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MDSPhase 2NCT02985190

A Study of Azacitidine in Myelodysplastic Syndrome (MDS) Associated to Systemic Auto-immune and Inflammatory Disorders

A Phase 2 study testing Azacitidine in people with MDS and Systemic Autoimmune Diseases, run by Groupe Francophone des Myelodysplasies at 59 sites in France.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This study is a phase II of effcicacy and tolerance of azacitidine in patients with myelodysplatic syndrome and steroid dependent or resistent systemic auto-immune and inflammatory disorders Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
MDS, Systemic Autoimmune Diseases
What is being tested?
Azacitidine
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
30 participants
Where
59 sitesFrance
Study dates
26 Jan 2017to 30 Aug 2022
Phase
Phase 2
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

Who may be able to join

  • Must understand and voluntarily sign the informed consent form
  • Age 18 years at the time of signing the informed consent form
  • Must be able to adhere to the study visit schedule and other protocol requirements
  • MDS or CMML or AML with 20-30% marrow blasts using 2008 WHO classification, with any of the following characteristics :
  • IPSS intermediate 2 or high, including AML with 20 to 30% marrow blasts and CMML with WBC<13G/L and marrow blasts >10%,
  • IPSS low or int 1 in need of treatment (transfusion dependent anemia resistant to ESAs and/or platelets below 30 G/l or below 50 G/l with bleeding or platelet transfusion requirement, and/or ANC < 0.5 G/l with infectious complications)
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  • Documented (by cytogenetic or molecular analysis) MDS /CMML not meeting those criteria, but with at least one significant cytopenia (Hb <10 g/dl, platelets <50G/l, ANC <1 G/l). In this situation, the underlying SAID should be severe and have resisted to a second line treatment (following steroids), if such treatment can be proposed for this particular SAID. Those cases should be discussed prior to inclusion with the trial sponsors complications)
  • SAID-associated with MDS defined according to usual international criteria for each SAID (ie ACR criteria for systemic lupus, Chapel Hill classification for systemic vasculitis, etc…)
  • Steroid dependence and/or resistance of SAID (steroid dependence being defined as the impossibility to decrease steroids during at least 2 months below 15 mg/day; steroid resistance as no response of SAID to at least 1 mg/kg/day of prednisone equivalent during one month)
  • Ineligibility for allogeneic stem cell transplantation during the following 12 months
  • Wash-out at least 6 months since a previous treatement with Lenalidomide
  • No previous use of hypomethylating agents
  • Life expectancy ≥ 6 months
  • Adequate liver function (serum transaminases ≤ 3N)
  • Adequate renal function (creatinine clearance with MDRD formula > 30 ml/min)
  • Women of child-bearing potential (i.e., women who are pre-menopausal or not surgically sterile) must :
  • Have a negative serum or urine pregnancy test within 2 weeks prior to beginning treatment on this study. Lactating patients are excluded.
  • Agree to use, and to be able to comply with, effective contraception without interruption, 4 weeks before starting study drug throughout the entire duration study drug therapy (including doses interruptions) and for 3 months after the end of the study drug therapy.
  • Male patients must :
  • Agree the need for the use of a condom if engaged in sexual activity with a woman of childbearing potential during the entire period of treatment, even if disruption of treatment and during 3 months after end of treatment.
  • Agree to learn about the procedures for preservation of sperm before starting treatment.

Who cannot join

  • IPSS low and intermediate-1 not meeting the criteria described above
  • Creatinine clearance with MDRD formula < 30 ml/min
  • Serum total bilirubin, or serum transaminases > 3.0 x upper limit of normal (ULN) (except for unconjugated hyperbilirubinemia due to Gilbert's disease or secondary to MDS)
  • Known hypersensitivity to the active substance or to any of the excipients of AZA
  • History of severe congestive heart failure, clinically unstable cardiac or pulmonary disease
  • Previous treatment with hypomethylating agents
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  • Life-expectancy of less than six months because of another debilitating disease
  • Uncontrolled invasive fungal infection at time of registration or active serious infection not controlled by oral or intravenous antibiotics
  • Known positive for HIV or acute infectious hepatitis, type B or C
  • Any serious medical condition or psychiatric illness that will prevent the subject from signing the informed consent form or will place the subject at unacceptable risk if he/she participates in the study.
  • Active cancer or prior history of malignancy other than MDS (except basal cell or squamous cell carcinoma or carcinoma in situ of the cervix or breast) unless the subject has been free of disease for ≥ 3 years
  • Pregnant or lactating females
  • No affiliation to an insurance system

Trial locations and contacts

59 sites in 1 country.

59 sites in 1 country

France59 sites

  • CHU Amiens

    Amiens, 80054

  • CHU d'Angers

    Angers, 49933

  • Centre hospitalier Victor Dupouy

    Argenteuil, 95107

  • CH Henri Duffaut d'Avignon

    Avignon, 84000

  • Centre hospitalier de la Côte Basque

    Bayonne, 64109

  • Hôpital Nord Franche-Comté

    Belfort, 90015

  • Hôpital Avicenne

    Bobigny, 93009

  • Centre Hospitalier de Boulogne Sur Mer

    Boulogne-sur-Mer, 62321

  • CHRU de Brest - Hôpital Morvan

    Brest, 29609

  • CHU Côte de Nacre

    Caen, 14033

  • CH René Dubos

    Cergy-Pontoise, 95303

  • Centre Hospitalier William Morey

    Chalon-sur-Saône, 71100

  • CHU Estaing

    Clermont-Ferrand, 63000

  • CHSF Gilles de Corbeil

    Corbeil-Essonnes, 91100

  • CHU Henri Mondor

    Créteil, 94010

  • CHU François Mitterrand

    Dijon, 21079

  • CHU de Grenoble

    Grenoble, 38043

  • Groupe Hospitalier de la Rochelle Ré Aunis

    La Rochelle, 17000

  • Clinique Victor Hugo - Centre Jean Bernard

    Le Mans, 72000

  • CH Le Mans

    Le Mans, 72037

  • Centre Hospitalier de Lens

    Lens, 62307

  • Hôpital Saint Vincent de Paul

    Lille, 59020

  • Hôpital Claude Huriez

    Lille, 59037

  • CHRU de Limoges - Hôpital Dupuytren

    Limoges, 87042

  • Institut Paoli Calmettes

    Marseille, 13273

  • Centre Hospitalier de Meaux

    Meaux, 77104

  • CH de Mont de Marsan

    Mont-de-Marsan, 40000

  • Clinique Beausoleil

    Montpellier, 34000

  • CHRU de Montpellier - Service de Médecine Interne

    Montpellier, 34295

  • CHU de Montpellier - Service d'hématologie Oncologie

    Montpellier, 34295

  • CHU Nantes - Hôtel Dieu

    Nantes, 44093

  • Centre Catherine de Sienne

    Nantes, 44277

  • Centre Antoine Lacassagne

    Nice, 06189

  • Hôpital Archet 1

    Nice, 06202

  • CHU de Nîmes

    Nîmes, 30029

  • CHR d'Orléans

    Orléans, 45067

  • Hôpital Saint-Louis

    Paris, 75010

  • Hôpital Saint Antoine - Service de Médecine Interne

    Paris, 75012

  • Hôpital de La Pitié-Salpêtrière

    Paris, 75013

  • Hôpital Saint Antoine - Service d'Hématologie Clinique

    Paris, 75571

  • Hôpital Cochin

    Paris, 75679

  • Hôpital Necker

    Paris, 75743

  • Centre Hospitalier Joffre

    Perpignan, 66 046

  • CHU de Haut-Lévèque

    Pessac, 33604

  • Centre Hospitalier Lyon-Sud

    Pierre-Bénite, 69495

  • CHU de Poitiers

    Poitiers, 86021

  • Centre Hospitalier Annecy Genevois

    Pringy, 74374

  • CHU de Reims

    Reims, 51092

  • Hôpital PONTCHAILLOU

    Rennes, 35033

  • Centre Hospitalier de Rochefort

    Rochefort, 17301

  • Centrer Hospitalier de Roubaix

    Roubaix, 59056

  • Centre Henri Becquerel

    Rouen, 76038

  • CH Yves Le Foll

    Saint-Brieuc, 22000

  • Hôpitaux Universitaires de Strasbourg

    Strasbourg, 67091

  • IUCT Oncopole

    Toulouse, 31059

  • Hôpital Bretonneau

    Tours, 37000

  • Centre Hospitalier de Troyes

    Troyes, 10003

  • CH Valence

    Valence, 26953

  • CHU Brabois

    Vandœuvre-lès-Nancy, 54511

Other trials in France

About this study

Official title: A Phase II Study of Efficacy and Tolerance of Azacitidine (AZA) In MDS-associated Steroid Dependent/Refractory Systemic Auto-immune and Inflammatory Disorders (SAID)

This study is a phase II of effcicacy and tolerance of azacitidine in patients with myelodysplatic syndrome and steroid dependent or resistent systemic auto-immune and inflammatory disorders

Read the detailed description

This trial will be a prospective French nationwide study analyzing the effect of treatment with azacitidine in patients with MDS-associated SAID with steroid dependence and/or resistance, and its correlation with possible changes in immunological parameters

Being tested
Azacitidine
Keywords
Azacitidine, MDS, Systemic auto-immune and inflammatory disorders

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • AzacitidineDrug

    Also called Vidaza

    Azacitidine at 75mg/m²/j for 7 days.

Study groups in NCT02985190
Study groupRoleWhat they receive
Azacitidine 75mg/m²/dayExperimentalAzacitidine 75mg/m²/j subcutaneously daily for 7 days every 4 weeks for a minimum of 6 cycles (unless overt disease progression, especially to Acute Myeloid Leukemia (AML) occure before 6 cycles) Azacitidine will be continued after 6 cycles * in patients with hematological response of myelodysplastic syndrome to azacitidine according to IWG2006 criteria by 6 cycles (Complete Response (CR), Partial Response (PR), marrow Complete Response (CRm), stable disease with Hematological Improvment (HI)), for another 6 cycles * in patients with complete or partial response of Systemic Auto-Immune Disorders (SAID) after 6 cycles of Azacitidine, even if Myelodysplastic Syndrome remains only stable per IWG2006 criteria
Receives: Drug: Azacitidine

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Overall response rate of Myelodysplastic syndrome and systemic autoimmune and inflammatory diseases (SAID)

    Overall response rate (including partial and complete response) of systemic autoimmune and inflammatory diseases associated with Myelodysplastic syndrome after 6 cycles of azacitidine

    Measured: 6 months

Secondary outcomes (1)

  1. Number of participants with treament-related adverse events as assessed by CTCAE v4.0

    Number of participants with treament-related adverse events as assessed by CTCAE v4.0

    Measured: up to 52 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted7 Dec 2016 (estimated)
  2. Study start26 Jan 2017
  3. Primary completion2 Sep 2021
  4. Study completion30 Aug 2022

Resources

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
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
30 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02985190
Sponsor's protocol ID
GFM-AZA-SAID
Other IDs
2016-000918-30, 2016
Status last verified
Oct 2022
First submitted
7 Nov 2016
Last updated
4 Oct 2022
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Undecided

Publications

  • Boy M, Bisio V, Zhao LP, Guidez F, Schell B, Lereclus E, Henry G, Villemonteix J, Rodrigues-Lima F, Gagne K, Retiere C, Larcher L, Kim R, Clappier E, Sebert M, Mekinian A, Fain O, Caignard A, Espeli M, Balabanian K, Toubert A, Fenaux P, Ades L, Dulphy N. Myelodysplastic Syndrome associated TET2 mutations affect NK cell function and genome methylation. Nat Commun. 2023 Feb 3;14(1):588. doi: 10.1038/s41467-023-36193-w.

    PubMed 36737440

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Azacitidine in people with MDS and Systemic Autoimmune Diseases, run by Groupe Francophone des Myelodysplasies at 59 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 21 inclusion and 13 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: finished, as of the sponsor's last update on 4 Oct 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 59 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.

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?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

Overall response rate of Myelodysplastic syndrome and systemic autoimmune and inflammatory diseases (SAID) (time frame: 6 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02985190, which also lists 1 linked publication.

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 4 Oct 2022.

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