Phase 2
N=173
Study of Deferasirox for Treatment of Transfusional Iron Overload in Myelodysplastic Patients
Myelodysplastic Syndrome · Iron Overload
Bottom Line
View on ClinicalTrials.gov: NCT00110266 ↗Enrolled (actual)
173
Serious AEs
44.3%
Results posted
Aug 2021
Primary outcome: Primary: Number of Participants Reporting Adverse Events — 171 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Deferasirox (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Novartis Pharmaceuticals
- Primary completion
- Mar 2008
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Reporting Adverse Events |
171 | — |
| SECONDARY Change in Serum Ferritin From Baseline to Weeks 13, 25, 37 and 53 |
2771.5; -146.5; -167.5; -505.0; -592.0 | — |
| SECONDARY Change in Labile Plasma Iron (LPI) |
0.30; -0.10; -0.20; -0.30; -0.45 | — |
| SECONDARY Directly Chelatable Iron (DCI) |
0.00; 0.00; 0.00; 0.00; 0.00 | — |
| SECONDARY Total Iron Levels |
194.0; 230.50; 222.0; 212.0; 218.0; 221.00 | — |
| SECONDARY Serum Transferrin Levels |
156.0; 161.0; 160.0; 158.0; 160.0; 161.00 | — |
| SECONDARY Transferrin Saturation |
91.00; -11.00; -11.00; -12.50; -12.00; -18.00 | — |
| SECONDARY Transfusion Requirements |
163; 136; 119; 98; 72 | — |
| SECONDARY Frequency of Hematologic Improvement During the Study |
7; 74; 10 | — |
| SECONDARY Trough Plasma Deferasirox Concentration |
21.200; 26.700; 25.200; 30.700 | — |
| SECONDARY Treatment Compliance to Deferasirox |
2; 1; 4; 89; 65; 12 | — |
| SECONDARY The Prevalence of Hereditary Hemochromatosis Gene (HFE) Gene Mutations |
85; 9; 70; 24; 92; 2 | — |
Summary
The purpose of this trial is to examine the safety and efficacy of deferasirox in patients with Myelodysplastic Syndrome (MDS) and chronic iron overload from blood transfusions.
Eligibility Criteria
Inclusion Criteria
- Male or female patients with low or intermediate (INT-1) risk MDS
- Patients can be EITHER naïve to iron chelation OR have had prior treatment with deferoxamine (DFO).
- Age greater than or equal to 18 years
- Availability of transfusion records for the 12 weeks prior to registration
- A lifetime minimum of 30 previous packed red blood cell transfusions
- Availability of at least three CBC values (pretransfusion) during the 12 weeks prior to registration
- Serum Ferritin:
For entry into the screening period, serum ferritin ≥ 1000 ng/mL on at least two occasions, at least two weeks apart, during the prior year.
Serum ferritin ≥ 1000 ng/mL at screening via the central lab.
- Life expectancy ≥ 6 months
- Sexually active women must use an effective method of contraception, or must have undergone clinically documented total hysterectomy and/or oophorectomy, or tubal ligation or be postmenopausal (defined as amenorrhea for at least 12 months)
- Able to provide written informed consent
Exclusion Criteria
- Serum creatinine above the upper limit of normal
- Alanine aminotransferase (ALT) > 500 U/L during screening
- Clinical or laboratory evidence of active Hepatitis B or C
- Urinary protein/creatinine ratio > 0.5 mg/mg
- History of HIV positive test result (ELISA or Western blot)
- Eastern Cooperative Oncology Group (ECOG) Performance Status > 2
- Patients with uncontrolled systemic hypertension
- Unstable cardiac disease not controlled by standard medical therapy
- Patients with a diagnosis of or history of clinically relevant ocular toxicity related to iron chelation
- Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent study treatment
- Pregnancy or breast feeding
- Treatment with systemic investigational drug within the past 4 weeks or topical investigational drug within the past 7 days
- Other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of study drug
- History of non-compliance to medical regimens or patients who are considered potentially unreliable and/or not cooperative
Data sourced from ClinicalTrials.gov (NCT00110266). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.