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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 : September 16, 2020
Sponsor:
Collaborator:
State University of New York - Downstate Medical Center
Information provided by (Responsible Party):
Invenux, LLC

Tracking Information
First Submitted Date  ICMJE February 23, 2015
First Posted Date  ICMJE March 5, 2015
Last Update Posted Date September 16, 2020
Actual Study Start Date  ICMJE February 2015
Estimated Primary Completion Date August 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 30, 2019)
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 (18 weeks) ]
Original Primary Outcome Measures  ICMJE
 (submitted: March 4, 2015)
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 ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: July 30, 2019)
  • 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 from baseline in functional capacity as measured by the 6-Minute Walk Test [ Time Frame: From the time the participant is accessed at baseline through the final follow-up (18 weeks) ]
Original Secondary Outcome Measures  ICMJE
 (submitted: March 4, 2015)
  • 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 ]
Current Other Pre-specified Outcome Measures
 (submitted: April 11, 2017)
  • 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) ]
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Dose-Escalation Study of SCD-101 in Sickle Cell Disease
Official Title  ICMJE 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.
Brief 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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description:
2x2 crossover
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE
  • Sickle Cell Disease
  • Sickle-Beta Zero Thalassemia
Intervention  ICMJE Drug: SCD-101
Administered as gelatin capsules
Study Arms  ICMJE
  • 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
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: April 11, 2017)
60
Original Estimated Enrollment  ICMJE
 (submitted: March 4, 2015)
24
Estimated Study Completion Date  ICMJE November 2021
Estimated Primary Completion Date August 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 55 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Robert Swift, PhD 970-567-8676 sponsor@invenux.com
Contact: Carolyn O' Reilly, BA 303-639-1157 carolyn@invenux.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02380079
Other Study ID Numbers  ICMJE INVX-SCD-101-11
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Invenux, LLC
Study Sponsor  ICMJE Invenux, LLC
Collaborators  ICMJE State University of New York - Downstate Medical Center
Investigators  ICMJE
Principal Investigator: Peter Gillette, MD King's County Hospital
PRS Account Invenux, LLC
Verification Date September 2020

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