NL-OMON46191
Completed
Not Applicable
An observational, prospective, multi-centre, natural history study of patients with mucopolysaccharidosis type IIIA - Natural history study of patients with MPS IIIA
ysogene SA0 sites5 target enrollmentTBD
Overview
- Phase
- Not Applicable
- Intervention
- Not specified
- Conditions
- or Mucopolysaccharidosis type III (MPS III)
- Sponsor
- ysogene SA
- Enrollment
- 5
- Status
- Completed
- Last Updated
- 2 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •\* Documented MPS IIIA diagnosis.
- •\* Children up to and including 9 years of age.
- •\* The patient is sufficiently able, in the opinion of the Investigator, to adhere to the study visit schedule and other protocol requirements.
- •\* The patient's parent(s) or legal guardian(s) has signed written informed consent, according to the local regulations and after all relevant aspects of the study have been explained and discussed.
Exclusion Criteria
- •\* The patient is participating in a clinical trial of any potential disease\-modifying investigational medicinal product or taking high dose (\>100 mg/kg/day) synthetic Genistein (patients on low dose or naturally derived genistein can be included in this study).
- •\* The patient has received a hemapoietic stem cell or bone marrow transplant or gene therapy.
- •\* The patient has received enzyme replacement therapy.
- •\* Homozygous or compound heterozygous for the S298P mutation or the investigator and/or trial steering committee considers the patient not to have the classical severe form of MPS IIIA.
- •\* Individuals with rare and unrelated serious comorbidities e.g. Down syndrome, intraventricular haemorrhage in the new\-born period, or extreme low birth weight (\<1500 grams).
- •\* Visual or hearing impairment sufficient, in the clinical judgment of the investigator, to preclude cooperation with neurodevelopmental testing. Use of hearing aids is permitted.
Outcomes
Primary Outcomes
Not specified
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