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

JRCT ID: jRCT1031200137

Registered date:05/10/2020

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: Gaucher disease
• Date of first enrollment: 01/10/2020
• Target sample size: 20
• Study type: Observational

Outcome(s)

Primary Outcome

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 Outcome

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

Key inclusion & exclusion criteria

• Age minimum: Not applicable
• Age maximum: Not applicable
• Gender: Both

Include 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.

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

Related Information

• Primary Sponsor: Contact for Clinical Trial Information