An Observational Study to Investigate the Relationship between Plasma Lyso-Gb1 Concentration and the Treatment Outcomes of Gaucher disease patients in Japan

An Observational Study to Investigate the Relationship between Plasma Lyso-Gb1 Concentration and the Treatment Outcomes of Gaucher disease patients in Japan - Lyso-Gb1

Status

Active, not recruiting

Phases

Phase 4

Study type

Observational

Source

JPRN

Registry ID

JPRN-jRCT1031200137

Enrollment

Registered

2020-10-05

Start date

2020-10-01

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

Gaucher disease

Interventions

None listed

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. In the opinion of the investigator, the participant is capable of understanding and complying with protocol requirements. 2. The participant or, when applicable, the participant's legally acceptable representative signs and dates a written, informed consent form and any required privacy authorization prior to the initiation of any study procedures. 3. Participants who are diagnosed as Gaucher disease. 4. Patients who have been treated with velaglucerase alfa for at least 3 months.

Exclusion criteria

Exclusion criteria: Any reason that, in the Investigator's opinion, makes the patient unsuitable to participate in this study.

Design outcomes

Primary

Measure	Time frame
1.Correlation of Lyso-Gb1 plasma concentrations in number of treatment targets by therapeutic indices (hepatomegaly, splenomegaly, anemia, thrombocytopenia, bone pain, and bone crisis). Time Frame: 24 Weeks	—

Secondary

Measure	Time frame
1.Similarity of Lyso-Gb1 plasma levels in Japanese Gaucher disease patients to previously reported data from other countries Time Frame: 24 Weeks 2.Correlation between the Gaucher disease types and plasma Lyso-Gb1 concentration Time Frame: 24 Weeks 3.Correlation between the Gaucher disease pathological mutations and plasma Lyso-Gb1 concentration Time Frame: 24 Weeks 4.Similarity of Lyso-Gb1 plasma levels to hepatomegaly evaluation Time Frame: 24 Weeks 5.Similarity of Lyso-Gb1 plasma levels to splenomegaly evaluation Time Frame: 24 Weeks 6.Similarity of Lyso-Gb1 plasma levels to therapeutic achievement of anemia Time Frame: 24 Weeks 7.Similarity of Lyso-Gb1 plasma levels to hemoglobin concentration Time Frame: 24 Weeks 8.Similarity of Lyso-Gb1 plasma levels to therapeutic achievement of thrombocytopenia Time Frame: 24 Weeks 9.Similarity of Lyso-Gb1 plasma levels to blood platelet count Time Frame: 24 Weeks 10.Similarity of Lyso-Gb1 plasma levels to therapeutic achievement of bone pain Time Frame: 24 Weeks 11.Similarity of Lyso-Gb1 plasma levels to therapeutic achievement of bone crisis Time Frame: 24 Weeks	—

Measure

Time frame

1.Similarity of Lyso-Gb1 plasma levels in Japanese Gaucher disease patients to previously reported data from other countries Time Frame: 24 Weeks 2.Correlation between the Gaucher disease types and plasma Lyso-Gb1 concentration Time Frame: 24 Weeks 3.Correlation between the Gaucher disease pathological mutations and plasma Lyso-Gb1 concentration Time Frame: 24 Weeks 4.Similarity of Lyso-Gb1 plasma levels to hepatomegaly evaluation Time Frame: 24 Weeks 5.Similarity of Lyso-Gb1 plasma levels to splenomegaly evaluation Time Frame: 24 Weeks 6.Similarity of Lyso-Gb1 plasma levels to therapeutic achievement of anemia Time Frame: 24 Weeks 7.Similarity of Lyso-Gb1 plasma levels to hemoglobin concentration Time Frame: 24 Weeks 8.Similarity of Lyso-Gb1 plasma levels to therapeutic achievement of thrombocytopenia Time Frame: 24 Weeks 9.Similarity of Lyso-Gb1 plasma levels to blood platelet count Time Frame: 24 Weeks 10.Similarity of Lyso-Gb1 plasma levels to therapeutic achievement of bone pain Time Frame: 24 Weeks 11.Similarity of Lyso-Gb1 plasma levels to therapeutic achievement of bone crisis Time Frame: 24 Weeks

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Contacts

Public ContactTrial Information Contact for Clinical

Takeda Pharmaceutical Company Limited

smb.Japanclinicalstudydisclosure@takeda.com+81-6-6204-2111

Outcome results