Dose-Finding Study of SC411 in Children With Sickle Cell Disease (SCOT Trial)

Phase 2

Waitlist Available

Led By Matthew Heeney, MD

Research Sponsored by Micelle BioPharma Inc

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 0 (baseline) through month 52 (end of treatment)

Awards & highlights

Approved for 5 Other Conditions

All Individual Drugs Already Approved

Summary

This is a Phase 2, randomized, double-blind, placebo-controlled, parallel-group, dose-finding study of SC411 in children with sickle cell disease (SCD). The primary objective of the study is to evaluate the safety and tolerability of three different doses of SC411 compared to a placebo. All patients will undergo eight weeks of oral study treatment and a four-week safety follow-up period. Patients will be randomized to one of three dose levels of SC411 or placebo.

Eligible Conditions

Sickle Cell Disease

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 0 (baseline) through month 52 (end of treatment)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~week 0 (baseline) through month 52 (end of treatment)

This trial's timeline: 3 weeks for screening, Varies for treatment, and week 0 (baseline) through month 52 (end of treatment) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Evaluate safety & tolerability of SC411 & determine change from Baseline in the blood cells omega-3 fatty acids index in subjects, treated with active SC411 doses or placebo in Part A and safety and tolerability in Part B.

Secondary study objectives

Measurement of the pharmacokinetic (PK) parameter of AUC will be performed for three dose levels of SC411 will be measured.

Measurement of the pharmacokinetic (PK) parameter of Cmax will be performed for three dose levels of SC411 will be measured.

Measurement of the pharmacokinetic (PK) parameter of Tmax will be performed for three dose levels of SC411 will be measured.

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Other study objectives

The intensity of painful crises on a scale of 0 to 10, reported by subjects receiving one of the three oral dose levels of SC411 compared to matching placebo will be evaluated from eDiary-records of patient reported outcomes.

The number of blood transfusions on subjects receiving one of the three oral dose levels of SC411 compared to matching placebo will be evaluated.

The number of painful crises had by subjects receiving one of the three oral dose levels of SC411 compared to matching placebo will be evaluated from eDiary-records of patient reported outcomes.

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