A Phase IIa, Double Blind, Randomized, Placebo Controlled, Parallel, Multicentre, Proof-of-Concept Study to Evaluate the Efficacy and Safety of Desidustat Oral Tablet for Treatment of Sickle Cell Disease.
Mohit Desai, Bhargav Solanki, Manish Hathila et al.
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In 30 seconds
This phase IIa, double-blind, randomised, placebo-controlled, multicentre proof-of-concept trial assessed oral desidustat in 24 Indian adults with sickle cell disease. Participants received one of three doses or placebo thrice weekly for 8 weeks. The primary outcome was a haemoglobin increase of at least 1 g/dL. This response occurred in 50% of patients in the 150 mg cohort versus 16.7% with placebo. Haemoglobin increased dose-dependently, with reduced LDH levels; HbS and most other haemolysis markers did not change significantly. Desidustat was reported as well tolerated, with mild, non-serious adverse events considered unrelated to treatment.
Key findings
- A haemoglobin increase of at least 1 g/dL occurred in 50% of the 150 mg cohort versus 16.7% of the placebo group.
- Haemoglobin increased dose-dependently, and LDH levels decreased.
- No significant changes were observed in HbS or most haemolysis markers; no transfusions or vaso-occlusive crises occurred during the study.
- Reported adverse events were mild, non-serious, and considered unrelated to treatment.
Why it matters
Anaemia is a key feature of sickle cell disease, and this early trial provides a preliminary signal that desidustat may increase haemoglobin, particularly at the highest studied dose. The lack of significant change in HbS or most haemolysis markers means the findings do not establish broader effects on disease biology. Larger and longer studies would be needed to clarify efficacy and safety.
What to keep in mind
The trial included only 24 participants and lasted 8 weeks, limiting the precision and duration of the efficacy and safety evidence.