*SMART in MPS-IH
Research type
Research Study
Full title
An Open-Label, Phase 1/2, Non-comparative, Clinical Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of MTL-CEBPA in Paediatric Participants with Mucopolysaccharidosis Type 1H (MPS1H, Hurler Syndrome) (SMART in MPS1H)
IRAS ID
1005674
Contact name
Nagy Habib
Contact email
Sponsor organisation
MiNA Alpha Ltd.
Eudract number
2022-001379-15
ISRCTN Number
ISRCTN10369994
Research summary
Mucopolysaccharidosis type IH (MPS1H or Hurler Syndrome) is an incurable rare genetic condition that occurs both in males and females. It is caused by lack of an enzyme (α-L-iduronidase) resulting in accumulation of certain sugars in many organs, including the skeleton, heart, and respiratory systems, and if untreated leads to early death. There is still a high, unmet medical need for improved treatment of MPS1H.
This study investigates the safety of a ‘small activating RNA’ treatment called MTL-CEBPA (which is not yet approved by regulatory authorities) given as a 30-minute intravenous infusion in children with MPS1H to establish a dose which is both well tolerated and boosts the production of the α-L-iduronidase enzyme.
6 children aged 12 to 16 years inclusive, will be recruited into Cohort 1, and 6 children aged 4 to 11 years will be recruited into Cohort 2. All children will have successfully undergone an allogeneic haematopoietic stem cell transplantation (bone marrow transplant) at least six months previously. Three increasing single dose levels of MTL-CEBPA will be investigated in 2 children each. The optimum dose will then be given at either 2-, 3- or 4-weekly intervals for six months. The chosen dosing interval will be determined from the single dose phase. At the end of the 6 months of treatment, children who appear to have benefitted and had no significant side effects can choose to continue receiving long-term treatment in an open-label extension.
During the study, patients will undergo assessments which include physical examinations, cognitive, motor and behavioural assessments, blood, urine, x-rays, electrocardiogram, ultrasound, walking and breathing tests. Children and their families will also be asked to complete questionnaires about their day-to-day experience of living with MPS1H.
The research will be carried out at Manchester University Hospitals NHS Foundation Trust.REC name
London - Hampstead Research Ethics Committee
REC reference
22/LO/0665
Date of REC Opinion
22 Nov 2022
REC opinion
Further Information Favourable Opinion