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    Summary
    EudraCT Number:2020-005287-60
    Sponsor's Protocol Code Number:GBT2104-132
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-06-01
    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 ConcernedGermany - PEI
    A.2EudraCT number2020-005287-60
    A.3Full title of the trial
    A Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants with Sickle Cell Disease and Recurrent Vaso occlusive Crises
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants with Sickle Cell Disease and Recurrent Vaso occlusive Crises
    A.4.1Sponsor's protocol code numberGBT2104-132
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04927247
    A.5.4Other Identifiers
    Name:US IND numberNumber:144073
    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 pointSupriya Rao
    B.5.3 Address:
    B.5.3.1Street Address181 Oyster Point Blvd.
    B.5.3.2Town/ citySouth San Francisco
    B.5.3.3Post codeCA 94080
    B.5.3.4CountryUnited States
    B.5.4Telephone number001650351 4760
    B.5.6E-mailsrao@gbt.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 nameInclacumab
    D.3.4Pharmaceutical form 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 INNInclacumab
    D.3.9.1CAS number 1256258-86-2
    D.3.9.3Other descriptive nameAnti P-selectin glycoprotein ligand-1 humanised monoclonal antibody
    D.3.9.4EV Substance CodeSUB205263
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500 mg/ 10 mL
    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 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 placeboSolution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Sickle Cell Disease
    E.1.1.1Medical condition in easily understood language
    Sickle Cell Disease and Recurrent Vaso occlusive Crises
    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.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this study is to evaluate the safety and efficacy of a single dose of inclacumab compared to placebo to reduce the incidence of re-admission to a healthcare facility for a vaso-occlusive crisis (VOC) after an admission for an index VOC in participants with sickle cell disease (SCD).
    E.2.2Secondary objectives of the trial
    Additional objectives of the study are to evaluate the pharmacokinetics (PK) and pharmacodynamics (PD) of inclacumab, the presence of anti-drug antibodies (ADAs), and changes in quality of life (QOL).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Participant has an index VOC. The index VOC is any VOC that required admission to a healthcare facility and treatment with parenteral pain medication. An admission for the index VOC includes:
    a. A hospital admission, or
    b. An admission to an emergency room, observation unit, or infusion center for ≥ 12 hours, or
    c. 2 visits to an emergency room, observation unit, or infusion center over a 72-hour period. For an acute episode of pain with no other cause other than a vaso-occlusive event that includes the following:
    • Uncomplicated VOC,
    • Acute chest syndrome (ACS),
    • Hepatic sequestration,
    • Splenic sequestration, or
    • Priapism.
    2. Participant has a confirmed diagnosis of SCD (any genotype).
    Documentation of SCD genotype is required and may be based on documented history of laboratory testing or confirmed by laboratory testing at Baseline.
    3. Participant is male or female, ≥ 18 years of age at the time of informed consent.
    4. Participant is able to complete screening and receive study drug within 5 days following investigator-assessed resolution of index VOC (for example, hospital discharge, discontinuation of parenteral pain medication, or transition to oral pain medication).
    5. Participant has experienced between 2 and 10 VOCs (inclusive) within the 12 months prior to Screening as determined by documented medical history. The index VOC is not to be considered as one of the 2 to 10 events. A prior VOC is defined as an acute episode of pain that:
    • Has no medically determined cause other than a vaso-occlusive event, and
    • Results in a visit to a medical facility (hospital, emergency department, urgent care center, outpatient clinic, or infusion center) or results in a remote contact with a healthcare provider; and
    • Requires parenteral narcotic agents, parenteral NSAIDs, or an increase in treatment with oral narcotics.
    6. Participants receiving ESA (eg, erythropoietin [EPO]) must be on a stable dose for at least 90 days prior to Screening and expected to continue with the stabilized regimen throughout the course of the study.
    7. Participants receiving HU, L-glutamine, or voxelotor must be on a stable dose for at least 30 days prior to Screening and expected to continue with the stabilized regimen throughout the course of the study.
    8. Participant has adequate venous access, in the opinion of the Investigator, to comply with study procedures.
    9. Participant understands the study procedures and agrees to participate in the study by giving written informed consent.
    10. Women of childbearing potential (WOCBP) are required to have a negative serum pregnancy test at the Screening Visit and negative urine pregnancy tests on all subsequent clinic visits and must agree to use a highly effective method of contraception throughout the study period and for at least 165 days after dosing. (Section 6.2).
    Female participants will not be considered of childbearing potential if they are pre-menarchal, surgically sterile (hysterectomy, bilateral salpingectomy, tubal ligation, or bilateral oophorectomy) or postmenopausal (no menses for 12 months without an alternative medical cause, confirmed by follicle-stimulating hormone [FSH] test results).
    E.4Principal exclusion criteria
    1. Participant is receiving regularly scheduled RBC transfusion therapy (also termed chronic, prophylactic, or preventative transfusion).
