Gangliosidoses PROspective Neurological disease TrajectOry Study
Research type
Research Study
Full title
Prospective Longitudinal Study of Neurological Disease trajectory in children living with Late-Infantile or Juvenile Onset of GM1 or GM2 Glangliosidosis (PRONTO study)
IRAS ID
304102
Contact name
Ruben Giorgino
Contact email
Sponsor organisation
Azafaros B.V.
ISRCTN Number
ISRCTN00000000
Clinicaltrials.gov Identifier
Duration of Study in the UK
3 years, 10 months, 0 days
Research summary
This study will follow and repeatedly examine patients living with GM1 or GM2 gangliosidosis. This is an inherited disorder that progressively destroys nerve cells (neurons) in the brain and spinal cord. Potential participants may include children from age two to twenty and may include adults lacking capacity to consent. It is expected to follow patients between 24 and 48 months. The main goal will be to characterize the neurological disease trajectory. Tests that will be done are clinical assessments based on neurological clinical routine as well as questionnaires and passive monitoring. Patients or their parents/caregivers will be asked to complete questionnaires and a wearable patch is used to monitor the patient’s heartbeat, breathing, and movement how the patient is moving and other information that may help to better understand the disease. It is planned to recruit a total of 75 patients in several countries including France, Germany, Italy, US, Brazil and United Kingdom. At least 25 patients with GM1 and 25 with GM2 gangliosidosis are planned to be enrolled. In the United Kingdom, 2 sites are expecting to enroll around 9 patients in total.
REC name
London - Brighton & Sussex Research Ethics Committee
REC reference
21/LO/0716
Date of REC Opinion
16 Dec 2021
REC opinion
Further Information Favourable Opinion