CompletedNot Applicable

GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)

United States, Brazil, France31 participantsStarted 2022-02-22

Plain-language summary

The study aims to characterize prospectively longitudinal progression of neurological domains in GM1 and GM2 Gangliosidosis patients with high-quality standards (GCP compliant).

Who can participate

Age range2 Years – 20 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Genetically confirmed GM1 Gangliosidosis or genetically confirmed Tay-Sachs or Sandhoff disease * Onset of neurological symptoms on or after the patient's first birthday * Achieved 12-month developmental milestones at normal developmental time points as per Principal Investigator's judgement * Abnormal gait and/or speech disturbance Exclusion Criteria: * Patients who have received (within 6 months before screening), are currently receiving or are planned to receive (within the following 6 months) gene therapy, stem cell transplantation, experimental drugs, or any drug, which, in the Investigator´s opinion, may (have) interfere(d) with disease progression

What they're measuring

Change in the Gait 9-point item score of the Scale for Assessment and Rating of Ataxia (SARA)

Timeframe: 0-4 years

Change in the Speech 7-point item score of SARA

Timeframe: 0-4 years

Trial details

NCT IDNCT05109793

SponsorAzafaros A.G.

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2025-09-16

View on ClinicalTrials.gov More GM1 Gangliosidosis trials