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

NCT ID: NCT03926793

Last Updated: 2021-09-01

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

8 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-02-04

Study Completion Date

2021-05-05

Brief Summary

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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

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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.

Conditions

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Pulmonary Arterial Hypertension

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators
Clinical site investigators, study personnel, and study subjects will be blinded to treatment assignment; however, the Sponsor will be unblinded.

Study Groups

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Cohort 1

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

Group Type EXPERIMENTAL

GB002

Intervention Type DRUG

GB002 low dose or high dose for inhalation

Placebo

Intervention Type DRUG

Placebo for inhalation

Generic Dry Powder Inhaler

Intervention Type DEVICE

Generic dry powder inhaler for GB002 or Placebo delivery

Cohort 2

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

Group Type EXPERIMENTAL

GB002

Intervention Type DRUG

GB002 low dose or high dose for inhalation

Placebo

Intervention Type DRUG

Placebo for inhalation

Generic Dry Powder Inhaler

Intervention Type DEVICE

Generic dry powder inhaler for GB002 or Placebo delivery

Open Label Extension

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

Group Type EXPERIMENTAL

GB002

Intervention Type DRUG

GB002 low dose or high dose for inhalation

Generic Dry Powder Inhaler

Intervention Type DEVICE

Generic dry powder inhaler for GB002 or Placebo delivery

Interventions

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GB002

GB002 low dose or high dose for inhalation

Intervention Type DRUG

Placebo

Placebo for inhalation

Intervention Type DRUG

Generic Dry Powder Inhaler

Generic dry powder inhaler for GB002 or Placebo delivery

Intervention Type DEVICE

Eligibility Criteria

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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)
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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GB002, Inc., a wholly owned subsidiary of Gossamer Bio, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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UCSD Medical Center

La Jolla, California, United States

Site Status

University of California Davis Medical Center

Sacramento, California, United States

Site Status

University of Colorado Hospital

Aurora, Colorado, United States

Site Status

Yale New Haven Hospital

New Haven, Connecticut, United States

Site Status

Mayo Clinic Florida

Jacksonville, Florida, United States

Site Status

Norton Pulmonary Specialists

Louisville, Kentucky, United States

Site Status

Brigham and Women's Hospital

Boston, Massachusetts, United States

Site Status

Mayo Clinic

Rochester, Minnesota, United States

Site Status

Duke Early Phase Research

Durham, North Carolina, United States

Site Status

Cleveland Clinic Foundation

Cleveland, Ohio, United States

Site Status

University of Pittsburgh Medical Center Montefiore University Hospital

Pittsburgh, Pennsylvania, United States

Site Status

VitaLink Research - Anderson

Anderson, South Carolina, United States

Site Status

Golden Jubilee National Hospital

Glasgow, , United Kingdom

Site Status

Hammersmith Hospital, Imperial Healthcare NHS Trust

London, , United Kingdom

Site Status

Countries

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United States United Kingdom

Other Identifiers

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GB002-1101

Identifier Type: -

Identifier Source: org_study_id

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