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Trial record 90 of 696 for:    sickle cell disease

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

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.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: single 1500 mg dose of Ferriprox Phase 1

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 8 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Official Title: The Pharmacokinetic Profile of a Single Dose of Ferriprox® in Patients With Sickle Cell Disease
Study Start Date : May 2013
Actual Primary Completion Date : April 2014
Actual Study Completion Date : April 2014

Resource links provided by the National Library of Medicine

Drug Information available for: Deferiprone

Arm Intervention/treatment
No Intervention: Ferriprox
single 1500 mg dose of Ferriprox
Drug: single 1500 mg dose of Ferriprox
A single dose of 1500mg of Ferriprox (three 500mg tablets) administered under fasting conditions
Other Name: deferiprone




Primary Outcome Measures :
  1. 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.

  2. 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).


  3. 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.

  4. 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.


Secondary Outcome Measures :
  1. Frequency of Adverse Events [ Time Frame: From Day 1 (Dosing) to Day 7 plus/minus 3 days (Follow-up) ]
  2. Frequency of Serious Adverse Events [ Time Frame: From Day 1 (Dosing) to Day 30 post-dose ]


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Ages Eligible for Study:   18 Years to 45 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

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): NCT01835496


Locations
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Canada, Quebec
CHUM-Hôpital Notre-Dame
Montreal, Quebec, Canada, H2L 4M1
Sponsors and Collaborators
ApoPharma
Investigators
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Principal Investigator: Denis Soulieres, MD CHUM - Hôpital Notre-Dame

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Responsible Party: ApoPharma
ClinicalTrials.gov Identifier: NCT01835496     History of Changes
Other Study ID Numbers: LA41-0412
First Posted: April 19, 2013    Key Record Dates
Results First Posted: July 24, 2015
Last Update Posted: July 24, 2015
Last Verified: April 2013
Keywords provided by ApoPharma:
sickle cell disease
iron overload
pharmacokinetics
Ferriprox
deferiprone
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Hematologic Diseases
Genetic Diseases, Inborn
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hemoglobinopathies
Deferiprone
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action