Clinical Research Studies

Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

• Conditions:
  Gm2 Gangliosidosis
• Age: Between 2 years - 99 years
• Gender: Male or Female

• Other Inclusion Criteria:
  

  1. Subjects have diagnosis of late-onset GM2 gangliosidosis
  2. Subjects ability to perform the 9-hole peg test (9-HPT)
  3. If subjects have history of seizures, they are well controlled under appropriate medication
  4. Subjects who are cooperative, able to ingest or chew and swallow oral medication, willing to travel to a study site (if applicable), and able to comply with all aspects of the study, including all assessments, according to the Investigator’s judgment
  5. Subjects who will use contraception methods as per the protocol
  6. For the juvenile and adolescent secondary population, subjects must have body weight =10 kg at the time of signing the informed consent.

  
  

You may not be eligible for this study if the following are true:

• 1. Subjects with clinical features of Tay-Sachs or Sandhoff disease
  2. Subjects who cannot understand and perform all study assessments with the exception of AIDS.
  3. Subjects with any clinically relevant medical disorders
  4. Subjects who have documented diagnosis of any of the following infections: hepatitis B, hepatitis C, human immunodeficiency virus 1 or 2
  5. A history of drug and/or alcohol abuse within the past year prior to the first screening visit.
  6. Subjects scheduled for in-patient hospitalization including elective surgery during the study
  7. Subjects with a cortical cataract more than one-quarter of the lens circumference (Grade cortical cataract-2) or a posterior subcapsular cataract >2 mm (Grade posterior subcapsular cataract-2). Participants with nuclear cataracts will not be excluded.
  8. Subjects uses invasive ventilatory support
  9. Subjects receives treatment with anticoagulants currently
  10. Received SRT within 3 months before study enrollment
  11. Received strong or moderate inducers or inhibitors of CYP3A4 within 14 days or 5 half-lives, whichever is longer, prior to study enrolment
  12. Currently receiving potentially cataractogenic medications as or any medication that may worsen the vision of participant with cataract
  13. Participating in another investigational interventional study
  14. Subjects who have renal insufficiency
  15. Subjects who use IMP before study enrollment
  16. Participant not suitable for participation, whatever the reason, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures.