Clinical trial NCT01326845: Myelodysplastic Syndrome (MDS) Gastrointestinal (GI) Tolerability Study
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
Overall status: TERMINATED
Phase: PHASE4
Conditions: Myelodysplastic Syndrome
Transfusional Iron Overload
Keywords: Gastrointestinal adverse events,
myelodysplastic syndrome,
transfusional iron overload diarrhea,
constipation abdominal pain,
vomiting
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2011-03-31
Enrollment: 12
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Difference in the Frequency of Overall Newly Occurring GI Adverse Events (AEs) in the Two Treatment Arms
timeFrame: 3 months
Secondary outcomes:
measure: Difference in Frequency of Overall Newly Occurring GI AEs Between the Two Treatment Groups at Month 6.
timeFrame: 6 months
measure: Difference in Frequency of Specific Commonly Reported GI AEs Between the Two Treatment Groups
timeFrame: months 3 and 6.
measure: Difference in Severity of Overall GI AEs Between the Two Treatment Groups
timeFrame: months 3 and 6.
measure: Difference in Severity of Specific Commonly Reported GI Symptoms Between the Two Treatment Groups
timeFrame: months 3 and 6
measure: Difference in Frequency and Severity of All Non-GI AEs Between the Two Treatment Groups
timeFrame: months 3 and 6
measure: the Difference Between the Time From Baseline to the First Occurrence of GI AEs Between the Two Treatment Groups
timeFrame: 3 months, 6 months
measure: Difference in Severity of GI Symptoms, Bowel Habits and Level of Satisfaction From the Patient's Perspective Between the Two Treatment Groups
timeFrame: 3 months, 6 months
measure: Difference in Reducing Serum Ferritin After Each Month of Study Drug Administration Between the Two Groups
timeFrame: 3 months, 6 months
Other outcomes:
Locations:
city: Caen
country: France
facility: Novartis Investigative Site
state: Cedex
city: Brest
country: France
facility: Novartis Investigative Site
city: Limoges cedex
country: France
facility: Novartis Investigative Site
city: Pessac Cedex
country: France
facility: Novartis Investigative Site
city: Vandoeuvre les Nancy
country: France
facility: Novartis Investigative Site
city: Alessandria
country: Italy
facility: Novartis Investigative Site
state: AL
city: Torino
country: Italy
facility: Novartis Investigative Site
state: TO
city: Roma
country: Italy
facility: Novartis Investigative Site
city: Bloemfontein
country: South Africa
facility: Novartis Investigative Site
city: Madrid
country: Spain
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: