Retrospective study of lyso-Gb3 plasma concentration in Fabry patients receiving ERT/Replagal

Retrospective study of lyso-Gb3 plasma concentration in Fabry patients receiving ERT/Replagal - Retrospective study of lyso-Gb3 plasma concentration in Fabry patients receiving ERT/Replagal

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

JPRN

Registry ID

JPRN-UMIN000017152

Enrollment

100

Registered

2015-04-17

Start date

2015-04-17

Completion date

Unknown

Last updated

2026-06-29

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

Conditions

Fabry disease

Interventions

None listed

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Patients should meet all the following criteria; 1) Patients with Fabry disease who participated in the Replagal Post Marketing Surveillance and have no history of any enzyme replacement therapies before the treatment with Replagal 2) Patients whose stored blood sample taken before the treatment with Replagal was at least 0.3 mL 3) Patients whose any stored blood samples taken after initiation of the treatment with Replagal were at least 0.2 mL 4) Patients who are fully informed of and understand the objectives, procedures, and possible risks of the study and provide the written voluntary consent to participate in the study. For patients aged 15 years or older and younger than 20 years at informed consent, consent should be obtained from the patients'legal acceptable representatives in addition to the consent from the patients themselves. For patients younger than 15 years at informed consent, consent should be obtained from the patients'legal acceptable representatives in addition to the assent from the patients themselves.

Exclusion criteria

Exclusion criteria: The following patients should not be enrolled to the study; Patients who are considered ineligible for the study by the investigator

Design outcomes

Primary

Measure	Time frame
As a diagnostic bio-marker; 1) Plasma concentration of lyso-Gb3 2) Plasma concentration of lyso-Gb3 analogs (lyso-Gb3 [-2], lyso-Gb3 [+34] and other analogs) 3) GLA activity in plasma As a therapeutic bio-marker;; 1) Plasma concentration of lyso-Gb3 and the change from baseline in the concentration after initiation of the treatment with Replagal 2) Plasma concentration of lyso-Gb3 analogs (lyso-Gb3 [-2], lyso-Gb3 [+34]) and other analogs) after initiation of the treatment with Replagal, and the change from baseline in the analog concentration after initiation of the treatment with Replagal	—

Measure

Time frame

As a diagnostic bio-marker; 1) Plasma concentration of lyso-Gb3 2) Plasma concentration of lyso-Gb3 analogs (lyso-Gb3 [-2], lyso-Gb3 [+34] and other analogs) 3) GLA activity in plasma As a therapeutic bio-marker;; 1) Plasma concentration of lyso-Gb3 and the change from baseline in the concentration after initiation of the treatment with Replagal 2) Plasma concentration of lyso-Gb3 analogs (lyso-Gb3 [-2], lyso-Gb3 [+34]) and other analogs) after initiation of the treatment with Replagal, and the change from baseline in the analog concentration after initiation of the treatment with Replagal

—

Countries

Japan

Contacts

Public ContactTadayasu Togawa

Meiji Pharmaceutical University Department of Functional Bioanalysis

tadayasu@my-pharm.ac.jp042-495-8992

Outcome results