Efficacy and Safety of SIL-8301 for Control of Hemolysis in a Uniform Sickle Cell Disease Endotype (RESCUE)

Biossil Inc.

Status and phase: Not yet enrolling; Phase 2

Conditions: Sickle Cell Anemia; Sickle Cell Disease; Sickle Cell Anaemia

Treatments: Drug: Placebo; Drug: Senicapoc

Study type: Interventional

Funder types: Industry

Identifiers

NCT07282210

SIL-8301-001

Details and patient eligibility

About

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.

Enrollment: 105 estimated patients

Sex: All

Ages: 16 to 35 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 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 criteria

• 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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105 participants in 2 patient groups, including a placebo group

Senicapoc (SIL-8301)

Experimental group

Description:

20 mg twice daily for 4 days, followed by 10 mg once daily for up to 24 weeks

Treatment:

Drug: Senicapoc

Placebo

Placebo Comparator group

Description:

Matching placebo tablets twice daily for 4 days, followed by once daily for up to 24 weeks

Treatment:

Drug: Placebo