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    Summary
    EudraCT Number:2020-004403-14
    Sponsor's Protocol Code Number:BCX9930-203
    National Competent Authority:Czechia - SUKL
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-09-24
    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 ConcernedCzechia - SUKL
    A.2EudraCT number2020-004403-14
    A.3Full title of the trial
    A Randomized, Double-Blind, Multicenter, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria
    Randomizovaná, dvojitě zaslepená, multicentrická, placebem kontrolovaná studie s paralelními skupinami k vyhodnocení účinnosti, bezpečnosti a snášenlivosti perorální monoterapie přípravkem BCX9930 pro léčbu paroxysmální noční hemoglobinurie
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    BCX9930 compared to placebo in the treatment of PNH
    A.3.2Name or abbreviated title of the trial where available
    REDEEM 2
    A.4.1Sponsor's protocol code numberBCX9930-203
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05116787
    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 SponsorBioCryst Pharmaceuticals 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 supportBioCryst Pharmaceuticals Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAMS Advanced Medical Services
    B.5.2Functional name of contact pointProject Management
    B.5.3 Address:
    B.5.3.1Street Address26-28 Hammersmith Grove
    B.5.3.2Town/ cityLondon
    B.5.3.3Post codeW67BA
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+44208834 1144
    B.5.5Fax number+44208834 1156
    B.5.6E-mailoperations@ams-europe.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 nameBCX9930
    D.3.2Product code BCX9930
    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.3.8INN - Proposed INNBCX9930
    D.3.9.2Current sponsor codeBCX9930
    D.3.9.3Other descriptive nameBCX9930
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Paroxysmal Nocturnal Hemoglobinuria
    E.1.1.1Medical condition in easily understood language
    PNH
    E.1.1.2Therapeutic area Body processes [G] - Immune system processes [G12]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10034042
    E.1.2Term Paroxysmal nocturnal haemoglobinuria
    E.1.2System Organ Class 10038359 - Renal and urinary disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    • To determine the efficacy of oral BCX9930 monotherapy administered for 12 weeks, as compared to placebo, in subjects with paroxysmal nocturnal hemoglobinuria (PNH)
    • To evaluate the long-term safety and tolerability of oral BCX9930 monotherapy administered for up to 52 weeks in subjects with PNH
    E.2.2Secondary objectives of the trial
    • To evaluate the safety and tolerability of BCX9930 monotherapy administered for 12 weeks, as compared to placebo, in subjects with PNH
    • To characterize the effects of BCX9930 monotherapy administered for 12 weeks, as compared to placebo, using clinical and laboratory measurements, including complement and thrombosis biomarkers, and PNH clone size
    • To evaluate the effects of BCX9930 monotherapy administered for 12 weeks, as compared to placebo, on FACIT-Fatigue scale and other PROs
    • To assess the effectiveness of BCX9930 monotherapy administered for up to 52 weeks
    • To characterize the effects of BCX9930 monotherapy administered for up to 52 weeks using clinical and laboratory measurements, including complement and thrombosis biomarkers, and PNH clone size
    • To evaluate the effects of BCX9930 monotherapy administered for up to 52 weeks on FACIT-Fatigue scale and other PROs
    • To characterize BCX9930 plasma concentrations and pharmacokinetic (PK) parameters
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    PK/PD substudy - within the same protocol V2, 27 May 2021
    E.3Principal inclusion criteria
    1. Male or female, aged 18 - 70 years old at screening.
    2. Body weight ≥ 50 kg.
    3. Documented diagnosis of PNH confirmed by flow cytometry with a PNH granulocyte or monocyte clone size of ≥ 10% during screening.
    4. Are either: (a) naïve to treatment with a complement inhibitor, or (b) have received no treatment with a complement inhibitor for at least 12 months prior to the screening visit.
    5. Recorded the following results during screening:
    a. Hb ≤ 105 g/L (≤ 10.5 g/dL).
    b. LDH ≥ 2 × upper limit of normal reference range (ULN)
    c. ARC of ≥ 100 × 109 cells/L (≥ 100,000 cells/µL; ≥ 100 G/L).
    d. Absolute neutrophil count (ANC) of ≥ 0.75 × 109 cells/L (≥ 750 cells/µL; ≥ 0.75 G/L).
    e. Platelet count of ≥ 30 × 109/L (≥ 30,000/µL; ≥ 30 G/L).
    f. Estimated glomerular filtration rate of ≥ 50 mL/min/1.73 m2 using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation (Levey and Stevens 2010).
    6. Contraception requirements:
    Female participants must meet at least one of the following requirements:
    a. Be a woman of nonchildbearing potential.
    b. Be a woman of childbearing potential who agrees to use a highly effective contraceptive method throughout the study and for a duration of 30 days after the last dose of study drug.
    c. Alternatively, true abstinence is acceptable for women of childbearing potential when it is in line with the subject’s preferred and usual lifestyle.
    Male participants must meet at least one of the following requirements:
    a. Males with a female partner of childbearing potential must use condoms throughout the study and for a duration of 90 days after the dose of study drug unless their partner is using a highly effective contraceptive method independent of the study.
    b. Alternatively, true abstinence is acceptable when it is in line with the subject’s preferred and usual lifestyle
    7. Documentation of current vaccinations against Neisseria meningitidis types A, C, W, and Y, and Streptococcus pneumoniae, or willingness to start vaccination series at least 14 days prior to Day 1.
    8. In the opinion of the investigator, the subject is expected to adequately comply with all required study procedures and restrictions for the study, including compliance with the BID dosing schedule for BCX9930.
    9. Willing and able to provide written informed consent
    E.4Principal exclusion criteria
    1. Known history of or existing diagnosis of hereditary complement deficiency.
    2. History of hematopoietic cell transplant or solid organ transplant or anticipated candidate for transplantation during the study.
    3. Myocardial infarction or cerebrovascular accident within 30 days prior to screening, or current and uncontrolled clinically significant cardiovascular or cerebrovascular condition, including unstable angina, severe congestive heart failure, unexplained syncope, arrhythmia, and critical aortic stenosis.
    4. History of malignancy within 5 years prior to the screening visit, with exception of adequately treated non-melanoma skin or superficial bladder cancer, curatively treated carcinoma in situ of the cervix, or other curatively treated solid tumor deemed by the investigator and medical monitor to be at low risk for recurrence.
    5. Active bacterial, viral, or fungal infection or any other serious infection within 14 days prior to screening.
    (Note: Suspected or confirmed coronavirus disease [COVID-19]; persistent or recurrent positive test(s) for severe acute respiratory syndrome coronavirus 2 [SARS CoV 2] nucleic acids or antigens; and worsening of dyspnea not due to PNH, vasculitic rash, and persistent fever or other symptoms consistent with multisystem inflammatory syndrome in adults [MIS A] are exclusionary.)
    6. Current participation in any other investigational drug study or participation in an investigational drug study within 30 days prior to the screening visit, or 5.5 half-lives of the investigational drug, whichever is longer.
    7. Treatment with anti-thymocyte globulin within 180 days prior to the screening visit.
    8. Initiation of treatment with an erythropoiesis-stimulating agent (eg, erythropoietin), a thrombopoietin receptor agonist (eg, eltrombopag), or danazol within 28 days prior to the screening visit.
    (Note: Treatment with these medications initiated > 28 days prior to the screening visit is not exclusionary, if the dose is stable and there is a reasonable expectation that treatment will be continued.)
    9. Receiving iron with an unstable dose in the 28 days prior to the screening visit.
    10. Clinically significant abnormal electrocardiogram (ECG) at the screening visit.
    (Note: This includes, but is not limited to, a QT interval corrected using Fridericia’s method [QTcF] of > 450 msec in males or > 470 msec in females, or ventricular and/or atrial premature contractions that are more frequent than occasional, and/or as couplets or higher in grouping.)
