Safety and Efficacy Clinical Study of Severe Hemophilia B Treatment With KL001

Study Identifier:
CP-KL001-001/01-NCT06125756
ClinicalTrials.gov Identifier:
NCT06125756
EudraCT Identifier:
N/A
Will Be Recruiting

Study Details

Medical Condition
  • Hemophilia B Without Inhibitor(s)
Study Drug
  • Drug: Low dose KL001
  • Drug: Middle dose KL001
  • Drug: High dose KL001
Date
Dec 2023 - Sep 2024
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Male
Age: 12+ years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Inclusion Criteria:
  • Male ≥12 years of age.
  • Moderate/severe or severe hemophilia B (baseline FIX activity ≤2% of normal or documented history of FIX activity ≤2%).
  • At least 3 bleeding episodes per year that require on-demand treatment with FIX or are treated with a prophylactic regimen of FIX.
  • At least 100 days exposure history to recombinant or plasma-derived FIX protein products.
  • No neutralizing antibodies to exogenous FIX protein products.
  • Willing and able to comply with study procedures and requirements.
  • Exclusion Criteria:
  • Suffering from chronic inflammatory muscle disease.
  • Positive in Hepatitis B or Hepatitis C.
  • Infection with HIV-1 or HIV-2 and CD4+T cell count ≤ 200/ μ L.
  • History of thrombosis or susceptibility to thrombosis.
  • Current or previous participation in another gene therapy study.
  • Has any other significant medical condition that the investigator feels would be a risk to the subject or would impede 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.