A Phase 2/3 Study of DNL310 on Neuronopathic or non-Neuronopathic Mucopolysaccharidosis Type II
Research type
Research Study
Full title
A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II
IRAS ID
1005494
Contact name
Nadia Ono
Contact email
Sponsor organisation
Denali Therapeutics Inc.
Eudract number
2021-005200-35
ISRCTN Number
ISRCTN11652897
Research summary
Mucopolysaccharidosis type II (MPS II or Hunter Syndrome) is a rare genetic condition that occurs almost exclusively in boys. MPS II is caused by lack of an enzyme resulting in accumulation of certain sugars in the body, causing abnormalities in many organs, including the skeleton, heart, and respiratory systems. In severe cases, this leads to early death.
There is no cure for MPS II. Approved enzyme replacement therapies (ERT) may improve some symptoms of MPS II, especially if started early in the disease. However, as standard of care ERT cannot cross the blood–brain barrier, it does not treat the cognitive impairment in patients with central nervous system (CNS) symptoms. There is still a high, unmet medical need for improved treatment of MPS II.
DNL310 is an investigational medicine (not yet approved by regulatory authorities). DNL310 will be given intravenously once a week. If DNL310 works as expected, it may help to reduce CNS and physical symptoms of MPS II.
Study DNLI-E-0007 is a Phase 2/3 multiregional, two-arm, double-blind, randomized, active (standard-of-care)–controlled study of the efficacy and safety of DNL310, an investigational CNS–penetrant ERT for MPS II. Approximately 54 participants aged 2 to under 17 years with MPS II who meet the study eligibility criteria will be enrolled.
During the study participants will undergo assessments which include physical exams, cognitive and behavioural assessments, blood, urine, cerebrospinal fluid analysis, hearing test, electrocardiogram, ultrasound and scales and questionnaires.REC name
South Central - Berkshire Research Ethics Committee
REC reference
22/SC/0156
Date of REC Opinion
22 Jun 2022
REC opinion
Further Information Favourable Opinion