Efficacy of Alphanate FVIII/VWF Concentrate in Type 3 Von Willebrand Patients

Study Identifier:
GBI 07-03
ClinicalTrials.gov Identifier:
NCT00555555
EudraCT Identifier:
N/A
Unmapped

Study Details

Medical Condition
  • Von Willebrand Disease
Study Drug
  • Biological: Alphanate SD/HT
Date
Sep 2007 - Dec 2028
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 7+ years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Male or female 7 years of age or older
  • The subject has been diagnosed of inherited VWD of Type 3 as determined by subject's medical records.
  • The subject needs a surgical procedure (at least 10 surgical procedures have to be considered as "Major" according to the criteria of the protocol).
  • The subject is expected to respond to exogenously administered FVIII/VWF according to Investigator's judgment.
  • The subject freely gives written informed consent. Patients who are not legally permitted to provide written consent must sign a form of assent for study participation, and written consent must be provided by a parent or legal guardian.
Exclusion Criteria
  • The subject has been diagnosed of acquired VWD.
  • The subject is known to have history of intolerance to any Alphanate® containing substance.
  • The subject is known to have history of anaphylactic reaction(s) to blood or blood components.
  • Liver function tests (AST, ALT, bilirubin) \> 2.5 x upper limit of normal (ULN).
  • Renal function test (creatinine, BUN) \> 1.5 x ULN.
  • The subject is known or suspected to have present or past inhibitor activity (antibodies) directed against FVIII or VWF.
  • The subject is known to abuse alcohol or illicit drug use within the past 12 months.
  • The subject is participating in another clinical study involving an investigational treatment, or participated within the past 4 weeks (except if the patient is participating in another Alphanate® study). Studies consisting of data and blood sampling collections on a regular or long-term basis are exempt from this exclusion.
  • The subject is unlikely to adhere to the protocol requirements of 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

No locations found.