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CTRI Number  CTRI/2024/11/077511 [Registered on: 29/11/2024] Trial Registered Prospectively
Last Modified On: 23/03/2026
Post Graduate Thesis  No 
Type of Trial  Interventional 
Type of Study   Drug 
Study Design  Randomized, Parallel Group, Multiple Arm Trial 
Public Title of Study   This study is a randomized, placebo-controlled, double-blind, multicenter Phase 2/3 study of patients, age 12 to 65 years, with sickle cell disease (SCD). 
Scientific Title of Study   An Adaptive, Randomized, Placebo-controlled, Double -blind, Multicenter Study of Oral Etavopivat, a Pyruvate Kinase Activator in Patients with Sickle Cell Disease. 
Trial Acronym  Forma Therapeutics Inc. 
Secondary IDs if Any  
Secondary ID  Identifier 
2020-003884-25  EudraCT 
4202-HEM-301 Version No. 06 Protocol Date 10-APR-2024  Protocol Number 
NCT04624659  ClinicalTrials.gov 
 
Details of Principal Investigator or overall Trial Coordinator (multi-center study)  
Name  Dr Meera Chakra 
Designation  principal Investigator 
Affiliation  Victoria hospital 
Address  Department of Clinical Hematology,
5th floor New OPD Block, Mysore road Near City Market, NewTharagupet Bangalore-560002,Kamataka,India
Bangalore
KARNATAKA
560002
India 
Phone  9845134167  
Fax    
Email  meerachakra@yahoo.com  
 
Details of Contact Person
Scientific Query  
Name  Sumit Gupta  
Designation  Director Clin Ops 
Affiliation  Syneos Health 
Address  4th Floor, Block 2, DLF Downtown, DLF City Phase 3 Rd,
Ambience Island, DLF Phase 3, Sector 24, Gurugram, Haryana -122022
Gurgaon
HARYANA
122022
India 
Phone  8532011805  
Fax    
Email  sumit.gupta@syneoshealth.com  
 
Details of Contact Person
Public Query  
Name  Ataur Rahman 
Designation  Manager, Clinical Operations 
Affiliation  Syneos Health 
Address  4th Floor, Block 2, DLF Downtown, DLF City Phase 3 Rd, Ambience Island, DLF Phase 3, Sector 24, Gurugram, Haryana -122022

Gurgaon
HARYANA
122022
India 
Phone  8532011805  
Fax    
Email  ataur.rahman@syneoshealth.com  
 
Source of Monetary or Material Support  
Forma Therapeutics, Inc. 
 
Primary Sponsor  
Name  Forma Therapeutics, Inc. 
Address  300 North Beacon Street, Suite 501 Watertown, MA 02472 Telephone: 617-679-1970 
Type of Sponsor  Pharmaceutical industry-Global 
 
Details of Secondary Sponsor  
Name  Address 
NIL  NIL 
 
Countries of Recruitment     Canada
Egypt
France
Germany
Ghana
Greece
Italy
Kenya
Lebanon
Nigeria
Oman
Saudi Arabia
Spain
Turkey
United Kingdom
United States of America
India  
Sites of Study  
No of Sites = 6  
Name of Principal Investigator  Name of Site  Site Address  Phone/Fax/Email 
Dr Tulika Seth  All India Institute of Medical Sciences  Room No. 10, 5th Floor, Teaching Block, Ansari Nagar, East, New Delhi- 11029, India
New Delhi
DELHI 		 9868397236

drtulikaseth@gmail.com 
Dr Alok Shrivastava  Christian Medical College  Department of Haematology, Vellore, Ranipet Campus, A Block, Fifth Floor, Kilminnal Village, Ranipet District, Ranipet -632517, Tamil Nadu, India
Vellore
TAMIL NADU 		 4162282352

aloks@cmcvellore.ac.in 
Dr Shrinath Kshirsagar  K.J Somaiya Hospital & Research Centre  Somaiya Ayurvibar Complex Eastern Express Highway, Sion (E), Mumbai 400022 Maharashtra, India
Mumbai
MAHARASHTRA 		 9821556030

