Study to evaluate the efficacy and safety of deferasirox film-coated tablet versus phlebotomy in patients with HH

Recruitment & Eligibility

Status: Not Recruiting

Sex: All

Target Recruitment: 150

Inclusion Criteria

- Male or female = 18-years-old
- Documented genotype testing confirming homozygous for the C282Y mutation (C282Y/C282Y)
- Transferrin saturation = 45% (at either screening visit)
- Serum Ferritin = 500 µg/L (at either screening visit)
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 120
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 30

Exclusion Criteria

1. Medical conditions that preclude inclusion:
- Iron overload not due to HH
- Condition which might significantly alter the absorption, distribution,
metabolism or excretion of oral deferasirox
- Systemic disease which prevents taking study treatment or any
contraindication to phlebotomy
- Inflammatory condition or immunological disease which may interfere
with the SF interpretation, such as an active infection, collagen vascular
disorders, irritable bowel syndrome, lupus, or immune thrombocytopenia
- Significantly impaired gastrointestinal function or disease that may
significantly alter the absorption of oral deferasirox, e.g. ulcerative
diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption
- Psychiatric or addictive disorder which prevent giving informed consent
or undergoing any of the treatment options or unwilling or unable to
comply with the protocol
- Uncontrolled or significant cardiac disease or symptomatic cardiac
arrhythmias, e.g., sustained ventricular tachycardia and clinically
significant second or third degree AV block without a pacemaker
- Illicit drug use and/or alcohol use, defined as an average alcohol
consumption greater than one standard drink a day for women or two
standard drinks a day for men within the 12 months prior to enrolment.
- Cirrhosis, including Child-Pugh class A, B, and C, diagnosed by liver
biopsy, elastography, radiologic exams, or clinical criteria.
- Active hepatitis B or C (hepatitis B carrier will be allowed)
- History of HIV seropositivity (ELISA or Western blot)
- Malignancy of any organ system, treated or untreated, within the past
5 years whether or not there is evidence of local recurrence or
metastases, except localized basal cell carcinoma of the skin.
Hepatocellular carcinoma at any time is excluded.
2. Prior iron chelation therapy, prohibited concomitant medications with deferasirox syndrome, or small bowel resection.
3. Abnormal laboratory values
4. Participation in an investigational study:
- Observational registry study is allowable
- Within 30 days prior to enrollment or within 5-half-lives of an investigational product, whichever is longer
5. Pregnancy and contraception:
- Pregnant or nursing (lactating) women
- Women of child-bearing potential unless using appropriate methods of contraception. Deferasirox may reduce the efficacy of hormonal contraception, thus it is recommended to use alternative methods of
contraception.
- Post-menopausal and not of childbearing potential if she has had 12 months of natural (spontaneous) amenorrhea with an expected clinical profile

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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