Study to investigate the benefits of an improved deferasirox formulatio

Phase 1

• Conditions: transfusion-dependent thalassemia or myelodysplastic syndrome at very low, low or intermediate (int-1) risk; MedDRA version: 16.1Level: LLTClassification code 10054658Term: ThalassemiaSystem Organ Class: 100000004850; MedDRA version: 16.1Level: LLTClassification code 10028534Term: Myelodysplastic syndrome NOSSystem Organ Class: 100000004864; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2013-004167-32-ES
• Lead Sponsor: ovartis Farmacéutica, S.A.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2014-03-31
• Last Update Posted: 29 August 2016

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 150

Inclusion Criteria

1. Written informed consent/assent before any study-specific procedures. For pediatric patients, consent will be obtained from parent(s) or legal patient?s representative. Investigators will also obtain assent of patients according to local, regional or national guidelines.
2. Male and female patients aged ? 10 years
3. Patients with transfusion-dependent thalassemia and iron overload, requiring deferasirox DT at doses of ? 30 mg/kg/day as per the investigator?s decision
OR
Patients with very low, low or intermediate (int-1) risk myelodysplastic syndrome (MDS) and iron overload, requiring deferasirox DT at doses of ? 20 mg/kg/day as per the investigator?s decision.
? The very low, low or intermediate (int-1) risk MDS should be determined by the Revised International Prognostic Scoring System (IPSS-R) and IPSS-R must be confirmed by a bone marrow examination within 6 months prior to study entry and must be hematologically stable with a patient?s life expectancy of at least 1 year.
4. History of transfusion of at least 20 PRBC units and anticipated to be transfused with at least 8 units of PRBCs annually during the study
5. Serum ferritin > 1000 ng/mL, measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria)
Are the trial subjects under 18? yes
Number of subjects for this age range: 30
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 105
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 15

Exclusion Criteria

1. Creatinine clearance below the contraindication limit in the locally approved prescribing information. Creatinine clearance will be estimated from serum creatinine at screening Visit 1 and screening Visit 2 and the mean value will be used for eligibility criteria.
2. Serum creatinine > 1.5 xULN at screening measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria).
3. ALT (SGPT) > 5xULN, unless if LIC confirmed as <10 mg Fe/dw within 6 months prior to screening visit 1.
4. Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 mg/mg in a non-first void urine sample at screening Visit 1 or screening Visit 2.
5. Patients with significant impaired gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral deferasirox (e.g. ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection).
6. 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).
7. Patients with psychiatric or addictive disorders which prevent them from giving their informed consent or undergoing any of the treatment options or patients unwilling or unable to comply with the protocol (including use of electronic devices for ePRO).
8. Patients with a known history of HIV seropositivity (Elisa or Western blot).
9. History of malignancy of any organ system, treated or untreated, within the past 5 years whether or not there is evidence of local recurrence or metastases, with the exception of localized basal cell carcinoma of the skin.
10. Patients participating in another clinical trial or receiving an investigational drug.
11. History of hypersensitivity to any of the study drug or excipients.
12. Significant medical condition interfering with the ability to partake in this study (e.g. systemic uncontrolled hypertension, unstable cardiac disease not controlled by standard medical therapy, systemic disease (cardiovascular, renal, hepatic, etc.).
13. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception during dosing of study treatment.
14. Sexually active males unless they use a condom during intercourse while taking drug and
for 28 days after stopping study medication and should not father a child in this period. A condom is required to be used also by vasectomized men in order to prevent delivery of the drug via seminal fluid.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified