Efficacy and Safety of SIL-8301 for Control of Hemolysis in a Uniform Sickle Cell Disease Endotype
About this study
SIL-8301 (senicapoc) is being developed for the chronic treatment of patients with sickle cell disease in both adults and children. The purpose of this study is to compare the effects of senicapoc to placebo in patients with sickle cell disease that have had fewer than 2 acute sickle-related painful crises per year over the preceding 2 years, and have a predominantly hemolytic phenotype, defined as presence or history of at least one hemolytic complication and a baseline Hb of 9 g/dL or less, despite receiving hydroxyurea (an oral drug used for treatment of sickle cell disease) as standard of care. Participants will take senicapoc or matching placebo daily and continue on hydroxyurea as prescribed for up to 24 weeks.
- Condition
- Sickle Cell Disease, Sickle Cell Anaemia, Sickle Cell Anemia
- Tested
- Senicapoc, Placebo
- Sponsor
- Biossil Inc.
Who can join
- Age
- 16 to 35 years
- Sex
- All sexes
- Healthy volunteers
- Not accepted
Inclusion 9
- Minimum age: 16 years
- Maximum age: 35 years
- Study condition: Sickle Cell Disease, Sickle Cell Anaemia, Sickle Cell Anemia
- Documented diagnosis of sickle cell disease
- 16-35 years of age
- Hb ≤ 9.0 g/dL
- History of no more than 1 acute SCD-related painful crises requiring a visit to a medical facility per year within the preceding 2 years
- History of at least one hemolytic complication
- Current treatment with hydroxyurea
Exclusion 6
- Receipt of senicapoc in a previous investigational study
- Current Red Blood Cell (RBC) transfusion or exchange transfusion program
- History of pulmonary hypertension
- Active cardiovascular, neurologic, endocrine, hepatic, or renal disorders
- Diagnosis of cancer (except non-melanoma skin cancer in situ, cervical cancer in situ, or breast cancer in situ) within the last 5 years
- History of liver disease
Where
4 sites, 4 recruiting
The Hospital for Sick Children
Toronto, Ontario, Canada
Centre Hospitalier de l'Université de Montréal (CHUM)
Montreal, Quebec, Canada
McGill University Health Centre
Montreal, Quebec, Canada
University Health Network, Toronto General Hospital
Toronto, Ontario, Canada
Contact
-
Head of Clinical Operations
(978) 245-7397 trials@biossil.ai
Potential match only. Final eligibility is determined by the study team.