Back to resultsSafety and Acceptability of Deferiprone Delayed Release Tablets in Patients With Systemic Iron Overload (TWICE)
Primary Purpose
Iron Overload Due to Repeated Red Blood Cell Transfusions
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Deferiprone DR tablets 1000 mg (Low dosage)
Deferiprone DR tablets 1000 mg (High dosage)
Sponsored by
About this trial
This is an interventional treatment trial for Iron Overload Due to Repeated Red Blood Cell Transfusions focused on measuring iron overload, chelation, deferiprone, Ferriprox
Eligibility Criteria
Inclusion Criteria:
- Male or female aged ≥ 18 years.
- Diagnosis of thalassemia syndrome, sickle cell disease, or other disorder requiring a regular regimen of red blood cell transfusions.
- On a stable regimen (≥3 months) of Ferriprox tablets for the treatment of systemic iron overload.
- Absolute neutrophil count ≥1.5 x 10^9/L at screening.
- A record of at least 12 measured alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels.
Exclusion Criteria:
- 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.
- ALT and/or AST value > 5 times the upper limit of normal (ULN) at screening
- Active case of hepatitis B or C at screening.
Sites / Locations
- Ann & Robert H. Lurie Children's Hospital of Chicago
- New York Presbyterian Hospital/Weill Cornell Medical Center
- St.Paul's Hospital
- National and Kapodistrian University of Athens, Aghia Sophia Children's Hospital
- San Luigi Gonzaga University Hospital Reparto Microcitemie-Pediatria
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
Low dosage
High dosage
Arm 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.
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.
Outcomes
Primary Outcome Measures
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
Secondary Outcome Measures
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.
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03802916
Brief Title
Safety and Acceptability of Deferiprone Delayed Release Tablets in Patients With Systemic Iron Overload
Acronym
TWICE
Official Title
Safety and Acceptability of Deferiprone Delayed Release Tablets in Patients With Systemic Iron Overload
Study Type
Interventional
2. Study Status
Record Verification Date
June 2021
Overall Recruitment Status
Completed
Study Start Date
March 6, 2019 (Actual)
Primary Completion Date
December 4, 2019 (Actual)
Study Completion Date
December 19, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
ApoPharma
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Iron Overload Due to Repeated Red Blood Cell Transfusions
Keywords
iron overload, chelation, deferiprone, Ferriprox
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Model Description
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
30 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Low dosage
Arm Type
Experimental
Arm 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.
Arm Title
High dosage
Arm Type
Experimental
Arm 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.
Intervention Type
Drug
Intervention Name(s)
Deferiprone DR tablets 1000 mg (Low dosage)
Intervention Description
Deferiprone DR tablets 1000 mg
Intervention Type
Drug
Intervention Name(s)
Deferiprone DR tablets 1000 mg (High dosage)
Intervention Description
Deferiprone DR tablets 1000 mg
Primary Outcome Measure Information:
Title
The Percentage of Patients in Each Treatment Group Who Experience Post-dose Increases in Liver Enzyme Levels That Are Considered a Safety Concern.
Description
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
Time Frame
Day 28
Secondary Outcome Measure Information:
Title
The Percentage of Patients in Each Treatment Group Who Report Post-dose Occurrences of Gastrointestinal (GI) Distress.
Description
Patients will be asked to report any events of GI distress during the study, such as nausea, vomiting, diarrhea, abdominal pain, and dyspepsia.
Time Frame
Day 28
Title
The Percentage of Patients in Each Group Who Indicate That They Prefer the Deferiprone DR Formulation Over the Immediate-release Formulation.
Description
At the end of the study, patients will complete a questionnaire to indicate which formulation they prefer.
Time Frame
Day 28
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Male or female aged ≥ 18 years.
Diagnosis of thalassemia syndrome, sickle cell disease, or other disorder requiring a regular regimen of red blood cell transfusions.
On a stable regimen (≥3 months) of Ferriprox tablets for the treatment of systemic iron overload.
Absolute neutrophil count ≥1.5 x 10^9/L at screening.
A record of at least 12 measured alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels.
Exclusion Criteria:
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.
ALT and/or AST value > 5 times the upper limit of normal (ULN) at screening
Active case of hepatitis B or C at screening.
Facility Information:
Facility Name
Ann & Robert H. Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
New York Presbyterian Hospital/Weill Cornell Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Facility Name
St.Paul's Hospital
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V6Z 1Y6
Country
Canada
Facility Name
National and Kapodistrian University of Athens, Aghia Sophia Children's Hospital
City
Goudí
State/Province
Athens
ZIP/Postal Code
11527
Country
Greece
Facility Name
San Luigi Gonzaga University Hospital Reparto Microcitemie-Pediatria
City
Orbassano (TO)
State/Province
Regione Gonzole
ZIP/Postal Code
10043
Country
Italy
12. IPD Sharing Statement
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Safety and Acceptability of Deferiprone Delayed Release Tablets in Patients With Systemic Iron Overload
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