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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02380079
Recruitment Status : Active, not recruiting
First Posted : March 5, 2015
Last Update Posted : March 21, 2023
Sponsor:
Collaborator:
State University of New York - Downstate Medical Center
Information provided by (Responsible Party):
Invenux, LLC

Study Description
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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.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Sickle-Beta Zero Thalassemia Drug: SCD-101 Phase 1

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 Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description: 2x2 crossover
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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.
Actual Study Start Date : February 2015
Estimated Primary Completion Date : November 2023
Estimated Study Completion Date : December 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia

Arms and Interventions
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Arm Intervention/treatment
Experimental: SCD-101
SCD-101 dosed TID for 28-days
 Drug: SCD-101
Administered as gelatin capsules
Placebo Comparator: Placebo
Placebo dosed TID for 28-days
 Drug: SCD-101
Administered as gelatin capsules


Outcome Measures
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Primary Outcome Measures :
  1. 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) ]

Secondary Outcome Measures :
  1. 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) ]
  2. 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) ]
  3. 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) ]
  4. 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) ]
  5. 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) ]
  6. 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) ]

Other Outcome Measures:
  1. 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) ]
  2. 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) ]
  3. 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) ]
  4. 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) ]
  5. 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) ]
  6. 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) ]

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 55 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02380079


Locations
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United States, New York
King's County Hospital
Brooklyn, New York, United States, 11203
Sponsors and Collaborators
Invenux, LLC
State University of New York - Downstate Medical Center
Investigators
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Principal Investigator: John Muthu, MD King's County Hospital
More Information
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Publications:
Swift, RA, Nathan, S, Tripathi, P, Chen, Q, Asakura,T (2009, September). Research Preview on Improving Nicosan™/Xickle™: A Phyto-Medicine for the Treatment of Sickle Cell Disease. Paper presented at the meeting of the Sickle Cell Disease Association of America, Orlando, FL.

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Invenux, LLC
ClinicalTrials.gov Identifier: NCT02380079    
Other Study ID Numbers: INVX-SCD-101-11
First Posted: March 5, 2015    Key Record Dates
Last Update Posted: March 21, 2023
Last Verified: March 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Invenux, LLC:
Homozygous Sickle Cell Disease S/Beta 0 Thalassemia
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
 Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn