Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias

ApoPharma

Status and phase: Terminated; Phase 4

Conditions: Iron Overload; Sickle Cell Disease; Other Anemias

Treatments: Drug: Deferiprone

Study type: Interventional

Funder types: Industry

Identifiers

NCT02443545

LA38-EXT

Details and patient eligibility

About

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.

Full description

Deferiprone (brand name Ferriprox®) is an iron chelator that is approved in the United States and over 60 other countries for the treatment of iron overload in patients with thalassemia, when other treatments are inadequate. This study has been designed to evaluate the long-term efficacy, safety, and tolerability of deferiprone to treat iron overload in patients who have sickle cell disease or other anemias.

Only patients who have completed an earlier study, LA38-0411, are eligible to enroll in this one.

Enrollment: 134 patients

Sex: All

Ages: 3+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

---

134 participants in 2 patient groups

Group 1: Deferiprone 3 years

Experimental group

Description:

Patients in this group are those who were randomized to the deferiprone arm in study LA38-0411, and hence will receive deferiprone for a total of 3 years (1 year in the initial study plus 2 years in the extension study).

Treatment:

Drug: Deferiprone

Group 2: Deferiprone 2 years

Experimental group

Description:

Patients in this group are those who were randomized to the deferoxamine arm in study LA38-0411, and hence will receive deferiprone for 2 years (both of them in the extension study).

Treatment:

Drug: Deferiprone