shrinathk2000@gmail.com 
Dr Rabindra Jena  Srirama Chandra Bhanja Medical College & Hospital  Department of Clinical Hematology, 1st Floor, Old medicine Building, Cuttack- 753007, Odisha, India
Cuttack
ORISSA 		 9437022343

rkjena@msn.com 
Dr Meera V  Victoria hospital  Department of Clinical Hematology, 5th floor New OPD Block, Mysore road Near City Market, NewTharagupet Bangalore-560002,Kamataka,India
Bangalore
KARNATAKA 		 9845134167

meerachakra@yahoo.com 
Dr Ganesh Jaishetwar  Yashoda Healthcare Services Pvt. Ltd.   Yashoda Hospitals, Cyber towers to JNTU Road, Hitech city, Hyderabad, Telangana-500084, India
Hyderabad
TELANGANA 		 9849388806

ganeshjaishetwar@gmail.com 
 
Details of Ethics Committee  
No of Ethics Committees= 3  
Name of Committee  Approval Status 
Ethics Committee of BMCRI  Approved 
Institutional Ethics Committee K.J Somaiya Hospital & Research Centre  Approved 
Institutional Ethics Committee Yashoda Academy of Medical Educational &Research Yashoda Group of Hospitals  Approved 
 
Regulatory Clearance Status from DCGI  
Status 
Approved/Obtained 
 
Health Condition / Problems Studied  
Health Type  Condition 
Patients  (1) ICD-10 Condition: D571||Sickle-cell disease without crisis,  
 
Intervention / Comparator Agent  
Type  Name  Details 
Intervention  • Drug: Etavopivat Tablets High dose  o 400 mg once daily 
Comparator Agent  • Drug: Placebo Tablets  o Placebo once daily 
Intervention  Drug: Etavopivat Tablets  Selected dose once daily 
Intervention  Drug: Etavopivat Tablets Low dose  o 200 mg once daily  
 
Inclusion Criteria  
Age From  12.00 Year(s)
Age To  65.00 Year(s)
Gender  Both 
Details  Patient has provided documented informed consent or assent (the informed consent form [ICF] must be reviewed and signed by each patient; in the case of adolescent patients, both the consent of the patient’s legal representative or legal guardian, and the patient’s assent must be obtained)
- Patient has a confirmed diagnosis of sickle cell disease Documentation of SCD genotype (HbSS, HbSβ0-thalassemia or other sickle cell syndrome variants) based on prior history of laboratory testing. Molecular genotyping is not required. SCD genotype may be determined from the results of Hb electrophoresis, HPLC, or similar testing. Note that Hb electrophoresis is performed by the central laboratory at Screening.
- Patient has 2–15 episodes of documented VOC (defined in protocol Section 8.2.2) within the 12 months prior to screening. Documentation must exist in the patient’s medical record prior to screening. Events based solely on patient recall without supporting documentation should not be counted towards eligibility.
- Hemoglobin ≥ 5.5 and ≤ 10.5 g/dL (≥ 55 and ≤ 105 g/L) during screening
- For participants taking HU, the dose of HU (mg/kg) must be stable (no more than a 20% change in dosing) for at least 90 days prior to start of study treatment with no anticipated need for dose adjustments during the study, in the opinion of the Investigator
- Patients on crizanlizumab or l-glutamine oral powder (Endari®) treatment at the time of consent may be eligible if they:
 Have been on a stable dose for ≥ 12 months at the time of consent (i.e., no changes to the dose except for changes to weight or for safety reasons)
 Have been ≥ 80% compliant with the planned regimen during the 12 months prior to the time of consent
ï‚· Meet the VOC eligibility requirement in Inclusion Criterion 4
- Patients, who if female and of childbearing potential, are using acceptable methods of contraception and agree not to donate ova from study start to 90 days after the last dose of study drug, and who if male are willing to use acceptable methods of contraception and agree not to donate sperm, from study start to 90 days after the last dose of study drug 
 
ExclusionCriteria 
Details  Medical Conditions
1. More than 15 VOCs (defined in protocol Section 8.2.2) within the past 12 months prior to screening
2. Hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of Screening
3. Female who is breast feeding or pregnant
4. Hepatic dysfunction characterized by
- Alanine aminotransferase (ALT) greater than 4.0 × upper limit of normal (ULN)
OR
- Direct bilirubin greater than 3.0 × ULN
5. Patients with clinically significant bacterial, fungal, parasitic, or viral infection requiring systemic therapy
- Patients with acute bacterial, fungal, parasitic, or viral infection requiring systemic therapy should delay screening or enrollment until active therapy has been completed
Note: Infection prophylaxis is allowed (see concomitant medication restrictions)
6. Known human immunodeficiency virus (HIV) positivity
7. Active infection with hepatitis B virus (hepatitis B surface antigen [HepBsAg] and hepatitis B core antibody [HepBcAb] positive)
8. Active hepatitis C infection
9. Severe renal dysfunction (estimated glomerular filtration rate at the Screening visit; calculated by the central laboratory less than 30 mL/min/1.73 m2) or on chronic dialysis
10. History of malignancy within the past 2 years prior to treatment Day 1 requiring systemic chemotherapy and/or radiation
ï‚· Patients with malignancy considered surgically cured are eligible (e.g., non-melanoma skin cancer, cancer of the cervix in-situ, ductal carcinoma in situ [stage 1], grade 1 endometrial cancer) 
 
Method of Generating Random Sequence   Computer generated randomization 
Method of Concealment   Pharmacy-controlled Randomization 
Blinding/Masking   Participant, Investigator, Outcome Assessor and Date-entry Operator Blinded 
Primary Outcome  
Outcome  TimePoints 
Hemoglobin response rate at Week 24 (increase of greater than 1 g/dL [grater than 10 g/L] from baseline) during the blinded treatment period


Annualized vaso-occlusive crisis rate during the 52-week blinded treatment period based on adjudicated vaso-occlusive crisis review 		 24 Weeks

52 Weeks 
 
Secondary Outcome  
Outcome  TimePoints 
Change from baseline in hemoglobin at Week 24 during the blinded treatment period  24 Weeks 
Change from baseline in hemoglobin at Week 52 during the blinded treatment period  52 Weeks 
Change in absolute reticulocyte count from baseline at Week 24 during the blinded treatment period  24 Weeks 
Change in unconjugated bilirubin from baseline at Week 24 during the blinded treatment period  24 Weeks 
Change in lactate dehydrogenase from baseline at Week 24 during the blinded treatment period  24 Weeks 
Time to first vaso-occlusive crisis during the blinded treatment period  52 Weeks 
Change in PROMIS Fatigue Scale from baseline in adult patients at Week 24 during the blinded treatment period  24 Weeks 
Change in PROMIS Fatigue Scale from baseline in adult patients at Week 52 during the blinded treatment period  52 Weeks 
 
Target Sample Size   Total Sample Size="450"
Sample Size from India="32" 
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 2/ Phase 3 
Date of First Enrollment (India)   01/01/2025 
Date of Study Completion (India) Applicable only for Completed/Terminated trials 
Date of First Enrollment (Global)  09/04/2021 
Date of Study Completion (Global) Applicable only for Completed/Terminated trials 
Estimated Duration of Trial   Years="3"
Months="5"
Days="0" 
Recruitment Status of Trial (Global)
Modification(s)  		 Open to Recruitment 
Recruitment Status of Trial (India)  Closed to Recruitment of Participants 
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   This clinical trial is a Phase 2/3 study that will evaluate the efficacy and safety of FT-4202 and test how well FT-4202 works compared to placebo to improve the amount of hemoglobin in the blood and to reduce the number of vaso-occlusive crises (times when the blood vessels become blocked and cause pain). 
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