Actively Recruiting

Age: 18Years - 99Years
All Genders
ID05992532

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

Led by Fundación Española de Hematología y Hemoterapía · Updated on 2026-02-11

210

Participants Needed

23

Research Sites

17 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

Researchers are studying patients with splenomegaly or those who have had a splenectomy without a known cause, focusing on the possible presence of Gaucher disease (GD) and Acid Sphingomyelinase Deficiency (ASMD). These conditions may be linked to monoclonal gammopathy of undetermined significance (MGUS) and multiple myeloma (MM), which have been found more frequently among Gaucher and ASMD patients. The study aims to improve diagnosis of unknown causes of splenomegaly and undiagnosed cases of MGUS or MM in these patients. The study involves using standard diagnostic procedures for unknown splenomegaly and splenectomy patients, including collecting blood samples for a dry drop test (DBS). This test measures enzymatic and genetic activity related to Gaucher disease and ASMD, alongside analysis of LisoGl1 and LisoSM biomarkers. No specific treatment or intervention is applied since this is an observational study. Participants will undergo clinical evaluations and blood sampling to help identify the prevalence of Gaucher disease and ASMD over a 36-month period. Researchers will track how many patients with unknown splenomegaly or MGUS/MM have these enzyme deficiencies. The study is observational, with no treatment assigned, and participants will be monitored during their routine medical consultations throughout the study duration.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 18Years - 99Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Adult patients of both sexes
  • Patients with splenomegaly (spleen palpable at 651cm from the costal margin) or splenectomy not related to any specific condition
  • Patients with thrombocytopenia present before treatment for their underlying condition
  • Patients who provide consent to participate in the study
Not Eligible

You will not qualify if you...

  • Splenomegaly due to portal hypertension caused by liver disease
  • Hematologic malignancy confirmed by physical exam and laboratory tests
  • Hemolytic anemia and/or thalassemia
  • Patients unable to comply due to mental or cognitive impairments or educational limitations
  • Patients who refuse to participate in the study

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

1
2
3
+1

Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Surveillance

Duration - Up to 36 months

Participants who undergo routine care are observed to assess the prevalence of Gaucher disease and acid sphingomyelinase deficiency.

Visit schedule may vary based on routine healthcare appointments

Trial Site Locations

Total: 23 locations

1

Hospital Universitario de Álava

Vitoria-Gasteiz, Alava, Spain

Actively Recruiting

2

Hospital Universitario Torrecárdenas

Almería, Almería, Spain

Actively Recruiting

3

Hospital Son Espases

Palma de Mallorca, Balearic Islands, Spain

Actively Recruiting

4

Hospital Parc Taulí

Sabadell, Barcelona, Spain

Actively Recruiting

5

Hospital Universitario de Burgos

Burgos, Burgos, Spain

Actively Recruiting

6

Hospital de Valdepeñas

Valdepeñas, Ciudad Real, Spain, 28040

Actively Recruiting

7

Hospital Universitario Dr. Josep Trueta

Girona, Girona, Spain

Actively Recruiting

8

Hospital Universitario Virgen de las Nieves

Granada, Granada, Spain

Actively Recruiting

9

Hospital San Jorge

Huesca, Huesca, Spain

Actively Recruiting

10

Hospital de Jaén

Jaén, Jaén, Spain

Actively Recruiting

11

Hospital del Bierzo

Ponferrada, León, Spain

Actively Recruiting

12

Hospital Arnau de Vilanova

Lleida, Lleida, Spain

Actively Recruiting

13

Hospital Universitario La Paz

Madrid, Madrid, Spain, 28040

Actively Recruiting

14

Hospital Universitario Ramón y Cajal

Madrid, Madrid, Spain, 28040

Actively Recruiting

15

Hospital Fundación Jiménez Díaz

Madrid, Madrid, Spain

Actively Recruiting

16

Hospital Regional Universitario de Málaga

Málaga, Málaga, Spain

Actively Recruiting

17

Complejo Hospitalario Universitario de Orense

Ourense, Orense, Spain

Actively Recruiting

18

Hospital Alvaro Cunqueiro

Vigo, Pontevedra, Spain

Actively Recruiting

19

Hospital Alcañiz

Alcañiz, Teruel, Spain

Actively Recruiting

20

Hospital de Manises

Valencia, Valencia, Spain

Actively Recruiting

21

Hospital Universitario y Politécnico La Fe

Valencia, Valencia, Spain

Actively Recruiting

22

Hospital Clínico Universitario de Valladolid

Valladolid, Zaragoza, Spain

Actively Recruiting

23

Complejo Asistencial de Ávila

Ávila, Ávila, Spain

Actively Recruiting

Loading map...

Research Team

F

FEHH SEHH, MD

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

0

Similar Trials

An Open-label, Dose-Finding, Phase 1/2 Study to Evaluate the...

Gaucher Disease

Actively Recruiting

9 locations

A Long-term Follow-up Study of Gaucher Disease

Gaucher Disease

Actively Recruiting

1 location

A Prospective Observational Study to Assess the Long-term Sa...

Niemann-Pick Diseases

Actively Recruiting

5 locations

Frequently Asked Questions

Have more questions? Get in touch with our team for quick support

Not the Right Trial for You?

Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.

Already have an account? Log in here