Dose-Escalation Study of SCD-101 in Sickle Cell Disease
Last updated on July 2021Recruitment
- Recruitment Status
- Recruiting
- Estimated Enrollment
- 24
Summary
- Conditions
- Sickle Cell Disease
- Sickle Beta Zero Thalassemia
- Type
- Interventional
- Phase
- Phase 1
- Design
- Allocation: RandomizedIntervention Model: Crossover AssignmentIntervention Model Description: 2x2 crossoverMasking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)Primary Purpose: Treatment
Participation Requirements
- Age
- Between 18 years and 55 years
- Gender
- Both males and females
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-rando...
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.
Tracking Information
- NCT #
- NCT02380079
- Collaborators
- State University of New York - Downstate Medical Center
- Investigators
- Principal Investigator: John Muthu, MD King's County Hospital
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