Clinical trial NCT01202812: A Randomized Trial of LOVAZA in Pediatric Sickle Cell Disease (SCD)
Official title: Phase II Randomized Double-Blind Placebo-Controlled Trial of the Omega-3 Fatty Acids Eicosapentaenoic (EPA) and Docosahexaenoic Acid (DHA) in Pediatric Sickle Cell Disease (SCD)
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Sickle Cell Disease
HEMOGLOBIN SS
Hemoglobin S Beta-0 Thalassemia
Inflammation
Quality of Life
Keywords: Sickle Cell Anemia
Sickle Cell Disease
Hemoglobin SS Disease
Hemoglobin S beta-0 Thalassemia
Inflammation
Quality of Life
Sickle Thalassemia
C-Reative Protein
Hemolytic Anemia
Hemostasis
Biomarkers
Coagulation
Omega-3 Fatty Acids
Eicosapentaenoic Acid
Docosahexaenoic Acid
Fish Oils
Drug: Placebo
Drug: LOVAZA
Lead sponsor: Thomas Jefferson University
Sponsor class: OTHER
Collaborators: name: Drexel University
name: GlaxoSmithKline
First posted: 2010-09-16
Enrollment: 48
Sex: ALL
Minimum age (years): 10
Maximum age (years): 19
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of the study is to determine the effectiveness of LOVAZA (fish oil capsules) to decrease inflammation in children and adolescents with Sickle Cell Disease (SCD). It has been found that besides the damage caused by sickle red blood cells themselves, the inflammatory response that occurs in SCD patients could potentially play a significant role in the occurrence of painful episodes or pain crises. The investigators will also study whether the subject/caregiver feels that there is an improvement in the child's quality of life by taking the medication. Besides the effect of LOVAZA on inflammation,the investigators are also testing whether the drug will have a beneficial effect on blood clotting ability (which is known to be increased in SCD) and on the anemia (low red blood cells) that is part of the disease entity.


Eligibility 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 or HbSβo Thal.
* History of ≥3 vasocclusive pain events in preceding 12 months.
* Regular compliance with comprehensive care.
* Aged 10 years or greater and less than 20 years.
* At enrollment, subject should be in his/her steady or baseline state.

Exclusion Criteria

* Subjects with Hb levels \<5.5gm/dL.
* Inability to take or tolerate oral medications.
* Poor compliance with previous treatment regimens.
* Hepatic dysfunction (SGPT also known as ALT \>2X upper limit of normal or conjugated bilirubin \>2X the patients baseline within the last 6 weeks).
* Renal dysfunction (A creatinine level within the past 6 weeks of ≥ 1.0mg/dL for children and ≥ 1.2mg/dL for a subject ≥ 18 years of age).
* Allergy to fish or shell fish.
* Triglyceride levels \<80mg/dL.
* Pregnancy.
* Chronic Transfusion Therapy.
* Transfusion within the last 30 days.
* Persistent pain from sickle-complications (e.g. avascular necrosis).
* A vasocclusive pain episode lasting longer than 2 weeks or \>12 pain episodes in preceding year.
* Daily narcotic usage.
* 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.
* Bleeding disorder or patient on concomitant anti-coagulation.
* Conditional or abnormal TCD result or stroke.
* Other chronic illness that could adversely affect subjects performance such as HIV or TB.
* Children in Care (CiC): A child in care is a child who has been placed under the control or protection of an agency, organization, institution or entity by the courts, the government or a government body, acting in accordance with powers conferred on them by law or regulation.

Primary outcomes:
measure: To determine whether supplementation with LOVAZA will exert an anti-inflammatory effect by decreasing levels of the inflammatory biomarker high sensitivity C Reactive Protein (hsCRP) in children and adolescents with Sickle Cell Disease (SCD).
timeFrame: 6 months
Secondary outcomes:
measure: To determine whether supplementation with LOVAZA will increase health-associated quality of life (QoL) responses as they relate to clinical vasocclusive events (VOC) in children and adolescents with Sickle Cell Disease (SCD).
timeFrame: 6 months
Other outcomes:
Locations:
city: Philadelphia
country: United States
facility: Thomas Jefferson University Hospital
state: Pennsylvania
city: Philadelphia
country: United States
facility: St. Christopher's Hospital for Children, Drexel University
state: Pennsylvania
Study officials:
affiliation: Thomas Jefferson University
name: Marie Stuart, M.D.
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: Krishnan S, Setty Y, Betal SG, Vijender V, Rao K, Dampier C, Stuart M. Increased levels of the inflammatory biomarker C-reactive protein at baseline are associated with childhood sickle cell vasocclusive crises. Br J Haematol. 2010 Mar;148(5):797-804. doi: 10.1111/j.1365-2141.2009.08013.x. Epub 2009 Dec 8.
citation: Dampier C, Lieff S, LeBeau P, Rhee S, McMurray M, Rogers Z, Smith-Whitley K, Wang W; Comprehensive Sickle Cell Centers (CSCC) Clinical Trial Consortium (CTC). Health-related quality of life in children with sickle cell disease: a report from the Comprehensive Sickle Cell Centers Clinical Trial Consortium. Pediatr Blood Cancer. 2010 Sep;55(3):485-94. doi: 10.1002/pbc.22497.