Omega-3 Fatty Acids in Sickle Cell Disease

NCT02947100 | Terminated Phase 1 Results FDA Drug

• 2 other identifiers: RM002P20GM109021
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Sickle Cell Disease

Keywords

Sickle Cell Disease; omega-3 fatty acids; Docosahexaenoic Acid (DHA); Eicosapentaenoic Acid (EPA)

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (3)

• Health-associated Quality of Life

  6 months

• Number of Days With Pain Measured by iPad Daily Report Pain Calendar

  8 months

• Changes in Individual Thermal Sensitivity Thresholds by QST

  8 months

Other Outcomes (12)

• Thrombin Generation as Assessed by Calibrated Automated Thrombogram

  6 months

• High Sensitivity C-reactive Protein (mg/L)

  6 months

• Plasma Lipidomic Analysis

  6 months

Study Arms (1)

SCD-Omegatex™

EXPERIMENTAL

single arm

Drug: SCD-Omegatex™

Interventions

SCD-Omegatex™ DRUG

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.

Also known as: Enteric Fish Oil 250 DHA/27 EPA Soft Gelatin Capsule

SCD-Omegatex™

Eligibility Criteria

• Age: 8 Years - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Robin E. Miller: lead
• National Institute of General Medical Sciences (NIGMS): collaborator
• Thomas Jefferson University: collaborator
• Solutex GC S.L.: collaborator

Study Sites (1)

Nemours/Alfred I duPont Hospital for Children

Wilmington, Delaware, 19899, United States

Location

Related Publications (4)

• Daak AA, Ghebremeskel K, Hassan Z, Attallah B, Azan HH, Elbashir MI, Crawford M. Effect of omega-3 (n-3) fatty acid supplementation in patients with sickle cell anemia: randomized, double-blind, placebo-controlled trial. Am J Clin Nutr. 2013 Jan;97(1):37-44. doi: 10.3945/ajcn.112.036319. Epub 2012 Nov 28.

  PMID: 23193009 BACKGROUND

• Serhan CN. Resolution phase of inflammation: novel endogenous anti-inflammatory and proresolving lipid mediators and pathways. Annu Rev Immunol. 2007;25:101-37. doi: 10.1146/annurev.immunol.25.022106.141647.

  PMID: 17090225 BACKGROUND

• Calder PC. Marine omega-3 fatty acids and inflammatory processes: Effects, mechanisms and clinical relevance. Biochim Biophys Acta. 2015 Apr;1851(4):469-84. doi: 10.1016/j.bbalip.2014.08.010. Epub 2014 Aug 20.

  PMID: 25149823 BACKGROUND

• Tomer A, Kasey S, Connor WE, Clark S, Harker LA, Eckman JR. Reduction of pain episodes and prothrombotic activity in sickle cell disease by dietary n-3 fatty acids. Thromb Haemost. 2001 Jun;85(6):966-74.

  PMID: 11434703 BACKGROUND

MeSH Terms

Conditions

Anemia, Sickle Cell

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

Results Point of Contact

• Title: Dr Robin Miller
• Organization: Nemours/AI duPont Hospital

rmiller@nemours.org

302-651-5500

Study Officials

• Robin E Miller, MD

  Nemours Children's Clinic

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Physician

Study Record Dates

• First Submitted: October 18, 2016
• First Posted: October 27, 2016
• Study Start: January 25, 2018
• Primary Completion: October 5, 2018
• Study Completion: October 15, 2018
• Last Updated: October 19, 2020
• Results First Posted: October 19, 2020

Record last verified: 2020-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)