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    Summary
    EudraCT Number:2019-003459-12
    Sponsor's Protocol Code Number:RB-NAIT-01-01
    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:2020-03-31
    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 ConcernedGermany - PEI
    A.2EudraCT number2019-003459-12
    A.3Full title of the trial
    A Single-blind, Placebo-controlled, Single-center Study Investigating the Dose of Human Anti-Human Platelet Antigen (HPA)-1a Immune Globulin (NAITgam) Needed to Eliminate HPA-1a Positive Platelets Transfused to HPA-1a Negative Healthy Male Volunteers
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study Investigating the Dose of NAITgam (a Human Anti-Human Platelet Antigen (HPA)-1a Immune Globulin) Needed to Eliminate Platelets Transfused to Healthy Male Volunteers
    A.4.1Sponsor's protocol code numberRB-NAIT-01-01
    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 SponsorRallybio, IPA, LLC
    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 supportRallybio
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRallybio, IPA, LLC
    B.5.2Functional name of contact pointCOO
    B.5.3 Address:
    B.5.3.1Street Address234 Church Street, Suite 1020
    B.5.3.2Town/ cityNew Haven
    B.5.3.3Post codeCT 06510
    B.5.3.4CountryUnited States
    B.5.6E-mailinfo@rallybio.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 numberEMA/OD/076/11 EMA/COMP/676058/2011
    D.3 Description of the IMP
    D.3.1Product nameNAITgam
    D.3.4Pharmaceutical form Solution for injection
    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 INNAnti-Human Platelet Antigen-1a Immune Globulin
    D.3.9.2Current sponsor codeNAITgam Bulk
    D.3.9.3Other descriptive nameHuman platelet antigen 1a immunoglobulin
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1.000
    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 Yes
    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 Yes
    D.2.5.1Orphan drug designation numberEMA/OD/076/11 EMA/COMP/676058/2011
    D.3 Description of the IMP
    D.3.1Product nameNAITgam
    D.3.4Pharmaceutical form Solution for injection
    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 INNAnti-Human Platelet Antigen-1a Immune Globulin
    D.3.9.2Current sponsor codeNAITgam Bulk
    D.3.9.3Other descriptive nameHuman platelet antigen 1a immunoglobulin
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number4.000
    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 Yes
    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: 3
    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 numberEMA/OD/076/11 EMA/COMP/676058/2011
    D.3 Description of the IMP
    D.3.1Product nameNAITgam
    D.3.4Pharmaceutical form Solution for injection
    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 INNAnti-Human Platelet Antigen-1a Immune Globulin
    D.3.9.2Current sponsor codeNAITgam Bulk
    D.3.9.3Other descriptive nameHuman platelet antigen 1a immunoglobulin
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10.000
    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 Yes
    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 injection
    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
    prevention of Fetal Neonatal Alloimmune Thrombocytopenia (FNAIT
    In mothers negative for HPA-1a antigen, fetal platelets positive for HPA-1a antigen, may enter the mother’s circulation and induce production of maternal HPA-1a alloantibodies. These can traverse the placenta and destroy the fetal platelets, resulting in fetal thrombocytopenia and increased risk of bleeding. FNAIT presents as isolated thrombocytopenia in otherwise healthy newborns and is the leading cause of severe thrombocytopenia
    E.1.1.1Medical condition in easily understood language
    Prevention of bleeding in fetus and newborn due to antibodies produced by the mother against a marker on fetal platelets
    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 20.1
    E.1.2Level LLT
    E.1.2Classification code 10075151
    E.1.2Term Fetal and neonatal alloimmune thrombocytopenia
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To establish the dose of human anti-human platelet antigen (HPA)-1a immune globulin (NAITgam) needed to markedly (10-fold or greater) accelerate the clearance of HPA-1a positive platelets transfused to HPA-1a negative healthy volunteers
    E.2.2Secondary objectives of the trial
    - To evaluate the safety of a single dose of NAITgam
    - To monitor alloimmune response
    - To establish the pharmacokinetic (PK) profile of NAITgam
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol
    2. Healthy male participants, ≥ 18 and ≤ 65 years of age
    3. Body mass index (BMI) < 35 kg/m2
    4. HPA-1a negative
    5. Cohorts 1, 1B and 2 only: HLA-A2 negative
    E.4Principal exclusion criteria
    1. History of hypersensitivity to platelet concentrates or human plasma proteins
    2. IgA levels < 0.06 g/L
    3. Blood donation received within 3 weeks of screening
    4. Platelet counts < 150 x 109/L or > 450 x 109/L
    5. Any type of known platelet function disorder
    6. Treatment with NSAIDs (eg, acetylsalicylic acid) or selective serotonin reuptake inhibitors within 7 days prior to screening
    7. Chronic or ongoing active infectious disease requiring systemic treatment including, but not limited to, chronic renal infection, chronic chest infection with bronchiectasis, and tuberculosis
    8. Individuals with increased risk of thrombotic events (eg, history of pulmonary embolism or thrombosis)
    9. Current participation in any other interventional clinical study (or within 90 days prior to screening)
    10. Participants known or suspected of not being able to comply with this study protocol (eg, due to alcoholism, drug dependency, or psychological disorder)
    11. Presence of HLA class I-antibodies at screening – for participants, not previously transfused, this is defined as MFI value > 3,000 on the One-Lambda’s LABScreen Single Antigen assay
    12. Ongoing active infection with HIV and/or hepatitis B and/or C virus
    13. Vaccination received within 1 month of screening
    14. Current diagnosis of diabetes mellitus or increased HbA1c at screening
    E.5 End points
    E.5.1Primary end point(s)
    half-life (t1/2) of transfused platelets
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline until latest day 7
    E.5.2Secondary end point(s)
    - type, seriousness, and incidence of adverse of events
    - vital signs
    - clinical laboratory values
    - electrocardiogram (ECG)
    - persistence (or development) of anti-HPA-1a antibodies after clearance of NAITgam
    - PK parameters of NAITgam:
    o t1/2 of NAITgam
    o initial NAITgam concentration after slow injection (C0)
    o clearance (CL) of NAITgam
    o area under the NAITgam concentration versus time curve (AUC)-
    E.5.2.1Timepoint(s) of evaluation of this end point
    at every study visit when assessed: day 1, day 3, day 7, week 2, 4, 8, 12, 16, 20, 24
    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 No
    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) Yes
    E.7.1.1First administration to humans Yes
    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 Yes
    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 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 No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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 years2
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    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: 28
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers Yes
    F.3.2Patients No
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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 state28
    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)
    Since this is a study with healthy subjects no further treatment after end of study is necessary and planned
    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 Decision2020-08-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-07-10
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2023-05-15
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