Pulmonary Arterial HypertensionPhase 1PAHNCT03926793
Clinical Study of Inhaled GB002 for Treatment of WHO Group I Pulmonary Arterial Hypertension
A Phase 1 study testing GB002 and Generic Dry Powder Inhaler in people with Pulmonary Arterial Hypertension, run by GB002, Inc., a wholly owned subsidiary of Gossamer Bio, Inc. at 14 sites in 2 countries.
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). Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Pulmonary Arterial Hypertension
- What is being tested?
- GB002 and Generic Dry Powder Inhaler
- Type of treatment
- Medical device
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 8 participants
- Where
- 14 sitesin 2 countries
- Study dates
- 4 Feb 2020to 5 May 2021
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adult (males and females) aged 18 to 75 years (inclusive) with pulmonary arterial hypertension (PAH) (Main study)
- A current diagnosis of symptomatic PAH classified by one of the following (Main and OLE study):
- Idiopathic (IPAH) or heritable pulmonary arterial hypertension (HPAH)
- 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)
Who cannot join
- 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.
Trial locations and contacts
14 sites in 2 countries.
14 sites in 2 countries
United States12 sites
VitaLink Research - Anderson
Anderson, South Carolina, 29621
University of Colorado Hospital
Aurora, Colorado, 80045
Brigham and Women's Hospital
Boston, Massachusetts, 02115
Cleveland Clinic Foundation
Cleveland, Ohio, 44195
Duke Early Phase Research
Durham, North Carolina, 27710
Mayo Clinic Florida
Jacksonville, Florida, 32224
UCSD Medical Center
La Jolla, California, 92037
Norton Pulmonary Specialists
Louisville, Kentucky, 40202
Yale New Haven Hospital
New Haven, Connecticut, 06510
University of Pittsburgh Medical Center Montefiore University Hospital
Pittsburgh, Pennsylvania, 15213
Mayo Clinic
Rochester, Minnesota, 55905
University of California Davis Medical Center
Sacramento, California, 95817
United Kingdom2 sites
Golden Jubilee National Hospital
Glasgow, G814DY
Hammersmith Hospital, Imperial Healthcare NHS Trust
London, W12 0HS
About this study
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)
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).
Read the 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.
- Conditions
- Pulmonary Arterial Hypertension
- Being tested
- GB002, Placebo, Generic Dry Powder Inhaler
- Medical subject headings
- Pulmonary Arterial Hypertension
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GB002Drug
GB002 low dose or high dose for inhalation
PlaceboDrug
Placebo for inhalation
Generic Dry Powder InhalerDevice
Generic dry powder inhaler for GB002 or Placebo delivery
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Patients will be randomized to receive inhaled GB002 or Placebo daily for 14 days Receives: Drug: GB002; Drug: Placebo; Device: Generic Dry Powder Inhaler |
| Cohort 2 | Experimental | Patients will be randomized to receive inhaled GB002 or Placebo daily for 14 days Receives: Drug: GB002; Drug: Placebo; Device: Generic Dry Powder Inhaler |
| Open Label Extension | Experimental | Eligible subjects may participate in the Open Label Extension (OLE) study for a period of 24 weeks. Receives: Drug: GB002; Device: Generic Dry Powder Inhaler |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with Treatment-Related Adverse Events GB002 (Main study)
To evaluate the safety and tolerability of GB002
Measured: Up to 45 days
Number of participants with Treatment-Related Adverse Events GB002 (OLE study)
To evaluate the long-term safety and tolerability and efficacy of GB002
Measured: Up to 200 days
Secondary outcomes (8)
Pharmacokinetics: Time to Reach Maximum Concentration (Tmax) of GB002 (Main study)
Measured: 14 days
Pharmacokinetics: Maximum Concentration (Cmax) of GB002 (Main study)
Measured: 14 days
Pharmacokinetics: Area Under the Concentration Curve (AUC) of GB002 (Main study)
Measured: 14 days
Changes from baseline in 6-Minute Walk Test (6MWT) (OLE study)
Measured: 169 days
Changes from baseline in WHO Functional Class (OLE study)
Measured: 197 days
Changes from baseline in N-terminal pro B-type natriuretic peptide (NT-proBNP) (OLE study)
Measured: 169 days
Changes from baseline in Pulmonary Arterial Hypertension - Symptoms and Impact (PAH-SYMPACT) Quality of Life questionnaire (OLE study)
Measured: 197 days
Changes from baseline in Right Ventricular function by imaging (OLE study)
Measured: 169 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Apr 2019
- Study start4 Feb 2020
- Primary completion5 May 2021
- Study completion5 May 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pulmonary Arterial HypertensionStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- GB002, Inc., a wholly owned subsidiary of Gossamer Bio, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03926793
- Sponsor's protocol ID
- GB002-1101
- Acronym
- PAH
- Status last verified
- Aug 2021
- First submitted
- 22 Apr 2019
- Last updated
- 1 Sep 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT03926793
- Acronym / sponsor ID
- PAH / GB002-1101
- Conditions
- Pulmonary Arterial Hypertension
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- GB002, Inc., a wholly owned subsidiary of Gossamer Bio, Inc.Industry (pharma, biotech, devices)
- Study start
- 4 Feb 2020 (actual)
- Primary completion
- 5 May 2021 (actual)
- Study completion
- 5 May 2021 (actual)
- First posted
- 25 Apr 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Drug: GB002, Drug: Placebo, Device: Generic Dry Powder InhalerPatients will be randomized to receive inhaled GB002 or Placebo daily for 14 days |
| Cohort 2 | Experimental | Drug: GB002, Drug: Placebo, Device: Generic Dry Powder InhalerPatients will be randomized to receive inhaled GB002 or Placebo daily for 14 days |
| Open Label Extension | Experimental | Drug: GB002, Device: Generic Dry Powder InhalerEligible subjects may participate in the Open Label Extension (OLE) study for a period of 24 weeks. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Adult (males and females) aged 18 to 75 years (inclusive) with pulmonary arterial hypertension (PAH) (Main study)
- A current diagnosis of symptomatic PAH classified by one of the following (Main and OLE study):
- systemic sclerosis, rheumatoid arthritis, mixed CTD or overlap syndrome, or systemic lupus erythematosus
- World Health Organization (WHO)/New York Heart Association (NYHA) functional class II-IV symptomatology (Main study)
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GB002GB002 low dose or high dose for inhalation | Drug | Cohort 1, Cohort 2, Open Label Extension |
| PlaceboPlacebo for inhalation | Drug | Cohort 1, Cohort 2 |
| Generic Dry Powder InhalerGeneric dry powder inhaler for GB002 or Placebo delivery | Device | Cohort 1, Cohort 2, Open Label Extension |
Primary outcome
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
8 secondary outcomes
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Sep 2021.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
14 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing GB002 and Generic Dry Powder Inhaler in people with Pulmonary Arterial Hypertension, run by GB002, Inc., a wholly owned subsidiary of Gossamer Bio, Inc. at 14 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Sep 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with Treatment-Related Adverse Events GB002 (Main study) (time frame: Up to 45 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Sep 2021; AzTrials last refreshed its copy on 8 Nov 2026.