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

Sickle Cell Disease Clinical Trial

Official title: 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 Active, not recruiting

Clinical Trial 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.

Clinical Trial 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. ;

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease
• Sickle-Beta Zero Thalassemia
• Thalassemia

• NCT number: NCT02380079
• Study type: Interventional
• Source: Invenux, LLC
• Status: Active, not recruiting
• Phase: Phase 1
• Start date: February 2015
• Completion date: December 2023