RESCUESIL-8301 for Hemolytic Sickle Cell Disease Patients
This study evaluates the effectiveness of SIL-8301 in increasing hemoglobin levels in adults with hemolytic sickle cell disease.
Senicapoc
+ Placebo
Anemia+6
+ Anemia, Hemolytic
+ Anemia, Hemolytic, Congenital
Treatment Study
Summary
Study start date: January 1, 2026
Actual date on which the first participant was enrolled.This study is focused on determining the effectiveness and safety of a treatment called SIL-8301 (senicapoc) for people with sickle cell disease, a condition that affects the blood. Specifically, it targets those who experience a type of sickle cell disease characterized by frequent destruction of red blood cells (hemolytic phenotype) and have had fewer than two painful crises related to sickle cell per year. The study is significant because it aims to find better ways to manage this condition in patients who are already receiving the standard treatment, hydroxyurea, but continue to have low hemoglobin levels, which can lead to various complications. Participants in the study will either receive senicapoc or a placebo, which is a substance with no therapeutic effect, every day for a period of up to 24 weeks. They will continue their usual treatment with hydroxyurea. The main focus is to see if senicapoc can increase the hemoglobin levels in their blood by more than 1 gram per deciliter from the starting point. This increase is an important sign of improved red blood cell health. The study is carefully designed to measure these changes while ensuring the safety of participants.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.105 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.From 16 to 35 Years
Range of ages for which participants are eligible to join.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
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
Study Plan
Find out more about all the medication administered in this study, their detailed description and what they involve.2 intervention groups are designated in this study
50% chance of being blinded to the placebo group
Treatment Groups
Group I
ExperimentalGroup II
Placebo