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Trial record 19 of 28 for:    sickle cell | "Sickle Cell Trait"

A Sickle CEll Disease ComplicatioN Trial (ASCENT)

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ClinicalTrials.gov Identifier: NCT02604368
Recruitment Status : Not yet recruiting
First Posted : November 13, 2015
Last Update Posted : February 1, 2019
Sponsor:
Information provided by (Responsible Party):
Micelle BioPharma Inc

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.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: SC411 Drug: Placebo Phase 3

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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 210 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 3, Prospective, Randomized, Double-Blind, Placebo Controlled, Multi-center Study of SC411 for Sickle Cell Disease
Estimated Study Start Date : March 2019
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : December 2020


Arm Intervention/treatment
Experimental: SC411
Omega-3 docosahexaenoic acid, soft gelatin capsule, administered once a day on a per weight basis.
Drug: SC411
Soft gelatin capsule
Other Name: Docosahexaenoic acid (DHA)

Placebo Comparator: Placebo
Soybean oil, soft gelatin capsule, administered once a day on a per weight basis.
Drug: Placebo
Soft gelatin capsule
Other Name: Soybean Oil




Primary Outcome Measures :
  1. 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.



Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.


Other Outcome Measures:
  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.

  7. 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.

    -


  8. 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.

  9. 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.

  10. 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.

  11. 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.

  12. 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.

  13. 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.

  14. 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.

  15. 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.

  16. 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.

  17. 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).

  18. 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.

  19. 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.

  20. 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.

  21. 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.

  22. 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.

  23. 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.

  24. 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.

  25. 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.



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.


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Ages Eligible for Study:   5 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

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): NCT02604368


Contacts
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Contact: Uche Sampson, MD 615-678-2336 usampson@micellebio.com
Contact: George Steinfels, PhD 301-742-5656 gsteinfels@micellebio.com

Sponsors and Collaborators
Micelle BioPharma Inc
Investigators
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Study Chair: Carton Dampier, MD Emory University
Study Chair: Matt Heeney, MD Harvard University
Study Chair: Beng Fuh, MD East Carolina University

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Responsible Party: Micelle BioPharma Inc
ClinicalTrials.gov Identifier: NCT02604368     History of Changes
Other Study ID Numbers: OMEG-411-01
First Posted: November 13, 2015    Key Record Dates
Last Update Posted: February 1, 2019
Last Verified: January 2019

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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 Micelle BioPharma Inc:
hemoglobin SS
hemoglobin SC
hemoglobin S/β°-thalassemia
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn