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Efficacy and safety of treatment with N-butyl-deoxynojirimycin (NB-DNJ-miglustat) in patients with Niemann-Pick disease type C. - ND

Efficacy and safety of treatment with N-butyl-deoxynojirimycin (NB-DNJ-miglustat) in patients with Niemann-Pick disease type C. - ND

Status
Not yet recruiting
Phases
Unknown
Study type
Interventional
Source
EU CTR
Registry ID
EUCTR2006-005842-35-IT
Enrollment
Unknown
Registered
2009-10-28
Start date
2006-11-22
Completion date
Unknown
Last updated
2012-04-03

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

Conditions

NIEMANN-PICK DISEASE TYPE C MedDRA version: 9.1 Level: LLT Classification code 10029403

Interventions

Trade Name: ZAVESCA Pharmaceutical Form: Capsule, hard INN or Proposed INN: Miglustat Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 100-

Sponsors

Dipartimento di Pediatria Universita` di Napoli Federico II
Lead Sponsor

Eligibility

Sex/Gender
All

Inclusion criteria

Inclusion criteria: Diagnosis of NPC confirmed by biochemical tests (filipin staining and LDL-cholesterol esterification) and/or molecular analysis Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: Patients unable to take the drug Bad compliance to study procedures Patients with renal failure Fertile women that do not consent to oral contraceptives during the study and until three months after discontinuation of treatment

Design outcomes

Primary

MeasureTime frame
Main Objective: Efficacy of treatment with N-butyl-deoxynojirimycin (NB-DNJ-miglustat) in patients with Niemann-Pick disease type C.;Secondary Objective: Safety of treatment with N-butyl-deoxynojirimycin (NB-DNJ-miglustat) in patients with Niemann-Pick disease type C.;Primary end point(s): Stop of progression or improvement of the neurological involvement Stop of progression or improvement of the swallowing abnormalities—

Countries

Italy

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026