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

NCT ID: NCT07282210

Last Updated: 2025-12-15

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 105 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2026-01-31
• Study Completion Date: 2029-01-31

Brief Summary

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.

Conditions

Sickle Cell Disease; Sickle Cell Anaemia; Sickle Cell Anemia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Senicapoc (SIL-8301)

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

Group Type: EXPERIMENTAL

Senicapoc

Intervention Type: DRUG

10 mg tablets; administered at a loading dose of 20 mg twice daily for 4 days, followed by a maintenance dose of 10 mg once daily for up to 24 weeks

Placebo

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

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Tablets similar in size and color; matching administration schedule

Interventions

Senicapoc

10 mg tablets; administered at a loading dose of 20 mg twice daily for 4 days, followed by a maintenance dose of 10 mg once daily for up to 24 weeks

Intervention Type: DRUG

Placebo

Tablets similar in size and color; matching administration schedule

Intervention Type: DRUG

Other Intervention Names

SIL-8301

Eligibility Criteria

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

• Minimum Eligible Age: 16 Years
• Maximum Eligible Age: 35 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Biossil Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Central Contacts

Head of Regulatory and Operations

Role: CONTACT

debora@biossil.ai

978-245-7397

Other Identifiers

SIL-8301-001

Identifier Type: -

Identifier Source: org_study_id