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    The EU Clinical Trials Register currently displays   43801   clinical trials with a EudraCT protocol, of which   7259   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2017-001276-27
    Sponsor's Protocol Code Number:GBT440-026
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2017-05-03
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedUK - MHRA
    A.2EudraCT number2017-001276-27
    A.3Full title of the trial
    A Phase II open label study to evaluate the effect of GBT440 on hypoxemia in subjects with Idiopathic Pulmonary Fibrosis (IPF) who are using supplemental oxygen at rest (ZEPHYR)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to determine if GBT440 can increase the level of oxygen in the blood of people with Idiopathic Pulmonary Fibrosis (IPF) who need to use supplemental oxygen when resting.
    A.3.2Name or abbreviated title of the trial where available
    ZEPHYR
    A.4.1Sponsor's protocol code numberGBT440-026
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorGlobal Blood Therapeutics Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportGlobal Blood Therapeutics Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGlobal Blood Therapeutics Inc
    B.5.2Functional name of contact pointDirector, Clinical Operations
    B.5.3 Address:
    B.5.3.1Street Address400 East Jamie Court
    B.5.3.2Town/ citySouth San Francisco
    B.5.3.3Post codeCA 94080
    B.5.3.4CountryUnited States
    B.5.4Telephone number0016503514758
    B.5.5Fax number001650-741-7701
    B.5.6E-mailboguno@globalbloodtx.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameGBT440 300 mg capsule
    D.3.2Product code GBT440
    D.3.4Pharmaceutical form Capsule
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 1446321-46-5
    D.3.9.2Current sponsor codeGBT440
    D.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number300
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameGBT440 300 mg tablet
    D.3.2Product code GBT440
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 1446321-46-5
    D.3.9.2Current sponsor codeGBT440
    D.3.9.4EV Substance CodeAS2
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number300
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Idiopathic Pulmonary Fibrosis (IPF)
    E.1.1.1Medical condition in easily understood language
    IPF is a chronic condition in which the lungs become scarred causing low oxygen levels in the blood and worsening breathlessness. It is not clear what causes it and there is no known cure.
    E.1.1.2Therapeutic area Diseases [C] - Respiratory Tract Diseases [C08]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10021240
    E.1.2Term Idiopathic pulmonary fibrosis
    E.1.2System Organ Class 10038738 - Respiratory, thoracic and mediastinal disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the effect of GBT440 on oxygen saturation at rest, breathing room air, on Days 30 and 90 compared to baseline.
    E.2.2Secondary objectives of the trial
    • To evaluate the effect of GBT440 on the requirement for supplemental oxygen (O2) at rest and post-exercise at Days 30 and 90 compared to baseline
    • To evaluate the effect of GBT440 on resting and post-exercise Alveolar-arterial O2 tension difference [P(A-a)O2] at Days 30 and 90 compared to baseline
    • To evaluate the effect of GBT440 on 6-minute walk distance (6MWD) at Days 30 and 90 compared to baseline
    • To evaluate the effect of GBT440 on IPF-related symptoms, using patient reported outcomes (PROs), at Days 30 and 90 compared to baseline
    • To evaluate pulmonary function at Day 90 compared to baseline
    • To evaluate the safety and tolerability of 900 mg and 1500 mg GBT440 dosed daily for 90 days
    • To evaluate the pharmacokinetics (PK) of GBT440
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1) 45 to 85 years of age inclusive, at randomization
    2) Able and willing to provide signed informed consent to participate in
    this study
    3) Documented diagnosis of IPF, as indicated in the American Thoracic
    Society (ATS)/European Respiratory Society (ERS)/Japanese Respiratory Society (JRS)/Latin American Respiratory Society (ALAT) 2011 Guidelines
    4) Receiving supplemental O2 for use at rest.
    • Subjects using O2 only with exercise will not be eligible.
    5) Resting oxygen saturation (SpO2) between 85 and 88% for at least
    30 consecutive seconds while breathing room air (refer to Section 5.1 and Section 5.5.2 for details on confirming eligibility)
    Or
    Resting SpO2 <85% for 10 consecutive seconds or, if in the opinion of the site staff, it is not safe or tolerable for the subject to continue without using their supplemental oxygen for 10 consecutive seconds whilst the SpO2 is <85%.
    Either of these 2 criteria should be met at both screening and Day 1 visits.
