CPLATFRM2104
Research type
Research Study
Full title
Biomarker study of adolescent sickle cell patients in acute vaso occlusive crisis and recovery
IRAS ID
185986
Contact name
Kenneth Huttner
Contact email
Sponsor organisation
Novartis Institutes for BioMedical Research, Inc.
Duration of Study in the UK
1 years, 0 months, 28 days
Research summary
The sponsor has a long-standing commitment to address the significant and life-long medical complications common to sickle cell disease (SCD) patients, including 15 ongoing/completed studies (www.clinicaltrials.gov). Fundamental to the severe clinical outcomes in SCD patients are the recurrence of vaso-occlusion/pain crises. The proposed study specifically targets the elucidation of biologic pathways activated during SCD crises. Through the use of cutting-edge protein, RNA and DNA analysis technologies (see details in full protocol), the primary study goal is identification of a crisis “molecular signature” that will lead to trials with pathway-specific therapeutics.
The study will enroll approximately 30 subjects, ages 12-17 years of age, in two cohorts. Cohort 1 will include 20 SCD patients admitted for acute pain crisis at one of two clinical sites (Toronto and London). Blood samples will be obtained during the acute crisis and at a convalescent timepoint. Biomarker changes will be correlated with patient-reported pain scores and associated DNA polymorphisms.
Samples are obtained at the time of clinically-indicated phlebotomy when possible. Other data collection includes standard laboratory values (when performed as routine care), demographics, medical history, adverse events, physical exams and patient vital signs. The study consists of 2 to 3 visits with a brief telephone interview at study end.
Cohort 2 will enroll approximately 10 HbSC patients at steady state. These subjects will have a single blood draw and the identical set of biomarker analyses to establish the levels of molecular markers in non-crisis patients.
Currently the sponsor has clinical-stage trials with therapeutic agents that have the potential to impact SCD pathophysiology, including trials targeting IL-1 biology (NCT01576367), cell-specific inflammation (NCT01245179, NCT01972776), and cell adhesion (NCT01895361). The proposed study is intended to provide a deeper understanding of the pathophysiology of SCD crises and will be key in taking the next steps in SCD crisis therapy.REC name
London - Stanmore Research Ethics Committee
REC reference
15/LO/1628
Date of REC Opinion
18 Nov 2015
REC opinion
Further Information Favourable Opinion