Myelodysplastic Syndrome (MDS) Gastrointestinal (GI) Tolerability Study

NCT01326845 | Terminated Phase 4 Results

• 2 other identifiers: CICL670A24172011-001077-13
• Study Type: interventional
• Target: 12
• Locations: 4 countries
• Sites: 10

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.

Conditions

Myelodysplastic Syndrome; Transfusional Iron Overload

Keywords

Gastrointestinal adverse events,; myelodysplastic syndrome,; transfusional iron overload diarrhea,; constipation abdominal pain,; vomiting

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (8)

• Difference in Frequency of Overall Newly Occurring GI AEs Between the Two Treatment Groups at Month 6.

  6 months

• Difference in Frequency of Specific Commonly Reported GI AEs Between the Two Treatment Groups

  months 3 and 6.

• Difference in Severity of Overall GI AEs Between the Two Treatment Groups

  months 3 and 6.

• Difference in Severity of Specific Commonly Reported GI Symptoms Between the Two Treatment Groups

  months 3 and 6

• Difference in Frequency and Severity of All Non-GI AEs Between the Two Treatment Groups

  months 3 and 6

Study Arms (2)

Deferasirox am

EXPERIMENTAL

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

Drug: Deferasirox

Deferasirox pm

EXPERIMENTAL

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

Drug: Deferasirox

Interventions

Deferasirox DRUG

Also known as: ICL670

Deferasirox am; Deferasirox pm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (10)

Novartis Investigative Site

Caen, Cedex, 14033, France

Location

Novartis Investigative Site

Brest, 29200, France

Location

Novartis Investigative Site

Limoges, 87042, France

Location

Novartis Investigative Site

Pessac, 33604, France

Location

Novartis Investigative Site

Vandœuvre-lès-Nancy, 54511, France

Location

Novartis Investigative Site

Alessandria, AL, 15100, Italy

Location

Novartis Investigative Site

Torino, TO, 10126, Italy

Location

Novartis Investigative Site

Roma, 00133, Italy

Location

Novartis Investigative Site

Bloemfontein, 901, South Africa

Location

Novartis Investigative Site

Madrid, Madrid, 28033, Spain

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes; Vomiting

Interventions

Deferasirox

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Signs and Symptoms, Digestive; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Benzoates; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Triazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

862-778-8300

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 30, 2011
• First Posted: March 31, 2011
• Study Start: December 1, 2011
• Primary Completion: September 1, 2012
• Study Completion: September 1, 2012
• Last Updated: March 3, 2017
• Results First Posted: November 15, 2013

Record last verified: 2017-03

Locations

ES (1); FR (5); IT (3); ZA (1)