Clinical Study of Inhaled GB002 for Treatment of WHO Group I Pulmonary Arterial Hypertension
- Conditions
- Pulmonary Arterial Hypertension
- Interventions
- Registration Number
- NCT03926793
- Lead Sponsor
- GB002, Inc., a wholly owned subsidiary of Gossamer Bio, Inc.
- Brief Summary
This is a Phase 1B, randomized, participant- and investigator-blinded, placebo-controlled, multi-center clinical trial to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and biomarkers of inhaled GB002 in adults with World Health Organization (WHO) Group 1 Pulmonary Arterial Hypertension (PAH).
- Detailed Description
The primary objective for this trial is to evaluate the safety and tolerability of GB002. The secondary objective for this trial is to evaluate the PK parameters of GB002. Exploratory objectives are to evaluate the PD readouts, change in WHO Group I functional class, and change in quality of life associated with GB002 treatment.
In this Phase 1B study, two dose levels of GB002 will be tested in adult participants with PAH. Four participants in cohort 1 will be randomized to receive up to two daily doses of either active drug or placebo, with 3 subjects receiving GB002 and 1 subject receiving placebo. An additional 4 participants (3 active and 1 placebo) may be added to cohort 1 at the discretion of the Data Review Committee (DRC). The dose and dosing interval (i.e., once daily or twice daily) for the second cohort will be determined by review of the safety, tolerability, and drug levels in the blood from cohort 1. Cohort 2 participants will also be randomized such that 6 subjects receive GB002 and 2 subjects receive placebo.
Eligible subjects who have completed the 2 week treatment period have the option to participate in a 24 week open label extension.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 8
-
Adult (males and females) aged 18 to 75 years (inclusive) with pulmonary arterial hypertension (PAH) (Main study)
-
A current diagnosis of symptomatic PAH classified by one of the following (Main and OLE study):
-
Idiopathic (IPAH) or heritable pulmonary arterial hypertension (HPAH)
-
PAH associated with one of the following connective tissue diseases (CTDs):
systemic sclerosis, rheumatoid arthritis, mixed CTD or overlap syndrome, or systemic lupus erythematosus
-
PAH associated with anorexigen or methamphetamine use
-
-
World Health Organization (WHO)/New York Heart Association (NYHA) functional class II-IV symptomatology (Main study)
- Clinically significant systemic hypertension or hypotension (Main and OLE study)
- History of left-sided heart disease and/or clinically significant cardiac disease (Main and OLE study)
- History of decompensated right heart failure within 30 days of screening (e.g., hospitalization for PAH or the need to add an additional PAH medication) (Main study)
NOTE: Additional inclusion/exclusion criteria may apply, per protocol.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Cohort 2 GB002 Patients will be randomized to receive inhaled GB002 or Placebo daily for 14 days Open Label Extension Generic Dry Powder Inhaler Eligible subjects may participate in the Open Label Extension (OLE) study for a period of 24 weeks. Cohort 1 GB002 Patients will be randomized to receive inhaled GB002 or Placebo daily for 14 days Cohort 1 Generic Dry Powder Inhaler Patients will be randomized to receive inhaled GB002 or Placebo daily for 14 days Cohort 2 Placebo Patients will be randomized to receive inhaled GB002 or Placebo daily for 14 days Cohort 2 Generic Dry Powder Inhaler Patients will be randomized to receive inhaled GB002 or Placebo daily for 14 days Cohort 1 Placebo Patients will be randomized to receive inhaled GB002 or Placebo daily for 14 days Open Label Extension GB002 Eligible subjects may participate in the Open Label Extension (OLE) study for a period of 24 weeks.
- Primary Outcome Measures
Name Time Method Number of participants with Treatment-Related Adverse Events GB002 (OLE study) Up to 200 days To evaluate the long-term safety and tolerability and efficacy of GB002
Number of participants with Treatment-Related Adverse Events GB002 (Main study) Up to 45 days To evaluate the safety and tolerability of GB002
- Secondary Outcome Measures
Name Time Method Pharmacokinetics: Maximum Concentration (Cmax) of GB002 (Main study) 14 days Changes from baseline in 6-Minute Walk Test (6MWT) (OLE study) 169 days Changes from baseline in Pulmonary Arterial Hypertension - Symptoms and Impact (PAH-SYMPACT) Quality of Life questionnaire (OLE study) 197 days Pharmacokinetics: Area Under the Concentration Curve (AUC) of GB002 (Main study) 14 days Changes from baseline in N-terminal pro B-type natriuretic peptide (NT-proBNP) (OLE study) 169 days Changes from baseline in WHO Functional Class (OLE study) 197 days Pharmacokinetics: Time to Reach Maximum Concentration (Tmax) of GB002 (Main study) 14 days Changes from baseline in Right Ventricular function by imaging (OLE study) 169 days
Trial Locations
- Locations (14)
Yale New Haven Hospital
🇺🇸New Haven, Connecticut, United States
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
Brigham and Women's Hospital
🇺🇸Boston, Massachusetts, United States
VitaLink Research - Anderson
🇺🇸Anderson, South Carolina, United States
University of California Davis Medical Center
🇺🇸Sacramento, California, United States
Norton Pulmonary Specialists
🇺🇸Louisville, Kentucky, United States
Duke Early Phase Research
🇺🇸Durham, North Carolina, United States
Golden Jubilee National Hospital
🇬🇧Glasgow, United Kingdom
Hammersmith Hospital, Imperial Healthcare NHS Trust
🇬🇧London, United Kingdom
Mayo Clinic Florida
🇺🇸Jacksonville, Florida, United States
University of Colorado Hospital
🇺🇸Aurora, Colorado, United States
UCSD Medical Center
🇺🇸La Jolla, California, United States
Cleveland Clinic Foundation
🇺🇸Cleveland, Ohio, United States
University of Pittsburgh Medical Center Montefiore University Hospital
🇺🇸Pittsburgh, Pennsylvania, United States