CompletedPhase 1/2

Senicapoc and Dehydrated Stomatocytosis

United States5 participantsStarted 2021-04-15

Plain-language summary

Dehydrated stomatocytosis is a genetic disorder characterized by chronic hemolysis, variable anemia and erythrocyte dehydration. Causative mutations have been identified in either the Gardos (KCNN4) channel or the mechanosensitive channel PIEZO1. Senicapoc is a selective blocker of the Gardos channel that has been extensively studied in sickle cell disease and shown to be safe with limited side-effects. However, senicapoc did not meet the designated clinical endpoints in a pivotal phase 3 trial. The present study is an explanatory, proof-of-concept study of Senicapoc administered once daily in patients with familial dehydrated stomatocytosis caused by autosomal dominant V282 mutations in the Gardos (KCNN4) channel.

Who can participate

Age range21 Years

SexALL

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Inclusion criteria

✓. Have a diagnosis of dehydrated stomatocytosis with a molecularly confirmed mutation in KCNN4.
✓. Are at least 21 years of age.
✓. Have hematological manifestations of dehydrated stomatocytosis such as elevated MCHC, compensated or uncompensated chronic hemolysis, with reticulocytosis. For enrollment, 3/5 of the following baseline value must meet enrollment criteria:
✓. Personally dated and signed informed consent detailing all the pertinent aspects of the study.
✓. Willingness to adhere to study visit schedule, treatment plan, blood draws and laboratory tests and other study procedures.

What they're measuring

chronic hemolysis biomarkers

Timeframe: 6 months

Trial details

NCT IDNCT04372498

SponsorBoston Children's Hospital

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2024-02-01

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