Efficacy of dietary suppletion genestein in patients with the MPS III (A follow-up study)

Efficacy of dietary suppletion genestein in patients with the MPS III (A follow-up study) - Follow-up study genestein in MPS III

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON35077

Enrollment

Registered

2010-06-28

Start date

2010-07-01

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

All participating patients will receive genestein capsules (10mg/kg) for a period of 12 months.

Eligibility

Age

2 Years to 99 Years

Inclusion criteria

Inclusion criteria: The patient should have a biochemically confirmed deficiency of heparan-N-Sulphatase (MPS IIIA) or A-N-acetylglucosaminidase (MPS IIIB) or Acetyl CoA: alpha-glucosaminide N-acetyltransefrase (MPS IIIC). All patients who participated in the placebo controlled cross-over trial with genestein are allowed to participate in this study.

Exclusion criteria

Exclusion criteria: The parent/legal reprensentative is unwilling to participate Patient underwent umbilical cord blood cell transplantation

Design outcomes

Secondary

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

Primary

Measure	Time frame
- urinary GAG excretion	—

Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)