ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 3 for:    scd-101
Previous Study | Return to List | Next Study

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
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

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 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.
Study Start Date : February 2015
Estimated Primary Completion Date : October 2018
Estimated Study Completion Date : December 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia

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




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 (16 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) ]

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) ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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

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


Contacts
Contact: Robert Swift, PhD 970-567-8676 sponsor@invenux.com
Contact: Carolyn O' Reilly, BA 303-639-1157 carolyn@invenux.com

Locations
United States, New York
King's County Hospital Recruiting
Brooklyn, New York, United States, 11203
Contact: Susanne Fryd, BS    718-613-8188    susanne@invenux.com   
Sponsors and Collaborators
Invenux, LLC
State University of New York - Downstate Medical Center
Investigators
Principal Investigator: Peter Gillette, MD King's County Hospital

Additional Information:
Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Invenux, LLC
ClinicalTrials.gov Identifier: NCT02380079     History of Changes
Other Study ID Numbers: INVX-SCD-101-11
First Posted: March 5, 2015    Key Record Dates
Last Update Posted: March 19, 2018
Last Verified: March 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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:
Anemia, Sickle Cell
Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn