A Sickle CEll Disease ComplicatioN Trial

NCT02604368 | Unknown Phase 3 DMC FDA Drug

• 1 other identifier: OMEG-411-01
• Study Type: interventional
• Target: 210
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Sickle Cell Disease

Keywords

hemoglobin SS; hemoglobin SC; hemoglobin S/β°-thalassemia

Outcome Measures

Primary Outcomes (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.

  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.

  52 weeks

Secondary Outcomes (4)

• Evaluation of the effect of SC411 compared to placebo by measuring the time until the patient's first sickle cell event.

  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.

  52 weeks

• 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.

  52 weeks

• Evaluation of the effect of SC411 compared to placebo by measuring the number of crisis-free days.

  52 weeks

Other Outcomes (25)

• 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.

  52 weeks

• Evaluation of the effect of SC411 compared to placebo by measuring the number of complications of SCD.

  52 weeks

• Evaluation of the effect of SC411 compared to placebo by measuring the Parent/guardian eDiary-recorded school attendance.

  52 weeks

Study Arms (2)

SC411

EXPERIMENTAL

Omega-3 docosahexaenoic acid, soft gelatin capsule, administered once a day on a per weight basis.

Drug: SC411

Placebo

PLACEBO COMPARATOR

Soybean oil, soft gelatin capsule, administered once a day on a per weight basis.

Drug: Placebo

Interventions

SC411 DRUG

Soft gelatin capsule

Also known as: Docosahexaenoic acid (DHA)

SC411

Placebo DRUG

Soft gelatin capsule

Also known as: Soybean Oil

Placebo

Eligibility Criteria

• Age: 5 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Patients who meet all of the following criteria will be eligible to participate in the study:
• \. Aged ≥ 5 years and ≤ 17 years at screening;
• Has been diagnosed with SCD (that includes the genotypes HbSS, HbSC, and HbS/β°-thalassemia, documented by hemoglobin HPLC or electrophoresis);
• 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.
• 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.
• 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
• 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.

You may not qualify if:

• Patients who meet any of the following criteria will be excluded from participation in the study:
• A known allergy or hypersensitivity to fish or shellfish;
• A known allergy or hypersensitivity to soy;
• Inability to swallow capsules;
• History of treatment with SC411;
• 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;
• Active infection with Human Immunodeficiency Virus (HIV), Hepatitis B, or Hepatitis C;
• Prothrombin time \> 1.5 x ULN at screening;
• Required regular anticoagulation or chronic aspirin therapy;
• Moderate thrombocytopenia, defined as platelets \< 80,000/µL at screening;
• History of stroke or Moyamoya syndrome;
• Abnormal results on most recent transcranial Doppler (TCD) evaluation;
• Received blood transfusion or blood products in the 2 months prior to the Screening Visit or on chronic blood transfusion;
• Chronic renal insufficiency, defined as a eGFR \< 30ml/min at screening as estimated by the Schwartz equation (Appendix H), or requiring peritoneal or hemodialysis;
• Abnormal liver function tests (ALT \> 3.0 x ULN) at screening;

Sponsors & Collaborators

• Micelle BioPharma Inc: lead

MeSH Terms

Conditions

Anemia, Sickle Cell; Hemoglobin SC Disease

Interventions

Docosahexaenoic Acids; Soybean Oil

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Fatty Acids, Omega-3; Dietary Fats, Unsaturated; Dietary Fats; Fats; Lipids; Fatty Acids, Unsaturated; Fatty Acids; Fish Oils; Oils; Fats, Unsaturated; Plant Oils; Plant Preparations; Biological Products; Complex Mixtures; Food; Diet, Food, and Nutrition; Physiological Phenomena; Food and Beverages

Study Officials

• Carton Dampier, MD

  Emory University

  STUDY CHAIR

• Matt Heeney, MD

  Harvard University

  STUDY CHAIR

• Beng Fuh, MD

  East Carolina University

  STUDY CHAIR

Central Study Contacts

Uche Sampson, MD

CONTACT

615-678-2336; usampson@micellebio.com

George Steinfels, PhD

CONTACT

301-742-5656; gsteinfels@micellebio.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 11, 2015
• First Posted: November 13, 2015
• Study Start: March 1, 2019
• Primary Completion: December 1, 2020
• Study Completion: December 1, 2020
• Last Updated: February 1, 2019

Record last verified: 2019-01