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    Clinical Trial Results:
    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

    Summary
    EudraCT number
    		 2019-003459-12
    Trial protocol
    		 DE  
    Global end of trial date
    04 Apr 2022

    Results information
    Results version number
    		 v1(current)
    This version publication date
    11 Oct 2024
    First version publication date
    11 Oct 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    RB-NAIT-01-01
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Rallybio IPA, LLC
    Sponsor organisation address
    234 Church Street, New Haven, United States, Suite 1020
    Public contact
    Head of Regulatory and Quality, Rallybio IPA, LLC, regulatory@rallybio.com
    Scientific contact
    Head of Regulatory and Quality, Rallybio IPA, LLC, regulatory@rallybio.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    04 Apr 2022
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    04 Apr 2022
    Global end of trial reached?
    Yes
    Global end of trial date
    04 Apr 2022
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main 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
    Protection of trial subjects
    Not Applicable
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    21 Sep 2020
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Germany: 12
    Worldwide total number of subjects
    12
    EEA total number of subjects
    12
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    12
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Results from Cohorts 1 and 1B demonstrated that 1,000 IU NAITgam markedly accelerated the elimination of transfused platelets compared to placebo (10-fold or greater) and that proof of concept (PoC) criteria were met. It was determined by the sponsor that Cohorts 2 and 3 were not required and study was terminated following completion of Cohort 1B.

    Pre-assignment
    Screening details
    		 A total of 12 participants were enrolled at a single site in Germany.

    Pre-assignment period milestones
    Number of subjects started
    		 12
    Number of subjects completed
    		 12

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Single blind
    Roles blinded
    		 Subject

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Cohort 1 NAITgm 1000 IU
    Arm description
    		 Participants received a single IV dose of NAITgam 1000 IU on Day 1 that was administered 1 hour following completion of a HPA-1a positive platelet transfusion, followed by a 24-week safety follow-up period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    NAITgam
    Investigational medicinal product code
    Other name
    Human Anti-Human Platelet Antigen (HPA)-1a Immune Globulin
    Pharmaceutical forms
    Solution for injection in vial
    Routes of administration
    Intravenous use
    Dosage and administration details
    Participants received a single IV dose of NAITgam 1000 IU.

    Arm title
    		 Cohort 1B NAITgam 1000 IU
    Arm description
    		 Participants received a single IV dose of NAITgam 1000 IU on Day 1 followed by a HPA-1a positive platelet transfusion on Day 8, with a 25-week safety follow-up period from day of NAITgam administration.
    Arm type
    		 Experimental

    Investigational medicinal product name
    NAITgam
    Investigational medicinal product code
    Other name
    Human Anti-Human Platelet Antigen (HPA)-1a Immune Globulin
    Pharmaceutical forms
    Solution for injection in vial
    Routes of administration
    Intravenous use
    Dosage and administration details
    Participants received a single IV dose of NAITgam 1000 IU.

    Arm title
    		 Cohort 1 and Cohort 1B Placebo
    Arm description
    		 Cohort 1: Participants received a single IV administration of placebo 1 hour following completion of a HPA-1a positive platelet transfusion, followed by a 24-week safety follow-up period. Cohort 1B: Participants received a single IV administration of placebo on Day 1 followed by a HPA-1a positive platelet transfusion on Day 8, with a 25-week follow- up period from day of placebo administration.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Sodium chloride injection, 0.9% (saline)
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    Participants received a single administration of placebo IV.

    Number of subjects in period 1
    		Cohort 1 NAITgm 1000 IU Cohort 1B NAITgam 1000 IU Cohort 1 and Cohort 1B Placebo
    Started
    6
    3
    3
    Completed
    6
    3
    3

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Cohort 1 NAITgm 1000 IU
    Reporting group description
    		 Participants received a single IV dose of NAITgam 1000 IU on Day 1 that was administered 1 hour following completion of a HPA-1a positive platelet transfusion, followed by a 24-week safety follow-up period.

    Reporting group title
    Cohort 1B NAITgam 1000 IU
    Reporting group description
    		 Participants received a single IV dose of NAITgam 1000 IU on Day 1 followed by a HPA-1a positive platelet transfusion on Day 8, with a 25-week safety follow-up period from day of NAITgam administration.

    Reporting group title
    Cohort 1 and Cohort 1B Placebo
    Reporting group description
    		 Cohort 1: Participants received a single IV administration of placebo 1 hour following completion of a HPA-1a positive platelet transfusion, followed by a 24-week safety follow-up period. Cohort 1B: Participants received a single IV administration of placebo on Day 1 followed by a HPA-1a positive platelet transfusion on Day 8, with a 25-week follow- up period from day of placebo administration.

