search
Back to results

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

Primary Purpose

Myelodysplastic Syndrome, Transfusional Iron Overload

Status
Terminated
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
Deferasirox
Sponsored by
Novartis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Myelodysplastic Syndrome focused on measuring Gastrointestinal adverse events,, myelodysplastic syndrome,, transfusional iron overload diarrhea,, constipation abdominal pain,, vomiting

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Deferasirox am

Deferasirox pm

Arm Description

Deferasirox 20 mg/kg/day taken in the morning, 30 minutes before food

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

Outcomes

Primary Outcome Measures

Difference in the Frequency of Overall Newly Occurring GI Adverse Events (AEs) in the Two Treatment Arms
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.
Study was prematurely terminated and not powered for efficacy.
Difference in Frequency of Specific Commonly Reported GI AEs Between the Two Treatment Groups
Study was prematurely terminated and not powered for efficacy.
Difference in Severity of Overall GI AEs Between the Two Treatment Groups
Study was prematurely terminated and not powered for efficacy.
Difference in Severity of Specific Commonly Reported GI Symptoms Between the Two Treatment Groups
Study was prematurely terminated and not powered for efficacy.
Difference in Frequency and Severity of All Non-GI AEs Between the Two Treatment Groups
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
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
Difference in Reducing Serum Ferritin After Each Month of Study Drug Administration Between the Two Groups
Study was prematurely terminated and not powered for efficacy.

Full Information

First Posted
March 30, 2011
Last Updated
March 1, 2017
Sponsor
Novartis Pharmaceuticals
search

1. Study Identification

Unique Protocol Identification Number
NCT01326845
Brief Title
Myelodysplastic Syndrome (MDS) Gastrointestinal (GI) Tolerability Study
Acronym
MACS1574
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.
Study Type
Interventional

2. Study Status

Record Verification Date
March 2017
Overall Recruitment Status
Terminated
Why Stopped
The trial was terminated due to insufficient enrollment.
Study Start Date
December 2011 (undefined)
Primary Completion Date
September 2012 (Actual)
Study Completion Date
September 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals

4. Oversight

5. Study Description

Brief Summary
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myelodysplastic Syndrome, Transfusional Iron Overload
Keywords
Gastrointestinal adverse events,, myelodysplastic syndrome,, transfusional iron overload diarrhea,, constipation abdominal pain,, vomiting

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
12 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Deferasirox am
Arm Type
Experimental
Arm Description
Deferasirox 20 mg/kg/day taken in the morning, 30 minutes before food
Arm Title
Deferasirox pm
Arm Type
Experimental
Arm Description
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
Intervention Type
Drug
Intervention Name(s)
Deferasirox
Other Intervention Name(s)
ICL670
Primary Outcome Measure Information:
Title
Difference in the Frequency of Overall Newly Occurring GI Adverse Events (AEs) in the Two Treatment Arms
Description
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.
Time Frame
3 months
Secondary Outcome Measure Information:
Title
Difference in Frequency of Overall Newly Occurring GI AEs Between the Two Treatment Groups at Month 6.
Description
Study was prematurely terminated and not powered for efficacy.
Time Frame
6 months
Title
Difference in Frequency of Specific Commonly Reported GI AEs Between the Two Treatment Groups
Description
Study was prematurely terminated and not powered for efficacy.
Time Frame
months 3 and 6.
Title
Difference in Severity of Overall GI AEs Between the Two Treatment Groups
Description
Study was prematurely terminated and not powered for efficacy.
Time Frame
months 3 and 6.
Title
Difference in Severity of Specific Commonly Reported GI Symptoms Between the Two Treatment Groups
Description
Study was prematurely terminated and not powered for efficacy.
Time Frame
months 3 and 6
Title
Difference in Frequency and Severity of All Non-GI AEs Between the Two Treatment Groups
Description
Study was prematurely terminated and not powered for efficacy.
Time Frame
months 3 and 6
Title
the Difference Between the Time From Baseline to the First Occurrence of GI AEs Between the Two Treatment Groups
Description
Study was prematurely terminated and not powered for efficacy.
Time Frame
3 months, 6 months
Title
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
Title
Difference in Reducing Serum Ferritin After Each Month of Study Drug Administration Between the Two Groups
Description
Study was prematurely terminated and not powered for efficacy.
Time Frame
3 months, 6 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Novartis Investigative Site
City
Caen
State/Province
Cedex
ZIP/Postal Code
14033
Country
France
Facility Name
Novartis Investigative Site
City
Brest
ZIP/Postal Code
29200
Country
France
Facility Name
Novartis Investigative Site
City
Limoges cedex
ZIP/Postal Code
87042
Country
France
Facility Name
Novartis Investigative Site
City
Pessac Cedex
ZIP/Postal Code
33604
Country
France
Facility Name
Novartis Investigative Site
City
Vandoeuvre les Nancy
ZIP/Postal Code
54511
Country
France
Facility Name
Novartis Investigative Site
City
Alessandria
State/Province
AL
ZIP/Postal Code
15100
Country
Italy
Facility Name
Novartis Investigative Site
City
Torino
State/Province
TO
ZIP/Postal Code
10126
Country
Italy
Facility Name
Novartis Investigative Site
City
Roma
ZIP/Postal Code
00133
Country
Italy
Facility Name
Novartis Investigative Site
City
Bloemfontein
ZIP/Postal Code
901
Country
South Africa
Facility Name
Novartis Investigative Site
City
Madrid
ZIP/Postal Code
28033
Country
Spain

12. IPD Sharing Statement

Learn more about this trial

Myelodysplastic Syndrome (MDS) Gastrointestinal (GI) Tolerability Study

We'll reach out to this number within 24 hrs