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
Conditions
Interventions
Product Name: NAITgam
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Anti-Human Platelet Antigen-1a Immune Globulin
Current Sponsor code: NAITgam Bulk
Other descriptive name: Human
Sponsors
Rallybio, IPA, LLC
Eligibility
Sex/Gender
Male
Inclusion criteria
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) =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. History of hypersensitivity to platelet concentrates or human plasma proteins 2. IgA levels 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 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;Secondary Objective: - To evaluate the safety of a single dose of NAITgam - To monitor alloimmune response - To establish the pharmacokinetic (PK) profile of NAITgam;Primary end point(s): half-life (t1/2) of transfused platelets;Timepoint(s) of evaluation of this end point: Baseline until latest day 7 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary 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)- ;Timepoint(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 | — |
Countries
Germany
Contacts
Public ContactCOO
Rallybio, IPA, LLC
Outcome results
None listed