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出境医 / 临床实验 / Clinical Study of Inhaled GB002 for Treatment of WHO Group I Pulmonary Arterial Hypertension (PAH)

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

Study Description
Brief 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).

Condition or disease Intervention/treatment Phase
Pulmonary Arterial Hypertension Drug: GB002 Drug: Placebo Device: Generic Dry Powder Inhaler Phase 1

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
Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 8 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Masking Description: Clinical site investigators, study personnel, and study subjects will be blinded to treatment assignment; however, the Sponsor will be unblinded.
Primary Purpose: Treatment
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)
Actual Study Start Date : February 4, 2020
Actual Primary Completion Date : May 5, 2021
Actual Study Completion Date : May 5, 2021
Arms and Interventions
Arm Intervention/treatment
Experimental: Cohort 1
Patients will be randomized to receive inhaled GB002 or Placebo daily for 14 days
 Drug: GB002
GB002 low dose or high dose for inhalation

Drug: Placebo
Placebo for inhalation

Device: Generic Dry Powder Inhaler
Generic dry powder inhaler for GB002 or Placebo delivery
Experimental: Cohort 2
Patients will be randomized to receive inhaled GB002 or Placebo daily for 14 days
 Drug: GB002
GB002 low dose or high dose for inhalation

Drug: Placebo
Placebo for inhalation

Device: Generic Dry Powder Inhaler
Generic dry powder inhaler for GB002 or Placebo delivery
Experimental: Open Label Extension
Eligible subjects may participate in the Open Label Extension (OLE) study for a period of 24 weeks.
 Drug: GB002
GB002 low dose or high dose for inhalation

Device: Generic Dry Powder Inhaler
Generic dry powder inhaler for GB002 or Placebo delivery
Outcome Measures
Primary Outcome Measures :
  1. 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

  2. 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


Secondary Outcome Measures :
  1. Pharmacokinetics: Time to Reach Maximum Concentration (Tmax) of GB002 (Main study) [ Time Frame: 14 days ]
  2. Pharmacokinetics: Maximum Concentration (Cmax) of GB002 (Main study) [ Time Frame: 14 days ]
  3. Pharmacokinetics: Area Under the Concentration Curve (AUC) of GB002 (Main study) [ Time Frame: 14 days ]
  4. Changes from baseline in 6-Minute Walk Test (6MWT) (OLE study) [ Time Frame: 169 days ]
  5. Changes from baseline in WHO Functional Class (OLE study) [ Time Frame: 197 days ]
  6. Changes from baseline in N-terminal pro B-type natriuretic peptide (NT-proBNP) (OLE study) [ Time Frame: 169 days ]
  7. Changes from baseline in Pulmonary Arterial Hypertension - Symptoms and Impact (PAH-SYMPACT) Quality of Life questionnaire (OLE study) [ Time Frame: 197 days ]
  8. Changes from baseline in Right Ventricular function by imaging (OLE study) [ Time Frame: 169 days ]

Eligibility Criteria
Layout table for eligibility information
Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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)

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

Contacts and Locations

Locations
Layout table for location information
United States, California
UCSD Medical Center
La Jolla, California, United States, 92037
University of California Davis Medical Center
Sacramento, California, United States, 95817
United States, Colorado
University of Colorado Hospital
Aurora, Colorado, United States, 80045
United States, Connecticut
Yale New Haven Hospital
New Haven, Connecticut, United States, 06510
United States, Florida
Mayo Clinic Florida
Jacksonville, Florida, United States, 32224
United States, Kentucky
Norton Pulmonary Specialists
Louisville, Kentucky, United States, 40202
United States, Massachusetts
Brigham and Women's Hospital
Boston, Massachusetts, United States, 02115
United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
United States, North Carolina
Duke Early Phase Research
Durham, North Carolina, United States, 27710
United States, Ohio
Cleveland Clinic Foundation
Cleveland, Ohio, United States, 44195
United States, Pennsylvania
University of Pittsburgh Medical Center Montefiore University Hospital
Pittsburgh, Pennsylvania, United States, 15213
United States, South Carolina
VitaLink Research - Anderson
Anderson, South Carolina, United States, 29621
United Kingdom
Golden Jubilee National Hospital
Glasgow, United Kingdom, G814DY
Hammersmith Hospital, Imperial Healthcare NHS Trust
London, United Kingdom, W12 0HS
Sponsors and Collaborators
GB002, Inc., a wholly owned subsidiary of Gossamer Bio, Inc.
Tracking Information
First Submitted Date  ICMJE April 22, 2019
First Posted Date  ICMJE April 25, 2019
Last Update Posted Date May 18, 2021
Actual Study Start Date  ICMJE February 4, 2020
Actual Primary Completion Date May 5, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 25, 2020)
  • 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
Original Primary Outcome Measures  ICMJE
 (submitted: April 22, 2019) 		Number of participants with Treatment-Related Adverse Events GB002 [ Time Frame: Up to 45 days ]
To evaluate the safety and tolerability of GB002
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: September 25, 2020)
  • 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 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: April 22, 2019)
  • Pharmacokinetics: Time to Reach Maximum Concentration (Tmax) of GB002 [ Time Frame: 14 days ]
  • Pharmacokinetics: Maximum Concentration (Cmax) of GB002 [ Time Frame: 14 days ]
  • Pharmacokinetics: Area Under the Concentration Curve (AUC) of GB002 [ Time Frame: 14 days ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Clinical Study of Inhaled GB002 for Treatment of WHO Group I Pulmonary Arterial Hypertension
Official Title  ICMJE 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)
Brief 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).
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Masking Description:
Clinical site investigators, study personnel, and study subjects will be blinded to treatment assignment; however, the Sponsor will be unblinded.
Primary Purpose: Treatment
Condition  ICMJE Pulmonary Arterial Hypertension
Intervention  ICMJE
  • Drug: GB002
    GB002 low dose or high dose for inhalation
  • Drug: Placebo
    Placebo for inhalation
  • Device: Generic Dry Powder Inhaler
    Generic dry powder inhaler for GB002 or Placebo delivery
Study Arms  ICMJE
  • Experimental: Cohort 1
    Patients will be randomized to receive inhaled GB002 or Placebo daily for 14 days
    Interventions:
    • Drug: GB002
    • Drug: Placebo
    • Device: Generic Dry Powder Inhaler
  • Experimental: Cohort 2
    Patients will be randomized to receive inhaled GB002 or Placebo daily for 14 days
    Interventions:
    • Drug: GB002
    • Drug: Placebo
    • Device: Generic Dry Powder Inhaler
  • Experimental: Open Label Extension
    Eligible subjects may participate in the Open Label Extension (OLE) study for a period of 24 weeks.
    Interventions:
    • Drug: GB002
    • Device: Generic Dry Powder Inhaler
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: May 17, 2021) 		8
Original Estimated Enrollment  ICMJE
 (submitted: April 22, 2019) 		24
Actual Study Completion Date  ICMJE May 5, 2021
Actual Primary Completion Date May 5, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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)

NOTE: Additional inclusion/exclusion criteria may apply, per protocol.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 75 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03926793
Other Study ID Numbers  ICMJE GB002-1101
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: Yes
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Gossamer Bio Inc. ( GB002, Inc., a wholly owned subsidiary of Gossamer Bio, Inc. )
Study Sponsor  ICMJE GB002, Inc., a wholly owned subsidiary of Gossamer Bio, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Gossamer Bio Inc.
Verification Date May 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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