Cystadane in the treatment of AG

Phase 1

• Conditions: Aspartylglucosaminuria; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2017-000645-48-FI
• Lead Sponsor: Minna Laine

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2017-05-31
• Last Update Posted: 16 October 2017

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

Patients below the age of 15 years who have aspartylglucosaminuria
Preferably homozygous for the Finnish major AGU mutation, AGU-Fin-major
Written informed consent of the parents
No known hypersensitivity or allergy against betain
Compliance of the patient

Are the trial subjects under 18? yes
Number of subjects for this age range: 15
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Age above 15 years
Patients who do not carry at least one allele of the Finnish major mutation
Known adverse reactions against Betain or components of Cystadane
Known history of cerebral oedema
Participating in other interventional clinical studies
Patients who have undergone bone marrow tranplantation
Co-medication which would interfere with administration of Betain

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified