A study to evaluate the efficacy and safety of Desidustat oral tablets for the treatment of sickle cell disease

Phase 2

Conditions: Health Condition 1: D572- Sickle-cell/Hb-C disease

Registration Number: CTRI/2024/06/068363

Lead Sponsor: Zydus Lifesciences Limited

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Yet Recruiting

Sex: Not specified

Target Recruitment: 0

Inclusion Criteria

1) Indian male and female participants with confirmed diagnosis of Sickle cell

disease (any genotype; Documentation of SCD genotype may be based on available

or history of confirmatory laboratory testing with hemoglobin electrophoresis, HPLC,

or genetic testing).

2)Hemoglobin level 7-11 g/dL for males and 6.5-11 g/dL for females (both inclusive) at

screening

3)Participants have had at least 1 episode of Vaso Occlusive Crisis (VOC) in the past

12 months. For study eligibility, VOC is defined as a previously documented episode

of Acute Chest Syndrome (ACS) or acute painful crisis (for which there was no

explanation other than VOC) which required prescription or healthcare professionalinstructed

use of analgesics for moderate to severe pain (documentation must exist in

the patient medical record prior to Screening).

4) Serum ferritin level =100 ng/mL or/and TSAT = 20 % at screening

5) No clinically significant vitamin B12 or Folate deficiency at screening

6) For participants taking hydroxyurea, L-glutamine, crizanlizumab, the dose of diseasemodifying therapies must be stable for at least 60 days prior to signing the ICF.

7) Men and women of childbearing potential must agree to use adequate birth control

measures during the study. Acceptable methods of birth control in this study include:

surgical sterilization, intrauterine device, oral contraceptive, contraceptive patch,

long-acting injectable contraceptive, partner’s vasectomy, double-barrier method

(condom or diaphragm with spermicide) during study participation and for 30 days

after their last dose of study drug.

8) Participant has provided signed informed written consent. In case of illiterate patients,

thumb impression of the patients will be obtained along with the signature of the

impartial witness on the consent form prior to patient’s participation in the trial.

Exclusion Criteria

1)Participants who have received blood transfusion within 30 days prior to signing of

ICF.

2) Hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of

signing the ICF (i.e., hospitalization and discharge for vaso-occlusive event cannot be

within 14 days prior to signing the ICF).

3) Participants who are currently receiving Voxelotor.

4)Diagnosis of cirrhosis

5)New arterial or venous thrombosis diagnosed within 6 months of signing the ICF.

6)Participants who have participated in clinical trial of any other investigational product

or medical device other than the present trial within 3 months prior to screening.

7)Major surgery within 90 days prior to randomization, and minor surgery within 30

days prior to randomization.

8)History of severe allergic or hypersensitivity reaction to investigational products and

its excipients

9)Unable to swallow tablets or disease significantly affecting gastrointestinal function

and/or inhibiting small intestine absorption such as; mal-absorption syndrome,resection of the small bowel or poorly controlled inflammatory bowel disease affecting the small intestine.

10)Medical, psychological or behavioural conditions, which in the opinion of the

investigator, may preclude safe participation, confound study interpretation, interfere

with compliance, or preclude informed consent.

11)Participant requiring treatment with intravenous antibiotic within 14 days of

enrolment.

12)History of Hepatitis A, B, C or HIV infection or positive test for Hbs Ag, HCV or

HIV at screening.

13)Receipt of erythropoietin, Voxelotor or other hemopoietic growth factor treatment

within 28 days of signing ICF or anticipated need for such agent during the study.

14)Prior history of pancreatitis or myocardial infarction within 06 months prior to

screening.

15)History of significant alcoholism or drug abuse within the past 1 year. History or

presence of significant smoking (more than 10 cigarettes per day) or consumption of

tobacco/nicotine products (more than 10 times per day).

16)History of difficulty with donating blood.

17)Intravenous iron within 14 days prior to enrolment.

18)History of previous or concurrent cancer.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
To assess the efficacy of <br/ ><br>Desidustat oral tablet in sickle cell <br/ ><br>disease patients i.e Proportion of patients with Hb response compared to PlaceboTimepoint: Baseline to Week 08

Secondary Outcome Measures

Name	Time	Method
Mean change in hemoglobin levelTimepoint: Baseline to Week 08;Proportion of patient requiring blood <br/ ><br>transfusionTimepoint: Baseline to Week 08;Proportion of patients experiencing vasoocclusive <br/ ><br>crisisTimepoint: Baseline to Week 08;To assess the effect of Desidustat on <br/ ><br>biomarkers related to hemolysisTimepoint: Baseline to Week 08