GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma

NCT ID: NCT05992532

Last Updated: 2026-02-11

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

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Total Enrollment

210 participants

Study Classification

OBSERVATIONAL

Study Start Date

2023-05-30

Study Completion Date

2026-05-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The study of splenomegaly, and the follow-up of splenectomized patients, is one of the causes of referral of these patients to pediatric gastroenterology and oncohematology clinics, and adult internal medicine and hematology. The study and management of splenomegaly is well described among the different medical specialties to which these patients arrive. After the application of the different algorithms and the different studies that are carried out, these splenomegaly are identified as being of hepatic, infectious, inflammatory, congestive, hematological origin and primary causes. Despite these studies of splenomegaly, approximately 10-15% of these patients still remain undiagnosed.

Several studies have suggested that there is an increased frequency of MGUS (monoclonal gammopathy of undetermined significance) and/or multiple myeloma (MM) among Gaucher patients. Regarding ASMD (Acid Sphingomyelinase Deficiency), few studies have been published but it seems the 21% of patient with ASMD has MGUS and 15% ASMD patients have MGUS. Moreover, patients with MGUS and Gaucher disease (GD) are at increased risk of developing MM.

The objective of the present study is to increase the diagnostic sensitivity of these unknown splenomegalys, or unknown splenomegaly patients with MGUS or multiple myeoloma who remain in consultations, using the usual diagnostic clinical procedures of unknown splenomegaly and unknown splenectomy patients, where we include the extraction of a blood sample for dry drop test (DBS), where the determination of the enzymatic/genetic activity will be carried out for Gaucher disease (GD) and acid sphingomyelinase deficiency (ASMD) , analysis of LisoGl1 and LisoSM.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Gaucher Disease Acid SphingoMyelinase Deficiency

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

COHORT

Study Time Perspective

CROSS_SECTIONAL

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Adult patients of both sexes.
* Patients with splenomegaly (spleen palpable at ≥ 1cm from the costal margin) or splenectomy not related to any specific condition, or patients with thrombocytopenia (with or without splenomegaly). In cases where the patient is undergoing treatment for their underlying condition, the thrombocytopenia must have been present prior to the start of the treatment.
* Patient who gives their consent to participate in the study.

Exclusion Criteria

* Splenomegaly due to portal hypertension (documented by abdominal ultrasound or other instrumental test) due to liver disease
* Hematologic malignancy \[documented by positive physical exam + blood smear or fine needle aspiration (FNA) or bone marrow biopsy\]
* Hemolytic anemia and/or thalassemia
* Patients who cannot meet the requirements of the protocol due to mental and/or cognitive alterations, uncooperative patients, educational limitations and understanding of written language
* Refusal of the patient to participate in the study
Minimum Eligible Age

18 Years

Maximum Eligible Age

99 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Fundación Española de Hematología y Hemoterapía

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Hospital Universitario de Álava

Vitoria-Gasteiz, Alava, Spain

Site Status RECRUITING

Hospital Universitario Torrecárdenas

Almería, Almería, Spain

Site Status RECRUITING

Hospital Son Espases

Palma de Mallorca, Balearic Islands, Spain

Site Status RECRUITING

Hospital Parc Taulí

Sabadell, Barcelona, Spain

Site Status RECRUITING

Hospital Universitario de Burgos

Burgos, Burgos, Spain

Site Status RECRUITING

Hospital de Valdepeñas

Valdepeñas, Ciudad Real, Spain

Site Status RECRUITING

Hospital Universitario Dr. Josep Trueta

Girona, Girona, Spain

Site Status RECRUITING

Hospital Universitario Virgen de las Nieves

Granada, Granada, Spain

Site Status RECRUITING

Hospital San Jorge

Huesca, Huesca, Spain

Site Status RECRUITING

Hospital de Jaén

Jaén, Jaén, Spain

Site Status RECRUITING

Hospital del Bierzo

Ponferrada, León, Spain

Site Status RECRUITING

Hospital Arnau de Vilanova

Lleida, Lleida, Spain

Site Status RECRUITING

Hospital Universitario La Paz

Madrid, Madrid, Spain

Site Status RECRUITING

Hospital Universitario Ramón y Cajal

Madrid, Madrid, Spain

Site Status RECRUITING

Hospital Fundación Jiménez Díaz

Madrid, Madrid, Spain

Site Status RECRUITING

Hospital Regional Universitario de Málaga

Málaga, Málaga, Spain

Site Status RECRUITING

Complejo Hospitalario Universitario de Orense

Ourense, Orense, Spain

Site Status RECRUITING

Hospital Alvaro Cunqueiro

Vigo, Pontevedra, Spain

Site Status RECRUITING

Hospital Alcañiz

Alcañiz, Teruel, Spain

Site Status RECRUITING

Hospital de Manises

Valencia, Valencia, Spain

Site Status RECRUITING

Hospital Universitario y Politécnico La Fe

Valencia, Valencia, Spain

Site Status RECRUITING

Hospital Clínico Universitario de Valladolid

Valladolid, Zaragoza, Spain

Site Status RECRUITING

Complejo Asistencial de Ávila

Ávila, Ávila, Spain

Site Status RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

Spain

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

FEHH SEHH, MD

Role: CONTACT

91 319 19 98

Facility Contacts

Find local site contact details for specific facilities participating in the trial.

Xabier Gutiérrez López de Ocáriz, MD

Role: primary

Alejandro Ponce Navarro, MD

Role: primary

Albert Pérez, MD

Role: primary

Marta Gomez Nuñez, MD

Role: primary

Beatriz Cuevas, MD

Role: primary

Bolívar Luis Díaz Jordán, MD

Role: primary

+034 913191998

Yolanda González, MD

Role: primary

Antonio Cruz, MD

Role: primary

María Flor Yus Cebrián, MD

Role: primary

Juan Antonio López López, MD

Role: primary

Erik de Cabo López, MD

Role: primary

Antonio García Guiñón, MD

Role: primary

Marta Morado Arias, MD

Role: primary

+034 913191998

María del Mar Meijón Ortigueira, MD

Role: primary

Amalia Domingo Gonzalez, MD

Role: primary

Alejandro Contento Gonzalo, MD

Role: primary

José Ángel Méndez Sánchez, MD

Role: primary

Carmen Albo López, MD

Role: primary

Andrés Medinaveitia, MD

Role: primary

Dolores Gómez Toboso, MD

Role: primary

Javier de la Rubia, MD

Role: primary

Carmen Pérez Martínez, MD

Role: primary

Abelardo Bárez García, MD

Role: primary

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

GammaGA

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.