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

Phase 1

• Conditions: Hereditary Hemochromatosis; MedDRA version: 20.0Level: LLTClassification code 10057874Term: Hereditary hemochromatosisSystem Organ Class: 100000012236; Therapeutic area: Diseases [C] - Eye Diseases [C11]

• Registration Number: EUCTR2016-002529-12-ES
• Lead Sponsor: ovartis Farmacéutica, S.A.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2017-08-02
• Last Update Posted: 30 October 2017

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• 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 = 700 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
2. Prior iron chelation therapy, prohibited concomitant medications with deferasirox
syndrome, or small bowel resection.
- 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, or any history of hepatocellular carcinoma
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
- Treatment with a systemic investigational drug within 4 weeks or topical investigational drug within 7 days of starting the study
5. Pregnancy and contraception:
- Pregnant or nursing (lactating) women
- Women of child-bearing potential unless using basic 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