Monoclonal Gammopathy of Undetermined SignificanceGAMYNCT06559033
Determine the Frequency of Variants in the GBA/PSAP Genes in Patients With MM or MGUS
An observational study of Monoclonal Gammopathy of Undetermined Significance and Myeloma Multiple, run by University Hospital, Rouen at 2 sites in France.
Trial summary
No effective specific treatment is currently available for the management of Multiple Myeloma (MM) and Monoclonal Gammopathy of Undetermined Significance (MGUS). A better understanding of the pathophysiological mechanisms would make it possible to propose treatments specifically targeting the deregulated pathways. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Monoclonal Gammopathy of Undetermined Significance, Myeloma Multiple
- What is being tested?
- Evaluation of the presence and number of mutated alleles of the GBA/PSAP genes in patients with MM or MGUS
- How many people
- 300 participants
- Where
- 2 sitesFrance
- Study dates
- 7 Oct 2025to 1 Apr 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: 100 patients with multiple myeloma (MM), 100 patients with monoclonal gammopathy of undetermined significance (MGUS), 100 controls with plasma samples available
Who may be able to join
- Major patients with multiple myeloma (MM) (defined by clonal proliferation of tumour plasma cells (>10%), presence of a monoclonal peak in serum or urine (excluding non-secretory myeloma) and organ involvement secondary to bone marrow invasion) or with MGUS (defined as bone marrow plasmacytosis of less than 10%, associated with a monoclonal protein of less than 30g/L and no clinical involvement).
- Membership of a social security scheme
- Adult having read and understood the information letter and signed the consent form
Who cannot join
- Person deprived of liberty by an administrative or judicial decision or person placed under court protection / sub-guardianship or guardianship
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
France2 sites, 2 recruiting
- Not yet recruiting
Centre Henri Becquerel
Rouen, 76000
Fabrice FJ JARDIN, Professor, 02.32.08.24.65 ext. +33, fabrice.jardin@chb.unicancer.fr
- Recruiting
University Rouen Hospital
Rouen, 76031
Gaétan GS SAUVETRE, Doctor, 0232889318 ext. +33, Gaetan.Sauvetre@chu-rouen.fr
Hélène HG GOMEZ, Doctor, 0232889318 ext. +33, Helene.Gomez@chu-rouen.fr
About this study
Official title: Determine the Frequency of Variants in the GBA/PSAP Genes in Patients With Multiple Myeloma (MM) or Monoclonal Gammopathy of Undetermined Significance (MGUS)
No effective specific treatment is currently available for the management of Multiple Myeloma (MM) and Monoclonal Gammopathy of Undetermined Significance (MGUS). A better understanding of the pathophysiological mechanisms would make it possible to propose treatments specifically targeting the deregulated pathways.
Read the detailed description
This study will characterise the links between rare diseases and complex, chronic diseases. Metabolism can be visualised as a complex network in which the various biomolecules represent metabolic nodes and are linked together by connections. The number of connections at a node influences the effect of that biomolecule on the metabolic network(s) as a whole. If a biomolecule has a large number of connections, altering a metabolic pathway involving it will have an effect that will spread throughout the network. On the other hand, metabolic pathways with a high flux have a major impact on the homeostasis of the network. Thus, alteration of such a metabolic pathway cannot be without consequence: a major alteration could induce a rare hereditary metabolic disease with an early-onset clinic, whereas an alteration with a moderate effect could participate in the pathogenesis of complex diseases, and may open up new therapeutic prospects for these tumour pathologies.
- Being tested
- Evaluation of the presence and number of mutated alleles of the GBA/PSAP genes in patients with MM or MGUS
- Medical subject headings
- Monoclonal Gammopathy of Undetermined Significance, Multiple Myeloma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Evaluation of the presence and number of mutated alleles of the GBA/PSAP genes in patients with MM or MGUSBiological
Also called Evaluation of plasma concentrations of LGL1 in patients with MM or MGUS compared to controls, Evaluation of the % Reactivity of plasma immunoglobulins from patients with MM or MGUS towards LGL1
Estimation of the frequency of variants in the PSAP/GBA genes in patients with MM or MGUS, then comparison with a reference frequency from databases such as the Exome Aggregation Consortium, the Exome Sequencing Project, the 1000 Genomes Project and the dbSNP.
| Study group | Role | What they receive |
|---|---|---|
| Multiple myeloma (MM) patient group | patients with multiple myeloma (MM) (defined by a clonal proliferation of tumour plasma cells (>10%), the presence of a monoclonal peak in the serum or urine (excluding non-secretory myeloma) and organ damage secondary to bone marrow invasion) Receives: Biological: Evaluation of the presence and number of mutated alleles of the GBA/PSAP genes in patients with MM or MGUS | |
| Monoclonal gammopathy of undetermined significance (MGUS) patient group | patients with MGUS (defined as bone marrow plasmacytosis of less than 10%, associated with a monoclonal protein of less than 30g/L and no clinical involvement) Receives: Biological: Evaluation of the presence and number of mutated alleles of the GBA/PSAP genes in patients with MM or MGUS | |
| Control group | patient with no pathology under study relating to the project Receives: Biological: Evaluation of the presence and number of mutated alleles of the GBA/PSAP genes in patients with MM or MGUS |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of variants in the GBA/PSAP genes in patients with MM or MGUS
The main aim of the research is to determine the frequency of variants in the GBA/PSAP genes in patients with MM or MGUS.
Measured: inclusion (one day)
Secondary outcomes (2)
Plasma concentrations of LGL1 in patients with MM or MGUS
Compare the plasma concentration of LGL1 in MM and MGUS patients versus control individuals
Measured: inclusion (one day)
Reactivity of monoclonal antibodies in MM and MGUS patients
Assess the % (proportion of patients with reactivity (% reactivity greater than 0) and those without (% reactivity equal to 0)) of reactivity of monoclonal antibodies from MM and MGUS patients to LGL1
Measured: inclusion (one day)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Aug 2024
- Study start7 Oct 2025
- Primary completion2 Oct 2026 (estimated)
- Study completion1 Apr 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Monoclonal Gammopathy of Undetermined SignificanceStudies of the same condition, open and under way.
- More trials run by University Hospital, RouenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 300 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Rouen
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06559033
- Sponsor's protocol ID
- 2020/0430/OB
- Other IDs
- 2022-A00306-37
- Acronym
- GAMY
- Status last verified
- Aug 2025
- First submitted
- 13 Aug 2024
- Last updated
- 10 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Determine the Frequency of Variants in the GBA/PSAP Genes in Patients With Multiple Myeloma (MM) or Monoclonal Gammopathy of Undetermined Significance (MGUS)
- ClinicalTrials.gov ID
- NCT06559033
- Acronym / sponsor ID
- GAMY / 2020/0430/OB
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- University Hospital, RouenUniversity, hospital or other
- Study start
- 7 Oct 2025 (actual)
- Primary completion
- 2 Oct 2026 (estimated)
- Study completion
- 1 Apr 2027 (estimated)
- First posted
- 19 Aug 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
No effective specific treatment is currently available for the management of Multiple Myeloma (MM) and Monoclonal Gammopathy of Undetermined Significance (MGUS). A better understanding of the pathophysiological mechanisms would make it possible to propose treatments specifically targeting the deregulated pathways.
Detailed description
This study will characterise the links between rare diseases and complex, chronic diseases. Metabolism can be visualised as a complex network in which the various biomolecules represent metabolic nodes and are linked together by connections. The number of connections at a node influences the effect of that biomolecule on the metabolic network(s) as a whole. If a biomolecule has a large number of connections, altering a metabolic pathway involving it will have an effect that will spread throughout the network. On the other hand, metabolic pathways with a high flux have a major impact on the homeostasis of the network. Thus, alteration of such a metabolic pathway cannot be without consequence: a major alteration could induce a rare hereditary metabolic disease with an early-onset clinic, whereas an alteration with a moderate effect could participate in the pathogenesis of complex diseases, and may open up new therapeutic prospects for these tumour pathologies.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Multiple myeloma (MM) patient group | – | Biological: Evaluation of the presence and number of mutated alleles of the GBA/PSAP genes in patients with MM or MGUSpatients with multiple myeloma (MM) (defined by a clonal proliferation of tumour plasma cells (>10%), the presence of a monoclonal peak in the serum or urine (excluding non-secretory myeloma) and organ damage secondary to bone marrow invasion) |
| Monoclonal gammopathy of undetermined significance (MGUS) patient group | – | Biological: Evaluation of the presence and number of mutated alleles of the GBA/PSAP genes in patients with MM or MGUSpatients with MGUS (defined as bone marrow plasmacytosis of less than 10%, associated with a monoclonal protein of less than 30g/L and no clinical involvement) |
| Control group | – | Biological: Evaluation of the presence and number of mutated alleles of the GBA/PSAP genes in patients with MM or MGUSpatient with no pathology under study relating to the project |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- 100 patients with multiple myeloma (MM), 100 patients with monoclonal gammopathy of undetermined significance (MGUS), 100 controls with plasma samples available
Key inclusion criteria (3 listed)
- Major patients with multiple myeloma (MM) (defined by clonal proliferation of tumour plasma cells (>10%), presence of a monoclonal peak in serum or urine (excluding non-secretory myeloma) and organ involvement secondary to bone marrow invasion) or with MGUS (defined as bone marrow plasmacytosis of less than 10%, associated with a monoclonal protein of less than 30g/L and no clinical involvement).
- Membership of a social security scheme
- Adult having read and understood the information letter and signed the consent form
Key exclusion criteria (1 listed)
- Person deprived of liberty by an administrative or judicial decision or person placed under court protection / sub-guardianship or guardianship
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Evaluation of the presence and number of mutated alleles of the GBA/PSAP genes in patients with MM or MGUSAlso: Evaluation of plasma concentrations of LGL1 in patients with MM or MGUS compared to controls, Evaluation of the % Reactivity of plasma immunoglobulins from patients with MM or MGUS towards LGL1Estimation of the frequency of variants in the PSAP/GBA genes in patients with MM or MGUS, then comparison with a reference frequency from databases such as the Exome Aggregation Consortium, the Exome Sequencing Project, the 1000 Genomes Project and the dbSNP. | Biological | Control group, Monoclonal gammopathy of undetermined significance (MGUS) patient group, Multiple myeloma (MM) patient group |
Primary outcome
Frequency of variants in the GBA/PSAP genes in patients with MM or MGUS
Time frame: inclusion (one day)
The main aim of the research is to determine the frequency of variants in the GBA/PSAP genes in patients with MM or MGUS.
2 secondary outcomes
Plasma concentrations of LGL1 in patients with MM or MGUS
Time frame: inclusion (one day)
Reactivity of monoclonal antibodies in MM and MGUS patients
Time frame: inclusion (one day)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jun 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Monoclonal Gammopathy of Undetermined Significance and Myeloma Multiple, run by University Hospital, Rouen at 2 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 10 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Abdellah AB TEBANI, Pr (02 32 88 81 24 ext. +33, Abdellah.Tebani@chu-rouen.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Frequency of variants in the GBA/PSAP genes in patients with MM or MGUS (time frame: inclusion (one day)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jun 2026.