Open-label Extension Study of GB002 in Adult Subjects With Pulmonary Arterial Hypertension (PAH)

Study Overview
=================
Brief Summary
-----------------
This open-label extension study will evaluate the long-term effects of GB002 (seralutinib) in subjects who previously participated in a GB002 PAH study.

Official Title
-----------------
An Open-label Extension Study Evaluating the Long-term Safety and Efficacy of Oral Inhalation of GB002 for the Treatment of WHO Group 1 Pulmonary Arterial Hypertension (PAH)

Conditions
-----------------
Pulmonary Arterial Hypertension

Intervention / Treatment
-----------------
* Drug: GB002 (seralutinib)
* Device: Generic Dry Powder Inhaler


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Type of Subject and Disease Characteristics 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. Treatment with standard of care PAH disease-specific background therapies (stable dose). Informed Consent Review and signature of an IRB-approved informed consent form. Exclusion Criteria: Medical Conditions Persistent and clinically significant systemic hypertension or hypotension. Interval history of newly developed left-sided heart disease. Potentially life-threatening cardiac arrhythmia with an ongoing risk. Uncontrolled bacterial, viral, or fungal infections which require systemic therapy. Other severe acute or chronic medical or laboratory abnormality that may increase the risk associated with study participation or GB002 administration or may interfere with the interpretation of study results and, in the judgment of the Investigator, would make the subject inappropriate for entry into this study. History of portopulmonary hypertension or portal hypertension due to cirrhosis classified as Child-Pugh Class A or higher. Subjects with a history of severe milk protein allergy. In addition, subjects with known intolerance or hypersensitivity to lactose who, in the opinion of the investigator, may experience severe symptoms following the ingestion of lactose. Current use of inhaled tobacco and/or inhaled marijuana. Ingestible or topical marijuana is allowed, per local restrictions and regulations. Current alcohol use disorder as defined by DSM-5, and/or history of current utilization of drugs of abuse (amphetamines, methamphetamines, cocaine, phencyclidine [PCP]). Have any other condition or reason that, in the opinion of the Investigator and/or the Sponsor's Medical Monitor (or designee), would prohibit the subject from participating in the study. Diagnostic Assessments Chronic renal insufficiency Hemoglobin (Hgb) concentration <8.5 g/dL. Absolute neutrophil count (ANC) < 1x 10^9/L. Platelet count <50 x 10^9/L. Prior Therapy Use of inhaled prostanoids. Chronic use of oral anticoagulants (ie, vitamin K antagonist such as warfarin or novel oral anticoagulant [NOAC]/direct oral anticoagulant [DOAC]). Chronic use of any prohibited medication. NOTE: Additional inclusion/exclusion criteria may apply, per protocol.

Ages Eligible for Study
-----------------
Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: GB002 (seralutinib)<br>GB002 (seralutinib) inhaled orally twice per day (BID) over 144 weeks | Drug: GB002 (seralutinib)<br>* Capsule containing GB002 (seralutinib)<br>Device: Generic Dry Powder Inhaler<br>* Generic dry powder inhaler for GB002 (seralutinib) delivery<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Treatment Emergent Adverse Events |  | From first dose of study drug up to 148 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from Baseline Over Time on the Six-Minute Walk Test (6MWT) | Change in the distance achieved on the 6MWT (Δ6MWT) | Baseline, up to 148 weeks | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
seralutinib