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NCT Number: NCT07282210

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

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.

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Key information

Conditions

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

Age range

16 year–35 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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

Other names: SIL-8301

Placebo

Drug

Tablets similar in size and color; matching administration schedule

Primary outcomes

  1. Hb response rate

    Time frame: 24 Weeks

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

Secondary outcomes

  1. Change from baseline in hemolytic markers

    Time frame: 24 Weeks

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

    Time frame: 24 Weeks

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

    Time frame: 24 Weeks

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

    Time frame: 24 Weeks

  5. 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)

    Time frame: 24 Weeks

  6. Change from baseline in overall score and subscale domain scores of the Participant-Reported Outcomes Measurement Information System (PROMIS)

    Time frame: 24 Weeks

    PROMIS-29 for adults; PROMIS Pediatric-25 for participants <18 years of age

  7. Sickle cell disease complication rate

    Time frame: 24 Weeks

    Proportion of participants experiencing at least one new or worsening hemolytic complication at any time during the study

  8. Proportion of participants with at least one category of improvement from baseline in Clinician and Patient Global Impression of Change

    Time frame: 24 Weeks

  9. Frequency of acute sickle cell-related painful crises

    Time frame: 28 Weeks

  10. Incidence of AEs, SAEs, and sickle cell disease related AEs

    Time frame: 28 Weeks

Study contacts

Contact information is provided by the study sponsor or research team.

Head of Regulatory and Operations

CONTACT

[email protected]

978-245-7397

Sponsors and collaborators

Lead sponsor

Biossil Inc.

Industry

Registry information

Official study title

A Multicenter, Randomized, Double-blind, Placebo-controlled Study to Determine Efficacy and Safety of SIL-8301 in Sickle Cell Disease (SCD) Patients With a Predominantly Hemolytic Phenotype

Acronym: RESCUE

Important dates

Study start
2026
Primary completion
2028
Study completion
2029
First posted
Dec 15, 2025
Registry last updated
Dec 15, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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