RESTORE
Research type
Research Study
Full title
An Interventional, Prospective Open-Label Study of Immunosuppressive Therapies to Mitigate Immune-Mediated Loss of Therapeutic Response to Asfotase Alfa (STRENSIQ®) for Hypophosphatasia (RESTORE)
IRAS ID
1008095
Contact name
Claudia Tomas
Contact email
Sponsor organisation
Alexion Pharmaceuticals, Inc.
Eudract number
2022-502793-17
Research summary
The administration of biological medicines to patients carries a risk that the patients produce antibodies, a protein produced by the body's immune system when it detects harmful substances, that prevent said medicines from effective treatment. This has been seen in the case of asfotase alfa treating hypophosphatasia.
This study will evaluate if immunosuppressive therapies (ISTs), methotrexate and rituximab, given with asfotase alfa (STRENSIQ®) can help reduce the body's immune response so that asfotase alfa can better treat hypophosphatasia. A third IST, bortezomib, can be added if the other two do not given enough benefit.
This study will specifically evaluate the effect of ISTs impact of asfotase alfa treatment in paediatric patients with hypophosphatasia. This follows the clinical development of asfotase alfa, which is indicated to treat patients with hypophosphatasia that started in childhood. Any potential risks associated with administration of the ISTs are justified by the anticipated benefits that may be afforded to asfotase alfa-treated participants with hypophosphatasia who are experiencing immune-mediated loss of effectiveness.
REC name
Wales REC 3
REC reference
23/WA/0349
Date of REC Opinion
15 Feb 2024
REC opinion
Further Information Favourable Opinion