    11. Subjects with any of the following results at the screening visit:
    a. Alanine aminotransferase (ALT; also serum glutamic-pyruvic transaminase [SGPT]) > 3 × ULN.
    b. AST (SGOT) > 3 × ULN.
    (Note: Subjects may be enrolled with AST > 3 × ULN if explained by hemolysis.)
    c. Total serum bilirubin > 2 × ULN
    (Note: Subjects may be enrolled with total serum bilirubin > 2 × ULN if explained by hemolysis or Gilbert’s syndrome. In the case of hemolysis, total serum bilirubin must be < 5 × ULN and in the case of Gilbert’s syndrome, total serum bilirubin must be < 11 × ULN.)
    12. Current use of a prohibited concomitant medication within 7 days prior to Day 1
    13. Positive serology for human immunodeficiency virus, or active infection with hepatitis B virus or hepatitis C virus.
    14. Positive drugs of abuse screen, unless by prescription or alcohol dependence, defined as
    consumption of ≥ 40 g pure alcohol per day for men and ≥ 20 g pure alcohol for women.
    15. Pregnant, planning to become pregnant, or breastfeeding.
    16. Known or suspected hypersensitivity to BCX9930 or any of its formulation excipients.
    17. History of severe hypersensitivity to any medicinal product, which was associated with swelling, severe rash requiring treatment/hospitalization, or anaphylaxis.
    18. Any other clinically significant medical or psychiatric condition that, in the opinion of the investigator, would interfere with the subject’s ability to participate in the study or increase the risk of participation for that subject.
    E.5 End points
    E.5.1Primary end point(s)
    Part1
    • Change from baseline (CFB) in hemoglobin (Hb)
    Part2
    • Number and proportion of subjects with a TEAE
    • Number and proportion of subjects who discontinue due to a TEAE
    • Number and proportion of subjects who experience a TESAE
    • Number and proportion of subjects who experience a CTCAE Grade 3 or Grade 4 TEAE
    • Number and proportion of subjects who experience a treatment-emergent CTCAE Grade 3 or Grade 4 laboratory abnormality
    E.5.1.1Timepoint(s) of evaluation of this end point
    Part 1 - Week 12
    Part 2 - week 52
    E.5.2Secondary end point(s)
    Part 1
    1. Proportion of subjects who are transfusion-free
    2. Number of units of packed red blood cells (pRBCs) transfused
    3. Percent CFB in lactate dehydrogenase (LDH)
    4. CFB in FACIT-Fatigue scale score
    Part 2
    • CFB in Hb
    • Proportion of subjects with Hb ≥ 12 g/dL
    • Proportion of subjects achieving Hb stabilization (avoidance of a > 2 g/dL decrease in the absence of transfusion)
    • Proportion of subjects who are transfusion-free
    • Number of units of pRBCs transfused
    • Percent reduction in rate of pRBCs transfused
    • Percent CFB in LDH
    • CFB in ARC
    • Proportion of subjects with ARC in the normal range
    • CFB in haptoglobin
    • Proportion of subjects with haptoglobin ≥ LLN
    • CFB in total PNH RBC clone size
    • CFB in total PNH RBC clone size relative to PNH WBC clone size
    E.5.2.1Timepoint(s) of evaluation of this end point
    Part 1 - Week 12
    Part 2 - week 52
    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 Yes
    E.6.4Safety Yes
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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 Yes
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA15
    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
    Albania
    Argentina
    Azerbaijan
    Brazil
    Canada
    China
    Czechia
    Italy
    Lithuania
    Poland
    Romania
    Serbia
    South Africa
    Spain
    Sweden
    Turkey
    United States
    E.8.7Trial has a data monitoring committee Yes
    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 months11
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    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.1.1.1In 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) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 52
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 57
    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 who are experiencing clinical benefit will be offered the opportunity to continue to receive BCX9930 via an access protocol
    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 Decision2022-03-31
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-01-19
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2023-09-18
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