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    Summary
    EudraCT Number:2017-001747-12
    Sponsor's Protocol Code Number:CSEG101B2201
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2019-03-06
    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 ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2017-001747-12
    A.3Full title of the trial
    A phase 2, Multicenter, Open-Label Study to Assess Appropriate Dosing and to Evaluate Safety of
    Crizanlizumab, with or without Hydroxyurea/Hydroxycarbamide, in Sequential,
    Descending Age Groups of Pediatric Sickle Cell Disease Patients with Vaso-Occlusive Crisis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients
    A.4.1Sponsor's protocol code numberCSEG101B2201
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03474965
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/533/2021
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorNovartis Pharma AG
    B.1.3.4CountrySwitzerland
    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 supportNovartis Pharma AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Pharma AG
    B.5.2Functional name of contact pointClinical Trial Information Desk
    B.5.3 Address:
    B.5.3.1Street AddressForum 1, Novartis Campus
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4056
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number+41 61 324 1111
    B.5.5Fax number+41 61 324 8001
    B.5.6E-mailclinicaltrial.enquiries@novartis.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/3/12/1034
    D.3 Description of the IMP
    D.3.1Product namecrizanlizumab
    D.3.2Product code SEG101
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCRIZANLIZUMAB
    D.3.9.2Current sponsor codeSEG101
    D.3.9.4EV Substance CodeSUB188615
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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
    Sickle Cell Disease with Vaso-Occlusive Crisis
    E.1.1.1Medical condition in easily understood language
    sickle cell disease
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level PT
    E.1.2Classification code 10040644
    E.1.2Term Sickle cell disease
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10002077
    E.1.2Term Anaemia sickle cell
    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
    - To confirm and establish appropriate dosing of crizanlizumab in patients ages 6 months to <18
    years at the time of study entry (Part A and B)
    - To evaluate the safety of crizanlizumab in patients ages 6 months to <18 years at the time of study entry (Parts A and B)
    E.2.2Secondary objectives of the trial
    - To assess the long-term efficacy of crizanlizumab in 6 months to < 18 year old patients at the time of study entry (Parts A and B)
    - To assess other safety measures in patients aged 6 months to < 18 years at the time of study entry
    - To characterize long-term PK and PD of crizanlizumab in patients ages 6 months to <18 years at the time of study entry
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    •Male or female patients aged 2 to <18 years (Group 3 will be expanded to allow enrolment of patients aged 6 to <24 months (and at least 7 kg) in Part B once the appropriate dose is confirmed in 2 to <6 year old participants)
    •Confirmed diagnosis of sickle cell disease (SCD) (e.g. any genotype including HbSS, HbSC, HbSβ0- thalassemia, HbSβ+-thalassemia, and others) by hemoglobin electrophoresis or high performance liquid chromatography (HPLC) performed locally. Confirmation of diagnosis by two accepted methods is recommended.
    •Experienced at least 1 VOC within the preceding 12 months prior to
    screening, as determined by medical history. Prior VOC must have resolved at least 7 days prior to the first dose in the study and must include all the following:
    1.the occurrence of appropriate symptoms (see VOC definition in protocol Section 7.2.1.1)
    2.either a visit to a medical facility or healthcare professional,
    3.receipt of oral/parenteral opioid or parenteral NSAIDs.
    •If receiving HU/HC, L-glutamine or erythropoietin stimulating agent, must have been receiving the drug consistently for at least 6 months prior to Screening and plan to continue taking it at the same dose and schedule during the trial. Patients who have not been receiving such
    drugs must have been off them for at least 6 months prior to screening. Dose alterations of HU/HC, L-glutamine or erythropoietin stimulating agent during Part A are not allowed, and if this occurs, the participant will enter directly to the Part B.
    •Received standard age-appropriate care for SCD, including penicillin prophylaxis, pneumococcal immunization, and parental education
    • Performance status: Karnofsky ≥ 50% for patients >10 years of age,and Lansky ≥ 50 for patients ≤ 10 years of age
    • Patient must meet the following laboratory values prior to Week 1 Day 1:
     Absolute Neutrophil Count ≥1.0 x 109/L
     Platelets ≥75 x 109/L
     Hemoglobin (Hgb) > 5.5 g/dL
    • Patient must have adequate renal and hepatic function as defined:
     Estimated Glomerular filtration rate (eGFRe) ≥ 75 mL/min/1.73 m2
    using Schwartz formula
     Direct (conjugated) bilirubin ≤ 2.0 x ULN
     Alanine transaminase (ALT) ≤ 3.0 x ULN
    •Transcranial Doppler (TCD) for patients aged 2 to < 16 years at time of screening) with HbSS, HbSβ0-thalassemia, and HbSD disease indicating low risk for stroke (per investigator). Please refer to Section 7.2.2.6 for details.

