An Open-Label Extension study of DNL310 in MPS II patients
Research type
Research Study
Full title
An Open-Label Extension to Investigate the Long-Term Safety, Tolerability, and Efficacy of DNL310 in Patients With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007
IRAS ID
1008391
Contact name
Nadia Ono
Contact email
Sponsor organisation
Denali Therapeutics Inc.
ISRCTN Number
ISRCTN11652897
Research summary
Mucopolysaccharidosis type II (MPS II or Hunter Syndrome) is a rare genetic condition which mainly affects 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, brain and respiratory systems. There is no cure for MPS II. and in severe cases, this leads to early death.
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 (a network of blood vessels and tissues that prevent harmful substances from reaching the brain), 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 that is designed to cross the blood brain barrier. Clinical trials are being carried out to determine whether DNL310 may help to reduce CNS and physical symptoms of MPS II. DNL310 will be given intravenously once a week.
DNLI-E-0008 is an open-label extension study to investigate the long-term safety, tolerability, and efficacy of DNL310 in patients with MPS II from study DNLI-E-0002 or Study DNLI-E-0007.
About 99 participants will take part in the DNLI-E--008 study. This study will be run at about 46 study sites in about 17 countries. The total duration of each participant’s involvement in the study will be approximately 5 years.
During the study participants will undergo assessments which include physical exams, cognitive and behavioural assessments, blood, urine, cerebrospinal fluid analysis, hearing test, electrocardiogram, echocardiogram, magnetic resonance imaging (MRI), and quality of life scales and questionnaires.
REC name
South Central - Hampshire A Research Ethics Committee
REC reference
23/SC/0351
Date of REC Opinion
22 Nov 2023
REC opinion
Further Information Favourable Opinion