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

Novartis

Status and phase: Terminated; Phase 4

Conditions: Transfusional Iron Overload; Myelodysplastic Syndrome

Treatments: Drug: Deferasirox

Study type: Interventional

Funder types: Industry

Identifiers

NCT01326845

2011-001077-13 (EudraCT Number)

CICL670A2417

Details and patient eligibility

About

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.

Enrollment: 12 patients

Sex: All

Ages: 18+ years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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12 participants in 2 patient groups

Deferasirox am

Experimental group

Description:

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

Treatment:

Drug: Deferasirox

Deferasirox pm

Experimental group

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

Treatment:

Drug: Deferasirox