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

• ClinicalTrials.gov Identifier: NCT03926793
• Recruitment Status: Completed
• First Posted: April 25, 2019
• Last Update Posted: September 1, 2021
• Sponsor: GB002, Inc., a wholly owned subsidiary of Gossamer Bio, Inc.
• Information provided by (Responsible Party): Gossamer Bio Inc. ( GB002, Inc., a wholly owned subsidiary of Gossamer Bio, Inc. )

Tracking Information

• First Submitted Date: April 22, 2019
• First Posted Date: April 25, 2019
• Last Update Posted Date: September 1, 2021
• Actual Study Start Date: February 4, 2020
• Actual Primary Completion Date: May 5, 2021 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: September 25, 2020)

• 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

Original Primary Outcome Measures (submitted: April 22, 2019)

Number of participants with Treatment-Related Adverse Events GB002 [ Time Frame: Up to 45 days ]

To evaluate the safety and tolerability of GB002

Current Secondary Outcome Measures (submitted: September 25, 2020)

• 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 ]

Original Secondary Outcome Measures (submitted: April 22, 2019)

• Pharmacokinetics: Time to Reach Maximum Concentration (Tmax) of GB002 [ Time Frame: 14 days ]
• Pharmacokinetics: Maximum Concentration (Cmax) of GB002 [ Time Frame: 14 days ]
• Pharmacokinetics: Area Under the Concentration Curve (AUC) of GB002 [ Time Frame: 14 days ]

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Clinical Study of Inhaled GB002 for Treatment of WHO Group I Pulmonary Arterial Hypertension
• 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)
• 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.

• Study Type: Interventional
• Study Phase: Phase 1

Study Design

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

• Condition: Pulmonary Arterial Hypertension

Intervention

• 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

Study Arms

• Experimental: Cohort 1
  Patients will be randomized to receive inhaled GB002 or Placebo daily for 14 days
  Interventions:

  • Drug: GB002
  • Drug: Placebo
  • Device: Generic Dry Powder Inhaler
• Experimental: Cohort 2
  Patients will be randomized to receive inhaled GB002 or Placebo daily for 14 days
  Interventions:

  • Drug: GB002
  • Drug: Placebo
  • Device: Generic Dry Powder Inhaler
• Experimental: Open Label Extension
  Eligible subjects may participate in the Open Label Extension (OLE) study for a period of 24 weeks.
  Interventions:

  • Drug: GB002
  • Device: Generic Dry Powder Inhaler

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: May 17, 2021): 8
• Original Estimated Enrollment (submitted: April 22, 2019): 24
• Actual Study Completion Date: May 5, 2021
• Actual Primary Completion Date: May 5, 2021 (Final data collection date for primary outcome measure)

Eligibility 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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 75 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United Kingdom, United States

Administrative Information

• NCT Number: NCT03926793
• Other Study ID Numbers: GB002-1101
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: Yes

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Gossamer Bio Inc. ( GB002, Inc., a wholly owned subsidiary of Gossamer Bio, Inc. )
• Original Responsible Party: GB002, Inc.
• Current Study Sponsor: GB002, Inc., a wholly owned subsidiary of Gossamer Bio, Inc.
• Original Study Sponsor: GB002, Inc.
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Gossamer Bio Inc.
• Verification Date: August 2021