Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03802916
Recruitment Status : Completed
First Posted : January 14, 2019
Last Update Posted : February 24, 2020
Sponsor:
Information provided by (Responsible Party):
ApoPharma

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

Condition or disease Intervention/treatment Phase
Iron Overload Due to Repeated Red Blood Cell Transfusions Drug: Deferiprone DR tablets 1000 mg (Low dosage) Drug: Deferiprone DR tablets 1000 mg (High dosage) Phase 2

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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 30 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety and Acceptability of Deferiprone Delayed Release Tablets in Patients With Systemic Iron Overload
Actual Study Start Date : March 6, 2019
Actual Primary Completion Date : December 4, 2019
Actual Study Completion Date : December 19, 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Iron
Drug Information available for: Deferiprone

Arm Intervention/treatment
Experimental: Low dosage
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.
Drug: Deferiprone DR tablets 1000 mg (Low dosage)
Deferiprone DR tablets 1000 mg

Experimental: High dosage
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.
Drug: Deferiprone DR tablets 1000 mg (High dosage)
Deferiprone DR tablets 1000 mg




Primary Outcome Measures :
  1. The percentage of patients in each treatment group who experience post-dose increases in liver enzyme levels that are considered a safety concern. [ Time Frame: Day 28 ]
    Liver enzymes will be assessed throughout the study to determine if any patients have post-dose increases that are considered to be a safety concern.


Secondary Outcome Measures :
  1. The percentage of patients in each treatment group who report post-dose occurrences of gastrointestinal (GI) distress. [ Time Frame: Day 28 ]
    Patients will be asked to report any events of GI distress during the study.

  2. The percentage of patients in each group who indicate that they prefer the deferiprone DR formulation over the immediate-release formulation. [ Time Frame: Day 28 ]
    At the end of the study, patients will complete a questionnaire to indicate which formulation they prefer.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03802916


Locations
Layout table for location information
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
United States, New York
New York Presbyterian Hospital/Weill Cornell Medical Center
New York, New York, United States, 10065
Canada, British Columbia
St.Paul's Hospital
Vancouver, British Columbia, Canada, V6Z 1Y6
Greece
National and Kapodistrian University of Athens, Aghia Sophia Children's Hospital
Goudí, Athens, Greece, 11527
Italy
San Luigi Gonzaga University Hospital Reparto Microcitemie-Pediatria
Orbassano (TO), Regione Gonzole, Italy, 10043
Sponsors and Collaborators
ApoPharma
Layout table for additonal information
Responsible Party: ApoPharma
ClinicalTrials.gov Identifier: NCT03802916    
Other Study ID Numbers: LA61-0218
First Posted: January 14, 2019    Key Record Dates
Last Update Posted: February 24, 2020
Last Verified: February 2020

Layout table for additional information
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
Keywords provided by ApoPharma:
iron overload
chelation
deferiprone
Ferriprox
Additional relevant MeSH terms:
Layout table for MeSH terms
Iron Overload
Iron Metabolism Disorders
Metabolic Diseases
Deferiprone
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action