Trial | NCT02380079  Report issue

Title

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.

  • Phase

    Phase 1

  • Study Type

    Interventional

  • Status

    Active, not recruiting

  • Intervention/Treatment

    scd-101 ...

  • Study Participants

    60
The purpose of this study is to determine the safety and clinical effects of SCD-101 when given to adults with sickle cell disease.
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.
Study Started
Feb 28
2015
Primary Completion
Nov 30
2023
Anticipated
Study Completion
Dec 31
2023
Anticipated
Last Update
Mar 21
2023

Drug SCD-101

Administered as gelatin capsules

SCD-101 Experimental

SCD-101 dosed TID for 28-days

Placebo Placebo Comparator

Placebo dosed TID for 28-days

Criteria 

Inclusion Criteria:

Male or female, 18-55 years of age
Homozygous sickle cell disease or S/beta 0 thalassemia
Hemoglobin F ≤10%
Hemoglobin ≥ 6.0 g/dL and ≤ 9.5 g/dL
Female participants of child bearing potential and male participants whose partner is a female of child bearing potential must be willing to use approved contraception during the trial and for 3 months following the end of treatment. Only barrier methods or complete abstinence are acceptable for this study. Participants using hormonal contraception (including morning-after-pill) and IUD are excluded unless willing/able to change to an acceptable form of contraception.
Ability to adhere to the study visit schedule and other protocol requirements
Ability to understand and the willingness to sign an informed consent document

Exclusion Criteria:

Red blood cell transfusion within 3 months of enrollment
Hydroxyurea treatment within 6 months of enrollment
Painful or other acute sickle cell event that required a hospitalization within 4-weeks of enrollment
AST and/or ALT >3x upper limit of normal and/or creatinine >2x upper limit of normal or any other significant renal or hepatic impairment
Estimated creatinine clearance (CrCl) < 60 mL/min (Cockcroft- Gault formula) at screening.
QTc interval of >470 msec at trial entry and participant with congenital long QT syndrome.
No other significant sickle cell or non-sickle cell illness that would confound the results of the trial
Any condition that, in the view of the investigator, places the participant at risk because of participation in the trial, or may influence the result of the trial or the participant's ability to participate in the trial
Participant pregnant or nursing an infant or planning pregnancy during the course of the trial
History of allergic reactions attributed to sorghum or compounds of similar chemical or biologic composition (such as Nicosan, Niprisan, Jobelyn or Xickle).
Other investigational drug use within 3 months of enrollment
PROMIS Fatigue Questionnaire 8a T-score ˂ 44.3
No Results Posted