Omega-3 Fatty Acids in Sickle Cell Disease

Study Overview
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Brief Summary
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The purpose of this study is to determine the safety of a new formulation of the omega-3 fatty acids Docosahexaenoic Acid (DHA) and Eicosapentaenoic Acid (EPA) and to assess whether it decreases inflammation and inflammatory pain in children and young adults with Sickle Cell Disease.

Official Title
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Phase I/II Safety and Dose Escalation Trial of the Omega-3 Fatty Acids Docosahexaenoic Acid (DHA) and Eicosapentaenoic Acid (EPA) in Children and Young Adults With Sickle Cell Disease (SCD)

Conditions
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Sickle Cell Disease

Intervention / Treatment
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* Drug: SCD-Omegatex™


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Subjects who meet all of the following criteria are eligible for enrollment into the study: Participant has signed the informed consent/assent with parent signing informed consent as age appropriate. Established diagnosis of HbSS, HbSC or HbSβo Thalassemia History of ≥1 vasoocclusive events (managed at home and/or in hospital) in preceding 12 months. Regular compliance with comprehensive care. Aged 8 years or greater and less than 26 years. At enrollment, subject should be in his/her baseline steady state and not in the midst of any acute complication due to SCD. Must be at least 2 weeks from infection or vasoocclusive crisis at time of screening labs Exclusion Criteria: Baseline hemoglobin levels <5.5 gm/dL. Inability to swallow capsules Poor compliance with previous treatment regimens. Hepatic dysfunction Renal dysfunction PT and/or PTT ≥ 20% outside of normal Allergy to fish, shell fish or soy Triglyceride levels <80mg/dL. Pregnancy. Chronic Transfusion Therapy. Transfusion within the last 30 days. Treatment with any investigational drug or regular fish oil supplementations in last 60 days. Currently receiving another investigational agent, or on such an agent with the last 60 days. Dosage changes in preceding 3 months if on hydroxyurea Diagnosed bleeding disorder or patient on concomitant anti-coagulation. Conditional or abnormal result on most recent transcranial doppler or history of stroke. Other active chronic illness that could adversely affect subjects performance Children in Care Platelet count less than 100,000

Ages Eligible for Study
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Minimum Age: 8 Years
Maximum Age: 25 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: SCD-Omegatex™<br>single arm | Drug: SCD-Omegatex™<br>* Subjects will receive SCD-Omegatex™ (Enteric Fish Oil 250 DHA/27 EPA Soft Gelatin Capsule, 450 mg) at one of two daily doses, orally, once a day for 6 months. The trial will follow a 3+3 design using two dose levels. In the phase I portion, subjects will be treated with a dose of 25 mg/kg/day DHA and EPA. If this is tolerated without dose limiting toxicity (DLT), a subsequent cohort of patients will be treated at a dose of 37.5 mg/kg/day with a maximum total daily dose of 4 grams. Once a maximum tolerated dose (MTD) is determined, subjects on the phase II portion of the study will be treated at that dose.<br>* Other names: Enteric Fish Oil 250 DHA/27 EPA Soft Gelatin Capsule;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Clinical Safety, in a Dose Escalation Trial of SCD-Omegatex™ as Evidenced by an Absence of Adverse Events. | No results are available from any outcome measures as study was stopped early due to manufacturing issues with study drug and will not be reopened. | 6 months with continuous monitoring | 
| Determine Whether 6 Months of Supplementation With SCD-Omegatex™ Will Reduce Thermal Sensitivity by Quantitative Sensory Testing to Below Pre-treatment Levels | No results are available from any outcome measures as study was stopped early due to manufacturing issues with study drug and will not be reopened. | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Health-associated Quality of Life | No results are available from any outcome measures as study was stopped early due to manufacturing issues with study drug and will not be reopened. | 6 months | 
| Number of Days With Pain Measured by iPad Daily Report Pain Calendar | No results are available from any outcome measures as study was stopped early due to manufacturing issues with study drug and will not be reopened. | 8 months | 
| Changes in Individual Thermal Sensitivity Thresholds by QST | No results are available from any outcome measures as study was stopped early due to manufacturing issues with study drug and will not be reopened. | 8 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Sickle Cell Disease, omega-3 fatty acids, Docosahexaenoic Acid (DHA), Eicosapentaenoic Acid (EPA)