    Reporting group values
    		 Cohort 1 NAITgm 1000 IU Cohort 1B NAITgam 1000 IU Cohort 1 and Cohort 1B Placebo Total
    Number of subjects
    		 6 3 3 12
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 6 3 3 12
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 46.8 ( 11.27 ) 46.7 ( 12.50 ) 39.0 ( 17.09 ) -
    Gender categorical
    Units: Subjects
        Male
    		 6 3 3 12
    Race
    Units: Subjects
        White
    		 6 3 3 12

    End points

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    End points reporting groups
    Reporting group title
    Cohort 1 NAITgm 1000 IU
    Reporting group description
    		 Participants received a single IV dose of NAITgam 1000 IU on Day 1 that was administered 1 hour following completion of a HPA-1a positive platelet transfusion, followed by a 24-week safety follow-up period.

    Reporting group title
    Cohort 1B NAITgam 1000 IU
    Reporting group description
    		 Participants received a single IV dose of NAITgam 1000 IU on Day 1 followed by a HPA-1a positive platelet transfusion on Day 8, with a 25-week safety follow-up period from day of NAITgam administration.

    Reporting group title
    Cohort 1 and Cohort 1B Placebo
    Reporting group description
    		 Cohort 1: Participants received a single IV administration of placebo 1 hour following completion of a HPA-1a positive platelet transfusion, followed by a 24-week safety follow-up period. Cohort 1B: Participants received a single IV administration of placebo on Day 1 followed by a HPA-1a positive platelet transfusion on Day 8, with a 25-week follow- up period from day of placebo administration.

    Primary: Elimination half-life (t 1/2) of transfused HPA-1a positive platelets

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    End point title
    		 Elimination half-life (t 1/2) of transfused HPA-1a positive platelets [1]
    End point description
    Half-life of transfused HPA-1a positive platelets in circulation in HPA-1a negative participants after IV administration of NAITgam or placebo, determined by flow cytometry. Proof of concept was elimination of HPA-1a positive platelets by 10-fold or greater compared to placebo, as defined by platelet elimination half-life.
    End point type
    Primary
    End point timeframe
    Cohort 1: Day 1 to Day 8 Cohort 1B: Day 8 to Day 15
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No statistical analysis was performed for this endpoint.
    End point values
    		 Cohort 1 NAITgm 1000 IU Cohort 1B NAITgam 1000 IU Cohort 1 and Cohort 1B Placebo
    Number of subjects analysed
    6
    3
    3
    Units: Hours
        median (full range (min-max))
    0.33 (0.28 to 0.37)
    0.59 (0.42 to 2.60)
    59.94 (49.34 to 71.11)
    No statistical analyses for this end point

    Secondary: Serious treatment emergent adverse events (serious TEAEs) and non-serious TEAEs

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    End point title
    		 Serious treatment emergent adverse events (serious TEAEs) and non-serious TEAEs
    End point description
    Treatment-emergent adverse events (TEAEs) were defined as adverse events that occurred from the time of study drug administration through 25 weeks of follow-up. The incidence of TEAEs were reported by seriousness (serious and non-serious). Safety analysis set (SAS) comprised of all participants randomly assigned to study intervention and who received platelets and/or study drug.
    End point type
    Secondary
    End point timeframe
    Cohort 1: 24 weeks from day of study drug administration Cohort 1B: 25 weeks from day of study drug administration
    End point values
    		 Cohort 1 NAITgm 1000 IU Cohort 1B NAITgam 1000 IU Cohort 1 and Cohort 1B Placebo
    Number of subjects analysed
    6
    3
    3
    Units: Participants
    number (not applicable)
        Serious TEAEs
    0
    0
    0
        non-Serious TEAEs
    5
    3
    3
    No statistical analyses for this end point

    Secondary: Vital signs, clinical laboratory values and electrocardiogram (ECG)

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    End point title
    		 Vital signs, clinical laboratory values and electrocardiogram (ECG)
    End point description
    Vital signs included pulse rate and blood pressure measurements. Clinical laboratory evaluations included of hematology, clinical chemistry, coagulation, and urinalysis; ECGs
    End point type
    Secondary
    End point timeframe
    Cohort 1: 24 weeks from day of study drug administration Cohort 1B: 25 weeks from day of study drug administration
    End point values
    		 Cohort 1 NAITgm 1000 IU Cohort 1B NAITgam 1000 IU Cohort 1 and Cohort 1B Placebo
    Number of subjects analysed
    6
    3
    3
    Units: Participants
        number (not applicable)
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Assessment for anti-HPA-1a Alloantibodies

