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

Sickle Cell Disease Clinical Trial

Official title:

Long-term Safety and Efficacy Study of Ferriprox® for the Treatment of Transfusional Iron Overload in Patients With Sickle Cell Disease or Other Anemias

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02443545
• Other study ID #: LA38-EXT
• Status: Terminated
• Phase: Phase 4
• Start date: May 21, 2015
• Est. completion date: August 21, 2019

Study information

• Verified date: December 2023
• Source: Chiesi Canada Corp
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 134
• Est. completion date: August 21, 2019
• Est. primary completion date: April 30, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Anemia
• Anemia, Sickle Cell
• Iron Overload
• Other Anemias
• Sickle Cell Disease

Intervention

Drug:
• Deferiprone

Locations

Country	Name	City	State
Canada	Hospital for Sick Kids	Toronto	Ontario
Egypt	Zagazig University	Alexandria
Egypt	Ain Shams University	Cairo
Egypt	Cairo University	Cairo
Egypt	Pediatric Hospital of Cairo University	Cairo
Saudi Arabia	Asser Central Hospital	Abha
United Kingdom	Barts and The London	London
United Kingdom	Evelina Children's Hospital	London
United States	University of Michigan Comprehensive Cancer Center	Ann Arbor	Michigan
United States	Medical University of South Carolina	Charleston	South Carolina
United States	Children's Hospital of Michigan	Detroit	Michigan
United States	UCSF Benioff Children's Hospital Oakland	Oakland	California
United States	The Children's Hospital of Philadephia	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
ApoPharma

Countries where clinical trial is conducted

United States,  Canada,  Egypt,  Saudi Arabia,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Patients With Adverse Events	Number of patients with at least one adverse event (AE) of any type; number of patients with at least one serious adverse event, and number of patients who withdrew from the study due to an AE	From the first day of the study until the last study visit (Week 104 or early termination)
Secondary	Change From Baseline in Liver Iron Concentration (LIC)	LIC was measured by MRI, in units of mg of iron per gram of liver (dry weight). The change from baseline in LIC was determined for three different periods of exposure to deferiprone: one year, two years, and three years.	One year, two years, and three years after the start of deferiprone therapy
Secondary	Change From Baseline in Cardiac MRI T2*	The change from baseline in cardiac MRI T2* was determined for three different periods of exposure to deferiprone: one year, two years, and three years	One year, two years, and three years after the start of deferiprone therapy
Secondary	Change From Baseline in Serum Ferritin	The change from baseline in serum ferritin (SF) was determined for three different periods of exposure to deferiprone: one year, two years, and three years.	One year, two years, and three years after the start of deferiprone therapy