PreleukemiaPhase 2NCT04741945
Repurposing Metformin As a Leukemia-preventive Drug in CCUS and LR-MDS
A Phase 2 study testing Metformin in people with Preleukemia and Myelodysplastic Neoplasm, run by Kirsten Grønbæk at 1 site in Denmark.
Trial summary
This is a single-arm pilot study of the feasibility and safety of metformin in patients with clonal cytopenia of undetermined significance (CCUS) or lower-risk myelodysplastic neoplasms (LR-MDS). Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Preleukemia, Myelodysplastic Neoplasm, Cytopenia
- What is being tested?
- Metformin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteDenmark
- Study dates
- 13 Dec 2021to Feb 2026
- Phase
- Phase 2
- 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
Who may be able to join
- A diagnosis of:
- LR-MDS according to the revised international prognostic scoring system (IPSS-R), i.e., very low- or low-risk disease (IPSS-R score ≤3) in addition to a bone marrow blast percentage <5 OR
- CCUS defined as the presence of somatic mutation(s) or cytogenetic abnormality not diagnostic of MDS or any other malignancy in the context of persistent cytopenia (>6 months) with other common causes of cytopenia ruled out in the setting of bone marrow morphology that is not diagnostic of MDS or any other malignancy, and hematolytic conditions have been ruled out. Peripheral blood cytopenia is defined as hemoglobin (hgb) <11.3 g/dL (7 mmol/L) in women and hgb <12.9 g/dL (8 mmol/L) in men, platelet count <150 x 109/L, or neutrophil count <1.8 x 109/L
- Menopause, if being a female, defined as females >45 years of age who have experienced amenorrhea for minimum 12 months, without any other obvious pathological or physiological cause
- ≥18 years of age
- Written informed consent
Show all 11Show fewer
- Willingness to comply with mandatory aspects of the protocol
- Ability to swallow pills
- Healthy individuals matched on age, sex, and BMI, if possible, to individual patient participants in WP1
- Written informed consent
- Willingness to comply with mandatory aspects of the protocol
Who cannot join
- Any prior treatment with metformin
- A diagnosis of diabetes mellitus
- Therapeutic radiation, immunosuppressive therapy (with the exception of corticosteroids), or chemotherapy within the past year
- Treatment with granulocyte colony-stimulating factor within the past 30 days
- Prior therapy with hypomethylating agents (i.e., azacitidine, decitabine)
- eGFR <45 mL/min
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- Performance status according to the Eastern Cooperative Oncology Group >2
- Other active malignancy within the past five years
- Uncontrolled comorbidity including impaired hepatic function (total serum bilirubin >1.5 × upper limit of the normal range (ULN), serum alanine transaminase >3 × ULN), chronic hepatitis with decompensated cirrhosis, disabling psychiatric disease, severe neurologic disease, uncontrolled metabolic disease, or severe cardiac disease (NYHA class 3-4)
- An eGFR calculation performed up to one month prior to inclusion may be used to assess renal function. If such an assessment is not available, it is performed at screening.
- Healthy volunteers are eligible to be included in WP0 if they meet all of the following criteria:
- Use of metformin within the past 3 years
- A diagnosis of diabetes mellitus, rheumatological disorders, autoimmune diseases or other inflammatory disorders, celiac disease, inflammatory bowel disease, or other gastrointestinal disorders or symptoms
- Treatment with immunosuppressive drugs (with the exception of corticosteroids) or chemotherapy within the past year or antibiotics within the past 6 months
- Any contraindications to MRS
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Denmark1 site, 1 recruiting
- Recruiting
Rigshospitalet
Copenhagen, Copenhagen N, 2200
Kirsten Grønbæk, Professor, MD, +45 35456060, Kirsten.Groenbaek@regionh.dk
Stine Ulrik Mikkelsen, MD
About this study
Official title: STOP-LEUKEMIA: Repurposing Metformin As a Leukemia-preventive Drug in CCUS and LR-MDS
This is a single-arm pilot study of the feasibility and safety of metformin in patients with clonal cytopenia of undetermined significance (CCUS) or lower-risk myelodysplastic neoplasms (LR-MDS).
Read the detailed description
The research plan is divided into three work packages (WP):
WP0: Bone Marrow Adipose Tissue, Gut Microbiota, and Intestinal Permeability in CCUS and LR-MDS Patients.
The aim of WP0 is to investigate biological features which the investigators hypothesize to be of pathogenetic relevance for MDS progression and may be possible targets of metformin treatment. For this purpose, 20 elderly (≥60 years) healthy controls will be included for comparison to patients with CCUS or LR-MDS from WP1.
The primary objectives are to investigate 1) the abundance and properties of bone marrow adipose tissue (BMAT) and bone marrow (BM) adipocytes, and 2) the gut microbiota and intestinal permeability of patients with CCUS or LR-MDS compared to age-, sex- and body mass index (BMI)-matched healthy controls. Secondary objectives are to characterize DNA methylation and hydroxymethylation (5-mC and 5-hmC) patterns, and hormone and cytokine levels in BM plasma from healthy controls and patients with CCUS or LR-MDS.
WP1: Safety, feasibility, and mechanisms of action of metformin in patients with CCUS or LR-MDS.
In this WP up to 40 patients with CCUS or LR-MDS will receive metformin 2000 mg daily or their maximum tolerated dose (MTD) for 12 months. The aim of WP1 is to investigate safety of metformin and feasibility of the protocol in patients with CCUS or LR-MDS. Potential mechanisms of anti-leukemic action of metformin will also be explored in order to identify a suitable outcome measure and estimate standard deviation of the outcome measure. All in order to inform the design of a future phase 3 RCT of the efficacy of metformin in CCUS and LR-MDS patients. Endpoints of WP1 are specified in the corresponding section.
WP2: Safety and efficacy of metformin compared to placebo. The primary objective of WP2 is to compare safety and preliminary efficacy of metformin in patients with CCUS or LR-MDS to a cohort of patients with CCUS or LR-MDS receiving placebo in context of our EVI-2 randomized, controlled pilot study (NCT03999723).
Data and samples from approximately 50 historical controls will be included from the EVI-2 study in which participants were randomized to receive placebo or oral vitamin C supplement for 12 months.
- Conditions
- Preleukemia, Myelodysplastic Neoplasm, Cytopenia, Preleukemic Anemia
- Being tested
- Metformin
- Keywords
- Metformin, Myelodysplastic Neoplasm, Clonal Cytopenia of Undetermined Significance, Safety, Feasibility, Bone Marrow Adipose Tissue, Gut Microbiota, Intestinal Permeability
- Medical subject headings
- Preleukemia, Anemia, Refractory, with Excess of Blasts, Cytopenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MetforminDrug
2000 mg/day metformin for 12 months (1000 mg b.i.d.) with a slow up-titration six weeks prior to full dose treatment.
| Study group | Role | What they receive |
|---|---|---|
| Metformin | Experimental | 2000 mg/day metformin for 12 months. Receives: Drug: Metformin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety as assessed by the number of serious adverse events including any suspected unexpected serious adverse reactions
To assess safety of metformin treatment in this off-label indication by the type, grade, and number of adverse events and serious adverse events including any suspected unexpected serious adverse reactions in the patients.
Measured: From inclusion to 12 months of study treatment
Safety as assessed by median maximum tolerated dose in mg/day
To assess safety of metformin treatment in this off-label indication by median maximum tolerated dose and a description of any changes in individual medication doses.
Measured: From inclusion to 12 months of study treatment
Feasibility as assessed by rates of recruitment/refusal rates
To assess feasibility of the study protocol in terms of recruitment and refusal rates.
Measured: From inclusion to 12 months of study treatment
Feasibility as assessed by 12 months follow-up, i.e., study completion, rate
To assess feasibility of the study protocol in terms of rate of study completion.
To assess safety of metformin treatment in this off-label indication by the type, grade, and number of adverse events and serious adverse events including any suspected unexpected serious adverse reactions in the patients; drop-out rates; and a description of any changes in individual medication doses including median maximum tolerated dose.
Measured: From inclusion to 12 months of study treatment
Feasibility as assessed by rate of compliance to protocol procedures
To assess feasibility of the study protocol in terms of rate of adherence to protocol procedures (study medication and study procedures).
Measured: From inclusion to 12 months of study treatment
Secondary outcomes (25)
Interim efficacy: Mutational burden as assessed by change in variant allele frequency
Change in variant allele frequency (ΔVAF) by next generation sequencing (NGS)
Measured: From inclusion to 12 months of study treatment
Interim efficacy: Patient-reported outcome measures based on the EORTC QLQ-C30
Change in patient-reported outcome measures (PROM), based on the validated European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30).
Measured: From inclusion to 4 months of study treatment
Interim efficacy: Patient-reported outcome measures based on the EORTC QLQ-C30
Change in patient-reported outcome measures (PROM), based on the validated European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30).
Measured: From inclusion to 12 months of study treatment
Interim efficacy: Patient-reported outcome measures based on the SF-36
Change in patient-reported outcome measures (PROM), based on the validated Short Form 36 Health Survey Questionnaire (SF-36).
Measured: From inclusion to 4 months of study treatment
Interim efficacy: Patient-reported outcome measures based on the SF-36
Change in patient-reported outcome measures (PROM), based on the validated Short Form 36 Health Survey Questionnaire (SF-36).
Measured: From inclusion to 12 months of study treatment
Interim efficacy: Patient-reported outcome measures based on the EQ-5D
Change in patient-reported outcome measures (PROM), based on the validated European Quality of Life Five Dimension questionnaire (EQ-5D).
Measured: From inclusion to 4 months of study treatment
Interim efficacy: Patient-reported outcome measures based on the EQ-5D
Change in patient-reported outcome measures (PROM), based on the validated European Quality of Life Five Dimension questionnaire (EQ-5D).
Measured: From inclusion to 12 months of study treatment
Interim efficacy: Bone marrow adipose tissue as assessed by MR spectroscopy ratio of adipose tissue and water phase
Change in bone marrow adipose tissue (BMAT) content in bone marrow (BM) measured by MR spectroscopy
Measured: From inclusion to 4 months of study treatment
Interim efficacy: Bone marrow adipose tissue as assessed by MR spectroscopy ratio of adipose tissue and water phase
Change in bone marrow adipose tissue (BMAT) content in bone marrow (BM) measured by MR spectroscopy
Measured: From inclusion to 12 months of study treatment
Interim efficacy: Bone mineral density as assessed by DEXA scan measured in grams per cubic centimeter with resulting Z score
Change in bone mineral density (BMD) measured by DEXA scan measured in grams per cubic centimeter with resulting Z score.
Measured: From inclusion to 12 months of study treatment
Interim efficacy: Body composition as assessed by DEXA scan presented as whole body bone mass and soft tissue composition
Change in body composition measured by DEXA scan presented as whole body bone mass and soft tissue composition with ratios of lean mass, body fat, and bone mass.
Measured: From inclusion to 12 months of study treatment
Interim efficacy: Gut microbiota composition as assessed by 16S rRNA sequencing
Change in gut microbiota by 16S rRNA sequencing or whole genome sequencing of intestinal bacteria.
Measured: From inclusion to 4 months of study treatment
Interim efficacy: Gut microbiota composition as assessed by 16S rRNA sequencing
Change in gut microbiota by 16S rRNA sequencing or whole genome sequencing of intestinal bacteria.
Measured: From inclusion to 12 months of study treatment
Interim efficacy: Small intestinal permeability as assessed by urine-lactulose/mannitol measurement and ion chromatography
Change in small intestinal permeability by functional assessment by urine-lactulose/mannitol measurement and ion chromatography or by indirect assessment by qPCR and 16S rRNA sequencing of whole blood.
Measured: From inclusion to 4 months of study treatment
Interim efficacy: Epigenetic regulation as assessed by levels of 5-mC and 5-hmC
Change in DNA methylation and hydroxymethylation (5-mC and 5-hmC) patterns in hematopoietic cells by global 5-hmC/5-mC assessment and EPIC arrays.
Measured: From inclusion to 4 months of study treatment
Interim efficacy: Epigenetic regulation as assessed by levels of 5-mC and 5-hmC
Change in DNA methylation and hydroxymethylation (5-mC and 5-hmC) patterns in hematopoietic cells by global 5-hmC/5-mC assessment and EPIC arrays.
Measured: From inclusion to 12 months of study treatment
Interim efficacy: Gene expression as assessed by RNA sequencing
Change in RNA expression in hematopoietic cells and BM adipocytes.
Measured: From inclusion to 4 months of study treatment
Interim efficacy: Gene expression as assessed by RNA sequencing
Change in RNA expression in hematopoietic cells and BM adipocytes.
Measured: From inclusion to 12 months of study treatment
Interim efficacy: Protein profiles as assessed by proteomics
Change in protein profiles in hematopoietic cells and BM adipocytes by proteomics.
Measured: From inclusion to 4 months of study treatment
Interim efficacy: Protein profiles as assessed by proteomics
Change in protein profiles in hematopoietic cells and BM adipocytes by proteomics.
Measured: From inclusion to 12 months of study treatment
Interim efficacy: Response and disease progression as according to the IWG response criteria in myelodysplastic neoplasms
Rates of response and disease progression as according to the International Working Group (IWG) response criteria in myelodysplastic neoplasms.
Measured: From inclusion to 12 months of study treatment
Interim efficacy: Bone marrow niche factor levels as assessed by ELISA
Change in niche factors in bone marrow by ELISA.
Measured: From inclusion to 4 months of study treatment
Interim efficacy: Bone marrow niche factor levels as assessed by ELISA
Change in niche factors in bone marrow by ELISA.
Measured: From inclusion to 12 months of study treatment
Interim efficacy: Cytokine levels as assessed by ELISA
Change in cytokine levels in peripheral blood and bone marrow plasma by ELISA.
Measured: From inclusion to 4 months of study treatment
Interim efficacy: Cytokine levels as assessed by ELISA
Change in cytokine levels in peripheral blood and bone marrow plasma by ELISA.
Measured: From inclusion to 12 months of study treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2021
- Study start13 Dec 2021
- Primary completionFeb 2026 (estimated)
- Study completionFeb 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PreleukemiaStudies of the same condition, open and under way.
- 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Kirsten Grønbæk
- Sponsor type
- University, hospital or other
- Collaborators
- Steno Diabetes Center Copenhagen, Zealand University Hospital, Van Andel Research Institute, Herlev Hospital, Technical University of Denmark, Region Hovedstadens Apotek, University of Copenhagen
- Responsible party
- Sponsor-investigator, Kirsten Grønbæk, Copenhagen University Hospital, Rigshospitalet
- Investigators
- Kirsten Grønbæk, Professor, MD, Study chair, Copenhagen University Hospital, Rigshospitalet
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04741945
- Sponsor's protocol ID
- STOP-LEUKEMIA
- Status last verified
- Nov 2024
- First submitted
- 19 Jan 2021
- Last updated
- 22 Nov 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- STOP-LEUKEMIA: Repurposing Metformin As a Leukemia-preventive Drug in CCUS and LR-MDS
- ClinicalTrials.gov ID
- NCT04741945
- Acronym / sponsor ID
- STOP-LEUKEMIA
- Conditions
- Preleukemia, Myelodysplastic Neoplasm, Cytopenia, Preleukemic Anemia
- Keywords
- Metformin, Myelodysplastic Neoplasm, Clonal Cytopenia of Undetermined Significance, Safety, Feasibility, Bone Marrow Adipose Tissue, Gut Microbiota, Intestinal Permeability
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Kirsten GrønbækUniversity, hospital or other
- Collaborators
- Steno Diabetes Center Copenhagen, Zealand University Hospital, Van Andel Research Institute, Herlev Hospital, Technical University of Denmark, Region Hovedstadens Apotek, University of Copenhagen
- Study start
- 13 Dec 2021 (actual)
- Primary completion
- Feb 2026 (estimated)
- Study completion
- Feb 2026 (estimated)
- First posted
- 5 Feb 2021 (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.
This is a single-arm pilot study of the feasibility and safety of metformin in patients with clonal cytopenia of undetermined significance (CCUS) or lower-risk myelodysplastic neoplasms (LR-MDS).
Detailed description
The research plan is divided into three work packages (WP):
WP0: Bone Marrow Adipose Tissue, Gut Microbiota, and Intestinal Permeability in CCUS and LR-MDS Patients.
The aim of WP0 is to investigate biological features which the investigators hypothesize to be of pathogenetic relevance for MDS progression and may be possible targets of metformin treatment. For this purpose, 20 elderly (≥60 years) healthy controls will be included for comparison to patients with CCUS or LR-MDS from WP1.
The primary objectives are to investigate 1) the abundance and properties of bone marrow adipose tissue (BMAT) and bone marrow (BM) adipocytes, and 2) the gut microbiota and intestinal permeability of patients with CCUS or LR-MDS compared to age-, sex- and body mass index (BMI)-matched healthy controls. Secondary objectives are to characterize DNA methylation and hydroxymethylation (5-mC and 5-hmC) patterns, and hormone and cytokine levels in BM plasma from healthy controls and patients with CCUS or LR-MDS.
WP1: Safety, feasibility, and mechanisms of action of metformin in patients with CCUS or LR-MDS.
In this WP up to 40 patients with CCUS or LR-MDS will receive metformin 2000 mg daily or their maximum tolerated dose (MTD) for 12 months. The aim of WP1 is to investigate safety of metformin and feasibility of the protocol in patients with CCUS or LR-MDS. Potential mechanisms of anti-leukemic action of metformin will also be explored in order to identify a suitable outcome measure and estimate standard deviation of the outcome measure. All in order to inform the design of a future phase 3 RCT of the efficacy of metformin in CCUS and LR-MDS patients. Endpoints of WP1 are specified in the corresponding section.
WP2: Safety and efficacy of metformin compared to placebo. The primary objective of WP2 is to compare safety and preliminary efficacy of metformin in patients with CCUS or LR-MDS to a cohort of patients with CCUS or LR-MDS receiving placebo in context of our EVI-2 randomized, controlled pilot study (NCT03999723).
Data and samples from approximately 50 historical controls will be included from the EVI-2 study in which participants were randomized to receive placebo or oral vitamin C supplement for 12 months.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Metformin | Experimental | Drug: Metformin2000 mg/day metformin for 12 months. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- A diagnosis of:
- Menopause, if being a female, defined as females >45 years of age who have experienced amenorrhea for minimum 12 months, without any other obvious pathological or physiological cause
- ≥18 years of age
- Written informed consent
- Willingness to comply with mandatory aspects of the protocol
Key exclusion criteria (15 listed)
- Any prior treatment with metformin
- A diagnosis of diabetes mellitus
- Therapeutic radiation, immunosuppressive therapy (with the exception of corticosteroids), or chemotherapy within the past year
- Treatment with granulocyte colony-stimulating factor within the past 30 days
- Prior therapy with hypomethylating agents (i.e., azacitidine, decitabine)
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 |
|---|---|---|
| Metformin2000 mg/day metformin for 12 months (1000 mg b.i.d.) with a slow up-titration six weeks prior to full dose treatment. | Drug | Metformin |
Primary outcome
Safety as assessed by the number of serious adverse events including any suspected unexpected serious adverse reactions
Time frame: From inclusion to 12 months of study treatment
To assess safety of metformin treatment in this off-label indication by the type, grade, and number of adverse events and serious adverse events including any suspected unexpected serious adverse reactions in the patients.
Safety as assessed by median maximum tolerated dose in mg/day
Time frame: From inclusion to 12 months of study treatment
To assess safety of metformin treatment in this off-label indication by median maximum tolerated dose and a description of any changes in individual medication doses.
Feasibility as assessed by rates of recruitment/refusal rates
Time frame: From inclusion to 12 months of study treatment
To assess feasibility of the study protocol in terms of recruitment and refusal rates.
Feasibility as assessed by 12 months follow-up, i.e., study completion, rate
Time frame: From inclusion to 12 months of study treatment
To assess feasibility of the study protocol in terms of rate of study completion. To assess safety of metformin treatment in this off-label indication by the type, grade, and number of adverse events and serious adverse events including any suspected unexpected serious adverse reactions in the patients; drop-out rates; and a description of any changes in individual medication doses including median maximum tolerated dose.
Feasibility as assessed by rate of compliance to protocol procedures
Time frame: From inclusion to 12 months of study treatment
To assess feasibility of the study protocol in terms of rate of adherence to protocol procedures (study medication and study procedures).
