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

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ClinicalTrials.gov Identifier: NCT01835496
Recruitment Status : Completed
First Posted : April 19, 2013
Results First Posted : July 24, 2015
Last Update Posted : July 24, 2015
Sponsor:
Information provided by (Responsible Party):
ApoPharma

Tracking Information
First Submitted Date  ICMJE April 11, 2013
First Posted Date  ICMJE April 19, 2013
Results First Submitted Date  ICMJE June 26, 2015
Results First Posted Date  ICMJE July 24, 2015
Last Update Posted Date July 24, 2015
Study Start Date  ICMJE May 2013
Actual Primary Completion Date April 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 22, 2015)
  • Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide [ Time Frame: 10-hour interval ]
    Cmax (maximum measured serum concentration) was assessed over a 10-hour interval for deferiprone and its 3-O-glucuronide metabolite. Blood samples were obtained pre-dose and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 8, and 10 hours post-dose.
  • Tmax for Deferiprone and Deferiprone 3-O-glucuronide [ Time Frame: 10-hour interval ]
    Tmax (time to the maximum measured serum concentration) was assessed over a 10-hour interval for deferiprone and its 3-O-glucuronide metabolite. Blood samples were obtained pre-dose and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 8, and 10 hours post-dose. The results of the Tmax parameter are reported as the median and range (other parameters are reported as mean and standard deviation).
  • AUC0-∞ for Serum Deferiprone and Deferiprone 3-O-glucuronide [ Time Frame: 10-hour interval ]
    AUC0-∞ (area under the curve, zero to infinity) was assessed over a 10-hour interval for deferiprone and its 3-O-glucuronide metabolite. Blood samples were obtained pre-dose and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 8, and 10 hours post-dose.
  • T1/2 for Serum Deferiprone and Deferiprone 3-O-glucuronide [ Time Frame: 10-hour interval ]
    T1/2 (apparent terminal elimination half-life) was assessed over a 10-hour interval for deferiprone and its 3-O-glucuronide metabolite. Blood samples were obtained pre-dose and at 0.25, 0.50, 0.75, 1, 1.33, 1.66, 2, 2.5, 3, 4, 6, 8, and 10 hours post-dose.
Original Primary Outcome Measures  ICMJE
 (submitted: April 16, 2013)
  • AUC of deferiprone and its 3-O-glucuronide metabolite [ Time Frame: Blood samples taken pre-dose and at specified intervals up to 10 hours post-dose; urine collected from -2 to 0 hours pre-dose and at specified intervals up to 10 hours post-dose ]
    Area under the curve, calculated over 10 hours
  • Cmax of deferiprone and its 3-O-glucuronide metabolite [ Time Frame: Blood samples taken pre-dose and at specified intervals up to 10 hours post-dose; urine collected from -2 to 0 hours pre-dose and at specified intervals up to 10 hours post-dose ]
    Maximum measured serum concentration, calculated over 10 hours
  • T 1/2 of deferiprone and its 3-O-glucuronide metabolite [ Time Frame: Blood samples taken pre-dose and at specified intervals up to 10 hours post-dose; urine collected from -2 to 0 hours pre-dose and at specified intervals up to 10 hours post-dose ]
    Apparent terminal elimination half-life, calculated over 10 hours
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: July 22, 2015)
  • Frequency of Adverse Events [ Time Frame: From Day 1 (Dosing) to Day 7 plus/minus 3 days (Follow-up) ]
  • Frequency of Serious Adverse Events [ Time Frame: From Day 1 (Dosing) to Day 30 post-dose ]
Original Secondary Outcome Measures  ICMJE
 (submitted: April 16, 2013)
  • Frequency of Adverse Events [ Time Frame: From Day 1 (Dosing) to Day 7 (Follow-up) ]
  • Frequency of Serious Adverse Events [ Time Frame: From Day 1 (Dosing) to Day 7 (Follow-up) ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Absorption, Metabolism, and Excretion of a Single Dose of Ferriprox® in Patients With Sickle Cell Disease
Official Title  ICMJE The Pharmacokinetic Profile of a Single Dose of Ferriprox® in Patients With Sickle Cell Disease
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 cell disease.
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 and to undergo baseline assessments, and will receive a single dose of 1500 mg Ferriprox under fasting conditions. Blood and urine samples for pharmacokinetic assessment will be collected over a 10-hour period. Standard safety assessments will be performed throughout the study, and patients will return for a safety follow-up.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Condition  ICMJE Sickle Cell Disease
Intervention  ICMJE Drug: single 1500 mg dose of Ferriprox
A single dose of 1500mg of Ferriprox (three 500mg tablets) administered under fasting conditions
Other Name: deferiprone
Study Arms  ICMJE No Intervention: Ferriprox
single 1500 mg dose of Ferriprox
Intervention: Drug: single 1500 mg dose of Ferriprox
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: April 16, 2013)
8
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE April 2014
Actual Primary Completion Date April 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Male or female, 18-45 years of age (inclusive)
  2. Diagnosis of sickle cell disease, confirmed by Hb electrophoresis
  3. Body weight ≥ 50 kg
  4. Body mass index (BMI) ≥ 18 and ≤ 32 kg/m^2
  5. Absolute neutrophil count (ANC) of >1.5 x 10^9/L
  6. 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
  7. 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 Criteria:

  1. History or presence of hypersensitivity or idiosyncratic reaction to Ferriprox
  2. Use of Ferriprox within the past 3 months
  3. History of malignancy
  4. 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)
  5. 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
  6. Hemodialysis during the week prior to dosing or planned for the day of dosing
  7. Known difficulty in providing blood samples
  8. 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.)
  9. 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)
  10. Use of tobacco/nicotine-containing products for at least 3 months prior to study drug administration
  11. 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
  12. 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
  13. Pregnant or nursing female
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 45 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01835496
Other Study ID Numbers  ICMJE LA41-0412
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party ApoPharma
Study Sponsor  ICMJE ApoPharma
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Denis Soulieres, MD CHUM - Hôpital Notre-Dame
PRS Account ApoPharma
Verification Date April 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP