A Two-year Study to Evaluate the Efficacy and Safety of Deferasirox Film-coated Tablet Versus Phlebotomy in Patients With Hereditary Hemochromatosis.

A Phase II, Multicenter, Open-label, Randomized Two-year Study to Evaluate the Efficacy and Safety of Deferasirox Film-coated Tablet Versus Phlebotomy in Patients With Hereditary Hemochromatosis.

ClinicalTrials.gov Identifier: NCT03203850

Novartis Reference Number: CICL670F2203

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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. 

Study Description

The purpose of this study is to evaluate the efficacy and safety of deferasirox film coated tablet (FCT) versus phlebotomy for the management of iron overload in adults with HH at risk of iron-related morbidity. This evaluation will provide information on the two treatment options in terms of the rate of response of proportion of patients reaching the study target SF ≤ 100 μg/L and their associated safety profiles.

In addition to exploring the safety and efficacy of deferasirox FCT in hereditary hemochromatosis (HH), this study is being conducted to fulfill an FDA post-marketing requirement [PMC 750-10 (Exjade) /PMR 2888-8 (Jadenu)] to provide additional randomized data to confirm the ocular safety profile of deferasirox through detailed ocular assessments in patients treated with deferasirox FCT for 2 years.

Condition 
Hereditary Hemochromatosis
Phase 
Phase 2
Overall status 
Recruiting
Enrollment count 
150 participants
Start date 
Jan 11, 2018
Completion date 
Apr 25, 2023
Gender 
All
Age(s)
18 Years and older (Adult, Older Adult)

Interventions

Drug
Deferasirox FCT
Taken orally once per day (QD)
Procedure
Phlebotomy
according to investigator's decision

Eligibility Criteria

Inclusion Criteria:

Written informed consent must be obtained prior to any screening procedures.

Patients eligible for inclusion must meet all following criteria prior to receiving study treatment:

1. Male or female ≥ 18-years-old 2. Documented genotype testing confirming homozygous for the C282Y mutation (C282Y/C282Y) 3. Transferrin saturation ≥ 45% (at either screening visit) 4. Serum ferritin (SF) ≥ 500 μg/L (at either screening visit)

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Exclusion Criteria:

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 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. A standard drink is generally considered to be 12 ounces of beer, 5 ounces of wine, or 1.5 ounces of 80-proof distilled spirits
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)
Organ transplant recipient
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

Concomitant therapy that precludes enrollment:

Prior iron chelation therapy
Prohibited concomitant medications with deferasirox

Abnormal Laboratory Values:

Significant anemia that contraindicates phlebotomy (males with hemoglobin < 130g/L, females with hemoglobin < 120g/L) in both screening visit samples
Platelets ≤ 50 x 109/L in both screening visit samples
Urine protein/urine creatinine ratio > 1.0 mg/mg in both non-first void urine screening visit samples
Creatinine clearance ≤ 40 ml/min, or use the locally approved contraindication limit in prescribing information if it is stricter, in both screening visit samples
Serum creatinine > 1.5 x ULN in both screening visit samples
ALT ≥ 5 x ULN in both screening visit samples
Total bilirubin > 1.5 x ULN in both screening visit samples

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

Pregnancy and contraception:

Pregnant or nursing (lactating) women
Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless using basic methods of contraception, such as:
Total abstinence Periodic abstinence (calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are unacceptable methods.
Female sterilization (bilateral oophorectomy with or without hysterectomy), total hysterectomy, or tubal ligation at least six weeks before taking study treatment. If oophorectomy alone, hormone levels must confirm menopause.
Male sterilization (at least 6 months prior to screening). The vasectomized male must be the sole partner.
Barrier methods of contraception: condom or occlusive cap For UK: spermicidal foam/gel/film/cream/vaginal suppository
Placement of an intrauterine device or intrauterine system
Women considered as post-menopausal and not of childbearing potential are allowed to be enrolled in the trial if they have had 12 months of natural (spontaneous) amenorrhea with an expected clinical profile, e.g., age appropriate and history of vasomotor symptoms.

Other protocol-defined inclusion/exclusion may apply. -

Study Locations

United States
Tufts Medical Center
Recruiting
Boston, 02111
Massachusetts
United States
Lahey Hospital and Medical Center Center Primary
Recruiting
Burlington, 01805
Massachusetts
United States
Henry Ford Hospital
Withdrawn
Detroit, 48202
Michigan
United States
Saint Louis University School of Medicine
Withdrawn
Saint Louis, 63110-0250
Missouri
United States
Lucas Research
Withdrawn
Morehead City, 28557
North Carolina
United States
Belgium
Novartis Investigative Site
Recruiting
Leuven, 3000
-
Belgium
France
Novartis Investigative Site
Recruiting
Limoges cedex, 87042
-
France
Novartis Investigative Site
Recruiting
Rennes, 35043
-
France
Germany
Novartis Investigative Site
Withdrawn
Bad Doberan, 18209
-
Germany
Novartis Investigative Site
Withdrawn
Chemnitz, 09113
-
Germany
Novartis Investigative Site
Withdrawn
Hannover, 30625
-
Germany
Novartis Investigative Site
Withdrawn
Heidelberg, 69120
-
Germany
Novartis Investigative Site
Withdrawn
Magdeburg, 39120
-
Germany
Novartis Investigative Site
Withdrawn
Oberhausen, 46045
-
Germany
Romania
Novartis Investigative Site
Recruiting
Bucharest, 022328
District 2
Romania
Novartis Investigative Site
Recruiting
Bucuresti, 11025
-
Romania
Novartis Investigative Site
Recruiting
Craiova, 200136
-
Romania
Novartis Investigative Site
Recruiting
Sibiu, 550245
-
Romania
Russian Federation
Novartis Investigative Site
Recruiting
Moscow, 125167
-
Russian Federation
Slovakia
Novartis Investigative Site
Recruiting
Bratislava, 831 01
-
Slovakia
Novartis Investigative Site
Recruiting
Bratislava, 85107
-
Slovakia
Spain
Novartis Investigative Site
Recruiting
Manresa, 08241
Espana
Spain
Novartis Investigative Site
Recruiting
Baracaldo, 48903
Vizcaya
Spain
Novartis Investigative Site
Withdrawn
Barcelona, 08041
-
Spain
Novartis Investigative Site
Recruiting
Las Palmas de Gran Canaria, 35010
-
Spain
Novartis Investigative Site
Withdrawn
Madrid, 28009
-
Spain
Switzerland
Novartis Investigative Site
Recruiting
Lugano, 6900
-
Switzerland
Novartis Investigative Site
Recruiting
St Gallen, 9007
-
Switzerland

Contacts

Name: 
Novartis Pharmaceuticals
Phone: 
1-888-669-6682
Name: 
Novartis Pharmaceuticals
Phone: 
+41613241111

Have a question?

Call 1-999-669-6682 or email [email protected]