Myelodysplastic Syndrome (MDS) Gastrointestinal (GI) Tolerability Study

Myelodysplastic Syndrome Clinical Trial

— 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.

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01326845
• Other study ID #: CICL670A2417
• Secondary ID: 2011-001077-13
• Status: Terminated
• Phase: Phase 4
• First received: March 30, 2011
• Last updated: March 1, 2017
• Start date: December 2011
• Est. completion date: September 2012

Study information

• Verified date: March 2017
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 12
• Est. completion date: September 2012
• Est. primary completion date: September 2012
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Iron Overload
• Myelodysplastic Syndrome
• Myelodysplastic Syndromes
• Preleukemia
• Syndrome
• Transfusional Iron Overload

Intervention

Drug:
• Deferasirox

Locations

Country	Name	City	State
France	Novartis Investigative Site	Brest
France	Novartis Investigative Site	Caen	Cedex
France	Novartis Investigative Site	Limoges cedex
France	Novartis Investigative Site	Pessac Cedex
France	Novartis Investigative Site	Vandoeuvre les Nancy
Italy	Novartis Investigative Site	Alessandria	AL
Italy	Novartis Investigative Site	Roma
Italy	Novartis Investigative Site	Torino	TO
South Africa	Novartis Investigative Site	Bloemfontein
Spain	Novartis Investigative Site	Madrid

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

France,  Italy,  South Africa,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	3 months
Secondary	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.	6 months
Secondary	Difference in Frequency of Specific Commonly Reported GI AEs Between the Two Treatment Groups	Study was prematurely terminated and not powered for efficacy.	months 3 and 6.
Secondary	Difference in Severity of Overall GI AEs Between the Two Treatment Groups	Study was prematurely terminated and not powered for efficacy.	months 3 and 6.
Secondary	Difference in Severity of Specific Commonly Reported GI Symptoms Between the Two Treatment Groups	Study was prematurely terminated and not powered for efficacy.	months 3 and 6
Secondary	Difference in Frequency and Severity of All Non-GI AEs Between the Two Treatment Groups	Study was prematurely terminated and not powered for efficacy.	months 3 and 6
Secondary	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.	3 months, 6 months
Secondary	Difference in Severity of GI Symptoms, Bowel Habits and Level of Satisfaction From the Patient's Perspective Between the Two Treatment Groups		3 months, 6 months
Secondary	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.	3 months, 6 months