Clinical Study of Inhaled GB002 for Treatment of WHO Group I Pulmonary Arterial Hypertension

Study Purpose

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).

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Interventional
Eligible Ages	18 Years - 75 Years
Gender	All

More Inclusion & Exclusion Criteria

Inclusion Criteria:

1. Adult (males and females) aged 18 to 75 years (inclusive) with pulmonary arterial hypertension (PAH) (Main study) 2. A current diagnosis of symptomatic PAH classified by one of the following (Main and OLE study): 1. Idiopathic (IPAH) or heritable pulmonary arterial hypertension (HPAH) 2. PAH associated with one of the following connective tissue diseases (CTDs): systemic sclerosis, rheumatoid arthritis, mixed CTD or overlap syndrome, or systemic lupus erythematosus. 3. PAH associated with anorexigen or methamphetamine use. 3. World Health Organization (WHO)/New York Heart Association (NYHA) functional class II-IV symptomatology (Main study)

Exclusion Criteria:

1. Clinically significant systemic hypertension or hypotension (Main and OLE study) 2. History of left-sided heart disease and/or clinically significant cardiac disease (Main and OLE study) 3. 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.

Trial Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT03926793
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.	Phase 1
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	GB002, Inc., a wholly owned subsidiary of Gossamer Bio, Inc.
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	N/A
Principal Investigator Affiliation	N/A
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Industry
Overall Status	Recruiting
Countries	United Kingdom, United States
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	Pulmonary Arterial Hypertension

Additional Details

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.

Arms & Interventions

Arms

Experimental: Cohort 1

Patients will be randomized to receive inhaled GB002 or Placebo daily for 14 days

Experimental: Cohort 2

Patients will be randomized to receive inhaled GB002 or Placebo daily for 14 days

Experimental: Open Label Extension

Eligible subjects may participate in the Open Label Extension (OLE) study for a period of 24 weeks.

Interventions

Drug: - GB002

GB002 low dose or high dose for inhalation

Drug: - Placebo

Placebo for inhalation

Device: - Generic Dry Powder Inhaler

Generic dry powder inhaler for GB002 or Placebo delivery

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

UCSD Medical Center, La Jolla, California

Status

Completed

Address

UCSD Medical Center

La Jolla, California, 92037

Site Contact

ClinicalTrials@gossamerbio.com

1-866-668-4083

Sacramento, California

Status

Completed