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

ApoPharma

Status and phase: Completed; Phase 2

Conditions: Iron Overload Due to Repeated Red Blood Cell Transfusions

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

Study type: Interventional

Funder types: Industry

Identifiers

NCT03802916

LA61-0218

Details and patient eligibility

About

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

Full 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.

Enrollment: 30 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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30 participants in 2 patient groups

Low dosage

Experimental group

Description:

Patients in this group will receive a total daily dosage of deferiprone DR tablets that is closer to 75 mg/kg/day. The total dosage will be divided into two equal parts, taken about 12 hours apart.

Treatment:

Drug: Deferiprone DR tablets 1000 mg (Low dosage)

High dosage

Experimental group

Description:

Patients in this group will receive a total daily dosage of deferiprone DR tablets that is closer to 100 mg/kg/day. The total dosage will be divided into two equal parts, taken about 12 hours apart.

Treatment:

Drug: Deferiprone DR tablets 1000 mg (High dosage)