Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias
Condition: Iron Overload; Sickle Cell Disease; Other Anemias
MeSH terms: Anemia; Anemia, Sickle Cell; Iron Overload
Intervention: Deferiprone
Intervention MeSH terms: Deferiprone
Phase: Phase 4
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 134.0
Sex: All
Minimum age: 3 Years
Healthy volunteers: No
Keywords: Iron overload; Sickle cell disease; Deferiprone; Ferriprox; Iron chelation

Summary:

      This is a long-term follow-up to an earlier study, LA38-0411. Its purpose is to gather more
      information about the safety and efficacy of deferiprone in patients with sickle cell disease
      or other anemias who suffer from iron overload caused by regular blood transfusions.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Completed study LA38-0411

          2. Females of childbearing potential must have a negative pregnancy test result at Visit
             1. In addition, if applicable, they must:

               -  Use an effective method of contraception according to local requirements, during
                  the study and within 30 days following their last dose of study medication, OR

               -  Have had a tubal ligation (supporting evidence required), OR

               -  Have had a hysterectomy (supporting evidence required), OR

               -  Participate in a non-heterosexual lifestyle, OR

               -  Have a male sexual partner who has been sterilized (supporting evidence required)

          3. Fertile heterosexual males and/or their partners must agree to use an effective method
             of contraception during the study and for 30 days following the last dose of study
             medication

          4. All patients and/or their authorized legal representatives must provide signed and
             dated written informed consent prior to the first study intervention, and assent will
             be obtained from patients who are considered to be minors. Patients must be able to
             adhere to study restrictions, appointments, and evaluation schedules.

        Exclusion Criteria:

          1. Plan to participate in another clinical trial at any time from the day of enrollment
             until 30 days post-treatment in the current study

          2. For only those patients who were treated with deferoxamine in study LA38-0411 (Group
             2): Presence of any medical condition (including clinically significant laboratory
             abnormalities, such as ALT (alanine aminotransferase) ≥ 5 x ULN or creatinine ≥ 2 x
             ULN), psychological condition, or psychiatric condition which in the opinion of the
             investigator would cause participation in the study to be unwise.

          3. Pregnant, breastfeeding, or planning to become pregnant during the study period.

          4. Treatment failure after 1 year on deferiprone which in the investigator's judgment
             indicates the need for the patient to be started on a different iron chelator