Phase 1 N=8

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

Sickle Cell Disease

Bottom Line

View on ClinicalTrials.gov: NCT01835496 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Jul 2015

Primary outcome: Primary: Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide — 17.56; 32.95 μg/mL

Study Design & Population

Study type: Interventional
Phase: Phase 1
Interventions: single 1500 mg dose of Ferriprox (Drug)
Age: Adult · 18+ yrs
Sex: All
Sponsor: ApoPharma
Primary completion: Apr 2014

Outcome Measures

Outcome	Result	p-value
PRIMARY Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide	17.56; 32.95	—
PRIMARY Tmax for Deferiprone and Deferiprone 3-O-glucuronide	1.000; 2.750	—
PRIMARY AUC0-∞ for Serum Deferiprone and Deferiprone 3-O-glucuronide	43.37; 142.7	—
PRIMARY T1/2 for Serum Deferiprone and Deferiprone 3-O-glucuronide	1.458; 1.575	—
SECONDARY Frequency of Adverse Events	2	—
SECONDARY Frequency of Serious Adverse Events	—	—

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.

Eligibility Criteria

Inclusion Criteria

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 Criteria

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01835496). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.