Immune Tolerance Induction with Methotrexate in Hurler Syndrome
Research type
Research Study
Full title
A Single Centre Study Investigating the Safety and Efficacy of an Immune Modulation Regimen in Mitigating the Alloimmune Response to Intravenous Laronidase in Infants With Severe Mucopolysaccharidosis type I (Hurler syndrome) Prior to Haematopoietic Stem Cell Transplantation
IRAS ID
168565
Contact name
Simon Jones
Contact email
Sponsor organisation
Central Manchester University Hospitals NHS Foundation Trust
Eudract number
2015-003031-35
Duration of Study in the UK
1 years, 5 months, 21 days
Research summary
The purpose of this study is to see if methotrexate has any effect on the antibodies to enzyme replacement therapy that develop in children with Hurler syndrome, the severe form of mucopolysaccharidosis type I (MPS I).
This study is open to patients between the age of 3 months and 2½ years who have been diagnosed with Hurler syndrome and who will be receiving enzyme replacement therapy with laronidase, followed by a bone marrow transplant.
All children enrolled in the study will receive the standard treatment for Hurler syndrome which involves weekly infusions of laronidase, followed by a bone marrow transplant. In addition, all children in the study will be given a very short course of methotrexate around their first infusion.
This will be a total of three doses of oral methotrexate around their first weekly infusion of laronidase. The first dose will be given in hospital 1 hour before the infusion. The second and third doses can be given at home and these will be at 24 hours and 48 hours after the infusion respectively. The dose of methotrexate will be 0.4 mg per kilogram of body weight per dose.
After this, children will stop methotrexate and continue with the standard treatment for Hurler syndrome. They will get weekly infusions of laronidase followed by a bone marrow transplant, and will receive the same care as all children with Hurler syndrome.
REC name
HSC REC B
REC reference
15/NI/0189
Date of REC Opinion
18 Sep 2015
REC opinion
Favourable Opinion