25 secondary outcomes
Interim efficacy: Mutational burden as assessed by change in variant allele frequency
Time frame: From inclusion to 12 months of study treatment
Interim efficacy: Patient-reported outcome measures based on the EORTC QLQ-C30
Time frame: From inclusion to 4 months of study treatment
Interim efficacy: Patient-reported outcome measures based on the EORTC QLQ-C30
Time frame: From inclusion to 12 months of study treatment
Interim efficacy: Patient-reported outcome measures based on the SF-36
Time frame: From inclusion to 4 months of study treatment
Interim efficacy: Patient-reported outcome measures based on the SF-36
Time frame: From inclusion to 12 months of study treatment
Interim efficacy: Patient-reported outcome measures based on the EQ-5D
Time frame: From inclusion to 4 months of study treatment
Interim efficacy: Patient-reported outcome measures based on the EQ-5D
Time frame: From inclusion to 12 months of study treatment
Interim efficacy: Bone marrow adipose tissue as assessed by MR spectroscopy ratio of adipose tissue and water phase
Time frame: From inclusion to 4 months of study treatment
Interim efficacy: Bone marrow adipose tissue as assessed by MR spectroscopy ratio of adipose tissue and water phase
Time frame: From inclusion to 12 months of study treatment
Interim efficacy: Bone mineral density as assessed by DEXA scan measured in grams per cubic centimeter with resulting Z score
Time frame: From inclusion to 12 months of study treatment
Interim efficacy: Body composition as assessed by DEXA scan presented as whole body bone mass and soft tissue composition
Time frame: From inclusion to 12 months of study treatment
Interim efficacy: Gut microbiota composition as assessed by 16S rRNA sequencing
Time frame: From inclusion to 4 months of study treatment
Interim efficacy: Gut microbiota composition as assessed by 16S rRNA sequencing
Time frame: From inclusion to 12 months of study treatment
Interim efficacy: Small intestinal permeability as assessed by urine-lactulose/mannitol measurement and ion chromatography
Time frame: From inclusion to 4 months of study treatment
Interim efficacy: Epigenetic regulation as assessed by levels of 5-mC and 5-hmC
Time frame: From inclusion to 4 months of study treatment
Interim efficacy: Epigenetic regulation as assessed by levels of 5-mC and 5-hmC
Time frame: From inclusion to 12 months of study treatment
Interim efficacy: Gene expression as assessed by RNA sequencing
Time frame: From inclusion to 4 months of study treatment
Interim efficacy: Gene expression as assessed by RNA sequencing
Time frame: From inclusion to 12 months of study treatment
Interim efficacy: Protein profiles as assessed by proteomics
Time frame: From inclusion to 4 months of study treatment
Interim efficacy: Protein profiles as assessed by proteomics
Time frame: From inclusion to 12 months of study treatment
Interim efficacy: Response and disease progression as according to the IWG response criteria in myelodysplastic neoplasms
Time frame: From inclusion to 12 months of study treatment
Interim efficacy: Bone marrow niche factor levels as assessed by ELISA
Time frame: From inclusion to 4 months of study treatment
Interim efficacy: Bone marrow niche factor levels as assessed by ELISA
Time frame: From inclusion to 12 months of study treatment
Interim efficacy: Cytokine levels as assessed by ELISA
Time frame: From inclusion to 4 months of study treatment
Interim efficacy: Cytokine levels as assessed by ELISA
Time frame: From inclusion to 12 months of study treatment
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 22 Nov 2024.
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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.
Kirsten Grønbæk, Professor, MD study chair
Copenhagen University Hospital, Rigshospitalet
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Metformin in people with Preleukemia and Myelodysplastic Neoplasm, run by Kirsten Grønbæk at 1 site in Denmark.
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 11 inclusion and 15 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 22 Nov 2024. 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 Denmark. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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 Kirsten Grønbæk, Professor, MD (+45 35456060, Kirsten.Groenbaek@regionh.dk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety as assessed by the number of serious adverse events including any suspected unexpected serious adverse reactions (time frame: From inclusion to 12 months of study treatment). The study lists 5 primary outcomes in all. 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 22 Nov 2024.