Clinical trial NCT03802916: Safety and Acceptability of Deferiprone Delayed Release Tablets in Patients With Systemic Iron Overload
Official title: Safety and Acceptability of Deferiprone Delayed Release Tablets in Patients With Systemic Iron Overload
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Iron Overload Due to Repeated Red Blood Cell Transfusions
Keywords: iron overload
chelation
deferiprone
Ferriprox
Lead sponsor: Chiesi Canada Corp
Sponsor class: INDUSTRY
First posted: 2019-01-14
Enrollment: 30
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Parallel Assignment
Primary purpose: TREATMENT

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

Detailed 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.

Eligibility criteria: 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.

Primary outcomes:
measure: The Percentage of Patients in Each Treatment Group Who Experience Post-dose Increases in Liver Enzyme Levels That Are Considered a Safety Concern.
timeFrame: Day 28
Secondary outcomes:
measure: The Percentage of Patients in Each Treatment Group Who Report Post-dose Occurrences of Gastrointestinal (GI) Distress.
timeFrame: Day 28
measure: The Percentage of Patients in Each Group Who Indicate That They Prefer the Deferiprone DR Formulation Over the Immediate-release Formulation.
timeFrame: Day 28
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: New York
country: United States
facility: New York Presbyterian Hospital/Weill Cornell Medical Center
state: New York
city: Vancouver
country: Canada
facility: St.Paul's Hospital
state: British Columbia
city: Goudí
country: Greece
facility: National and Kapodistrian University of Athens, Aghia Sophia Children's Hospital
state: Athens
city: Orbassano (TO)
country: Italy
facility: San Luigi Gonzaga University Hospital Reparto Microcitemie-Pediatria
state: Regione Gonzole
Study officials:
References: