Myelodysplastic SyndromesAMICUSNCT02804984
Identification of Molecular Defects in Idiopathic Cytopenia of Undetermined Significance
An observational study of Myelodysplastic Syndromes, run by Centre Hospitalier Universitaire, Amiens at 1 site in France.
Trial summary
The project's objective is to identify and characterize somatic mutations in cases of idiopathic cytopenia of undetermined significance (ICUS) on the basis of molecular defects found in myelodysplastic syndrome (MDS), in order to validate the hypothesis whereby ICUS may be a precursor of MDS Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- ICUS
- How many people
- 2 participants
- Where
- 1 siteFrance
- Study dates
- 9 Jul 2015to 12 Aug 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
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
- No healthy volunteers
- Adult, Older adult
Study population: patients with myelodysplastic syndrome
Who may be able to join
- Age 18 or over.
- Haemoglobin <11 g/dl and/or a polynuclear neutrophil count <1.5.109/L and/or a platelet count <100.109/L
- Full clinical biochemistry/haematological profiling: complete blood count, blood smear, reticulocyte count, iron status, folates, B12, TSH, creatinine, liver enzymes, ANAs, rheumatoid factor, anticardiolipin antibodies, Coombs test, EPO assay, serological tests for HIV, HVB and HVC.
- Availability of a bone marrow differential cell count and an evaluation of myelopoiesis disorders (number of lineages, percentage of cells affected, etc.) plus Perls staining.
- Availability of a cytogenetic analysis.
- Voluntary provision of written, informed consent
- Life expectancy >6 months
- Social security coverage
Who cannot join
- An obvious cause of anaemia (if isolated): iron deficiency, chronic kidney failure (clearance <60 ml/min), regenerative anaemia (reticulocytes >150G/L)
- Vitamin B12 or B9 deficiency
- Hepatomegaly, or clinical and/or ultrasound signs of portal hypertension
- Clinical and/or ultrasound signs of splenomegaly
- Abnormal liver enzyme levels: total bilirubin, alkaline phosphatases or transaminases > 1.5N; gammaGT > 2N. A history of (or diagnostic criteria during screening) auto-immune diseases such as systemic erythematous lupus, antiphospholipid syndrome or Evans syndrome.
- An abnormal bone marrow differential cell count
Show all 9Show fewer
- A bone marrow karyotype revealing MDS
- Medical, psychological or social conditions that prevent the participant from correctly understanding the study procedures.
- Legal guardianship and incarceration.
Trial locations and contacts
1 site in 1 country.
About this study
The project's objective is to identify and characterize somatic mutations in cases of idiopathic cytopenia of undetermined significance (ICUS) on the basis of molecular defects found in myelodysplastic syndrome (MDS), in order to validate the hypothesis whereby ICUS may be a precursor of MDS
Read the detailed description
The project's objective is to identify and characterize somatic mutations in cases of idiopathic cytopenia of undetermined significance (ICUS) on the basis of molecular defects found in myelodysplastic syndrome (MDS), in order to validate the hypothesis whereby ICUS may be a precursor of MDS. To this end, high-throughput exon sequencing (using next-generation sequencing (NGS)) will be used to target the genes known to be mutated in MDS. This study is important for two reasons. Firstly, it will help to optimise the clinical monitoring of patients with molecular defects and considered to be at risk of progression. Secondly, it will provide a better understanding of the fundamental molecular mechanisms underlying the progression of ICUS to MDS.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- ICUS
- Keywords
- idiopathic cytopenia of undetermined significance (ICUS), molecular defects
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ICUSGenetic
| Study group | Role | What they receive |
|---|---|---|
| ICUS | idiopathic cytopenia of undetermined significance (ICUS) Receives: Genetic: ICUS |
What is being measured
The primary outcome is the main question the study is designed to answer.
high-throughput sequencing
The presence or absence of one or several of the following molecular defects, as detected by high-throughput sequencing: DNMT3A, TET2, IDH1/2, ASXL1, EZH2, RUNX1, EVI1, GATA2, P53, JAK2, CBL, KRAS, SF3B1, SRSF2, U2AF1, and ZRSR2.
Measured: Day 0
Secondary outcomes (3)
phenotypic defects
Analysis of phenotypic defects detected by flow cytometry.
Measured: Day 0
growth of erythroid progenitors
Analysis of the growth of erythroid progenitors (BFU-E) and granulocyte-monocyte progenitors (CFU-GM).
Measured: Day 0
Appearance of MDS
Appearance of MDS during follow-up: a bone marrow differential cell count at 6 months and whenever cytopenia appears.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jun 2016 (estimated)
- Study start9 Jul 2015
- Primary completion12 Aug 2022
- Study completion12 Aug 2022
Why it stopped: Difficult recruitment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by Centre Hospitalier Universitaire, AmiensThe 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
- Case only
- Time perspective
- Prospective
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Centre Hospitalier Universitaire, Amiens
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Bérengère GRUSON, PhD, Principal investigator, CHU Amiens
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02804984
- Sponsor's protocol ID
- PI2014_843_0008
- Acronym
- AMICUS
- Status last verified
- Jun 2026
- First submitted
- 15 Jun 2016
- Last updated
- 15 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Identification of Molecular Defects in Idiopathic Cytopenia of Undetermined Significance
- ClinicalTrials.gov ID
- NCT02804984
- Acronym / sponsor ID
- AMICUS / PI2014_843_0008
- Conditions
- Myelodysplastic Syndromes
- Keywords
- idiopathic cytopenia of undetermined significance (ICUS), molecular defects
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Difficult recruitment
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Centre Hospitalier Universitaire, AmiensUniversity, hospital or other
- Study start
- 9 Jul 2015 (actual)
- Primary completion
- 12 Aug 2022 (actual)
- Study completion
- 12 Aug 2022 (actual)
- First posted
- 17 Jun 2016 (estimated)
- 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.
The project's objective is to identify and characterize somatic mutations in cases of idiopathic cytopenia of undetermined significance (ICUS) on the basis of molecular defects found in myelodysplastic syndrome (MDS), in order to validate the hypothesis whereby ICUS may be a precursor of MDS
Detailed description
The project's objective is to identify and characterize somatic mutations in cases of idiopathic cytopenia of undetermined significance (ICUS) on the basis of molecular defects found in myelodysplastic syndrome (MDS), in order to validate the hypothesis whereby ICUS may be a precursor of MDS. To this end, high-throughput exon sequencing (using next-generation sequencing (NGS)) will be used to target the genes known to be mutated in MDS. This study is important for two reasons. Firstly, it will help to optimise the clinical monitoring of patients with molecular defects and considered to be at risk of progression. Secondly, it will provide a better understanding of the fundamental molecular mechanisms underlying the progression of ICUS to MDS.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ICUS | – | Genetic: ICUSidiopathic cytopenia of undetermined significance (ICUS) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- patients with myelodysplastic syndrome
Key inclusion criteria (8 listed)
- Age 18 or over.
- Haemoglobin <11 g/dl and/or a polynuclear neutrophil count <1.5.109/L and/or a platelet count <100.109/L
- Full clinical biochemistry/haematological profiling: complete blood count, blood smear, reticulocyte count, iron status, folates, B12, TSH, creatinine, liver enzymes, ANAs, rheumatoid factor, anticardiolipin antibodies, Coombs test, EPO assay, serological tests for HIV, HVB and HVC.
- Availability of a bone marrow differential cell count and an evaluation of myelopoiesis disorders (number of lineages, percentage of cells affected, etc.) plus Perls staining.
- Availability of a cytogenetic analysis.
Key exclusion criteria (9 listed)
- An obvious cause of anaemia (if isolated): iron deficiency, chronic kidney failure (clearance <60 ml/min), regenerative anaemia (reticulocytes >150G/L)
- Vitamin B12 or B9 deficiency
- Hepatomegaly, or clinical and/or ultrasound signs of portal hypertension
- Clinical and/or ultrasound signs of splenomegaly
- Abnormal liver enzyme levels: total bilirubin, alkaline phosphatases or transaminases > 1.5N; gammaGT > 2N. A history of (or diagnostic criteria during screening) auto-immune diseases such as systemic erythematous lupus, antiphospholipid syndrome or Evans syndrome.
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 |
|---|---|---|
| ICUS | Genetic | ICUS |
Primary outcome
high-throughput sequencing
Time frame: Day 0
The presence or absence of one or several of the following molecular defects, as detected by high-throughput sequencing: DNMT3A, TET2, IDH1/2, ASXL1, EZH2, RUNX1, EVI1, GATA2, P53, JAK2, CBL, KRAS, SF3B1, SRSF2, U2AF1, and ZRSR2.
3 secondary outcomes
phenotypic defects
Time frame: Day 0
growth of erythroid progenitors
Time frame: Day 0
Appearance of MDS
Time frame: 6 months
Results and safety
The study was stopped early (reason given: Difficult recruitment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jun 2026.
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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. This study is not recruiting at the moment.
Bérengère GRUSON, PhD principal investigator
CHU Amiens
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Myelodysplastic Syndromes, run by Centre Hospitalier Universitaire, Amiens at 1 site 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 8 inclusion and 9 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: stopped early, as of the sponsor's last update on 15 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 1 site 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?
The study was stopped early (reason given: Difficult recruitment). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
high-throughput sequencing (time frame: Day 0). 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 15 Jun 2026.