Safety and Acceptability of Deferiprone Delayed Release Tablets in Patients With Systemic Iron Overload

Iron Overload Due to Repeated Red Blood Cell Transfusions Clinical Trial

— TWICE  

Official title:

Safety and Acceptability of Deferiprone Delayed Release Tablets in Patients With Systemic Iron Overload

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03802916
• Other study ID #: LA61-0218
• Status: Completed
• Phase: Phase 2
• Start date: March 6, 2019
• Est. completion date: December 19, 2019

Study information

• Verified date: June 2021
• Source: Chiesi Canada Corp
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Safety, tolerability, and acceptability of twice-daily dosing with deferiprone delayed-release (DR) tablets in patients with systemic iron overload.

Description:

This study is looking at the safety, tolerability, and acceptability of twice-daily dosing with deferiprone delayed-release (DR) tablets in patients with systemic iron overload who are currently taking deferiprone immediate-release tablets (Ferriprox) three times a day. Ferriprox doses range from 75 milligrams per kilogram of body weight (mg/kg) per day to 100 mg/kg per day. Half the patients in the study will be on a dosage that is closer to the low end of the range, and half will be on a dosage that is closer to the high end. Both groups will be switched for one month to deferiprone DR tablets at approximately the same total daily dosage that they have been taking for Ferriprox.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 30
• Est. completion date: December 19, 2019
• Est. primary completion date: December 4, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

1. Male or female aged = 18 years.

2. Diagnosis of thalassemia syndrome, sickle cell disease, or other disorder requiring a regular regimen of red blood cell transfusions.

3. On a stable regimen (=3 months) of Ferriprox tablets for the treatment of systemic iron overload.

4. Absolute neutrophil count =1.5 x 10^9/L at screening.

5. A record of at least 12 measured alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels.

Exclusion Criteria:

1. Receipt of any iron chelator other than Ferriprox (i.e., combination therapy) in the last 3 months, or planning to receive it at any time during the period of the study.

2. ALT and/or AST value > 5 times the upper limit of normal (ULN) at screening

3. Active case of hepatitis B or C at screening.

Related Conditions & MeSH terms

• Iron Overload
• Iron Overload Due to Repeated Red Blood Cell Transfusions

Intervention

Drug:
• Deferiprone DR tablets 1000 mg (Low dosage)
Deferiprone DR tablets 1000 mg
• Deferiprone DR tablets 1000 mg (High dosage)
Deferiprone DR tablets 1000 mg

Locations

Country	Name	City	State
Canada	St.Paul's Hospital	Vancouver	British Columbia
Greece	National and Kapodistrian University of Athens, Aghia Sophia Children's Hospital	Goudí	Athens
Italy	San Luigi Gonzaga University Hospital Reparto Microcitemie-Pediatria	Orbassano (TO)	Regione Gonzole
United States	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	New York Presbyterian Hospital/Weill Cornell Medical Center	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
ApoPharma

Countries where clinical trial is conducted

United States,  Canada,  Greece,  Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The Percentage of Patients in Each Treatment Group Who Experience Post-dose Increases in Liver Enzyme Levels That Are Considered a Safety Concern.	Levels of the liver enzymes alanine aminotransferase (ALT) and aspartate aminotransferase (AST) will be assessed throughout the study to determine if any patients have post-dose increases that are considered to be a safety concern. The criteria for being considered a safety concern are meeting one of the following: For a patient whose level was within the normal range at baseline, the criterion is reaching a value of 5 times the upper limit of normal (ULN); For a patient whose level was above the ULN at baseline, the criterion is reaching either 5 times the baseline value or 10 x ULN	Day 28
Secondary	The Percentage of Patients in Each Treatment Group Who Report Post-dose Occurrences of Gastrointestinal (GI) Distress.	Patients will be asked to report any events of GI distress during the study, such as nausea, vomiting, diarrhea, abdominal pain, and dyspepsia.	Day 28
Secondary	The Percentage of Patients in Each Group Who Indicate That They Prefer the Deferiprone DR Formulation Over the Immediate-release Formulation.	At the end of the study, patients will complete a questionnaire to indicate which formulation they prefer.	Day 28