    Other inclusion criteria as per protocol may apply
    E.4Principal exclusion criteria
    •History of stem cell transplant.
    •Received any blood products within 30 days prior to Day 1 dosing.
    •Plan to participate in a chronic transfusion program (preplanned series of transfusions for prophylactic purposes) or undergo exchange transfusions/plasmapheresis during the study. Patients requiring episodic transfusion (simple or exchange) in response to worsened anemia or VOC are permitted.
    •Patients with bleeding disorders
    •Contraindication or hypersensitivity to any drug from similar class as study drug or to any excipients of the study drug formulation.
    •Planning to initiate or terminate HU/HC or L-glutamine while on study (except if needed to terminate for safety reasons)
    •Patient with active human immunodeficiency virus (HIV) infection
    (detectable viral load)
    •Significant active infection or immune deficiency (including chronic use of immunosuppressive drugs) in the opinion of the investigator.
    •Patients having taken voxelotor less than 30 days prior to Screening, or planning to take voxelotor while on study are not allowed
    Other exclusion criteria as per protocol may apply
    E.5 End points
    E.5.1Primary end point(s)
    1.PK (AUCd15) after 1st dose
    Confirm appropriate dosing of crizanlizumab in participants aged 2 to < 18 years (Parts A)

    2.PD (AUCd15) after 1st dose
    Confirm appropriate dosing of crizanlizumab in participants aged 2 to < 18 years (Parts A)

    3.PK (AUCtau) after multiple dose
    Confirm appropriate dosing of Crizanlizumab in participants aged 2 to < 18 years old

    4.PD (AUCtau) after multiple dose
    Confirm appropriate dosing of Crizanlizumab in participants aged 2 to < 18 years old

    5.PK (Cmax) after 1st dose and multiple dose
    Confirm appropriate dosing of crizanlizumab in participants aged 2 to < 18 years (Parts A)

    6.PK pre-dose concentrations
    Confirm appropriate dosing of crizanlizumab in participants aged 6 months to less than 24 months of age (Part B)

    7.Frequency of any adverse events (AEs) as a measure of safety and tolerability
    Safety of crizanlizumab in participants aged 6 months to < 18 years (Parts A and B)
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. Day 15
    2. Day 15
    3. Week 15
    4. Week 15
    5. Week 1 and Week 15
    6. Week 1 and Week 19
    7. 6 months, 2 years
    E.5.2Secondary end point(s)
    1. Annualized rate Vaso Occusive Crisis (VOC) events leading to healthcare visit in clinic/ER/hospital
    To assess the long-term efficacy of crizanlizumab in 6 months to < 18 year old participants at the time of study entry (Parts A and B)

    2. Annualized rate Vaso Occusive Crisis (VOC) events treated at home (based on documentation by health care provider following phone contact with the patient)
    To assess the long-term efficacy of crizanlizumab in 6 months to < 18 year old participants at the time of study entry (Parts A and B)

    3. Annualized rate each subcategory of VOC event (uncomplicated pain crisis, acute chest syndrome, hepatic sequestration, splenic sequestration, priapism)
    To assess the long-term efficacy of crizanlizumab in 6 months to < 18 year old participants at the time of study entry (Parts A and B)

    4. Annualized rate hospitalizations and ER visits (both overall and VOC-related)
    To assess the long-term efficacy of crizanlizumab in 6 months to < 18 year old participants at the time of study entry (Parts A and B)

    5. Annualized rate days of ER/hospitalization (both overall and VOC-related)
    To assess the long-term efficacy of crizanlizumab in 6 months to < 18 year old participants at the time of study entry (Parts A and B)

    6. Annualized rate dactylitis events
    To assess the long-term efficacy of crizanlizumab in 6 months to < 18 year old participants at the time of study entry (Parts A and B)

    7.Number, seriousness, severity, and causality assessments of treatement emergent adverse events and other data as considered appropiate.
    To assess other safety measures in participants aged 6 months to < 18 years at the time of study entry


    8.Absolute change from baseline in hemoglobin
    To assess other safety measures in participants aged 6 months to < 18 years at the time of study entry

    9.Immunogenicity: measurement of anti-drug antibodies (ADA) to crizanlizumab
    To assess other safety measures in participants aged 6 months to < 18 years at the time of study entry

    10.Electrocardiogram (ECGs) at relevant PK time points
    To assess other safety measures in participants aged 6 months to < 18 years at the time of study entry

    11.Growth and sexual maturation assessments (Tanner stage)
    To assess other safety measures in participants aged 6 months to < 18 years at the time of study entry

    12.PK pre-dose concentrations prior to each study drug dose.
    Characterize long-term PK and PD of crizanlizumab in participants aged 6 months to < 18 years

    13.PD pre-dose concentrations prior to each study drug dose.
    Characterize long-term PK and PD of crizanlizumab in participants aged 6 months to < 18 years

    14.Percentage P-selectin inhibition prior to dosing
    Characterize long-term PK and PD of crizanlizumab in participants aged 6 months to < 18 years
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. 6 months, 2 years
    2. 6 months, 2 years
    3. 6 months, 2 years
    4. 6 months, 2 years
    5. 6 months, 2 years
    6. 6 months, 2 years
    7. 6 months, 2 years
    8. 6 months, 2 years
    9. Week 1, Week 3, Week 15, Week 27 and End of Treatment (EOT)
    10. Screening, Week 7, Week 11, week 15, week 27 and Week 51
    11. Screening, Week 51 and End of Treatment (EOT)
    12. Week 1, Week 3, Week 7, Week 15, Week 19, Week 23, Week 27, Week 31, Week 35, Week 39, Week 43, Week 47 and Week 51
    13. Week 1, Week 3, Week 7, Week 15, Week 19, Week 23, Week 27, Week 31, Week 35, Week 39, Week 43, Week 47 and Week 51
    14. Week 3, Week 15, Week 27 and Week 51
    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 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 trial3
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA20
    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
    Colombia
    Oman
    Switzerland
    Brazil
    Canada
    India
    Lebanon
    Turkey
    United Kingdom
    United States
    Belgium
    France
    Germany
    Italy
    Spain
    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 end of the study is defined as the date of last visit of the last participant in the study and will occur when all the participants have either completed or
    discontinued the study treatment and/or have completed the last scheduled procedure (105-days post-treatment follow-up visit when applicable)
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days23
    E.8.9.2In all countries concerned by the trial years7
    E.8.9.2In all countries concerned by the trial months2
    E.8.9.2In all countries concerned by the trial days16
    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: 100
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 6
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 44
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 50
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 0
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 35
    F.4.2.2In the whole clinical trial 100
    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)
    Novartis will make every effort to supply SEG101 to patients who may benefit from continued treatment after the end of study, as per the Investigator’s opinion; Novartis will provide the study treatment to the investigator as per local regulations and safety will be monitored and reported to the Health Authorities per regulatory requirements.
    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 Decision2018-12-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-01-21
    P. End of Trial
    P.End of Trial StatusOngoing
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