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

Pulmonary Arterial Hypertension Clinical Trial

— PAH  

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03926793
• Other study ID #: GB002-1101
• Status: Completed
• Phase: Phase 1
• Start date: February 4, 2020
• Est. completion date: May 5, 2021

Study information

• Verified date: August 2021
• Source: Gossamer Bio Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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 information / eligibility

• Status: Completed
• Enrollment: 8
• Est. completion date: May 5, 2021
• Est. primary completion date: May 5, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

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.

Related Conditions & MeSH terms

• Familial Primary Pulmonary Hypertension
• Hypertension
• Pulmonary Arterial Hypertension

Intervention

Drug:
• GB002
GB002 low dose or high dose for inhalation
• Placebo
Placebo for inhalation

Device:
• Generic Dry Powder Inhaler
Generic dry powder inhaler for GB002 or Placebo delivery

Locations

Country	Name	City	State
United Kingdom	Golden Jubilee National Hospital	Glasgow
United Kingdom	Hammersmith Hospital, Imperial Healthcare NHS Trust	London
United States	VitaLink Research - Anderson	Anderson	South Carolina
United States	University of Colorado Hospital	Aurora	Colorado
United States	Brigham and Women's Hospital	Boston	Massachusetts
United States	Cleveland Clinic Foundation	Cleveland	Ohio
United States	Duke Early Phase Research	Durham	North Carolina
United States	Mayo Clinic Florida	Jacksonville	Florida
United States	UCSD Medical Center	La Jolla	California
United States	Norton Pulmonary Specialists	Louisville	Kentucky
United States	Yale New Haven Hospital	New Haven	Connecticut
United States	University of Pittsburgh Medical Center Montefiore University Hospital	Pittsburgh	Pennsylvania
United States	Mayo Clinic	Rochester	Minnesota
United States	University of California Davis Medical Center	Sacramento	California

Sponsors (1)

Lead Sponsor	Collaborator
GB002, Inc., a wholly owned subsidiary of Gossamer Bio, Inc.

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of participants with Treatment-Related Adverse Events GB002 (Main study)	To evaluate the safety and tolerability of GB002	Up to 45 days
Primary	Number of participants with Treatment-Related Adverse Events GB002 (OLE study)	To evaluate the long-term safety and tolerability and efficacy of GB002	Up to 200 days
Secondary	Pharmacokinetics: Time to Reach Maximum Concentration (Tmax) of GB002 (Main study)		14 days
Secondary	Pharmacokinetics: Maximum Concentration (Cmax) of GB002 (Main study)		14 days
Secondary	Pharmacokinetics: Area Under the Concentration Curve (AUC) of GB002 (Main study)		14 days
Secondary	Changes from baseline in 6-Minute Walk Test (6MWT) (OLE study)		169 days
Secondary	Changes from baseline in WHO Functional Class (OLE study)		197 days
Secondary	Changes from baseline in N-terminal pro B-type natriuretic peptide (NT-proBNP) (OLE study)		169 days
Secondary	Changes from baseline in Pulmonary Arterial Hypertension - Symptoms and Impact (PAH-SYMPACT) Quality of Life questionnaire (OLE study)		197 days
Secondary	Changes from baseline in Right Ventricular function by imaging (OLE study)		169 days