Clinical Study of Inhaled GB002 for Treatment of WHO Group I Pulmonary Arterial Hypertension (PAH)
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ClinicalTrials.gov Identifier: NCT03926793 |
Recruitment Status :
Completed
First Posted : April 25, 2019
Last Update Posted : September 1, 2021
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Condition or disease | Intervention/treatment | Phase |
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Pulmonary Arterial Hypertension | Drug: GB002 Drug: Placebo Device: Generic Dry Powder Inhaler | Phase 1 |
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.
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 8 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Triple (Participant, Care Provider, Investigator) |
Masking Description: | Clinical site investigators, study personnel, and study subjects will be blinded to treatment assignment; however, the Sponsor will be unblinded. |
Primary Purpose: | Treatment |
Official Title: | A Phase 1b, Randomized, Subject- and Investigator-Blinded, Placebo-Controlled, Multi-Center Clinical Trial to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Biomarkers of Inhaled GB002 in Subjects With WHO Group 1 Pulmonary Arterial Hypertension (PAH) |
Actual Study Start Date : | February 4, 2020 |
Actual Primary Completion Date : | May 5, 2021 |
Actual Study Completion Date : | May 5, 2021 |

Arm | Intervention/treatment |
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Experimental: Cohort 1
Patients will be randomized to receive inhaled GB002 or Placebo daily for 14 days
|
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 |
Experimental: Cohort 2
Patients will be randomized to receive inhaled GB002 or Placebo daily for 14 days
|
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 |
Experimental: Open Label Extension
Eligible subjects may participate in the Open Label Extension (OLE) study for a period of 24 weeks.
|
Drug: GB002
GB002 low dose or high dose for inhalation Device: Generic Dry Powder Inhaler Generic dry powder inhaler for GB002 or Placebo delivery |
- Number of participants with Treatment-Related Adverse Events GB002 (Main study) [ Time Frame: Up to 45 days ]To evaluate the safety and tolerability of GB002
- Number of participants with Treatment-Related Adverse Events GB002 (OLE study) [ Time Frame: Up to 200 days ]To evaluate the long-term safety and tolerability and efficacy of GB002
- Pharmacokinetics: Time to Reach Maximum Concentration (Tmax) of GB002 (Main study) [ Time Frame: 14 days ]
- Pharmacokinetics: Maximum Concentration (Cmax) of GB002 (Main study) [ Time Frame: 14 days ]
- Pharmacokinetics: Area Under the Concentration Curve (AUC) of GB002 (Main study) [ Time Frame: 14 days ]
- Changes from baseline in 6-Minute Walk Test (6MWT) (OLE study) [ Time Frame: 169 days ]
- Changes from baseline in WHO Functional Class (OLE study) [ Time Frame: 197 days ]
- Changes from baseline in N-terminal pro B-type natriuretic peptide (NT-proBNP) (OLE study) [ Time Frame: 169 days ]
- Changes from baseline in Pulmonary Arterial Hypertension - Symptoms and Impact (PAH-SYMPACT) Quality of Life questionnaire (OLE study) [ Time Frame: 197 days ]
- Changes from baseline in Right Ventricular function by imaging (OLE study) [ Time Frame: 169 days ]

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years to 75 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Adult (males and females) aged 18 to 75 years (inclusive) with pulmonary arterial hypertension (PAH) (Main study)
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A current diagnosis of symptomatic PAH classified by one of the following (Main and OLE study):
- Idiopathic (IPAH) or heritable pulmonary arterial hypertension (HPAH)
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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)
Exclusion Criteria:
- 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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03926793
United States, California | |
UCSD Medical Center | |
La Jolla, California, United States, 92037 | |
University of California Davis Medical Center | |
Sacramento, California, United States, 95817 | |
United States, Colorado | |
University of Colorado Hospital | |
Aurora, Colorado, United States, 80045 | |
United States, Connecticut | |
Yale New Haven Hospital | |
New Haven, Connecticut, United States, 06510 | |
United States, Florida | |
Mayo Clinic Florida | |
Jacksonville, Florida, United States, 32224 | |
United States, Kentucky | |
Norton Pulmonary Specialists | |
Louisville, Kentucky, United States, 40202 | |
United States, Massachusetts | |
Brigham and Women's Hospital | |
Boston, Massachusetts, United States, 02115 | |
United States, Minnesota | |
Mayo Clinic | |
Rochester, Minnesota, United States, 55905 | |
United States, North Carolina | |
Duke Early Phase Research | |
Durham, North Carolina, United States, 27710 | |
United States, Ohio | |
Cleveland Clinic Foundation | |
Cleveland, Ohio, United States, 44195 | |
United States, Pennsylvania | |
University of Pittsburgh Medical Center Montefiore University Hospital | |
Pittsburgh, Pennsylvania, United States, 15213 | |
United States, South Carolina | |
VitaLink Research - Anderson | |
Anderson, South Carolina, United States, 29621 | |
United Kingdom | |
Golden Jubilee National Hospital | |
Glasgow, United Kingdom, G814DY | |
Hammersmith Hospital, Imperial Healthcare NHS Trust | |
London, United Kingdom, W12 0HS |
Responsible Party: | GB002, Inc., a wholly owned subsidiary of Gossamer Bio, Inc. |
ClinicalTrials.gov Identifier: | NCT03926793 |
Other Study ID Numbers: |
GB002-1101 |
First Posted: | April 25, 2019 Key Record Dates |
Last Update Posted: | September 1, 2021 |
Last Verified: | August 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | Yes |
Pulmonary Arterial Hypertension Familial Primary Pulmonary Hypertension Hypertension Vascular Diseases |
Cardiovascular Diseases Hypertension, Pulmonary Lung Diseases Respiratory Tract Diseases |