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

ConditionsSickle Cell DiseaseSickle Cell AnaemiaSickle Cell Anemia
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age16-35
SponsorBiossil Inc.

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Parallel

Treatments tested in this trial

  • 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

  • Placebo

    Drug

    Tablets similar in size and color; matching administration schedule

Treatment groups

105 Participants
are divided into 2 treatment groups
Group A: Senicapoc (SIL-8301)Experimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Hb response rate

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

Time frame
24 Weeks

Secondary outcomes

1

Change from baseline in Hb (g/dL)

Time frame
24 Weeks
2

Change from baseline in lactate dehydrogenase (LDH) (u/L)

Time frame
24 Weeks
3

Change from baseline in indirect bilirubin (µmol/L)

Time frame
24 Weeks
4

Change from baseline in reticulocyte count (%)

Time frame
24 Weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Biossil Inc.

Lead sponsor

Source ClinicalTrials.gov
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