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

NCT07282210 | Not Yet Recruiting Phase 2 DMC

• 1 other identifier: SIL-8301-001
• Study Type: interventional
• Target: 105
• Locations: 0 countries
• Sites: N/A

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

Keywords

senicapoc; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Anemia, Sickle Cell; Anaemia, Sickle Cell

Outcome Measures

Primary Outcomes (1)

• Hb response rate

  Proportion of participants achieving an increase in Hb of \> 1 g/dL from baseline

  24 Weeks

Secondary Outcomes (10)

• Change from baseline in hemolytic markers

  24 Weeks

• Proportion of participants with a Hb increase of > 2g/dL from baseline

  24 Weeks

• Percent change from baseline in urine albumin-creatinine ratio (uACR)

  24 Weeks

• Change from baseline in the 6-minute walk test (6mwt)

  24 Weeks

• Change from baseline in participant reported quality of life assessment overall score and subscale domain scores of the Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-ME)

  24 Weeks

Study Arms (2)

Senicapoc (SIL-8301)

EXPERIMENTAL

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

Drug: Senicapoc

Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

Senicapoc 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

Also known as: SIL-8301

Senicapoc (SIL-8301)

Placebo DRUG

Tablets similar in size and color; matching administration schedule

Placebo

Eligibility Criteria

• Age: 16 Years - 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Documented diagnosis of sickle cell disease
• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Biossil Inc.: lead

MeSH Terms

Conditions

Anemia, Sickle Cell; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Interventions

senicapoc

Central Study Contacts

Head of Regulatory and Operations

CONTACT

978-245-7397; debora@biossil.ai

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 13, 2025
• First Posted: December 15, 2025
• Study Start: January 1, 2026
• Primary Completion (Estimated): November 1, 2028
• Study Completion (Estimated): January 1, 2029
• Last Updated: December 15, 2025

Record last verified: 2025-11