Phase 1b Study of Inhaled GB002 in Pulmonary Arterial Hypertension

• Research type

  Research Study

• Full title

  A Phase 1b, Randomized, Subject- and Investigator-Blinded,\nPlacebo-Controlled, Multi-Center Clinical Trial to Evaluate the Safety,\nPharmacokinetics, Pharmacodynamics, and Biomarkers of Inhaled GB002 \nin Subjects with WHO Group 1 Pulmonary Arterial Hypertension (PAH)

• IRAS ID

  266934

• Contact name

  Luke Howard

• Contact email

  l.howard@imperial.ac.uk

• Sponsor organisation

  GB002, Inc.

• Eudract number

  2018-003093-27

• Clinicaltrials.gov Identifier

  NCT03926793

• Duration of Study in the UK

  1 years, 0 months, 0 days

• Research summary

  This is an early phase randomised, blinded, placebo-controlled, multicentre study to investigate the safety, the pharmacokinetics and pharmacodynamics of the novel compound GB002 for the treatment of pulmonary arterial hypotension (PAH). \n\nPAH is a rare disease. Despite recent treatment advances, it remains a frequently fatal condition. Therefore, more effective treatments are needed. Platelet-derived growth factor (PDGF) and its receptor (PDGFR) have been implicated in the biological causes of pulmonary hypertension. GB002 is a PDGFR inhibitor and its mode of action\nmay help improve the cardiopulmonary haemodynamics for PAH patients.\n\nA maximum of 24 subjects who have been diagnosed with a specific type of PAH (called WHO Group 1) will participate.\n\nThe randomisation ratio is 3 GB002: 1 placebo. The study has 2 cohorts: Cohort 1 - Up to 6 GB002 subjects and 2 placebo subjects; Cohort 2 - Up to 12 GB002 subjects and 4 placebo subjects. A Data Review Committee (DRC) will review the findings on an ongoing basis and determine the dose and dose frequency for Cohort 2. The DRC will also decide if/when Cohort 2 can be opened.\n\nSubjects will undergo screening evaluations within 30 days prior to their first dose. Subjects who complete screening and satisfy the inclusion/exclusion criteria will receive treatment for 14 days. The pharmaceutical form of GB002 or placebo is an inhalation powder in a hard capsule. Subjects will be trained how to prepare and take the drug using an inhaler. \n\nSubjects will be followed up post-treatment on days 15 and 42 to assess adverse events and concomitant medications. The total time on the study will be approximately 72 days.\n\nThe study will be conducted in approximately 20 specialist centres in the US and Europe. \n

• REC name

  South Central - Oxford B Research Ethics Committee

• REC reference

  19/SC/0350

• Date of REC Opinion

  16 Aug 2019

• REC opinion

  Further Information Favourable Opinion