Sickle Cell DiseasePhase 1NCT01835496
Absorption, Metabolism, and Excretion of a Single Dose of Ferriprox® in Patients With Sickle Cell Disease
A Phase 1 study testing single 1500 mg dose of Ferriprox in people with Sickle Cell Disease, run by ApoPharma at 1 site in Canada.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 45 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- single 1500 mg dose of Ferriprox
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteCanada
- Study dates
- May 2013to Apr 2014
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 45 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: The Pharmacokinetic Profile of a Single Dose of Ferriprox® in Patients With Sickle Cell Disease
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.
Read the 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.
- Conditions
- Sickle Cell Disease
- Being tested
- single 1500 mg dose of Ferriprox
- Keywords
- sickle cell disease, iron overload, pharmacokinetics, Ferriprox, deferiprone
- Medical subject headings
- Anemia, Sickle Cell, Iron Overload
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
single 1500 mg dose of FerriproxDrug
Also called deferiprone
A single dose of 1500mg of Ferriprox (three 500mg tablets) administered under fasting conditions
| Study group | Role | What they receive |
|---|---|---|
| Ferriprox | No intervention | single 1500 mg dose of Ferriprox Receives: Drug: single 1500 mg dose of Ferriprox |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide
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.
Measured: 10-hour interval
Tmax for Deferiprone and Deferiprone 3-O-glucuronide
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).
Measured: 10-hour interval
AUC0-∞ for Serum Deferiprone and Deferiprone 3-O-glucuronide
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.
Measured: 10-hour interval
T1/2 for Serum Deferiprone and Deferiprone 3-O-glucuronide
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.
Measured: 10-hour interval
Secondary outcomes (2)
Frequency of Adverse Events
Measured: From Day 1 (Dosing) to Day 7 plus/minus 3 days (Follow-up)
Frequency of Serious Adverse Events
Measured: From Day 1 (Dosing) to Day 30 post-dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Apr 2013 (estimated)
- Study startMay 2013
- Primary completionApr 2014
- Study completionApr 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- ApoPharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Denis Soulieres, MD, Principal investigator, CHUM - Hôpital Notre-Dame
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01835496
- Sponsor's protocol ID
- LA41-0412
- Status last verified
- Apr 2013
- First submitted
- 11 Apr 2013
- Last updated
- 24 Jul 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Soulieres D, Mercier-Ross J, Fradette C, Rozova A, Tsang YC, Tricta F. The pharmacokinetic and safety profile of single-dose deferiprone in subjects with sickle cell disease. Ann Hematol. 2022 Mar;101(3):533-539. doi: 10.1007/s00277-021-04728-0. Epub 2022 Jan 4.
PubMed 34981144
Study at a glance
From the registry record, last updated by the sponsor on 24 Jul 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Pharmacokinetic Profile of a Single Dose of Ferriprox® in Patients With Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT01835496
- Acronym / sponsor ID
- LA41-0412
- Conditions
- Sickle Cell Disease
- Keywords
- sickle cell disease, iron overload, pharmacokinetics, Ferriprox, deferiprone
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- ApoPharmaIndustry (pharma, biotech, devices)
- Study start
- May 2013
- Primary completion
- Apr 2014 (actual)
- Study completion
- Apr 2014 (actual)
- First posted
- 19 Apr 2013 (estimated)
- Results
- Posted 24 Jul 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ferriprox | No intervention | Drug: single 1500 mg dose of Ferriproxsingle 1500 mg dose of Ferriprox |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (13 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| single 1500 mg dose of FerriproxAlso: deferiproneA single dose of 1500mg of Ferriprox (three 500mg tablets) administered under fasting conditions | Drug | Ferriprox |
Primary outcome
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.
2 secondary outcomes
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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jul 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 34981144Soulieres D, Mercier-Ross J, Fradette C, Rozova A, Tsang YC, Tricta F. The pharmacokinetic and safety profile of single-dose deferiprone in subjects with sickle cell disease. Ann Hematol. 2022 Mar;101(3):533-539. doi: 10.1007/s00277-021-04728-0. Epub 2022 Jan 4.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Denis Soulieres, MD principal investigator
CHUM - Hôpital Notre-Dame
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing single 1500 mg dose of Ferriprox in people with Sickle Cell Disease, run by ApoPharma at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Jul 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 24 Jul 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide (time frame: 10-hour interval). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01835496, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jul 2015.