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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01326845
First received: March 30, 2011
Last updated: January 24, 2013
Last verified: January 2013
History of Changes
  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:

Drug Information available for: Deferasirox
U.S. FDA Resources

Further study details as provided by Novartis:

Primary Outcome Measures:
  • difference in the frequency of overall newly occurring GI adverse events in the two treatment arms [ Time Frame: 3 months ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • difference in frequency of overall GI adverse events between the two treatment groups based on clinical assessments of events at Month 6. [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
  • difference in frequency of each of diarrhea, vomiting, nausea, abdominal pain and constipation between the two treatment groups based on clinical assessment of events [ Time Frame: months 3 and 6. ] [ Designated as safety issue: Yes ]
  • difference in severity of overall GI adverse events between the two treatment groups based on clinical assessment of events [ Time Frame: months 3 and 6. ] [ Designated as safety issue: Yes ]
  • difference in severity of diarrhea, vomiting, nausea, abdominal pain and constipation in the two treatment groups using the CTC AE v4 grading tool [ Time Frame: months 3 and 6 ] [ Designated as safety issue: Yes ]
  • difference in frequency and severity of all non-GI adverse events between the two treatment groups based on clinical assessment [ Time Frame: months 3 and 6 ] [ Designated as safety issue: Yes ]

Enrollment: 21
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 Drug: Deferasirox
Other Name: ICL670
Experimental: Deferasirox pm 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
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
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
Last Updated: January 24, 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
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 21, 2013