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

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ClinicalTrials.gov Identifier: NCT02380079
Recruitment Status : Recruiting
First Posted : March 5, 2015
Last Update Posted : March 19, 2018
Sponsor:
Collaborator:
State University of New York - Downstate Medical Center
Information provided by (Responsible Party):
Invenux, LLC

February 23, 2015
March 5, 2015
March 19, 2018
February 2015
October 2018   (Final data collection date for primary outcome measure)
Determine the safety, tolerability, and dose limiting toxicities of escalating doses of SCD-101, assessed by frequency and severity of adverse events (AEs), and changes in vital signs, 12-lead ECGs and laboratory assessments as compared to baseline [ Time Frame: From the time the participant is administered the first dose through the final follow-up (16 weeks) ]
Determine the safety, tolerability, and dose limiting toxicities of escalating doses of SCD-101, assessed by frequency and severity of adverse events (AEs), and changes in vital signs, 12-lead ECGs and laboratory assessments as compared to baseline [ Time Frame: From the time the participant is administered the first dose through the final follow-up clinic visit, for a total of 6 weeks ]
Complete list of historical versions of study NCT02380079 on ClinicalTrials.gov Archive Site
  • Determine the effect of escalating doses of SCD-101 on the mean change in hemoglobin form base line [ Time Frame: From the time the participant is accessed at baseline through the final follow-up (18 weeks) ]
  • Determine the effect of escalating doses of SCD-101 on the mean change in percent reticulocytes from baseline [ Time Frame: From the time the participant is accessed at baseline through the final follow-up (18 weeks) ]
  • Determine the effect of escalating doses of SCD-101 on the mean change from baseline in red blood cell hemolysis as measured by lactate dehydrogenase (LDH) and indirect bilirubin. [ Time Frame: From the time the participant is accessed at baseline through the final follow-up (18 weeks) ]
  • Determine the effect of escalating doses of SCD-101 on the mean change from baseline in fatigue as measured by the PROMIS fatigue questionnaire [ Time Frame: From the time the participant is accessed at baseline through the final follow-up (18 weeks) ]
  • Determine the effect of escalating doses of SCD-101 on the mean change from baseline in the percent of venous circulating sickle red blood cells [ Time Frame: From the time the participant is accessed at baseline through the final follow-up (18 weeks) ]
  • Determine the effect of escalating doses of SCD-101 on the mean change in hemoglobin form base line. [ Time Frame: From the time the participant is accessed at baseline through the final follow-up clinic visit, for a total of 8 weeks ]
  • Determine the effect of escalating doses of SCD-101 on the mean change in percent reticulocytes from baseline [ Time Frame: From the time the participant is accessed at baseline through the final follow-up clinic visit, for a total of 8 weeks ]
  • Determine the effect of escalating doses of SCD-101 on the mean change from baseline in red blood cell hemolysis as measured by lactate dehydrogenase (LDH) and direct bilirubin. [ Time Frame: From the time the participant is accessed at baseline through the final follow-up clinic visit, for a total of 8 weeks ]
  • Exploratory Outcome Measure: Change from baseline of the ex-vivo percent of red blood cells that sickle under hypoxic conditions [ Time Frame: From the time the participant is accessed at baseline through the final follow-up clinic visit, for a total of 8 weeks ]
  • Exploratory Outcome Measure: Change from baseline in fatigue as measured by the PROMIS fatigue questionnaire score [ Time Frame: From the time the participant is accessed at baseline through the final follow-up clinic visit, for a total of 8 weeks ]
  • Part B: Exploratory Outcome Measure the mean change from baseline in sleep interference as measured by the PROMIS sleep interference questionnaire [ Time Frame: From the time the participant is accessed at baseline through the final follow-up (18 weeks) ]
  • Part B: Exploratory Outcome Measure the mean change from baseline in pain interference as measured by the PROMIS pain interference questionnaire [ Time Frame: From the time the participant is accessed at baseline through the final follow-up (18 weeks) ]
  • Part B: Exploratory Outcome Measure the mean change from baseline in patient reported daily pain as measured by a Numeric Rating Scale (NRS 0-10) [ Time Frame: From the time the participant is accessed at baseline through the final follow-up (18 weeks) ]
  • Part B: Exploratory Outcome Measure the mean change from baseline in analgesic usage as measured by patient medication diary [ Time Frame: From the time the participant is accessed at baseline through the final follow-up (18 weeks) ]
  • Part B: Exploratory Outcome Measure the mean:change from baseline in exercise and sleep activity as measured by wrist actigraphy [ Time Frame: From the time the participant is accessed at baseline through the final follow-up (18 weeks) ]
  • Part B: Exploratory Outcome Measure the mean:change from baseline in plasma inflammatory cytokines as measured by ELISA [ Time Frame: From the time the participant is accessed at baseline through the final follow-up (18 weeks) ]
Not Provided
 
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.
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.

Interventional
Phase 1
Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description:
2x2 crossover
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
  • Sickle Cell Disease
  • Sickle-Beta Zero Thalassemia
Drug: SCD-101
Administered as gelatin capsules
  • Experimental: SCD-101
    SCD-101 dosed TID for 28-days
    Intervention: Drug: SCD-101
  • Placebo Comparator: Placebo
    Placebo dosed TID for 28-days
    Intervention: Drug: SCD-101

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
60
24
December 2018
October 2018   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  1. Male or female, 18-55 years of age
  2. Homozygous sickle cell disease or S/beta 0 thalassemia
  3. Hemoglobin F ≤10%
  4. Hemoglobin ≥ 6.0 g/dL and ≤ 9.5 g/dL
  5. 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.
  6. Ability to adhere to the study visit schedule and other protocol requirements
  7. Ability to understand and the willingness to sign an informed consent document

Exclusion Criteria:

  1. Red blood cell transfusion within 3 months of enrollment
  2. Hydroxyurea treatment within 6 months of enrollment
  3. Painful or other acute sickle cell event that required a hospitalization within 4-weeks of enrollment
  4. 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
  5. Estimated creatinine clearance (CrCl) < 60 mL/min (Cockcroft- Gault formula) at screening.
  6. QTc interval of >470 msec at trial entry and participant with congenital long QT syndrome.
  7. No other significant sickle cell or non-sickle cell illness that would confound the results of the trial
  8. 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
  9. Participant pregnant or nursing an infant or planning pregnancy during the course of the trial
  10. History of allergic reactions attributed to sorghum or compounds of similar chemical or biologic composition (such as Nicosan, Niprisan, Jobelyn or Xickle).
  11. Other investigational drug use within 3 months of enrollment
  12. PROMIS Fatigue Questionnaire 8a T-score ˂ 44.3
Sexes Eligible for Study: All
18 Years to 55 Years   (Adult)
No
Contact: Robert Swift, PhD 970-567-8676 sponsor@invenux.com
Contact: Carolyn O' Reilly, BA 303-639-1157 carolyn@invenux.com
United States
 
 
NCT02380079
INVX-SCD-101-11
No
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Plan to Share IPD: No
Invenux, LLC
Invenux, LLC
State University of New York - Downstate Medical Center
Principal Investigator: Peter Gillette, MD King's County Hospital
Invenux, LLC
March 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP