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Completed

NCT Number: NCT03802916

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

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

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Key information

Conditions

Iron Overload Due to Repeated Red Blood Cell Transfusions Iron Metabolism Disorders Iron Overload Metabolic Diseases Nutritional and Metabolic Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

St.Paul's Hospital, Vancouver, British Columbia, Canada

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Deferiprone DR tablets 1000 mg (Low dosage)

Drug

Deferiprone DR tablets 1000 mg

Deferiprone DR tablets 1000 mg (High dosage)

Drug

Deferiprone DR tablets 1000 mg

Primary outcomes

  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

    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 outcomes

  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, such as nausea, vomiting, diarrhea, abdominal pain, and dyspepsia.

  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.

Sponsors and collaborators

Lead sponsor

ApoPharma

Industry

Registry information

Acronym: TWICE

Important dates

Study start
2019
Primary completion
2019
Study completion
2019
First posted
Jan 14, 2019
Registry last updated
Jul 16, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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