NCT07282210

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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
105

participants targeted

Target at P50-P75 for phase_2

Timeline
28mo left

Started Aug 2026

Geographic Reach
1 country

4 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress6%
Aug 2026Feb 2029

First Submitted

Initial submission to the registry

November 13, 2025

Completed
1 month until next milestone

First Posted

Study publicly available on registry

December 15, 2025

Completed
8 months until next milestone

Study Start

First participant enrolled

August 13, 2026

Completed
2.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2029

Expected
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2029

Last Updated

August 17, 2026

Status Verified

August 1, 2026

Enrollment Period

2.4 years

First QC Date

November 13, 2025

Last Update Submit

August 13, 2026

Conditions

Sickle Cell DiseaseSickle Cell AnaemiaSickle Cell Anemia

Keywords

senicapocAnemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesAnemia, Sickle CellAnaemia, Sickle Cell

Outcome Measures

Primary Outcomes (1)

  • Hb response rate

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

    24 Weeks

Secondary Outcomes (13)

  • Change from baseline in Hb (g/dL)

    24 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

  • +8 more secondary outcomes

Study Arms (2)

Senicapoc (SIL-8301)

EXPERIMENTAL

20 mg twice daily for 4 days, followed by 10 mg once daily for up to 24 weeks

Drug: Senicapoc

Placebo

PLACEBO COMPARATOR

Matching placebo tablets twice daily for 4 days, followed by once daily for up to 24 weeks

Drug: Placebo

Interventions

SenicapocDRUG

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

Also known as: SIL-8301
Senicapoc (SIL-8301)
PlaceboDRUG

Tablets similar in size and color; matching administration schedule

Placebo

Eligibility Criteria

Age16 Years - 35 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Documented diagnosis of sickle cell disease
  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

The Hospital for Sick Children

Toronto, Ontario, Canada

RECRUITING

University Health Network, Toronto General Hospital

Toronto, Ontario, Canada

NOT YET RECRUITING

Centre Hospitalier de l'Université de Montréal (CHUM)

Montreal, Quebec, Canada

NOT YET RECRUITING

McGill University Health Centre

Montreal, Quebec, Canada

NOT YET RECRUITING

MeSH Terms

Conditions

Anemia, Sickle CellAnemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Interventions

senicapoc

Central Study Contacts

Head of Clinical Operations

CONTACT

(978) 245-7397trials@biossil.ai

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 13, 2025

First Posted

December 15, 2025

Study Start

August 13, 2026

Primary Completion (Estimated)

January 1, 2029

Study Completion (Estimated)

February 1, 2029

Last Updated

August 17, 2026

Record last verified: 2026-08

Locations

CA(4)