A Study of PEGylated Recombinant Factor VIII (BAX855) in Previously Untreated Young Children With Severe Hemophilia A

Study Identifier:
261203
ClinicalTrials.gov Identifier:
NCT02615691
EudraCT Identifier:
2015-002136-40
Unmapped

Study Details

Medical Condition
  • Hemophilia A
Study Drug
  • Biological: PEGylated Recombinant Factor VIII
  • Biological: ITI
Date
Nov 2015 - Oct 2024
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: N/A - 5 Years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Inclusion Criteria
  • Participant is \<6 years old at the time of screening.
  • Participant is previously untreated with \<3 exposure days (EDs) to ADVATE, BAX 855 or plasma transfusion at any time prior to screening.
  • Participant has severe hemophilia A (Factor VIII (FVIII) \<1%) as determined by the central laboratory, or a historical FVIII level \<1% as determined at any local laboratory, optionally supported by an additional FVIII gene mutation consistent with severe hemophilia A.
  • Participant is immune competent with a cluster of differentiation 4 (CD4+) count \> 200 cells per cubic millimeter (mm\^3), as confirmed by the central laboratory at screening.
  • Parent or legally authorized representative is willing and able to comply with the requirements of the protocol.
  • Additional inclusion criteria for Part B (immune tolerance induction \[ITI\]).
  • Parent or legal representative has/have voluntarily provided signed informed consent for ITI portion.
  • Participant has a confirmed positive high titer inhibitor (\> 5.00 Bethesda unit (BU)) or has a positive confirmed low titer inhibitor (greater than or equal to \[\>=\] 0.6 BU) as determined by the central laboratory based on a second repeat blood sample with
  • poorly controlled bleeding despite increased BAX 855 doses, or
  • requires bypassing agents to treat bleeding.
  • Exclusion Criteria
  • Participant has detectable FVIII inhibitory antibodies (\>=0.6 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening.
  • Participant has a history of FVIII inhibitory antibodies (\>=0.6 BU using the Nijmegen modification of the Bethesda assay or the Bethesda assay) at any time prior to screening.
  • Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease).
  • Participant has been previously treated with any type of FVIII concentrate other than ADVATE or BAX 855, or was administered ADVATE, BAX 855 or plasma transfusion for \>=3 EDs at any time prior to screening.
  • Participant receives \> two EDs of ADVATE in total during the periods prior to enrollment and during the screening period, until the baseline infusion.
  • The participant's weight is anticipated to be \<5 kilogram (kg) at the baseline visit.
  • Participant's platelet count is \<100,000 per milliliter (mL).
  • Participant has known hypersensitivity towards mouse or hamster proteins, polyethylene glycol (PEG) or Tween 80.
  • Participant has severe chronic hepatic dysfunction (eg, \>5 times upper limit of normal alanine aminotransferase \[ALT\], aspartate aminotransferase \[AST\], or a documented international normalized ratio \[INR\] \>1.5) in his medical history or at the time of screening.
  • Participant has severe renal impairment (serum creatinine \>1.5 times the upper limit of normal).
  • Participant has current or recent (\<30 days) use of other PEGylated drugs prior to study participation or is scheduled to use such drugs during study participation.
  • Participant is scheduled to receive during the course of the study a systemic immunomodulating drug (e.g. corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day or α-interferon) other than anti-retroviral chemotherapy.
  • Participant has participated in another clinical study involving an investigational product (IP) or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.
  • Parent or legally authorized representative has a medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant safety or compliance.
  • Parent, legally authorized representative or participant are a member of the team conducting this study or is in a dependent relationship with one of the study team members. Dependent relationships include close relatives (ie, children, partner/spouse, siblings, parents) as well as employees of the investigator or site personnel conducting the study.
  • Additional exclusion criteria for Part B (ITI)
  • Spontaneous disappearance of the inhibitor prior to ITI.
  • FVIII inhibitor titer \>=0.6 BU is not confirmed by a second new blood sample and determined at the central laboratory.
  • Inability or unwillingness to comply with the protocol.

Protocol Summary

This study is looking at how safe it is to switch from emicizumab to Mim8, in people with haemophilia A. Mim8 is a new medicine that is used to prevent bleeding episodes in people with haemophilia A. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). Mim8 will be injected under the skin using a pen-injector either once every week, once every two weeks or once every month. The participants will be trained in using the pen injector. The participants can choose themselves, in collaboration with the study doctor how often they get Mim8 in this study. When the participant will get their first Mim8 injection depends on their current treatment with emicizumab. The participants will get their first Mim8 injection at Visit 2. Participants will have between 6 and 27 Mim8 injections. The total number of injections participants will have depends on their dosing frequency. The study will last for about 6-12 months. While taking part in this study, there are some restrictions about what medicine participant can use. The study doctor will tell the participants more about this. In case the participants experience bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor. Female participants cannot take part if they are pregnant, breast-feeding or plan to get pregnant during the study period.

Trial Locations

Location
Status
Location
Phoenix Childrens Hospital
Phoenix, Arizona, United States, 85016
Status
N/A
Location
Kaiser Permanente Oakland M.C.
Cupertino, California, United States, 95014
Status
N/A
Location
Kaiser Permanente Oakland M.C.
Oakland, California, United States, 94611
Status
N/A
Location
Kaiser Permanente Oakland M.C.
Roseville, California, United States, 95661
Status
N/A
Location
UC Davis Health System
Sacramento, California, United States, 95817
Status
N/A
Location
Connecticut Children's Med Ctr
Hartford, Connecticut, United States, 06106
Status
N/A
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