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A Sickle CEll Disease ComplicatioN Trial (ASCENT)

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: NCT02604368
Recruitment Status : Unknown
Verified January 2019 by Micelle BioPharma Inc.
Recruitment status was:  Not yet recruiting
First Posted : November 13, 2015
Last Update Posted : February 1, 2019
Sponsor:
Information provided by (Responsible Party):
Micelle BioPharma Inc

Tracking Information
First Submitted Date  ICMJE November 11, 2015
First Posted Date  ICMJE November 13, 2015
Last Update Posted Date February 1, 2019
Estimated Study Start Date  ICMJE March 2019
Estimated Primary Completion Date December 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 30, 2019)		 Assessment of the efficacy of SC411 in reducing the number of sickle cell crisis (SCC) events in subjects compared to placebo will be measured by counting the number of sickle cell crises that occur after randomization. [ Time Frame: 52 weeks ]
Assessment of the efficacy of SC411 in reducing the number of sickle cell crisis (SCC) events in subjects compared to placebo will be measured by counting the number of sickle cell crises that occur after randomization. The primary objective of this study is to assess the efficacy of orally administered SC411 in reducing the number of sickle cell crisis (SCC) events in sickle cell disease (SCD) subjects compared to placebo. A SCC event will be defined as either an acute painful crisis or an acute chest syndrome.
Original Primary Outcome Measures  ICMJE
 (submitted: November 12, 2015)		 Annualized sickle cell crisis rate [ Time Frame: 52 weeks ]
The total number of adjudicated acute sickle cell crises divided by the total number of months in the study from randomization then multiplied by 12
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 30, 2019)
  • Evaluation of the effect of SC411 compared to placebo by measuring the time until the patient's first sickle cell event. [ Time Frame: 52 weeks ]
    Evaluation of days to the first event from randomization for each patient.
  • Evaluation of the effect of SC411 compared to placebo by measuring the the number of visits to a medical facility (hospital, clinic, or emergency room) for SCC event or complications of SCD. [ Time Frame: 52 weeks ]
    Evaluation of the effect of SC411 compared to placebo by measuring the the number of visits to a medical facility (hospital, clinic, or emergency room) for SCC event or complications of SCD.
  • Evaluation of the effect of SC411 compared to placebo by measuring the number of days with electronic diary (eDiary)-recorded opioid or non opioid analgesic use at home to manage sickle cell pain. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the number of days with electronic diary (eDiary)-recorded opioid or non opioid analgesic use at home to manage sickle cell pain.
  • Evaluation of the effect of SC411 compared to placebo by measuring the number of crisis-free days. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the number of crisis-free days. A crisis-free day is defined as any day with a zero entry on the eDiary pain intensity scale.
Original Secondary Outcome Measures  ICMJE
 (submitted: November 12, 2015)
  • Annualized rate of diary-recorded painful crises [ Time Frame: 52 weeks ]
  • Annualized rate of emergency room/medical facility visits [ Time Frame: 52 weeks ]
  • Annualized rate of hospitalizations for sickle cell crises [ Time Frame: 52 weeks ]
  • Cumulative number of hospitalization days for sickle cell crises [ Time Frame: 52 weeks ]
  • Cumulative number of days out of school (ie, absence) due to SCD [ Time Frame: 52 weeks ]
Current Other Pre-specified Outcome Measures
 (submitted: January 30, 2019)
  • Evaluation of the effect of SC411 compared to placebo by measuring the time to second SCC event among subjects who had experienced at least one crisis while enrolled in the study. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the time to second SCC event among subjects who had experienced at least one crisis while enrolled in the study.
  • Evaluation of the effect of SC411 compared to placebo by measuring the number of complications of SCD. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the number of complications of SCD.
  • Evaluation of the effect of SC411 compared to placebo by measuring the Parent/guardian eDiary-recorded school attendance. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the Parent/guardian eDiary-recorded school attendance.
  • Evaluation of the effect of SC411 compared to placebo by measuring the percent of days free of eDiary-recorded sickle cell pain out of total number of Treatment Period days. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the percent of days free of eDiary-recorded sickle cell pain out of total number of Treatment Period days.
  • Evaluation of the effect of SC411 compared to placebo by measuring the subjects eDiary-recorded sickle cell pain score on the days that pain is recorded analyzed over time for intensity and diminution. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the subjects eDiary-recorded sickle cell pain score (scale from 0-10) on the days that pain is recorded analyzed over time for intensity and diminution.
  • Evaluation of the effect of SC411 compared to placebo by measuring the subjects eDiary-recorded sickle cell pain score on the days during a SCC event analyzed over time for intensity and diminution. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the subjects eDiary-recorded sickle cell pain score (from 0-10) on the days during a SCC event analyzed over time for intensity and diminution.
  • Evaluation of the effect of SC411 compared to placebo by measuring the number of hospitalization days due to SCC events. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the number of hospitalization days due to SCC events. -
  • Evaluation of the effect of SC411 compared to placebo by measuring the number of hospitalizations due to SCC events. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the number of hospitalizations due to SCC events.
  • Evaluation of the effect of SC411 compared to placebo by measuring the number of acute painful crisis events. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the number of acute painful crisis events.
  • Evaluation of the effect of SC411 compared to placebo by measuring the number of acute chest syndrome events. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the number of acute chest syndrome events.
  • Evaluation of the effect of SC411 compared to placebo by measuring the number of simple and exchange blood transfusions. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the number of simple and exchange blood transfusions.
  • Evaluation of the effect of SC411 compared to placebo by measuring the changes in hemoglobin. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the changes in hemoglobin.
  • Evaluation of the effect of SC411 compared to placebo by measuring the changes in hematocrit. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the changes in hematocrit.
  • Evaluation of the effect of SC411 compared to placebo by measuring the changes in hemoglobin phenotype. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the changes in hemoglobin phenotype.
  • Evaluation of the effect of SC411 compared to placebo by measuring the changes in white blood cells. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the changes in white blood cells.
  • Evaluation of the effect of SC411 compared to placebo by measuring the changes in blood platelets. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the changes in blood platelets.
  • Evaluation of the effect of SC411 compared to placebo by measuring the changes in the RBC membrane omega-3 fatty acids index. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the changes in the RBC membrane omega-3 fatty acids index (arachidonic acid, docosahexaenoic acid, and eicosapentaenoic acid).
  • Evaluation of the effect of SC411 compared to placebo by measuring the changes in the reticulocyte count. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the changes in the reticulocyte count.
  • Evaluation of the effect of SC411 compared to placebo by measuring the changes in lactate dehydrogenase. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the changes in lactate dehydrogenase.
  • Evaluation of the effect of SC411 compared to placebo by measuring the changes in haptoglobin. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the changes in haptoglobin.
  • Evaluation of the effect of SC411 compared to placebo by measuring the changes in indirect bilirubin. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the changes in indirect bilirubin.
  • Evaluation of the effect of SC411 compared to placebo by measuring the changes in high sensitivity C-reactive protein. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the changes in high sensitivity C-reactive protein.
  • Evaluation of the effect of SC411 compared to placebo by measuring the changes in D-dimer. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the changes in D-dimer.
  • Evaluation of the effect of SC411 compared to placebo by measuring the changes in whole blood adhesion to vascular cell adhesion molecule-1. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the changes in whole blood adhesion to vascular cell adhesion molecule-1.
  • Evaluation of the effect of SC411 compared to placebo by measuring the changes in E-selectin. [ Time Frame: 52 weeks ]
    To evaluate the effect of SC411 compared to placebo by measuring the changes in E-selectin.
Original Other Pre-specified Outcome Measures
 (submitted: November 12, 2015)
  • Intensity of diary-recorded painful crises [ Time Frame: 52 weeks ]
  • Frequency of analgesic use at home [ Time Frame: 52 weeks ]
  • Time to first and second sickle cell disease crisis [ Time Frame: 52 weeks ]
  • Hematological parameters [ Time Frame: 52 weeks ]
    Hemoglobin , hematocrit, reticulocyte count, dense Red Blood Cells, Serum lactate dehydrogenase , and indirect bilirubin
 
Descriptive Information
Brief Title  ICMJE A Sickle CEll Disease ComplicatioN Trial
Official Title  ICMJE A Phase 3, Prospective, Randomized, Double-Blind, Placebo Controlled, Multi-center Study of SC411 for Sickle Cell Disease
Brief Summary The objective of this study is to assess the efficacy of SC411 in reducing the number of sickle cell crisis (SCC) events in sickle cell disease (SCD) subjects receiving SC411 compared to those subjects receiving placebo.
Detailed Description

This Phase 3, prospective, randomized, double-blinded, placebo-controlled, multi-center study will enroll approximately 210 subjects at up to 70 sites in the United States. Participation will consist of a Screening Period, followed by a minimum 12-month Treatment Period. SC411 is administered orally as a soft gel mini capsule.

This study will enroll subjects aged ≥5 to ≤17 years who have a diagnosis of SCD that includes the phenotypes hemoglobin SS homozygous (HbSS), hemoglobin SC (HbSC), and hemoglobin S/β°-thalassemia (HbS/ β°-thalassemia); and have had at least 2 but no more than 10 documented SCC events (as defined above) within 12 months prior to the Screening Visit.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Sickle Cell Disease
Intervention  ICMJE
  • Drug: SC411
    Soft gelatin capsule
    Other Name: Docosahexaenoic acid (DHA)
  • Drug: Placebo
    Soft gelatin capsule
    Other Name: Soybean Oil
Study Arms  ICMJE
  • Experimental: SC411
    Omega-3 docosahexaenoic acid, soft gelatin capsule, administered once a day on a per weight basis.
    Intervention: Drug: SC411
  • Placebo Comparator: Placebo
    Soybean oil, soft gelatin capsule, administered once a day on a per weight basis.
    Intervention: Drug: Placebo
Publications * Not Provided

*   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 Unknown status
Estimated Enrollment  ICMJE
 (submitted: January 30, 2019)		 210
Original Estimated Enrollment  ICMJE
 (submitted: November 12, 2015)		 162
Estimated Study Completion Date  ICMJE December 2020
Estimated Primary Completion Date December 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

Patients who meet all of the following criteria will be eligible to participate in the study:

  1. 1. Aged ≥ 5 years and ≤ 17 years at screening;
  2. Has been diagnosed with SCD (that includes the genotypes HbSS, HbSC, and HbS/β°-thalassemia, documented by hemoglobin HPLC or electrophoresis);
  3. Has had between ≥ 2 to ≤10 episodes of acute SCC (as defined above) within 12 months of the Screening Visit. At least one crisis must have been managed in a hospital, clinic, or emergency room. For at least 2 of the episodes, the site must obtain the documentation created in a medical record at the time of the event.
  4. Must not be receiving HU or L-Glutamine, or if receiving HU and/or L-Glutamine must be at a stable weight-based treatment regimen (mg/kg), for at least 3 months prior to the Screening Visit with the intent to continue at a weight-based dose level at the discretion of the treating physician for the duration of the study, other than for safety reasons. If taking HU and/or L-glutamine, subjects must have had at least one SCC event (as previously defined) while on HU and/or L-Glutamine.
  5. Has a parent or guardian who is able to give written informed consent, and the potential pediatric subject must be able to provide assent in a manner approved by the Institutional Review Board (IRB) and comply with the requirements of the study; and
  6. If post pubertal in the opinion of the Investigator, must agree to use a reliable method of birth control (e.g., barrier, birth control pills, abstinence) during the study and for 1 month following the last dose of study drug.

Exclusion Criteria

Patients who meet any of the following criteria will be excluded from participation in the study:

  1. A known allergy or hypersensitivity to fish or shellfish;
  2. A known allergy or hypersensitivity to soy;
  3. Inability to swallow capsules;
  4. History of treatment with SC411;
  5. Confirmed diagnosis of chronic pain or chronic opioid use: Defined as pain experienced ≥3 days per week over a 6-month period or daily opioid use for pain management;
  6. Active infection with Human Immunodeficiency Virus (HIV), Hepatitis B, or Hepatitis C;
  7. Prothrombin time > 1.5 x ULN at screening;
  8. Required regular anticoagulation or chronic aspirin therapy;
  9. Moderate thrombocytopenia, defined as platelets < 80,000/µL at screening;
  10. History of stroke or Moyamoya syndrome;
  11. Abnormal results on most recent transcranial Doppler (TCD) evaluation;
  12. Received blood transfusion or blood products in the 2 months prior to the Screening Visit or on chronic blood transfusion;
  13. Chronic renal insufficiency, defined as a eGFR < 30ml/min at screening as estimated by the Schwartz equation (Appendix H), or requiring peritoneal or hemodialysis;
  14. Abnormal liver function tests (ALT > 3.0 x ULN) at screening;
  15. Received any organ transplant;
  16. Has a recent acute illness or other concomitant chronic medical or psychiatric condition that in the opinion of the Investigator would compromise participation in the study, prevent adherence to the protocol or confound the evaluation of the study outcome;
  17. Is pregnant or lactating, or has the intention of becoming pregnant during the study (if a female of child-bearing potential or partner of a female of child-bearing potential and sexually active and not willing to use an effective means of birth control);
  18. Is currently taking or has been treated with any form of omega-3 fatty acid or fish oil supplement within 30 days of the Screening Visit, or intends to do so during the course of the study;
  19. Has been treated with any investigational product within 30 days of the Screening Visit or intends to receive an investigational product during the course of this study; and
  20. There are factors that, in the judgment of the Investigator, would make it difficult for the patient to comply with the requirements of the study
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 5 Years to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02604368
Other Study ID Numbers  ICMJE OMEG-411-01
Has Data Monitoring Committee Yes
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 Not Provided
Current Responsible Party Micelle BioPharma Inc
Original Responsible Party Sancilio and Company, Inc.
Current Study Sponsor  ICMJE Micelle BioPharma Inc
Original Study Sponsor  ICMJE Sancilio and Company, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Chair: Carton Dampier, MD Emory University
Study Chair: Matt Heeney, MD Harvard University
Study Chair: Beng Fuh, MD East Carolina University
PRS Account Micelle BioPharma Inc
Verification Date January 2019

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