Myelodysplastic Syndrome (MDS) Gastrointestinal (GI) Tolerability Study (MACS1574)

This study has been terminated.
(The trial was terminated due to insufficient enrollment.)
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01326845
First received: March 30, 2011
Last updated: September 11, 2013
Last verified: September 2013
  Purpose

The objective of the study is to evaluate and compare the frequency and severity of GI adverse events in different dose administration regimens. The patient population consists of low or intermediate (int-1) risk myelodysplastic syndrome (MDS) patients with transfusional iron overload. The study patients are randomized to either a morning dose of 20 mg/kg/day deferasirox or an evening dose of the same. Patients are then followed up for 6 months for any GI events and are assessed using patient reported outcomes tools e.g. a patient diary.


Condition Intervention Phase
Myelodysplastic Syndrome,
Transfusional Iron Overload
Drug: Deferasirox
Phase 4

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Multicenter, Randomized, Comparative Study of Different Deferasirox Administration Regimens on Gastrointestinal (GI) Tolerability in Low or Intermediate (Int-1) Risk MDS Myelodysplastic Syndrome Patients With Transfusional Iron Overload.

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • Difference in the Frequency of Overall Newly Occurring GI Adverse Events (AEs) in the Two Treatment Arms [ Time Frame: 3 months ] [ Designated as safety issue: Yes ]
    Study was prematurely terminated and not powered for efficacy. Frequency of GI AEs during the overall study period is available in the AE tables reported in the safety section.


Secondary Outcome Measures:
  • Difference in Frequency of Overall Newly Occurring GI AEs Between the Two Treatment Groups at Month 6. [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
    Study was prematurely terminated and not powered for efficacy.

  • Difference in Frequency of Specific Commonly Reported GI AEs Between the Two Treatment Groups [ Time Frame: months 3 and 6. ] [ Designated as safety issue: Yes ]
    Study was prematurely terminated and not powered for efficacy.

  • Difference in Severity of Overall GI AEs Between the Two Treatment Groups [ Time Frame: months 3 and 6. ] [ Designated as safety issue: Yes ]
    Study was prematurely terminated and not powered for efficacy.

  • Difference in Severity of Specific Commonly Reported GI Symptoms Between the Two Treatment Groups [ Time Frame: months 3 and 6 ] [ Designated as safety issue: Yes ]
    Study was prematurely terminated and not powered for efficacy.

  • Difference in Frequency and Severity of All Non-GI AEs Between the Two Treatment Groups [ Time Frame: months 3 and 6 ] [ Designated as safety issue: Yes ]
    Study was prematurely terminated and not powered for efficacy.

  • the Difference Between the Time From Baseline to the First Occurrence of GI AEs Between the Two Treatment Groups [ Time Frame: 3 months, 6 months ] [ Designated as safety issue: Yes ]
    Study was prematurely terminated and not powered for efficacy.

  • Difference in Severity of GI Symptoms, Bowel Habits and Level of Satisfaction From the Patient's Perspective Between the Two Treatment Groups [ Time Frame: 3 months, 6 months ] [ Designated as safety issue: No ]
  • Difference in Reducing Serum Ferritin After Each Month of Study Drug Administration Between the Two Groups [ Time Frame: 3 months, 6 months ] [ Designated as safety issue: No ]
    Study was prematurely terminated and not powered for efficacy.


Enrollment: 12
Study Start Date: December 2011
Study Completion Date: September 2012
Primary Completion Date: September 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Deferasirox am
Deferasirox 20 mg/kg/day taken in the morning, 30 minutes before food
Drug: Deferasirox
Other Name: ICL670
Experimental: Deferasirox pm
Deferasirox 20 mg/kg/day taken in the evening, no less than 2 hours after the last food intake or at least 30 minutes before the evening meal
Drug: Deferasirox
Other Name: ICL670

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Written informed consent prior to any screening procedures
  • Male or female patients ≥ 18 years of age
  • Patient must weigh between 45-135 kg
  • Patients with low or intermediate (int-1) risk MDS, as determined by IPSS score or RA, RARS by WHO criteria. IPSS must be confirmed by a bone marrow examination within 6 months prior to study entry and must be hematologically stable

Deferasirox naïve:

Sexually active pre-menopausal female patients must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation

Exclusion Criteria:

  • History or current GI disease
  • Systemic diseases which could prevent study treatments
  • Left ventricular ejection fraction< 50 % by echo cardiography
  • Serum creatinine > 1.2 x ULN at screening
  • Platelet counts < 25x 109/L except in cases where guidance is already given in the local deferasirox label
  • AST or ALT > 2.5 xULN at screening

Other protocol-defined inclusion/exclusion criteria may apply

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01326845

Locations
France
Novartis Investigative Site
Caen, Cedex, France, 14033
Novartis Investigative Site
Brest, France, 29200
Novartis Investigative Site
Limoges cedex, France, 87042
Novartis Investigative Site
Pessac Cedex, France, 33604
Novartis Investigative Site
Vandoeuvre les Nancy, France, 54511
Italy
Novartis Investigative Site
Alessandria, AL, Italy, 15100
Novartis Investigative Site
Torino, TO, Italy, 10126
Novartis Investigative Site
Roma, Italy, 00133
South Africa
Novartis Investigative Site
Bloemfontein, South Africa, 901
Spain
Novartis Investigative Site
Madrid, Spain, 28033
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01326845     History of Changes
Other Study ID Numbers: CICL670A2417, 2011-001077-13
Study First Received: March 30, 2011
Results First Received: September 11, 2013
Last Updated: September 11, 2013
Health Authority: United States: Food and Drug Administration
France: Agence française de sécurité sanitaire des produits de santé
Germany: Bundesinstitut fϋr Arzneimittel und Medizinprodukte
Hungary: National Institute of Pharmacy
Spain: Agencie Española de Medicamentos y Productos Sanitarios

Keywords provided by Novartis:
Gastrointestinal adverse events,
myelodysplastic syndrome,
transfusional iron overload diarrhea,
constipation abdominal pain,
vomiting

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Preleukemia
Iron Overload
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms
Iron Metabolism Disorders
Metabolic Diseases
Deferasirox
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on August 20, 2014