Senicapoc and Dehydrated Stomatocytosis

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

5

Participants

Timeline

Start Date

April 15, 2021

Primary Completion Date

February 1, 2024

Study Completion Date

February 1, 2024

Conditions
Dehydrated Hereditary Stomatocytosis
Interventions
DRUG

Senicapoc (synonyms: ICA-17043; 2,2-bis-(4-fluorophenyl)-2-phenylacetamide)

This study is conducted as open label, with each patient's response determined with an adaptive Bayesian model based on historical and baseline values for the primary endpoints of the trial. 3 monthly dose escalation steps will assess the efficacy of senicapoc in a dose range (10-40 mg/day) which has been used in prior trials for other indications.

Trial Locations (1)

02115-5724

Boston Children's Hospital, Boston

All Listed Sponsors
lead

Boston Children's Hospital

OTHER