    6) Able, in the Investigator’s opinion, to walk a total of at least 100 meters at completion of the baseline 6-minute walk test (6MWT)
    7) Weight ≥40 kg
    8) Able, in the Investigator’s opinion, to complete the O2 titration study unassisted at baseline (Day 1) and Days 30, 90, and 120
    9) Able, in the Investigator’s opinion, to comply with the study procedures, including attending the assessment visits and adhering to study requirements and restrictions
    10) Male or female of childbearing potential willing and able to use highly effective methods of contraception from study start to 30 days after the last dose of study drug
    E.4Principal exclusion criteria
    1) Forced expiratory volume in 1 second (FEV1) / forced vital capacity (FVC) <70%
    2) History of interstitial lung diseases secondary to other medical conditions (e.g., scleroderma, sarcoidosis, or rheumatoid arthritis) or resulting from clinically significant environmental exposures including but not limited to, drug toxicity, hypersensitivity pneumonitis, or asbestos
    3) Hospitalization due to an exacerbation of IPF within 30 days of
    screening
    4) Documented pulmonary hypertension that is severe (World Health Organization [WHO] Functional Class IV) and/or clinically unstable,
    as determined by the study Investigator
    • Subjects with documented mild to moderate pulmonary hypertension on a stable regimen of therapy for at least 3 months prior to screening will be eligible for the study
    5) Subject plans to begin, or has commenced, pulmonary rehabilitation
    within 30 days of screening
    • Subject who is on a stable exercise regimen at screening or whose regimen, in the opinion of the Investigator, is not expected to change at any time during the entire study will be considered eligible for the study
    6) Corticosteroid therapy for the treatment of IPF, >10 mg per day of
    prednisone (or an equivalent), administered for 7 days or longer, within 30 days of screening.
    • Subjects receiving a stable dose of ≤10 mg per day of prednisone (or an equivalent) for at least 14 days prior to screening, and in the opinion of the Investigator not anticipated to require a dose adjustment during the study, are eligible for the study
    7) Corticosteroid therapy for treatment of non-IPF diseases, unless:
    • Receiving a stable dose of prednisone (or an equivalent) for at least 14 days prior to screening, and in the opinion of the Investigator not anticipated to require a dose adjustment during the study
    8) Participated in another clinical trial of an investigational drug (or medical device) within 30 days or 5-half-lives, whichever is longer, prior to screening, or is currently participating in another trial of an investigational drug (or medical device).
    9) Aspartate aminotransferase (AST), alanine aminotransferase (ALT)
    or total bilirubin >2 × upper limit of normal (ULN)
    10) Serum creatinine >2.0 mg/dL
    11) Clinical evidence of active infection, within 14 days of screening, which may include but is not limited to bronchitis, pneumonia, urinary tract infection, or cellulitis.
    12) Active viral hepatitis within the last 6 months
    13) Active tuberculosis within the last 6 months
    • Testing for latent tuberculosis is not required
    14) Electrocardiogram (ECG) with a corrected QT interval using the Fridericia formula (QTcF) >450 ms (males) or QTcF >470 ms (females)
    • If ventricular pacing is noted on ECG, then QTcF intervals will not be calculated
    15) Family or personal history of congenital long QT syndrome
    16) Female who is breast-feeding or pregnant
    17) Known current malignancy or current evaluation for a potential
    malignancy or history of malignancy within the past 2 years prior to
    screening, except for appropriately treated non-melanoma skin carcinoma, carcinoma in situ of the cervix, Stage 1 uterine cancer
    18) Current smoker (including use of eCigarettes or vaporizing) or history of smoking within 3 months from screening
    19) History of unstable or deteriorating cardiac or pulmonary disease (other than IPF) within 3 months of screening, including but not limited to, congestive heart failure requiring hospitalization or
    uncontrolled clinically significant arrhythmias
    20) History of mental illness within the last 5 years, unless the subject fulfills one of the following conditions:
    • The subject has not required or been prescribed any psychiatric medication (including but not limited to antidepressants or anxiolytics) within 12 months before screening and, in the
    opinion of the Investigator, the subject is able and safe to participate in the study
    • The subject has been on a fixed regimen of psychiatric medications for at least 6 months before screening and displays no sign of acute mental illness and, in the opinion of the Investigator, the subject is able and safe to participate in the study
    21) Other clinically significant medical disease that is uncontrolled despite treatment and is likely, in the study Investigator’s opinion, to significantly impact the study’s efficacy and safety assessments
    22) Any condition affecting drug absorption, such as major surgery
    involving the stomach or small intestine (prior cholecystectomy is acceptable)
    23) Known hypersensitivity to any component of the study drug
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is change and % change from baseline in oxygen saturation at rest (SpO2 or SaO2), measured while breathing room air.
    E.5.1.1Timepoint(s) of evaluation of this end point
    at Days 30 and 90.
    E.5.2Secondary end point(s)
    • Change and % change from baseline in O2 flow rate (L/min), measured at rest, to maintain a target oxygen saturation (SpO2) of 89 to 92%, at Days 30 and 90.
    • Change and % change from baseline in O2 flow rate (L/min), measured post-exercise to maintain a target oxygen saturation (SpO2 or SaO2) of 89 to 92%, at Days 30 and 90
    • Change and % change from baseline in resting P(A-a) O2 at Days 30 and 90
    • Change and % change from baseline in post-exercise P(A-a) O2 at Days 30 and 90
    • Change and % change from baseline in 6MWD, at Days 30 and 90
    • Change in patient-reported IPF-related symptoms from baseline, measured by ATAQ and SGRQ, at Days 30 and 90
    • Change from baseline in FVC and DLco at Day 90
    E.5.2.1Timepoint(s) of evaluation of this end point
    Day 30 and Day 90.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety No
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned3
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1Number of subjects for this age range: 0
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) No
    F.1.1.6.1Number of subjects for this age range: 0
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 8
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 24
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 8
    F.4.2.2In the whole clinical trial 32
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    No treatment after subject has ended participation in the trial
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2017-06-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-08-21
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2017-12-15
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