Name |
Archita R |
| Designation |
Assistant Professor |
| Affiliation |
Christian Medical College Vellore Ranipet campus |
| Address |
Department of Haematology,
Room no 25, A Block Fifth floor
TAMIL NADU
632517
India |
| Phone |
04172224553 |
| Fax |
|
| Email |
archita.r@cmcvellore.ac.in |
|
|
Source of Monetary or Material Support
|
| Fluid Research Grant,
Christian Medical College Vellore, Office of Research, Vellore 632002, Tamil Nadu |
|
|
Primary Sponsor
|
| Name |
Fluid Research Grant, Christian Medical College Vellore, |
| Address |
Office of Research, Vellore 632002, Tamil Nadu |
| Type of Sponsor |
Other [Charitable trust hospital] |
|
|
Details of Secondary Sponsor
|
| Name |
Address |
| Not applicable |
Not applicable |
|
|
Countries of Recruitment
|
India |
|
Sites of Study
|
| No of Sites = 1 |
| Name of Principal
Investigator |
Name of Site |
Site Address |
Phone/Fax/Email |
| Archita R |
Christian Medical College Vellore Ranipet campus |
Department of Haematology, Ranipet, Room no: 25, A Block, 5th Floor
Vellore
TAMIL NADU |
04172224553
archita.r@cmcvellore.ac.in |
|
|
Details of Ethics Committee
|
| No of Ethics Committees= 1 |
| Name of Committee |
Approval Status |
| Institutional Review Board, CMC Vellore |
Approved |
|
|
Regulatory Clearance Status from DCGI
|
|
|
Health Condition / Problems Studied
|
| Health Type |
Condition |
| Patients |
(1) ICD-10 Condition: D758||Other specified diseases of bloodand blood-forming organs, |
|
|
Intervention / Comparator Agent
|
| Type |
Name |
Details |
| Comparator Agent |
Not applicable |
Not applicable |
| Intervention |
Tab Desidustat |
Tab Desidustat 100mg x 3 times a week x 6 months
After 2 months if no response increase Tab Desidustat to 150mg x 3 times a week x 4 months |
|
|
Inclusion Criteria
|
| Age From |
18.00 Year(s) |
| Age To |
75.00 Year(s) |
| Gender |
Both |
| Details |
a)Very low, low, or intermediate-risk MDS based on IPSS-R score with
less than 5% bone marrow blasts; baseline Hb of less than or equal to 9 g/dL;
b)MPN with anemia;
c)18 years and above and
d)Low RBC transfusion burden, defined as 1–4 packed red blood cell (pRBC) units per 8-week period or 1 pRBC transfusion per 8-week period for 2 consecutive 8-week periods before randomization.
|
|
| ExclusionCriteria |
| Details |
a)Higher – risk MDS; Del 5q cytogenetic abnormality; or have anemia of a non-MDS etiology (e.g., iron deficiency).
b)Children
c)Patients who are unable to come for a visit atleast every 2 months in the initial 6 month period of the study.
|
|
|
Method of Generating Random Sequence
|
Not Applicable |
|
Method of Concealment
|
Not Applicable |
|
Blinding/Masking
|
Not Applicable |
|
Primary Outcome
|
| Outcome |
TimePoints |
| The primary efficacy endpoint will be the proportion of patients who were TI for more than or equal to 8 consecutive weeks during the first 28 treatment weeks. |
2 months
6 months
12 months
18 months
24 months |
|
|
Secondary Outcome
|
| Outcome |
TimePoints |
(1) Study the proportion of patients who had above 50% reduction in the number of RBC transfusions over any 8 weeks compared with baseline,
(2) Study the proportion of patients who were TI for more than 20 consecutive weeks.
(3) Study the proportion of patients who develop Grade 3 or more toxicity
(4) To explore whether HIFa expression in peripheral blood at baseline or other time points correlates with response.
(5) Explore if EPO levels and iron related parameters predict response to therapy.
|
2 months
6 months
12 months
18 months
24 months |
|
|
Target Sample Size
|
Total Sample Size="40"
Sample Size from India="40"
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)
|
10/09/2025 |
| 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
|
N/A |
|
Individual Participant Data (IPD) Sharing Statement
|
Will individual participant data (IPD) be shared publicly (including data dictionaries)?
Response - NO
|
|
Brief Summary
|
Background: Anemia
is the predominant symptom in lower risk myelodysplastic syndrome (LR-MDS) and
some myeloproliferative illnesses especially primary myelofibrosis [MPN].
Response to standard medicines is seen in only 30-40% of patients and many
patients end up requiring multiple transfusions. Desidustat is a novel hypoxia
inducing factor [HIF-PHD] inhibitor which is used mainly for the treatment of
anemia in patients with chronic kidney disease [both dialysis dependent and
non-dependent]. It mimics the body’s natural response to the hypoxic condition
by inhibiting HIF-PH, thus preventing hydroxylation of HIF-alpha and allowing for the
transcription and expression of genes necessary for erythropoiesis, such as EPO
and iron metabolism factors. Based on the effectiveness of Desidustat in the
treatment of anemia in CKD, we hypothesized a potential clinical benefit in anemia
of LR-MDS and MPN.
Methodology: This is a
single arm, Phase II, Safety and Efficacy trial
where we will study the efficacy of Desidustat in treating transfusion
dependent anemia in patients with lower risk MDS or MPN. Desidustat will be orally
administered 100 mg 3 days in a week for 6 months. If there is inadequate
response after 2 months of initiation of therapy, the dose will be increased to
150mg three days a week for the subsequent 4 months, along with best supportive
care. We will also collect blood samples from patients at 0, 2 and 6 months to
look for HIFa expression, erythropoietin levels and iron parameters.
Results: The
primary efficacy endpoint will be the proportion of patients with transfusion
independence (TI) for more than 8 consecutive weeks in the first 24 treatment weeks.
Secondary efficacy endpoint will be the proportion of patients with a more than 50%
reduction in RBC transfusions over an 8-week period compared with baseline. We
will also study if HIFa expression correlated with hematological response.
Conclusions:
We hope to show that Desidustat will be able to ameliorate anemia in a group of
patients with MDS and MPN.
|