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Myelodysplastic SyndromesPhase 2NCT01058369

Exjade-Early-Trial

A Phase 2 study testing Deferasirox (Novartis Pharma) in people with Myelodysplastic Syndromes, run by University of Erlangen-Nürnberg Medical School at 1 site in Germany.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

Study outline: Deferasirox (Exjade®) is regularly used in severe iron overload in order to avoid organ damage of liver, heart and other organs. It has been proposed, that iron overload may not only impose damage to other organs but also to the bone marrow and thus worsen hematopoietic insufficiency in patients with… 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?
Deferasirox (Novartis Pharma)
Could I receive a placebo?
No, everyone receives an active treatment
How many people
2 participants
Where
1 siteGermany
Study dates
Apr 2010to Jan 2013
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

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

  • MDS of subtype RA, RARS, RCMD, RCMD-RS (i.e. lower risk)
  • RAEB I allowed, if clinically stable for > 3 months
  • 5q-minus syndrome allowed, if lenalidomide unsuccessful or unavailable at the time of inclusion
  • IPSS score < intermediate-1
  • transfusion dependent or Hb < 10,5 g/dl
  • History of less than 20 units of red blood cell transfusions or 100mL/kg of prepacked red blood cells (PRBCs), except for transfusions for acute bleeding
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  • Serum ferritin > 300 µg/l and < 1500 μg/l. This level should have been verified at least at two occasions within 3 months. Samples must be obtained in the absence of concomitant severe infection
  • no indication for EPO (due to high endogenous EPO levels) or EPO without benefit in the past
  • no indication and/or no plans for cytostatic drugs
  • no previous exposure to cytostatic drugs, thalidomide, lenalidomide, G-CSF or EPO or exposure to any of these drugs has been terminated since > 8 weeks (4 weeks for G-CSF).
  • no indication and/or no plans for stem cell transplantation
  • stable or worsening cytopenia during the past 8 weeks. If in doubt, extend screening period to >= 8 weeks
  • Patients of either gender and age > 18 years
  • Life expectancy > 12 months
  • Females of childbearing potential must use double-barrier contraception (for example orale contraception and condom).
  • Mental ability of the patient to understand explications concerning the study and to understand and follow instructions of the investigating physician
  • Written informed consent by the patient

Who cannot join

  • Treatment with deferasirox or other chelation therapy for periods > 4 weeks before study start
  • Patients with intolerance to Deferasirox
  • Patients with a concomitant second malignant disease, possibly interfering with life expectancy
  • Patients with mean levels of alanine aminotransferase (ALT) > 5x ULN
  • Patients with uncontrolled systemic hypertension
  • Patients with serum creatinine > 1.5x the upper limit of normal (ULN) or a creatinine clearance < 60 ml/min according to the MDRD formula (Levey 2005)
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  • History of nephrotic syndrome
  • Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent the patient from undergoing study treatment
  • Patients with psychiatric or addictive disorders which prevent them from giving their informed consent or undergoing study treatment
  • Patients treated with systemic investigational drugs within the past 4 weeks or topical investigational drug within the past 7 days
  • Any other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of any drug. The investigator should be guided by evidence of any of the following:
  • history of inflammatory bowel syndrome, gastritis, ulcers, gastrointestinal or rectal bleeding;
  • history of major gastrointestinal tract surgery such as gastrectomy, gastroenterostomy, or bowel resection;
  • history of pancreatic injury or pancreatitis; indications of impaired pancreatic function/injury as indicated by abnormal lipase or amylase;
  • history of urinary obstruction or difficulty in voiding
  • History of non-compliance to medical regimens and patients who are considered potentially unreliable and/or not cooperative
  • History of drug or alcohol abuse within the 12 months prior to dosing or evidence of such abuse as indicated by laboratory assays conducted during the screening period
  • Patients with active uncontrolled infectious disease
  • Pregnancy or breast feeding
  • QT > 470 msec on screening ECG
  • Patients with a history of Torsades de Pointes

Trial locations and contacts

1 site in 1 country.

Germany1 site

  • Medizinische Klinik 5, Universitätsklinikum Erlangen

    Erlangen, Bavaria, 91054

Other trials in Germany

About this study

Official title: Early Treatment With Deferasirox (Exjade®) in Low Risk MDS - a Prospective Multicentre Single-arm Single-stage Phase II Study -

Study outline: Deferasirox (Exjade®) is regularly used in severe iron overload in order to avoid organ damage of liver, heart and other organs. It has been proposed, that iron overload may not only impose damage to other organs but also to the bone marrow and thus worsen hematopoietic insufficiency in patients with MDS. Patients presenting with low or INT-1 risk MDS with only mild iron overload will be treated with deferasirox in this study. It will be analyzed if hematological improvement can be observed during this treatment.

Being tested
Deferasirox (Novartis Pharma)
Medical subject headings
Myelodysplastic Syndromes

What participants receive

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

  • Deferasirox (Novartis Pharma)Drug

    Also called Exjade(R)

    Treatment period 102 weeks. Starting dose 10mg/kg/day. Up to 30/mg/kg according to dose adjustment table as specified in the protocol

Study groups in NCT01058369
Study groupRoleWhat they receive
DeferasiroxExperimentalNot described
Receives: Drug: Deferasirox (Novartis Pharma)

What is being measured

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

  1. Fraction of Patients With Hematologic Improvement According to Modified IWG Criteria (Reduction of Transfusions and/or Increase in Hb, Improvement of Neutropenia and Thrombocytopenia)

    Measured: within two years

Secondary outcomes (7)

  1. Evaluate the Safety and Tolerability Profile of Deferasirox in MDS Patients

    Measured: within two years

  2. Effectiveness of Iron Depletion

    Measured: within two years

  3. Correlation Between Hematological Improvement and Effectiveness of Iron Depletion

    Measured: two years

  4. Development of Bone Marrow Morphology

    Measured: two years

  5. Correlation Between Hematological Improvement and Pretreatment Parameters. Extension of This Analysis to MDS Patients on Deferasirox Within the Licensed Indication (More Severe Iron Overload)

    Measured: two years

  6. Overall Survival

    Measured: within two years

  7. AML-free Survival

    Measured: within two years

Timeline

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

  1. First posted28 Jan 2010 (estimated)
  2. Study startApr 2010
  3. Primary completionJan 2013
  4. Study completionJan 2013

Why it stopped: Insufficient patient recruitment (only 2 patients)

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
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
2 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01058369
Sponsor's protocol ID
CICL670ADE06T
Status last verified
Sep 2020
First submitted
27 Jan 2010
Last updated
6 Oct 2020

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Deferasirox (Novartis Pharma) in people with Myelodysplastic Syndromes, run by University of Erlangen-Nürnberg Medical School at 1 site in Germany.

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 17 inclusion and 21 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 6 Oct 2020. 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 Germany. 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?

Yes. Results are posted in the registry (first posted 6 Oct 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Fraction of Patients With Hematologic Improvement According to Modified IWG Criteria (Reduction of Transfusions and/or Increase in Hb, Improvement of Neutropenia and Thrombocytopenia) (time frame: within two years). 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 NCT01058369.

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 6 Oct 2020; AzTrials last refreshed its copy on 2 Nov 2026.

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