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Dose-Finding Study of SC411 in Children With Sickle Cell Disease (SCOT)

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: NCT02973360
Recruitment Status : Active, not recruiting
First Posted : November 25, 2016
Last Update Posted : January 31, 2019
Sponsor:
Information provided by (Responsible Party):
Micelle BioPharma Inc

Brief Summary:
This is a Phase 2, randomized, double-blind, placebo-controlled, parallel-group, dose-finding study of SC411 in children with sickle cell disease (SCD). The primary objective of the study is to evaluate the safety and tolerability of three different doses of SC411 compared to a placebo. All patients will undergo eight weeks of oral study treatment and a four-week safety follow-up period. Patients will be randomized to one of three dose levels of SC411 or placebo.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: Docosahexaenoic Acid Drug: Placebo Phase 2

Detailed Description:
This Phase 2 study is to be conducted in two parts. Part A is a randomized, double-blind, placebo-controlled, parallel-group, dose-finding study of SC411 in children with SCD. This part of the study will consist of a screening period of up to 2 weeks, followed by an eight-week treatment period. Part B is an optional 49-month open-label extension for patients who have completed Part A.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 68 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Finding Study and Open-Label Extension of SC411 in Children With Sickle Cell Disease
Actual Study Start Date : March 2, 2017
Actual Primary Completion Date : September 26, 2017
Estimated Study Completion Date : January 2, 2022

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Dose Level 1
Target dose 20 mg/kg Docosahexaenoic acid
Drug: Docosahexaenoic Acid
Oral Capsule
Other Name: SC411

Experimental: Dose Level 2
Target dose 36 mg/kg Docosahexaenoic acid
Drug: Docosahexaenoic Acid
Oral Capsule
Other Name: SC411

Experimental: Dose Level 3
Target dose 60 mg/kg Docosahexaenoic acid
Drug: Docosahexaenoic Acid
Oral Capsule
Other Name: SC411

Placebo Comparator: Placebo
Soybean oil
Drug: Placebo
Oral Capsule
Other Name: Soybean oil




Primary Outcome Measures :
  1. Evaluate safety & tolerability of SC411 & determine change from Baseline in the blood cells omega-3 fatty acids index in subjects, treated with active SC411 doses or placebo in Part A and safety and tolerability in Part B. [ Time Frame: Week 0 (baseline) through Month 52 (end of treatment) ]
    Evaluate the safety and tolerability of three different doses of SC411 and determine the change from Baseline in the blood cells omega-3 fatty acids index in subjects, treated with either one of the three active doses of SC411 or matching placebo in Part A. Safety and tolerability will continue to be evaluated and reported during Part B.


Secondary Outcome Measures :
  1. Measurement of the pharmacokinetic (PK) parameter of AUC will be performed for three dose levels of SC411 will be measured. [ Time Frame: 2 weeks before baseline (screening) through Week 52 ]

    Measurement of the pharmacokinetic (PK) parameter of AUC will be performed for three dose levels of SC411 will be measured.

    The steady-state pharmacokinetic (PK) profile of the three doses and area under the curve will be reported. The purpose is to assess the steady-state pharmacokinetic (PK) profile of the three dose levels of SC411, following two months of dosing, and at the end of treatment (EOT).


  2. Measurement of the pharmacokinetic (PK) parameter of Tmax will be performed for three dose levels of SC411 will be measured. [ Time Frame: 2 weeks before baseline (screening) through Week 52 ]

    Measurement of the pharmacokinetic (PK) parameter of Tmax will be performed for three dose levels of SC411 will be measured.

    The steady-state pharmacokinetic (PK) profile of the three doses and Tmax will be reported. The purpose is to assess the steady-state pharmacokinetic (PK) profile of the three dose levels of SC411, following two months of dosing, and at the end of treatment (EOT).


  3. Measurement of the pharmacokinetic (PK) parameter of Cmax will be performed for three dose levels of SC411 will be measured. [ Time Frame: 2 weeks before baseline (screening) through Week 52 ]
    The steady-state pharmacokinetic (PK) profile of the three doses and Cmax will be reported. The purpose is to assess the steady-state pharmacokinetic (PK) profile of the three dose levels of SC411, following two months of dosing, and at the end of treatment (EOT).

  4. The safety & long term tolerability of SC411 will be evaluated in Part A, & a selected dose in Part B. The change from Baseline in blood cells omega-3 fatty acids index in subjects, treated with SC411 or placebo in Part A will be determined. [ Time Frame: Week 0 (baseline) through Month 52 (end of treatment) ]
    Evaluate the safety and long term tolerability of three different doses of SC411 and determine the change from Baseline in the blood cells omega-3 fatty acids index in subjects, treated with either one of the three active doses of SC411 or matching placebo in Part A. Safety and long term tolerability will continue to be evaluated and reported during Part B.


