A Safety Study of GSK3039294 in Healthy Volunteers and Patients With Systemic Amyloidosis
Status:
Terminated
Trial end date:
2017-05-10
Target enrollment:
Participant gender:
Summary
GSK3039294 has been developed in order to offer an orally available alternative to parenteral
CPHPC (GSK2315698 [metabolite of GSK3039294]) for plasma serum amyloid P component (SAP)
depletion prior to use of anti SAP monoclonal antibody (mAb) in the treatment of systemic
amyloidosis. This phase 1 study is intended to study safety, tolerability and pharmacokinetic
(PK) profile of GSK3039294 in humans. This study consists of three parts. Part A will
evaluate safety and tolerability of single doses of GSK3039294 in healthy subjects, Part B
will evaluate safety and tolerability of repeat doses of GSK3039294 in healthy subjects, and
Part C will evaluate safety and tolerability of repeat doses of GSK3039294 in subjects with
systemic amyloidosis. Part A is a single dose, open label, dose escalation study. Two cohorts
of subjects will be enrolled to provide data from 6 subjects per cohort and up to 4 different
doses (2 dose levels per cohort) of GSK3039294 will be tested. For Cohorts 1 and 2, each
subject may take part in two dosing periods. Part B is repeat dose, open label, dose
escalation study. Sufficient number of subjects will be enrolled in Cohort 3a to ensure 6
completers (Cohort 3b will be conducted if required) and GSK3039294 will be administered
repeatedly for a total of 21 days. Each subject will take part in a single study period. In
Part C a single dose level of GSK3039294 will be tested for 21 days repeat dose, in 12
subjects with systemic amyloidosis. Each subject will take part in a single study period. The
total duration for Part A is approximately 8 weeks, Part B is approximately 8-9 weeks, and
Part C is approximately 13 weeks.