    2. Participant is taking or has received crizanlizumab (ADAKVEO®) within 90 days prior to Screening.
    3. Participant weighs > 133 kg (292 lbs.).
    4. Participant has a significant active and poorly controlled (unstable) hepatic disorder clearly unrelated to SCD.
    5. Participant has any of the following laboratory values at Screening:
    a. Absolute neutrophil count (ANC) < 1.0 × 109/L
    b. Platelet count < 80 × 109/L
    c. Hemoglobin < 4.0 g/dL
    d. Estimated glomerular filtration rate (eGFR) < 30 mL/min using Chronic Kidney Disease-Epidemiology Collaboration (CKD-EPI)
    NOTE: Laboratory assessments conducted during Screening must be done by the local laboratory and must include CBC with total and differential leukocyte count, platelet count, hemoglobin, and a chemistry panel with blood urea nitrogen, bilirubin (total, direct, and indirect), alkaline phosphatase, aspartate aminotransferase, alanine aminotransferase, serum albumin, sodium, potassium, calcium, chloride, glucose, bicarbonate, serum creatinine, and total protein to assess participant eligibility. Laboratory assessments conducted during the index VOC admission that are obtained within 7 days prior to screening may be used for Screening assessments if done as part of standard medical care.
    6. Participant has known active (symptomatic) COVID-19 infection or tests positive for COVID-19 at any time during their index admission.
    7. Participant has a history of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to Screening including severe or unstable pulmonary hypertension.
    8. Participant has had treatment for a malignancy within the 12 months prior to Screening (except non-melanoma skin cancer and in situ cervical cancers).
    9. Participant has had a stroke within the 2 years prior to the Screening Visit.
    10. Participant has a positive test indicative of active malaria infection at Screening. Testing to be conducted at local laboratories in malaria-endemic regions at the discretion of the Investigator.
    11. Participant has any confirmed clinically significant drug allergy and/or known hypersensitivity to monoclonal antibody therapeutics or formulation components of the study drug or a related drug.
    12. Participant has been treated with another investigational agent within 30 days or 5 half-lives of the investigational agent (whichever is greater) prior to Screening.
    13. Participant has had a major surgery within 8 weeks prior to the Screening Visit.
    14. Participant is pregnant, breastfeeding, or planning to become pregnant during the 90-day treatment period.
    15. Participant, parent, or legal guardian are unlikely to comply with the study procedures.
    16. Participant has any other medical, or psychological, or behavioral conditions that, in the opinion of the Investigator, would: confound or interfere with evaluation of safety, efficacy, and/or PK of the investigational drug; prevent compliance with the study protocol; preclude informed consent; or render the participant, parent, or caretaker unable/unlikely to comply with the study procedures.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint for this study is, following an index VOC, the proportion of participants with at least 1 VOC that required admission to a healthcare facility and treatment with parenteral pain medication within 90 days of randomization.
    An admission for a VOC includes:
    • A hospital admission, or
    • An admission to an emergency room, observation unit, or infusion center for ≥ 12 hours, or
    • 2 visits to an emergency room, observation unit, or infusion center over a 72-hour period.
    for an acute episode of pain with no other cause other than a vaso-occlusive event that includes the following:
    • Uncomplicated VOC, or
    • Acute chest syndrome, or
    • Hepatic sequestration, or
    • Splenic sequestration, or
    • Priapism
    The definition of the index VOC requiring admission is the same as the primary endpoint VOC requiring admission.
    To ensure consistency across all sites, on-study VOCs to be used for the primary endpoint will be confirmed by adjudication by an independent, blinded panel comprised of experts in SCD. The primary efficacy analysis will be performed on adjudicated data.
    E.5.1.1Timepoint(s) of evaluation of this end point
    within 90 days of randomization
    E.5.2Secondary end point(s)
    The secondary efficacy endpoints of the study are the following:
    • Time to first VOC that required admission to a healthcare facility and treatment with parenteral pain medication within 90 days of randomization.
    • Proportion of participants with at least 1 VOC that required admission to a healthcare facility and treatment with parenteral pain medication within 30 days of randomization.
    • Rate of VOCs leading to a healthcare visit (hospital, emergency room, clinic visit, or remote contact with a healthcare provider) that requires parenteral pain medication (eg, parenteral narcotic agents or parenteral nonsteroidal anti-inflammatory drugs [NSAIDs]), or an increase in treatment with oral narcotics within 90 days following randomization.
    E.5.2.1Timepoint(s) of evaluation of this end point
    within 90 days of randomization
    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 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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 Yes
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA5
    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
    Brazil
    Colombia
    Egypt
    Ghana
    Kenya
    Lebanon
    Nigeria
    Oman
    Saudi Arabia
    United States
    Zambia
    France
    Germany
    Italy
    Turkey
    United Kingdom
    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 months10
    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 months2
    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: 255
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 37
    F.4.2.2In the whole clinical trial 280
    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)
    Participants not enrolling in the OLE study will complete the study at the time of the last scheduled study procedures at the Day 161 visit. For participants who enroll in the OLE study, the Day 91 visit will be the final study visit. Participants may receive their first dose in the OLE study on the same day as the completion of the study assessments for the Day 91 visit.
    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 Decision2021-11-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-07-28
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2023-11-24
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