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Not yet recruiting NCT07282210

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

Phase II Interventional Sickle Cell Disease Sickle Cell Anaemia Sickle Cell Anemia

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Senicapoc, Placebo.
Who it may be relevant to
Registry conditions: Sickle Cell Disease, Sickle Cell Anaemia, Sickle Cell Anemia. Basic parameters: 16 years — 35 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Center list to be confirmed — check the primary protocol.
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official 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

Overview

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.

Interventions

  • Drug 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
  • Drug Placebo
    Tablets similar in size and color; matching administration schedule

Primary outcome measures

  • Hb response rate [Time frame: 24 Weeks]
Secondary outcome measures (10)
  • Change from baseline in hemolytic markers [Time frame: 24 Weeks]
  • Proportion of participants with a Hb increase of > 2g/dL from baseline [Time frame: 24 Weeks]
  • Percent change from baseline in urine albumin-creatinine ratio (uACR) [Time frame: 24 Weeks]
  • Change from baseline in the 6-minute walk test (6mwt) [Time frame: 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) [Time frame: 24 Weeks]
  • Change from baseline in overall score and subscale domain scores of the Participant-Reported Outcomes Measurement Information System (PROMIS) [Time frame: 24 Weeks]
  • Sickle cell disease complication rate [Time frame: 24 Weeks]
  • Proportion of participants with at least one category of improvement from baseline in Clinician and Patient Global Impression of Change [Time frame: 24 Weeks]
  • Frequency of acute sickle cell-related painful crises [Time frame: 28 Weeks]
  • Incidence of AEs, SAEs, and sickle cell disease related AEs [Time frame: 28 Weeks]

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Quadruple blind
Primary purpose
Treatment

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT07282210 · SIL-8301-001

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