Status:

COMPLETED

Absorption, Metabolism, and Excretion of a Single Dose of Ferriprox® in Patients With Sickle Cell Disease

Lead Sponsor:

ApoPharma

Conditions:

Sickle Cell Disease

Eligibility:

All Genders

18-45 years

Phase:

PHASE1

Brief Summary

The objective of this study is to evaluate the pharmacokinetics of deferiprone and its 3-O-glucuronide metabolite following administration of a single 1500 mg dose of Ferriprox in patients with sickle...

Detailed Description

This is a single-arm, single-dose study of Ferriprox in patients with sickle cell disease. Patients found to be eligible will visit the clinic the day before receiving the drug, in order to reconfirm ...

Eligibility Criteria

Inclusion

  • Male or female, 18-45 years of age (inclusive)
  • Diagnosis of sickle cell disease, confirmed by Hb electrophoresis
  • Body weight ≥ 50 kg
  • Body mass index (BMI) ≥ 18 and ≤ 32 kg/m\^2
  • Absolute neutrophil count (ANC) of \>1.5 x 10\^9/L
  • Women of childbearing potential must agree to either be sexually inactive or use an acceptable method of birth control for 14 days prior to dosing and for 30 days afterwards
  • A fertile heterosexual male must agree that he or his partner will use an effective method of contraception for 14 days prior to dosing and for 30 days afterwards

Exclusion

  • History or presence of hypersensitivity or idiosyncratic reaction to Ferriprox
  • Use of Ferriprox within the past 3 months
  • History of malignancy
  • Evidence of abnormal liver function (serum Alanine Transaminase (ALT)level \> 5 times upper limit of normal or creatinine levels \>2 times upper limit of normal)
  • A serious, unstable illness, as judged by the Investigator, within the past 3 months before screening visit including but not limited to hepatic, renal, gastro-enterologic, respiratory, cardiovascular, endocrinologic, neurologic or immunologic disease
  • Hemodialysis during the week prior to dosing or planned for the day of dosing
  • Known difficulty in providing blood samples
  • Disorders or surgery of the gastrointestinal tract that may interfere with drug absorption or may otherwise influence the Pharmacokinetic (PK) results (e.g., resection of the small or large intestine, febrile conditions, chronic diarrhea, chronic vomiting, endocrine disease, severe infections, acute inflammations, etc.)
  • Clinically significant abnormalities on 12-lead ECG (e.g., QT interval corrected using Fridericia's formula (QTcF) ≥ 430 ms in males or ≥ 450 ms in females)
  • Use of tobacco/nicotine-containing products for at least 3 months prior to study drug administration
  • Use of any drugs within the past 14 days that are metabolized by the Uridine diphosphate glucosyltransferase enzyme (UGT1A6) and hence could affect the PK of Ferriprox
  • Treatment with an investigational drug within the past 30 days or 5 half-lives of that drug (whichever is longer) prior to study drug administration
  • Pregnant or nursing female

Key Trial Info

Start Date :

May 1 2013

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

April 1 2014

Estimated Enrollment :

8 Patients enrolled

Trial Details

Trial ID

NCT01835496

Start Date

May 1 2013

End Date

April 1 2014

Last Update

July 24 2015

Active Locations (1)

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Page 1 of 1 (1 locations)

1

CHUM-Hôpital Notre-Dame

Montreal, Quebec, Canada, H2L 4M1