Sickle Cell Anaemia, Sickle Cell Anemia, Sickle Cell Disease
Conditions
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
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.
Interventions
DRUGSenicapoc
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
DRUGPlacebo
Tablets similar in size and color; matching administration schedule
Sponsors
Biossil Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
16 Years to 35 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Hb response rate | 24 Weeks | Proportion of participants achieving an increase in Hb of \> 1 g/dL from baseline |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in overall score and subscale domain scores of the Participant-Reported Outcomes Measurement Information System (PROMIS) | 24 Weeks | The PROMIS-29 assessment for adult participants assesses depressive symptoms, anxiety, physical function, pain interference, fatigue, sleep disturbance, and ability to participate in social roles and activities. The PROMIS Pediatric-25 assessment for participants \<18 years of age assesses depressive symptoms, anxiety, physical function and mobility, pain interference, fatigue, and peer relationships. For both PROMIS assessments, a higher score indicates worse symptoms except for higher physical function values which indicate better physical function. |
| Sickle cell disease complication rate | 24 Weeks | Proportion of participants experiencing at least one new or worsening hemolytic complication at any time during the study |
| Proportion of participants with at least one category of improvement from baseline in Clinician and Patient Global Impression of Change | 24 Weeks | — |
| Frequency of acute sickle cell-related painful crises | 28 Weeks | — |
| Incidence of AEs, SAEs, and sickle cell disease related AEs | 28 Weeks | — |
| Change from baseline in lactate dehydrogenase (LDH) (u/L) | 24 Weeks | — |
| Change from baseline in indirect bilirubin (µmol/L) | 24 Weeks | — |
| Change from baseline in reticulocyte count (%) | 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 | The ASCQ-ME is a patient-reported outcome measurement system that evaluates and monitors the physical, mental, and social well-being of patients with SCD. For each subscale, there are 5 questions using a 5 point Likert scale. Scores for each subscale range from 0 to 100, where lower scores connote worse disease impact. |
| Change from baseline in Hb (g/dL) | 24 Weeks | — |
Countries
Canada
Contacts
CONTACTHead of Clinical Operations
Outcome results
None listed