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    End point title
    		 Assessment for anti-HPA-1a Alloantibodies
    End point description
    Assessment for alloimmune response to HPA-1a positive platelets.
    End point type
    Secondary
    End point timeframe
    Cohort 1: 24 weeks from day of study drug administration Cohort 1B: 25 weeks from day of study drug administration
    End point values
    		 Cohort 1 NAITgm 1000 IU Cohort 1B NAITgam 1000 IU Cohort 1 and Cohort 1B Placebo
    Number of subjects analysed
    6
    3
    3
    Units: Participants
        number (not applicable)
    0
    0
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Cohort 1: 24 weeks from day of study drug administration Cohort 1B: 25 weeks from day of study drug administration
    Adverse event reporting additional description
    Serious TEAEs and TEAEs were collected based on all participants randomly assigned to study intervention and who received platelets and/or study drug.
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23.1
    Reporting groups
    Reporting group title
    Cohort 1 1000 International Units (IU) NAITgam
    Reporting group description
    		 Participants received 1000 IU NAITgam IV 1 hour after completion of HPA-1a positive platelet transfusion on Day 1, with a 24-week follow-up period.

    Reporting group title
    Cohort 1B 1000 IU NAITgam
    Reporting group description
    		 Participants received 1000 IU NAITgam IV 7 days prior to HPA-1a positive platelet transfusion, with a 25-week follow-up period from day of NAITgam administration.

    Reporting group title
    Placebo (Cohort 1 and Cohort 1B)
    Reporting group description
    		 For Cohort 1, participants received single IV administration of placebo 1 hour after completion of HPA-1a positive platelet transfusion with a 24-week follow-up period. For Cohort 1B, participants received single IV administration of placebo 7 days prior to HPA-1a positive platelet transfusion, with a 25-week follow-up period from day of NAITgam administration.

    Serious adverse events
    		 Cohort 1 1000 International Units (IU) NAITgam Cohort 1B 1000 IU NAITgam Placebo (Cohort 1 and Cohort 1B)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Cohort 1 1000 International Units (IU) NAITgam Cohort 1B 1000 IU NAITgam Placebo (Cohort 1 and Cohort 1B)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    5 / 6 (83.33%)
    3 / 3 (100.00%)
    3 / 3 (100.00%)
    Injury, poisoning and procedural complications
    Skin laceration
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Muscle strain
         subjects affected / exposed
    2 / 6 (33.33%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    2
    0
    0
    Vascular disorders
    Vasodilatation
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Hypertension
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Nervous system disorders
    Sciatica
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Headache
         subjects affected / exposed
    2 / 6 (33.33%)
    2 / 3 (66.67%)
    1 / 3 (33.33%)
         occurrences all number
    2
    2
    3
    Migraine
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Ageusia
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Anosmia
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    General disorders and administration site conditions
    Oedema peripheral
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Oropharyngeal pain
         subjects affected / exposed
    2 / 6 (33.33%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    2
    0
    0
    Nasal polyps
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Psychiatric disorders
    Alcoholic hangover
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Musculoskeletal and connective tissue disorders
    Joint swelling
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Neck pain
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Coccydynia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Tendonitis
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Muscle tightness
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Osteonecrosis
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
         occurrences all number
    0
    1
    0
    Myalgia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0
    0
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    1
    0
    1
    Otitis media
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Sinusitis
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Bronchitis bacterial
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1
    Gingivitis
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
         occurrences all number
    0
    0
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    12 Jun 2020
    Updates to address review comments from the Ethics Committee and for administrative/editorial changes.
    29 Jun 2020
    Updates to address Health Authority review comments and for administrative/editorial changes.
    07 Aug 2020
    Updates to harmonize both Ethics Committee and Health Authority review comments in single protocol.
    11 May 2021
    Inclusion of Cohort 1B to characterize the duration of the observed pharmacodynamic effect of NAITgam and to assess whether NAITgam retains the ability to efficiently clear platelets in the days after administration of NAITgam.
    18 Oct 2021
    Eligibility criteria updated for clarification purposes; editorial changes to Guidance on remote Source Data Verification; Serious Adverse Event reporting guidance updated to align with General Data Protection Regulation guidelines.

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported. Trial ended prematurely having established proof-of-concept at the lowest NAITgam dose (1000 IU)
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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