Other Outcome Measures:
  1. The number of painful crises had by subjects receiving one of the three oral dose levels of SC411 compared to matching placebo will be evaluated from eDiary-records of patient reported outcomes. [ Time Frame: Week 0 (baseline) through Week 8 plus one day (end of treatment on Part A) ]
    The exploratory objective of this study are to evaluate the effect of the three oral dose levels of SC411 compared to matching placebo from eDiary-records of patient reported outcomes of painful crises. The number of painful crises will be reported by the patient and evaluated based on SC411 compared to matching placebo.

  2. The intensity of painful crises on a scale of 0 to 10, reported by subjects receiving one of the three oral dose levels of SC411 compared to matching placebo will be evaluated from eDiary-records of patient reported outcomes. [ Time Frame: Week 0 (baseline) through Week 8 plus one day (end of treatment on Part A) ]
    The exploratory objective of this study are to evaluate the effect of the three oral dose levels of SC411 compared to matching placebo from eDiary-records of patient reported outcomes of intensity of painful crises on a scale of 0 to 10, reported by the patient. Painful crises will be reported by the patient and evaluated based on level of intensity.

  3. The number of times the subject reports taking an analgesic at home by subjects receiving one of the three oral dose levels of SC411 compared to matching placebo will be evaluated from eDiary-records of patient reported outcomes. [ Time Frame: Week 0 (baseline) through Week 8 plus one day (end of treatment on Part A) ]
    The exploratory objective of this study are to evaluate the effect of the three oral dose levels of SC411 compared to matching placebo from eDiary-records of patient reported outcomes of the number of times the patient reports taking an analgesic at home. The number of times the patient reports taking an analgesic at home will be reported by the patient and evaluated.

  4. The number of school days missed because of pain by subjects receiving one of the three oral dose levels of SC411 compared to matching placebo will be evaluated from eDiary-records of patient reported outcomes. [ Time Frame: Week 0 (baseline) through Week 8 plus one day (end of treatment on Part A) ]
    The exploratory objective of this study are to evaluate the effect of the three oral dose levels of SC411 compared to matching placebo from eDiary-records of patient reported outcomes of the number of times the patient reports taking an analgesic at home. The number of school days missed because of pain will be reported by the patient and evaluated.

  5. The number of blood transfusions on subjects receiving one of the three oral dose levels of SC411 compared to matching placebo will be evaluated. [ Time Frame: Week 0 (baseline) through Week 8 plus one day (end of treatment on Part A) ]
    The exploratory objectives of this study are to evaluate the effect of the three oral dose levels of SC411 compared to matching placebo on the number of blood transfusions. (acute simple blood transfusions and exchange blood transfusions).



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:

  1. Aged greater than or equal to 5 years and less than or equal to 17 years at screening;
  2. Has been diagnosed with SCD that includes the phenotypes HbSS, hemoglobin SC, and HbS/beta-thalassemia. Hemoglobin phenotyping must be previously documented by either hemoglobin high-performance liquid chromatography [HPLC] or electrophoresis at time of Screening. If a patient does not have documented hemoglobin phenotyping at the time of Screening, or has received a blood transfusion within the two months prior to the Screening Visit, hemoglobin phenotyping should be documented by hemoglobin HPLC;
  3. Has had at least two and no more than ten documented episodes of clinical sickle cell crises within 12 months prior to the Screening Visit. A sickle cell crisis is defined as an episode of vaso-occlusive event:

    • Painful crisis defined as new onset of pain that lasts two or more hours for which there is no explanation other than vaso-occlusion, and which requires therapy with oral or parenteral opioids, non-steroidal anti-inflammatory drugs, or other analgesics prescribed by a healthcare provider in a medical setting such as a hospital, clinic, or emergency room visit, or documented telephone management (Ballas, 2010; Heeney, 2016; Jacob, 2005); and
    • Acute chest syndrome defined as acute illness characterized by a new segmental pulmonary infiltrate on a chest x-ray, and fever (greater than or equal to 38.5°C) or respiratory symptoms such as hypoxia, chest pain, tachypnea, wheezing, or cough (Ballas, 2010);
  4. Is either not on hydroxyurea at the Screening Visit and does not plan on receiving it during the course of the first 12 weeks of the study (Part A), or has received hydroxyurea for a minimum of 6 months and, except for safety reasons, will remain on the same weight-based dose of hydroxyurea from screening throughout the duration of Part A of the study;
  5. Parent or guardian is able to give written informed consent, and the potential pediatric patient is able to provide assent in a manner approved by the Institutional Review Board (IRB) and comply with the requirements of the study other than for safety reasons; and
  6. If sexually active, agrees to use a reliable method of birth control (eg, barrier, birth control pills, abstinence) during the study and for one month following the last dose of study drug.

Exclusion Criteria:

  1. Has a significant medical condition that required hospitalization (other than sickle cell crisis) within two months of the Screening Visit;
  2. Has a known allergy or hypersensitivity to fish or shellfish;
  3. Has a known allergy or hypersensitivity to soy;
  4. Is planning to initiate, terminate, or alter the dosing of hydroxyurea during the first 12 weeks of the study, other than for safety reasons;
  5. Has chronic daily use (more than 30 consecutive days during the last six months prior to enrollment) of opioid analgesia for any reason;
  6. Has a diagnosis of chronic pain or chronic pain syndrome (eg, chronic pain from the repeated vaso-occlusive events, chronic pain from avascular necrosis);
  7. Has a history of Human Immunodeficiency Virus (HIV), Hepatitis B, or Hepatitis C infection;
  8. Has a history of documented episode(s) of priapism within 12 months of the Screening Visit;
  9. Has a history of atrial or ventricular arrhythmia;
  10. Has an international normalized ratio (INR) >2.0, or is on regular anticoagulation therapy, or has a history of a known bleeding diathesis;
  11. Has thrombocytopenia (platelets less than 80,000) or is on chronic acetylsalicylic acid therapy;
  12. Has increased risk of stroke: documented abnormal or "high conditional" transcranial Doppler (TCD) mean velocity (TCD V) by STOP criteria (Adams, 1998) within the preceding year or has a history of known cerebrovascular disease:

    • "High conditional" = TCD V greater than or equal to 185 to 199 cm/sec, or TCDi V greater than or equal to 170 to 184 cm/sec, or TCD maximum V greater than or equal to 250 cm/sec; or
    • Abnormal = TCD V greater than or equal to 200 cm/sec, or abnormal high TCDi V greater than or equal to 185 cm/sec, or TCD maximum V greater than or equal to 250 cm/sec;
  13. Has received a blood transfusion or exchange transfusion in the three months prior to the Screening Visit;
  14. Has renal insufficiency (creatinine greater than 1.5 times upper limit of normal [ULN], or requiring peritoneal dialysis or hemodialysis);
  15. Has liver dysfunction (ALT greater than 2.0 times ULN);
  16. Has other concomitant chronic medical or psychiatric condition that, in the opinion of the Investigator, would compromise participation in the study 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 patient participating in the study);
  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 during the course of the study;
  19. Has been treated with an experimental anti-sickling medication/treatment within 30 days of the Screening Visit or during the course of the study;
  20. Is currently taking or has been treated with any investigational drug for any disease within 30 days of the Screening Visit or during the course of the study;
  21. Is currently enrolled in an investigational drug or device study and/or has participated in such a study within 30 days of the Screening Visit or during the course of the study; or
  22. There are factors that would, in the judgment of the Investigator, make it difficult for the patient to comply with the requirements of the study (eg, inability to swallow capsules due to past history of stroke, or poor compliance).

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): NCT02973360


Locations
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United States, Alabama
Children's of Alabama - University of Alabama
Birmingham, Alabama, United States, 35233
United States, California
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States, 94609
United States, Florida
University of Florida Health at Shands
Gainesville, Florida, United States, 32610
Batchelor Children's Research Institute - University of Miami
Miami, Florida, United States, 33136
United States, Georgia
Children's Healthcare of Atlanta - Emory University
Atlanta, Georgia, United States, 30322
United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
United States, Michigan
Children's Hospital of Michigan
Detroit, Michigan, United States, 48201
United States, Mississippi
University of Mississippi Medical Center
Jackson, Mississippi, United States, 39216
United States, North Carolina
East Carolina University
Greenville, North Carolina, United States, 27834
United States, South Carolina
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
United States, Texas
Texas Children's Hospital - Baylor College of Medicine
Houston, Texas, United States, 77030
Sponsors and Collaborators
Micelle BioPharma Inc
Investigators
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Principal Investigator: Matthew Heeney, MD Boston Children’s Hospital
Additional Information:
Publications of Results:
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Responsible Party: Micelle BioPharma Inc
ClinicalTrials.gov Identifier: NCT02973360    
Other Study ID Numbers: OMEG-411-02
First Posted: November 25, 2016    Key Record Dates
Last Update Posted: January 31, 2019
Last Verified: January 2019
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
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn