Clinical trial NCT04816604: Open-label Extension Study of GB002 in Adult Subjects With Pulmonary Arterial Hypertension (PAH)
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)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Pulmonary Arterial Hypertension
Keywords: seralutinib
Lead sponsor: Gossamer Bio Inc.
Sponsor class: INDUSTRY
First posted: 2021-03-25
Enrollment: 100
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

Type of Subject and Disease Characteristics

1. 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.
2. Treatment with standard of care PAH disease-specific background therapies (stable dose).

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

Exclusion Criteria:

Medical Conditions

1. Persistent and clinically significant systemic hypertension or hypotension.
2. Interval history of newly developed left-sided heart disease.
3. Potentially life-threatening cardiac arrhythmia with an ongoing risk.
4. Uncontrolled bacterial, viral, or fungal infections which require systemic therapy.
5. 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.
6. History of portopulmonary hypertension or portal hypertension due to cirrhosis classified as Child-Pugh Class A or higher.
7. 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.
8. Current use of inhaled tobacco and/or inhaled marijuana. Ingestible or topical marijuana is allowed, per local restrictions and regulations.
9. Current alcohol use disorder as defined by DSM-5, and/or history of current utilization of drugs of abuse (amphetamines, methamphetamines, cocaine, phencyclidine \[PCP\]).
10. 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
11. Chronic renal insufficiency
12. Hemoglobin (Hgb) concentration \<8.5 g/dL.
13. Absolute neutrophil count (ANC) \< 1x 10\^9/L.
14. Platelet count \<50 x 10\^9/L.

    Prior Therapy
15. Use of inhaled prostanoids.
16. Chronic use of oral anticoagulants (ie, vitamin K antagonist such as warfarin or novel oral anticoagulant \[NOAC\]/direct oral anticoagulant \[DOAC\]).
17. Chronic use of any prohibited medication.

NOTE: Additional inclusion/exclusion criteria may apply, per protocol.

Primary outcomes:
measure: Number of Participants With Treatment Emergent Adverse Events
timeFrame: From first dose of study drug up to 80 months or availability of commercial product
Secondary outcomes:
measure: Change from Baseline Over Time on the Six-Minute Walk Test (6MWT)
timeFrame: Baseline, up to 80 months or availability of commercial product
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Dept. of Veterans Affairs Greater Los Angeles Healthcare System
state: California
city: Sacramento
country: United States
facility: University of California, Davis Medical Center
state: California
city: Santa Barbara
country: United States
facility: Medical Corporation
state: California
city: Stanford
country: United States
facility: Stanford Health Care
state: California
city: Torrance
country: United States
facility: Lundquist Institute for Biomedical Innovation at Harbor UCLA
state: California
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Louisville
country: United States
facility: Norton Pulmonary Specialists
state: Kentucky
city: Boston
country: United States
facility: Tufts Medical Center
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Omaha
country: United States
facility: University of Nebraska Medical Center
state: Nebraska
city: New York
country: United States
facility: NYU Langone Health
state: New York
city: New York
country: United States
facility: New York Presbyterian Hospital - Weill Cornell Medicine
state: New York
city: Rochester
country: United States
facility: University of Rochester Medical Center
state: New York
city: Columbus
country: United States
facility: The Ohio State University Wexner Medical Center
state: Ohio
city: Oklahoma City
country: United States
facility: INTEGRIS Baptist Medical Center, Inc.
state: Oklahoma
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Houston
country: United States
facility: Houston Methodist Hospital
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah Health
state: Utah
city: Darlinghurst
country: Australia
facility: St Vincent's Hospital, Heart & Lung Transplant Unit
state: New South Wales
city: Hobart
country: Australia
facility: Royal Hobart Hospital
state: Tasmania
city: Fitzroy
country: Australia
facility: St. Vincent's Hospital Melbourne
state: Victoria
city: Leuven
country: Belgium
facility: University Hospital of Leuven
city: Praha
country: Czechia
facility: Všeobecná fakultní nemocnice v Praze
city: Le Kremlin-Bicêtre
country: France
facility: AP-HP Hôpital de Bicêtre
city: Gießen
country: Germany
facility: Universitaetsklinikum Giessen und Marburg GmbH - Medizinische Klinik IV und V
city: Hannover
country: Germany
facility: Klinik für Pneumologie, Klinisches Studienzentrum Medizinische Hochschule
city: Heidelberg
country: Germany
facility: Thoraxklinik-Heidelberg gGmbH
city: Regensburg
country: Germany
facility: Klinik und Poliklinik f. Innere Medizin II Universitätsklinikum Regensburg
city: Madrid
country: Spain
facility: Hospital Universitario 12 de Octubre
city: Santander
country: Spain
facility: Hospital Universitario Marqués de Valdecilla
city: Cambridge
country: United Kingdom
facility: Royal Papworth Hospital NHS Foundation Trust
city: London
country: United Kingdom
facility: Imperial College Healthcare NHS Trust - Hammersmith Medicines Research Limited
Study officials:
affiliation: Gossamer Bio Inc.
name: Richard Aranda
role: STUDY_DIRECTOR
References: