Phase 1b study enrollment is anticipated to restart soon, with minor adjustments.
The FDA has cleared the way for Fulcrum Therapeutics to continue its Phase 1b clinical trial of FTX-6058, an oral treatment for sickle cell disease (SCD). The trial will now proceed with stricter eligibility criteria.
Key Points:
- The Phase 1b clinical trial (NCT05169580) will resume with modified, stricter eligibility criteria.
- FTX-6058 aims to increase fetal hemoglobin (HbF) levels, which are more effective at oxygen transport than adult hemoglobin.
- Early data showed dose-dependent increases in HbF levels.
Additional Points:
- The clinical hold was initially due to concerns about the potential risk of blood cancers.
- Eligible participants must have experienced at least four SCD pain crises in the past year or two crises in the past six months.
- The trial is limited to patients who have not responded to, were intolerant of, or ineligible for other approved SCD therapies like hydroxyurea, Oxbryta, and Adakveo.
Conclusion:
- The lifting of the FDA’s clinical hold allows for the resumption of a promising trial that could offer a new oral treatment option for SCD patients, albeit with stricter eligibility criteria.
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“Based on the initial data from the Phase 1b trial, which showed increasing levels of HbF with each dose escalation, we believe in the potential of FTX-6058 to not only shift the current standard of care but importantly, offer these patients a differentiated oral option.”
– Alex C. Sapir, President and CEO of Fulcrum Therapeutics
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