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A Study Measuring Substances Potentially Indicating Bone Problems in Adults With Type 1 Gaucher Condition

NCT ID: NCT05702814

Last Updated: 2024-12-11

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

125 participants

Study Classification

OBSERVATIONAL

Study Start Date

2023-03-27

Study Completion Date

2024-05-24

Brief Summary

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Substances in the body, so-called biomarkers, can help predict the severity of Gaucher disease (GD)-related bone problems in adults. The main aim of the study is to determine if certain biomarkers found in the body at the time of diagnosing GD can help predict the risk of bone problems after 4-5 years.

There is no treatment involved in this study. The study will review previously collected participants' data using a database. Data from both adults with type 1 Gaucher condition as well as healthy adults will be compared.

Detailed Description

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This is a non-interventional and retrospective study of participants with GD1 and healthy participants whose data is available in the Aragon Health Systems Biobank (BSSA) from the date of diagnosis to 4-5 years after diagnosis. The main objective of this study is to validate the prognosis value of a set of potential biomarkers related to bone disease in participants with GD1.

The study will enroll approximately 120 participants, and it would be divided into 4 groups as given below:

* Group A: GD1 with Low-Normal Bone Disease
* Group B: GD1 with Mild Bone Disease
* Group C: GD1 with Severe Bone Disease
* Group D: Healthy Participants

This study will have a retrospective data collection from the date of diagnosis of GD1 until 4-5 years from diagnosis by using data available in BSSA.

This single-centre trial will be conducted in Spain. The overall time for data collection in this study will be approximately 9 months.

Conditions

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Gaucher Disease

Keywords

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Drug Therapy

Study Design

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Observational Model Type

CASE_CONTROL

Study Time Perspective

RETROSPECTIVE

Study Groups

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Group A: GD1 with Low-normal Bone Disease

Participants with GD1 with low-normal bone disease whose samples are available for analysis in BSSA will be collected retrospectively up to approximately 5 years from diagnosis.

No Intervention

Intervention Type OTHER

As this is an observational study, no intervention will be administered.

Group B: GD1 with Mild Bone Disease

Participants with GD1 with mild bone disease whose samples are available for analysis in BSSA will be collected retrospectively up to approximately 5 years from diagnosis.

No Intervention

Intervention Type OTHER

As this is an observational study, no intervention will be administered.

Group C: GD1 with Severe Bone Disease

Participants with GD1 with severe bone disease whose samples are available for analysis in BSSA will be collected retrospectively up to approximately 5 years from diagnosis.

No Intervention

Intervention Type OTHER

As this is an observational study, no intervention will be administered.

Group D: Healthy Participants

Healthy Participants whose samples are available for analysis in BSSA will be collected retrospectively up to approximately 5 years.

No Intervention

Intervention Type OTHER

As this is an observational study, no intervention will be administered.

Interventions

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No Intervention

As this is an observational study, no intervention will be administered.

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Participants with confirmed diagnosis of GD1.
* Determination of Glucocerebrosidase (GCase) blood activity at diagnosis of GD1.
* DNA analysis result demonstrating pathogenic variants in the Glucocerebrosidase gene (GBA1) gene.
* Available data of clinical state at diagnosis and at 4-5 years from diagnosis, including S-MRI, Dual energy x-ray absorptiometry (DXA), and GD1 severity scoring system (GD1-DS3) indexes (or data to calculate it).

Exclusion Criteria

• Evidence of hepatitis B, hepatitis C infection or other chronic infectious diseases.

For Healthy Volunteers


* Evidence of hepatitis B, hepatitis C infection or other chronic infectious diseases.
* Evidence of bone disease.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Takeda

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

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Fundación Española para el Estudio y Tratamiento de la Enfermedad de Gaucher (FEETEG)

Zaragoza, Aragon, Spain

Site Status

Countries

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Spain

Other Identifiers

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TAK-669-5010

Identifier Type: -

Identifier Source: org_study_id