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

NCT02973360 | Unknown Phase 2 DMC FDA Drug

• 1 other identifier: OMEG-411-02
• Study Type: interventional
• Target: 68
• Locations: 1 country
• Sites: 11

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.

Conditions

Sickle Cell Disease

Outcome Measures

Primary Outcomes (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.

  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.

  Week 0 (baseline) through Month 52 (end of treatment)

Secondary Outcomes (4)

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

  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.

  2 weeks before baseline (screening) through Week 52

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

  2 weeks before baseline (screening) through Week 52

• 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.

  Week 0 (baseline) through Month 52 (end of treatment)

Other Outcomes (5)

• 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.

  Week 0 (baseline) through Week 8 plus one day (end of treatment on Part A)

• 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.

  Week 0 (baseline) through Week 8 plus one day (end of treatment on Part A)

• 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.

  Week 0 (baseline) through Week 8 plus one day (end of treatment on Part A)

Study Arms (4)

Dose Level 1

EXPERIMENTAL

Target dose 20 mg/kg Docosahexaenoic acid

Drug: Docosahexaenoic Acid

Dose Level 2

EXPERIMENTAL

Target dose 36 mg/kg Docosahexaenoic acid

Drug: Docosahexaenoic Acid

Dose Level 3

EXPERIMENTAL

Target dose 60 mg/kg Docosahexaenoic acid

Drug: Docosahexaenoic Acid

Placebo

PLACEBO COMPARATOR

Soybean oil

Drug: Placebo

Interventions

Docosahexaenoic Acid DRUG

Oral Capsule

Also known as: SC411

Dose Level 1; Dose Level 2; Dose Level 3

Placebo DRUG

Oral 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:

• Aged greater than or equal to 5 years and less than or equal to 17 years at screening;
• 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;
• 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);
• 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;
• 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
• 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.

You may not qualify if:

• Has a significant medical condition that required hospitalization (other than sickle cell crisis) within two months of the Screening Visit;
• Has a known allergy or hypersensitivity to fish or shellfish;
• Has a known allergy or hypersensitivity to soy;
• Is planning to initiate, terminate, or alter the dosing of hydroxyurea during the first 12 weeks of the study, other than for safety reasons;
• Has chronic daily use (more than 30 consecutive days during the last six months prior to enrollment) of opioid analgesia for any reason;
• Has a diagnosis of chronic pain or chronic pain syndrome (eg, chronic pain from the repeated vaso-occlusive events, chronic pain from avascular necrosis);
• Has a history of Human Immunodeficiency Virus (HIV), Hepatitis B, or Hepatitis C infection;
• Has a history of documented episode(s) of priapism within 12 months of the Screening Visit;
• Has a history of atrial or ventricular arrhythmia;
• Has an international normalized ratio (INR) \>2.0, or is on regular anticoagulation therapy, or has a history of a known bleeding diathesis;
• Has thrombocytopenia (platelets less than 80,000) or is on chronic acetylsalicylic acid therapy;
• 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;
• Has received a blood transfusion or exchange transfusion in the three months prior to the Screening Visit;

Sponsors & Collaborators

• Micelle BioPharma Inc: lead

Study Sites (11)

Children's of Alabama - University of Alabama

Birmingham, Alabama, 35233, United States

Location

UCSF Benioff Children's Hospital Oakland

Oakland, California, 94609, United States

Location

University of Florida Health at Shands

Gainesville, Florida, 32610, United States

Location

Batchelor Children's Research Institute - University of Miami

Miami, Florida, 33136, United States

Location

Children's Healthcare of Atlanta - Emory University

Atlanta, Georgia, 30322, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Children's Hospital of Michigan

Detroit, Michigan, 48201, United States

Location

University of Mississippi Medical Center

Jackson, Mississippi, 39216, United States

Location

East Carolina University

Greenville, North Carolina, 27834, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Texas Children's Hospital - Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Related Publications (1)

• Daak AA, Dampier CD, Fuh B, Kanter J, Alvarez OA, Black LV, McNaull MA, Callaghan MU, George A, Neumayr L, Hilliard LM, Sancilio F, Rabinowicz AL, Heeney MM. Double-blind, randomized, multicenter phase 2 study of SC411 in children with sickle cell disease (SCOT trial). Blood Adv. 2018 Aug 14;2(15):1969-1979. doi: 10.1182/bloodadvances.2018021444.

  PMID: 30097463 RESULT

Related Links

• EFFECTS OF SC411 ON BLOOD CELL MEMBRANE OMEGA-3 INDEX AND SELECT SICKLE CELL DISEASE BIOMARKERS IN THE SCOT TRIAL: A PHASE 2 RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP, MULTI-CENTER STUDY
• CLINICAL EFFECT OF SC411 ON CHILDREN WITH SICKLE CELL DISEASE IN THE SCOT TRIAL: A PHASE 2 RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP, DOSE-FINDING MULTI-CENTER STUDY

MeSH Terms

Conditions

Anemia, Sickle Cell

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

• Matthew Heeney, MD

  Boston Children's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• 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 22, 2016
• First Posted: November 25, 2016
• Study Start: March 2, 2017
• Primary Completion: September 26, 2017
• Study Completion: January 2, 2022
• Last Updated: January 31, 2019

Record last verified: 2019-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (11)