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    Summary
    EudraCT Number:2018-001984-21
    Sponsor's Protocol Code Number:PTG-300-02
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-08-28
    Trial results View 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 number2018-001984-21
    A.3Full title of the trial
    Phase 2 Study of PTG-300 in Non-Transfusion Dependent (NTD) and Transfusion-Dependent (TD) β-Thalassemia Subjects with Chronic Anemia
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study to evaluate the safety and effect of PTG-300 in Non-Transfusion Dependent (NTD) and Transfusion-Dependent (TD) β-Thalassemia Patients with Chronic Anemia.
    A.4.1Sponsor's protocol code numberPTG-300-02
    A.5.4Other Identifiers
    Name:IND numberNumber:137605
    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 SponsorProtagonist 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 supportProtagonist Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationProtagonist Therapeutics, Inc.
    B.5.2Functional name of contact pointClinical-Regulatory Info Group
    B.5.3 Address:
    B.5.3.1Street Address7707, Gateway Blvd, Ste 140
    B.5.3.2Town/ cityNewark
    B.5.3.3Post code94560
    B.5.3.4CountryUnited States
    B.5.4Telephone number001510 4740170
    B.5.5Fax number0015103283232
    B.5.6E-mailclinregops@ptgx-inc.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/18/2058
    D.3 Description of the IMP
    D.3.1Product namePTG-300
    D.3.2Product code PN-8518A
    D.3.4Pharmaceutical form Injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codePTG-300
    D.3.9.3Other descriptive name1-(3-METHYLBUTANOYL)-L-ASPARTYL-L-THREONYL-L-HISTIDYL-L-PHENYLALANYL-L-PROLYL-(L-CYSTINYL-L-ISOLEUCYL-[(N6-(S)-4-CARBOXY-4-PALMITAMIDOBUTANOYL)-L-LYSINYL]-L-PHENYLALANYL-L-GLUTAMYL-L-PROLYL-L-ARGINYL-L-SERINYL-L-LYSINYL-L-GLYCINYL-L-CYSTINYL)-L-LYSINAMIDE, DISULFIDE, ACETATE
    D.3.9.4EV Substance CodeSUB194130
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number3 to 40
    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
    β-Thalassemia Subjects with Chronic Anemia (transfusion and non transfusion dependent)
    E.1.1.1Medical condition in easily understood language
    Blood disease that reduces the production of hemoglobin (protein needed to carry oxygen in the red blood cells) causing anemia.
    E.1.1.2Therapeutic area Body processes [G] - Genetic Phenomena [G05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10043391
    E.1.2Term Thalassaemia beta
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    1. To evaluate the safety and tolerability of PTG-300 in subjects with NTD and TD β-thalassemia.
    2. To obtain preliminary evidence of PTG-300’s efficacy for treating chronic anemia in subjects with β-thalassemia.
    3. To identify the optimal starting dose, titration algorithm, dose range and dose regimen to be used in Phase 3 studies.
    E.2.2Secondary objectives of the trial
    1. To evaluate the pharmacokinetics (PK) of PTG-300.
    2. To evaluate the pharmacodynamic (PD) effects of PTG-300.
    3. To evaluate the immunogenicity of PTG-300.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male and female subjects aged 18 to 65 years, inclusive (Cohorts 1-4b).
    2. Male and female subjects aged 12-<18 years, with a minimum weight of 30 kg (Cohorts 5 and 6).
    3. Documented diagnosis of β-thalassemia.
    4. Documented diagnosis of β-thalassemia with no other Hgb abnormality.
    5. Women of childbearing potential (WOCBP) and men agree to use a highly effective contraceptive measure (based on the Clinical Trial Facilitation Group [CTFG]) during the duration of the study and for 28 days after the last dose of study drug in the case of women and 90 days after the last dose of study drug in the case of men, as described in Appendix 1.
    6. For WOCBP, a negative serum pregnancy test at screening and a negative urine pregnancy test within 24 hours prior to the first dose of study medication.
    7. Subjects or legal guardians (in the case of minors) understand the study procedures and agree to participate in the study by giving written informed consent.
    8. Subjects, or legal representative (in the case of minors), are willing and able to adhere to the study visit schedule and other protocol requirements.
    9. Subjects between 12-<18 years of age understand and provide the assent to participate in the study, according to local guidelines.
    Inclusion criteria applicable only for NTD β-thalassemia subjects:
    1. Mean Hgb < 10.0 g/dL of two measurements (one performed 7–28 days prior to dosing and the other performed within 7 days prior to dosing).
    2. Requirement of < 6 units RBC transfusion in a 24 week period with the last transfusion at least 8 weeks before screening.
    Inclusion criteria applicable only for TD β-thalassemia subjects:
    1. Transfusion requirement of at least 6 units of RBC in the 24 weeks prior to screening with no transfusion free period > 45 days.
    2. Last RBC transfusion 5–15 days prior to dosing.
    E.4Principal exclusion criteria
    1. Subjects with Sickle Cell disease, Hgb H, Hb Bart’s hydrops foetalis or hemoglobin S
    2. Infection requiring hospitalization or IV antimicrobial therapy, or opportunistic infection within 6 months of dosing, any infection requiring antimicrobial therapy within 2 weeks of dosing; history of infection with human immunodeficiency virus (HIV).
    3. Subject has a concurrent clinically significant, unstable or uncontrolled cardiovascular, pulmonary, hepatic, renal, gastrointestinal, genitourinary, hematological, coagulation, immunological, endocrine/metabolic or other medical disorder that, in the opinion of the Investigator, might confound the results of the study or pose additional risk to the subject by their participation in the study.
    4. Known primary or secondary immunodeficiency.
    5. History within 6 months of screening of any of the following: myocardial infarction, unstable angina, transient ischemic attack, decompensated heart failure requiring hospitalization, congestive heart failure (New York Heart Association Class 3 or 4), uncontrolled arrhythmias, cardiac revascularization, stroke, uncontrolled hypertension (resting systolic blood pressure [BP] > 160mmHg or resting diastolic BP > 100mmHg on more than one occasion) or uncontrolled diabetes (Hgb A1c > 9% or > one episode of severe hypoglycemia).
    6. Clinically meaningful laboratory abnormalities at screening including, but not limited to, the ranges below:
    a. Absolute neutrophil count < 1000/μL
    b. Platelet count < 100,000/μL
    c. Estimated glomerular filtration rate (eGFR) < 60
    d. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥ 2.5 x upper limit of normal (ULN)
    7. Treatment with hydroxyurea ≤ 24 weeks prior to screening (unless approved by the Medical Monitor).
    8. Use of erythropoiesis-stimulating agent (ESA) ≤ 24 weeks prior to screening.
    9. Chronic use of systemic glucocorticoids (anti-inflammatory dose for more than 14 days) ≤ 12 weeks prior to screening (physiologic replacement therapy for adrenal insufficiency is allowed).
    10. Pregnant or lactating females.
    11. Any surgical procedure requiring general anesthesia within 1 month prior to screening or planned elective surgery during the study.
    12. History of malignant neoplasms within 5 years prior to screening. Subjects who are cancer-free for the 5 years before screening may be enrolled. Subjects with carcinoma in situ, adequately treated non-metastatic basal cell skin cancer, or squamous cell skin cancer that has not recurred for at least 1 year prior to screening, may be enrolled.
    13. Current or recent history of alcohol dependence or illicit drug use within 1 year prior to screening.
    14. Subject is mentally or legally incapacitated at the time of screening visit or has a history of clinically significant psychiatric disorders that would impact the subject’s ability to participate in the trial according to the Investigator. Note: Subjects who have had situational depression or adjustment disorder or treated depression may be enrolled at the discretion of the Investigator.
    15. Concurrent participation in any other interventional study.
    16. Having undergone splenectomy ≤ 24 weeks prior to screening.
    17. NTD subjects receiving iron chelation therapy (unless approved by the Medical Monitor).
    18. TD subjects who started regular transfusions before age 2 years.
    19. TD subjects who required more than 140 mL pure RBC/kg in the year prior to screening.
    E.5 End points
    E.5.1Primary end point(s)
    NTD:
    • Proportion of responders, where responders are defined as:
    - Subjects who achieve an increase in Hgb ≥ 1.0 g/dL without transfusion, confirmed by a successive measurement at least 1 week later
    • Hgb change from baseline

    TD:
    • Proportion of responders, where responders are defined as:
    − Subjects who achieve ≥ 20% reduction in the RBC units required over an 8-week period
    • Change from baseline in the number of units of RBC required
    E.5.1.1Timepoint(s) of evaluation of this end point
    NTD: Every Visit.
    TD: over an 8-week period under the same dose.
    E.5.2Secondary end point(s)
    NTD:
    • Hgb level
    • Proportion of subjects who achieve an increase in Hgb ≥ 1.5 g/dL without transfusion, confirmed by a successive measurement at least 1 week later
    • Duration of Hgb change of ≥1.0 g/dL from baseline without transfusion
    • Duration of Hgb change of ≥1.5 g/dL from baseline without transfusion
    • Change from baseline in the following PD parameters: serum iron, ferritin, transferrin saturation (TSAT)

    TD:
    • Proportion of subjects who achieve ≥ 33% reduction in the RBC units required over an 8-week period
    • Duration of response (defined as ≥ 20% reduction in the RBC units required over an 8-week period)
    • Percent change from baseline in the RBC units required
    • Best percent change from baseline in the RBC units required over an 8-week period
    • Number of RBC units required
    • Change from baseline to in the following PD parameters: serum iron, ferritin, transferrin, TSAT
    E.5.2.1Timepoint(s) of evaluation of this end point
    NTD: Every Visit.
    TD: over an 8-week period under the same dose.
    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 Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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 Yes
    E.8.1.7.1Other trial design description
    Multiple ascending dose
    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 trial1
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA11
    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
    Greece
    Italy
    Lebanon
    Malaysia
    Thailand
    Tunisia
    Turkey
    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
    The study will be considered completed when all subjects have either completed all study assessments or have withdrawn early.
    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 days7
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial days27
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 60
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 60
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 132
    F.1.3Elderly (>=65 years) No
    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 state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 107
    F.4.2.2In the whole clinical trial 192
    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)
    Subjects completing this Phase 2 study and who did not meet a stopping criterion, will have the opportunity to continue PTG-300 treatment in a separate open-label extension study, where the safety/ tolerability and efficacy of long term administration of PTG-300 will be assessed for a period of at least one year. 
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2018-10-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-12-17
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-03-31
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