Effect of dietary suppletion with genestein in patients with the MPS III

Effect of dietary suppletion with genestein in patients with the MPS III - genestein in MPS III

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON32555

Enrollment

Registered

2009-01-19

Start date

2009-01-01

Completion date

Unknown

Last updated

2024-05-06

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

MPS III Sanfilippo

Interventions

Patients will receive capsules for 2 periods of 6 months, one period the capsules will contain genestein, the other 6 months period patients will receive placebo.

Eligibility

Age

2 Years to 99 Years

Inclusion criteria

Inclusion criteria: The patient should have a biochemically confirmed deficiency of heparan-N-sulfatase (MPSIIIA) or A-N-acetylglucosaminidase (MPS IIIB) or Acetyl CoA:*-glucosaminide N-acetyltransferase (MPS IIIC).

Exclusion criteria

Exclusion criteria: The parent/legal representative is unwilling to participate Patient is already using genestein Estimated developmental age is below 1 year Patient underwent umbrilical cord blood cell transplantation

Design outcomes

Primary

Measure	Time frame
-urinary GAG excretion	—

Secondary

Measure	Time frame
-serum heparan sulphate levels -GAG-accumulation in skin biopsies. -hair morphology -behaviour -cognitive function	—

Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)