GB002 for Pulmonary Arterial Hypertension

Recruiting at 36 trial locations

Overseen By GB002, Inc.

Age: 18+

Sex: Any

Trial Phase: Phase 2

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

Must be taking: PAH therapies

Must not be taking: Inhaled prostanoids, Anticoagulants

Disqualifiers: Hypertension, Heart disease, Infections, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

74 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial will study the long-term effects of GB002 (seralutinib) in patients who were part of an earlier study for Pulmonary Arterial Hypertension (PAH). Seralutinib is a new treatment developed for PAH and has shown better results compared to another treatment in early studies.

Will I have to stop taking my current medications?

The trial requires that participants continue their standard PAH (Pulmonary Arterial Hypertension) medications at a stable dose. However, you cannot use inhaled prostanoids or chronic oral anticoagulants like warfarin. If you are on these, you may need to stop them to participate.

Is seralutinib (GB002) safe for humans?

Seralutinib (also known as GB002) is being tested in a Phase 2 clinical trial for pulmonary arterial hypertension, which includes evaluating its safety. This trial involves 80 participants and will assess safety over a 24-week period, with an optional 72-week extension. ^{1 2 3 4 5}

Who Is on the Research Team?

Richard Aranda

Principal Investigator

Gossamer Bio Inc.

Are You a Good Fit for This Trial?

This trial is for adults who've completed a previous GB002 study for Pulmonary Arterial Hypertension (PAH) and were compliant. They must be on stable PAH medications, not have life-threatening heart issues, severe hypertension or hypotension, new left-sided heart disease, substance abuse problems, certain blood disorders or infections. They can't use inhaled tobacco/marijuana but may use ingestible/topical marijuana.

Inclusion Criteria

Subjects must have completed a prior GB002 PAH study and, in the opinion of the Investigator and Sponsor, have been compliant with study procedures and have completed treatment with IP through parent study end-of-treatment (EOT) visit.

I am on a stable dose of standard treatment for PAH.

Review and signature of an IRB-approved informed consent form.

Exclusion Criteria

I have long-term kidney problems.

Your body has very low levels of a type of white blood cells called neutrophils.

I have high blood pressure in my lungs or liver due to cirrhosis, and it's at least moderate in severity.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Open-label Extension

Participants continue receiving GB002 (seralutinib) inhaled orally twice per day for long-term evaluation

Up to 80 months or until availability of commercial product

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• GB002 (seralutinib)

Trial Overview The trial tests the long-term effects of an inhaler drug called GB002 (seralutinib) in patients with PAH who participated in earlier GB002 studies. It's an open-label extension which means everyone knows they're getting the actual drug and there's no placebo.

How Is the Trial Designed?

Group I: GB002 (seralutinib) Experimental Treatment 2 Interventions

GB002 (seralutinib) inhaled orally twice per day (BID)

GB002 (seralutinib) is already approved in European Union, United States for the following indications:

Approved in European Union as Seralutinib for:

• Pulmonary arterial hypertension (PAH)

Approved in United States as Seralutinib for:

• Pulmonary arterial hypertension (PAH)

Who Is Running the Clinical Trial?

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

Lead Sponsor

Trials

4

Recruited

260+

Published Research Related to This Trial

Inhaled seralutinib effectively inhibited key receptors involved in pulmonary arterial hypertension (PAH) and demonstrated significant improvements in heart and lung function in two rat models of PAH, outperforming the proof-of-concept drug imatinib.

The treatment with seralutinib led to reduced pulmonary artery muscularization and right ventricle hypertrophy, along with restoration of beneficial protein levels, indicating its potential as a promising therapy for severe PAH.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Inhaled seralutinib exhibits potent efficacy in models of pulmonary arterial hypertension. Galkin, A., Sitapara, R., Clemons, B., et al.[2023]

In the PATENT-2 study involving 396 patients with pulmonary arterial hypertension, riociguat treatment for over 2 years was well tolerated, with serious adverse events occurring in 60% of patients but only 11% discontinuing due to these events.

Key efficacy parameters such as 6-minute walking distance (6MWD), WHO functional class, and NT-proBNP concentrations were significantly associated with overall survival, highlighting their importance in assessing long-term outcomes in patients receiving riociguat.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Predictors of long-term outcomes in patients treated with riociguat for pulmonary arterial hypertension: data from the PATENT-2 open-label, randomised, long-term extension trial. Ghofrani, HA., Grimminger, F., Grünig, E., et al.[2022]

In a 1-year study of patients with pulmonary arterial hypertension (PAH), sitaxsentan at 100 mg showed a high overall survival rate of 96% and a lower risk of clinical worsening events (34%) compared to bosentan, which had an 88% survival rate and a 40% risk of worsening.

Sitaxsentan also demonstrated a lower incidence of elevated liver enzymes (6% risk for AST/ALT > 3 x ULN) and discontinuation due to adverse events (15%) compared to bosentan, which had a 14% risk for elevated liver enzymes and a 30% risk of discontinuation due to adverse events.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Sitaxsentan for the treatment of pulmonary arterial hypertension: a 1-year, prospective, open-label observation of outcome and survival. Benza, RL., Barst, RJ., Galie, N., et al.[2021]

In a randomized controlled trial involving 120 patients with pulmonary arterial hypertension (PAH) due to left heart disease, the combination of sacubitril/valsartan and dapagliflozin significantly improved heart function and exercise tolerance compared to sacubitril/valsartan alone after 6 months of treatment.

The combination therapy also led to greater reductions in pulmonary artery pressures and inflammatory markers, with no significant increase in adverse drug reactions, indicating that this treatment regimen is both effective and safe.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A study of the efficacy of sacubitril/valsartan plus dapagliflozin combination treatment in pulmonary arterial hypertension due to left heart disease. Ge, T., Yang, Y., Zhao, Y.[2023]

Seralutinib is a novel inhaled drug targeting PDGFRα/β, CSF1R, and c-KIT pathways, which are implicated in the progression of pulmonary arterial hypertension, and is currently being tested in an ongoing Phase 2 trial with 80 participants.

The primary goal of the trial is to assess changes in pulmonary vascular resistance after 24 weeks of treatment, while secondary outcomes include improvements in exercise capacity measured by the 6-minute walk test.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

TORREY, a Phase 2 study to evaluate the efficacy and safety of inhaled seralutinib for the treatment of pulmonary arterial hypertension. Frantz, RP., Benza, RL., Channick, RN., et al.[2021]

Citations

1. England pubmed.ncbi.nlm.nih.gov

Inhaled seralutinib exhibits potent efficacy in models of pulmonary arterial hypertension. [2023]

2. England pubmed.ncbi.nlm.nih.gov

Predictors of long-term outcomes in patients treated with riociguat for pulmonary arterial hypertension: data from the PATENT-2 open-label, randomised, long-term extension trial. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Sitaxsentan for the treatment of pulmonary arterial hypertension: a 1-year, prospective, open-label observation of outcome and survival. [2021]

4. England pubmed.ncbi.nlm.nih.gov

A study of the efficacy of sacubitril/valsartan plus dapagliflozin combination treatment in pulmonary arterial hypertension due to left heart disease. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

TORREY, a Phase 2 study to evaluate the efficacy and safety of inhaled seralutinib for the treatment of pulmonary arterial hypertension. [2021]