NCT02603172

A Safety Study of GSK3039294 in Healthy Volunteers and Patients With Systemic Amyloidosis

A Three-part Open-label, Non-randomised, Dose-escalation Study to Investigate the Safety and Tolerability of GSK3039294 Administered as a Single Dose to Healthy Volunteers, and as Repeat Dose to Healthy Volunteers and Patients With Systemic Amyloidosis

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 23 (actual)
• Sponsor: GlaxoSmithKline · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Accepted

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.

Conditions

• Amyloidosis

Interventions

Type	Name	Description
DRUG	GSK3039294	GSK3039294 will be provided as white, opaque capsules. A single capsule or multiple capsules (20 mg to 200 mg), depending on the dosage required, will be taken orally with water.

Timeline

• Start date: 2016-05-12
• Primary completion: 2017-05-10
• Completion: 2017-05-10
• First posted: 2015-11-11
• Last updated: 2019-09-23
• Results posted: 2019-09-23

Locations

1 site across 1 country: United Kingdom