NCT02380079

Dose-Escalation Study of SCD-101 in Sickle Cell Disease

Part A: Phase IB, Single Site, Dose-Escalation of SCD-101 and Part B: Randomized, Double-Blind, Placebo-Controlled Crossover of SCD-101 in Adults With Homozygous Sickle Cell Disease or S/Beta 0 Thalassemia.

• Status: Unknown
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 60 (estimated)
• Sponsor: Invenux, LLC · Industry
• Sex: All
• Age: 18 Years – 55 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to determine the safety and clinical effects of SCD-101 when given to adults with sickle cell disease.

Detailed description

This is single site, dose- escalation study of SCD-101 in participants with homozygous sickle cell disease (S/S) or S/beta 0 Thalassemia. All participants will be monitored for safety, tolerability, and dose-limiting toxicities. The study is divided into two parts. Part A is an open-label, non-randomized, non-placebo-controlled dose escalation study with a 28-day treatment phase and 14-day follow-up phase with five cohorts . Part B is a randomized, placebo-controlled, confirmatory 2x2 crossover cohort with a 28 day washout between periods, and a 28-day follow-up phase.

Conditions

• Sickle Cell Disease
• Sickle-Beta Zero Thalassemia

Interventions

Type	Name	Description
DRUG	SCD-101	Administered as gelatin capsules

Timeline

• Start date: 2015-02-01
• Primary completion: 2023-11-01
• Completion: 2023-12-01
• First posted: 2015-03-05
• Last updated: 2023-03-21

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study