MO41787 - Subcutaneous Emicizumab in Paediatric Haemophilia
Research type
Research Study
Full title
A PHASE IIIb, MULTICENTER, OPEN-LABEL, SINGLE-ARM STUDY TO EVALUATE THE EFFICACY, SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF SUBCUTANEOUS EMICIZUMAB IN PATIENTS FROM BIRTH TO 12 MONTHS OF AGE WITH HEMOPHILIA A WITHOUT INHIBITORS
IRAS ID
283985
Contact name
Peter Collins
Contact email
Sponsor organisation
F Hoffmann-La Roche Ltd
Eudract number
2020-001733-12
Duration of Study in the UK
8 years, 3 months, 0 days
Research summary
Haemophilia A is an X-linked recessive bleeding disorder that occurs in approximately 1 in 5000 live male births. Patients with haemophilia A have a deficiency or absence of blood coagulation factor VIII (FVIII). Haemophilia A is most commonly caused by an inherited FVIII gene mutation of the X chromosome. It occurs mostly in males with one defective copy of the relevant gene on their X chromosome.
Because an affected male will transmit a normal Y chromosome to all his sons and an abnormal X chromosome to all his daughters, his sons will not be affected and all of his daughters will be carriers. The offspring of a future mother who is a carrier will have a 50% chance to receive a mutated FVIII gene; thus, haemophilia A could be transmitted to one half of the male infants, and one-half of female infants will be carriers.
Although the primary indication for routine prophylaxis in haemophilia is to prevent joint damage and to decrease the frequency of joint and other bleeds, the high rate of intracranial haemorrhage (bleeds) (ICH) observed provide a compelling argument for the initiation of early prophylaxis in infants to prevent a first occurrence of ICH.
The administration of prophylactic factor concentrate at birth is recommended because it is highly effective to prevent bleeds.
The aim of this study is to investigate emicizumab prophylaxis (evaluating the safety, efficacy, pharmacokinetics, and pharmacodynamics of emicizumab) enrolling patients from birth to 12 months with severe haemophilia. They will receive treatment initially for 1 year and then continue receiving treatment for a long term follow-up period of 7 years.
The study will last for 8 years.
51 patients aged between birth and 12 months will be enrolled globally with 5 patients from 5 sites in the UK.
The study is sponsored by F. Hoffman La Roche
Research Summary; Version 1.0, dated 11-Dec-2020
REC name
Wales REC 3
REC reference
21/WA/0004
Date of REC Opinion
5 Feb 2021
REC opinion
Favourable Opinion