Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS)

Study Identifier:
Pro00103838
ClinicalTrials.gov Identifier:
NCT04278404
EudraCT Identifier:
N/A
Recruiting

Study Details

Medical Condition
  • Ignored
  • Unmapped
  • Hemophilia
Study Drug
  • Drug: The POP02 study is collecting bodily fluid samples (i.e., whole blood, effluent samples) of children prescribed the following drugs of interest per standard of care:
Date
Mar 2020 - Sep 2026
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 0 - 20 Years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Inclusion Criteria:
  • Participant is \< 21 years of age
  • Parent/ Legal Guardian/ Adult Participant can understand the consent process and is willing to provide informed consent/HIPAA:
  • (a) Participant is receiving one or more of the study drugs of interest at the time of enrollment or (b) Participant is NOT receiving one or more of the study drugs of interest but is SARS-COV-2 positive within 60 days prior to enrollment
  • Exclusion Criteria:
  • Participant has a known pregnancy
  • Below exclusion criteria apply only to:
  • Participants receiving one or more of the study drugs of interest at the time of enrollment, DOI administration or PK sampling: (Refer to DOI specific appendices for details on enrollment cohort specifications and additional eligibility criteria)
  • Has had intermittent dialysis within previous 24 hours
  • Has had a kidney transplant within previous 30 days
  • Has had a liver transplant within previous 1 year
  • Has had a stem cell transplant within previous 1 year
  • Has had therapeutic hypothermia within previous 24 hours
  • Has had plasmapheresis within the previous 24 hours
  • Has a Ventricular Assist Device
  • Has any condition which would make the participant, in the opinion of the investigator, unsuitable for the study

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 Children's Hospital
Phoenix, Arizona, United States, 85016
Status
Active, not recruiting
Location
Arkansas Children's Hospital Research Institute
Little Rock, Arkansas, United States, 72202
Status
Recruiting
Location
University of California, Los Angeles Medical Center
Los Angeles, California, United States, 90095
Status
Recruiting
Location
Lucile Packard Children's Hospital
Palo Alto, California, United States, 94304
Status
Recruiting
Location
Colorado University Denver
Aurora, Colorado, United States, 80045
Status
Recruiting
Location
The Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Status
Recruiting
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