Study for the Treatment of Transfusional Iron Overload in Myelodysplastic Patients

NCT00117507 | Completed Phase 4 Results

• 1 other identifier: CICL670AUS02
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 3

Brief Summary

Thirty patients were to be enrolled and 24 patients were actually enrolled into this open-label, single-arm trial designed to assess the safety and tolerability of oral deferasirox in adult transfusion dependent myelodysplastic syndrome (MDS) patients with iron overload. Patients enrolled in this study had low or intermediate (INT-1) risk MDS per International Prognostic Scoring System (IPSS) criteria. All patients initiated treatment with 20mg/kg/day deferasirox. Deferasirox were administered orally once per day for 12 months.

Conditions

Myelodysplastic Syndromes; Iron Overload

Keywords

MDS; Myelodysplastic Syndrome; ICL-670; ICL-670 and Myelodysplastic Syndrome

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Adverse Events and Serious Adverse Events

  An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign \[including abnormal laboratory findings\], symptom or disease) in a subject or clinical investigation subject after providing written informed consent for participation in the study. Any sign or symptom that occured from first dose of study treatment until end of study treatment.

  Up To Week 52

Secondary Outcomes (7)

• Absolute Change in Serum Ferritin From Baseline to Week 52

  Baseline to Week 52

• Absolute Change in Liver Iron Concentration (LIC) From Baseline to End of Study

  Baseline to Week 52

• To Evaluate Change in Transfusion Requirements

  Baseline to Week 52

• Absolute Change in Serum Erythropoietin

  Baseline to Week 52

• Absolute Change in Urinary Hepcidin

  Baseline to Week 52

Study Arms (1)

Deferasirox

EXPERIMENTAL

Participants received deferasirox 20mg/kg/day OD for 12 months. Deferasirox was taken every morning 30 minutes before breakfast, if possible consistently around the same time between 7:00 and 9:00 AM. The tablets was dropped into water or orange juice and gently stirred for 1 to 3 minutes until completely dispersed.

Drug: Deferasirox

Interventions

Deferasirox DRUG

Also known as: Chelator, Iron chelator

Deferasirox

Eligibility Criteria

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

You may qualify if:

• Male or female patients with low or intermediate (INT-1) risk MDS, determined via IPSS criteria, with transfusional iron overload. NOTE: Bone marrow morphology and cytogenetic studies completed within 3 months prior to screening can be used if the patient has been hematologically stable. Every attempt to obtain cytogenetics studies should be made; however, if there is culture failure, repeat marrow aspiration will not be mandated. In this case, RAEB with less than 11% marrow blasts will be accepted.
• Patients on chelation therapy at the time of screening required a 1-day wash out prior to the first dose of study drug.
• Age: greater than or equal to 18 years
• Serum ferritin:
• For entry into the screening period: serum ferritin greater than or equal to 1000 µg/mL on at least two occasions, at least two weeks apart, during the prior year. Samples must be obtained in the absence of concomitant infection;
• For enrollment into the study: serum ferritin greater than or equal to 1000 µg/mL at screening (via the central lab) obtained in the absence of concomitant infection
• A lifetime minimum of 20 previous packed red cell transfusions
• Life expectancy greater than or equal to 6 months
• Women must have a negative serum or urine pregnancy test and use an effective method of contraception, or must have undergone clinically documented total hysterectomy and/or oophorectomy, or tubal ligation or be postmenopausal (defined by amenorrhea for at least 12 months).
• Able to provide written informed consent

You may not qualify if:

• Serum creatinine greater than 2 × upper limit of normal (ULN)
• ALT or AST greater than 5 × ULN.
• Clinical or laboratory evidence of active hepatitis B or hepatitis C (HBsAg in the absence of HBsAb -OR- HCV Ab positive with HCV RNA positive and ALT above the normal range)
• Significant proteinuria as indicated by a urinary protein/creatinine ratio greater than 0.5 mg/mg in a non-first void urine sample during screening (or alternatively in two of three samples obtained for screening)
• History of HIV positive test result (ELISA or Western blot)
• ECOG performance status greater than 2
• Uncontrolled systemic hypertension
• Unstable cardiac disease not controlled by standard medical therapy
• Third degree atrioventricular (AV) block or QT interval prolongation above the normal range
• History of clinically relevant ocular toxicity related to iron chelation
• Pregnancy or breast feeding
• Treatment with a systemic investigational drug within the past 4 weeks or a topical investigational drug within the past 7 days.
• Other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of any drug. The investigator should be guided by evidence of any of the following:
• inflammatory bowel syndrome, gastritis, ulcers, gastrointestinal or rectal bleeding;
• major gastrointestinal tract surgery, such as gastrectomy, gastroenterostomy, or bowel resection;

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (3)

Stanford University Medical Center

Stanford, California, 94305-5821, United States

Location

Karmanos Cancer Center

Detroit, Michigan, 48201, United States

Location

MD Anderson Cancer Center

Houston, Texas, 77030-4009, United States

Location

Related Publications (1)

• Greenberg PL, Koller CA, Cabantchik ZI, Warsi G, Glynos T, Paley C, Schiffer C. Prospective assessment of effects on iron-overload parameters of deferasirox therapy in patients with myelodysplastic syndromes. Leuk Res. 2010 Dec;34(12):1560-5. doi: 10.1016/j.leukres.2010.06.013. Epub 2010 Jul 8.

  PMID: 20615548 BACKGROUND

Related Links

• Results for CICL670AUS02 from the Novartis Clinical Trials website
• Results for CICL670AUS02 from the Novartis Clinical Trials website

MeSH Terms

Conditions

Myelodysplastic Syndromes; Iron Overload

Interventions

Deferasirox; Chelating Agents

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Iron Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

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; Sequestering Agents; Molecular Mechanisms of Pharmacological Action; Pharmacologic Actions; Chemical Actions and Uses; Specialty Uses of Chemicals

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

Novartis.email@novartis.com

862-778-8300

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 30, 2005
• First Posted: July 7, 2005
• Study Start: September 1, 2005
• Primary Completion: January 1, 2008
• Study Completion: January 1, 2008
• Last Updated: June 24, 2021
• Results First Posted: June 24, 2021

Record last verified: 2021-06

Locations

US (3)