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Omega-3 Fatty Acids in Sickle Cell Disease

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: NCT02947100
Recruitment Status : Terminated (manufacturing problem with study drug)
First Posted : October 27, 2016
Last Update Posted : October 18, 2018
Sponsor:
Collaborators:
National Institute of General Medical Sciences (NIGMS)
Thomas Jefferson University
Solutex GC, S.L.
Information provided by (Responsible Party):
Robin E. Miller, Nemours Children's Clinic

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.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: SCD-Omegatex™ Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 4 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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)
Actual Study Start Date : January 25, 2018
Actual Primary Completion Date : October 5, 2018
Actual Study Completion Date : October 15, 2018

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: SCD-Omegatex™
single arm
Drug: SCD-Omegatex™
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.
Other Name: Enteric Fish Oil 250 DHA/27 EPA Soft Gelatin Capsule




Primary Outcome Measures :
  1. Clinical safety, in a dose escalation trial of SCD-Omegatex™ as evidenced by an absence of adverse events. [ Time Frame: 6 months with continuous monitoring ]
  2. Determine whether 6 months of supplementation with SCD-Omegatex™ will reduce thermal sensitivity by Quantitative Sensory Testing to below pre-treatment levels [ Time Frame: 6 months ]

Secondary Outcome Measures :
  1. Health-associated Quality of Life [ Time Frame: 6 months ]
  2. Number of days with pain measured by iPad daily report pain calendar [ Time Frame: 8 months ]
  3. Changes in individual thermal sensitivity thresholds by QST [ Time Frame: 8 months ]

Other Outcome Measures:
  1. Thrombin generation as assessed by Calibrated Automated Thrombogram [ Time Frame: 6 months ]
  2. high sensitivity C-reactive protein (mg/L) [ Time Frame: 6 months ]
  3. plasma lipidomic analysis [ Time Frame: 6 months ]
  4. urine Resolvin D1 (pg/mg creatinine) [ Time Frame: 6 months ]
  5. plasma levels of lactate dehydrogenase (IU/L) [ Time Frame: 6 months ]
  6. fetal hemoglobin (%) [ Time Frame: 6 months ]
  7. analysis of pro and anti-inflammatory cytokines in plasma including interleukin (IL)1-beta, IL-4, IL-6, IL-8, IL-10 and tumor necrosis factor (TNF) alpha ( pg/ml) [ Time Frame: 6 months ]
  8. plasma Endothelin-1(pg/ml) [ Time Frame: 6 months ]
  9. plasma levels soluble vascular adhesion molecule -1 (VCAM-1) (ng/ml) [ Time Frame: 6 months ]
  10. plasma levels of soluble P-selectin (ng/ml) [ Time Frame: 6 months ]
  11. plasma levels of soluble L-selectin (ng/ml) [ Time Frame: 6 months ]
  12. hemostatic markers in plasma including d-dimers and prothrombin fragment 1.2 (nmol/L) [ Time Frame: 6 months ]


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Ages Eligible for Study:   8 Years to 25 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

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): NCT02947100


Locations
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United States, Delaware
Nemours/Alfred I duPont Hospital for Children
Wilmington, Delaware, United States, 19899
Sponsors and Collaborators
Robin E. Miller
National Institute of General Medical Sciences (NIGMS)
Thomas Jefferson University
Solutex GC, S.L.
Investigators
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Principal Investigator: Robin E Miller, MD Nemours Children's Clinic

Publications:
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Responsible Party: Robin E. Miller, Physician, Nemours Children's Clinic
ClinicalTrials.gov Identifier: NCT02947100     History of Changes
Other Study ID Numbers: RM002
P20GM109021 ( U.S. NIH Grant/Contract )
First Posted: October 27, 2016    Key Record Dates
Last Update Posted: October 18, 2018
Last Verified: October 2018
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
Keywords provided by Robin E. Miller, Nemours Children's Clinic:
Sickle Cell Disease
omega-3 fatty acids
Docosahexaenoic Acid (DHA)
Eicosapentaenoic Acid (EPA)
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn