Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Senicapoc (SIL-8301) | Experimental | 20 mg twice daily for 4 days, followed by 10 mg once daily for up to 24 weeks |
|
| Placebo | Placebo Comparator | Matching placebo tablets twice daily for 4 days, followed by once daily for up to 24 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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 |
|
| Measure | Description | Time Frame |
|---|---|---|
| Hb response rate | Proportion of participants achieving an increase in Hb of > 1 g/dL from baseline | 24 Weeks |
| Measure | Description | Time Frame |
|---|---|---|
| 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) |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Head of Regulatory and Operations | Contact | 978-245-7397 | debora@biossil.ai |
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| C472774 | senicapoc |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
|
| Placebo | Drug | Tablets similar in size and color; matching administration schedule |
|
| 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 |
| Change from baseline in overall score and subscale domain scores of the Participant-Reported Outcomes Measurement Information System (PROMIS) | PROMIS-29 for adults; PROMIS Pediatric-25 for participants <18 years of age | 24 Weeks |
| Sickle cell disease complication rate | Proportion of participants experiencing at least one new or worsening hemolytic complication at any time during the study | 24 Weeks |
| 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 |