Chronic Iron OverloadPhase 2DEEP-1NCT01740713
Pharmacokinetic Study of Deferiprone in Paediatric Patients
A Phase 2 study testing Deferiprone, dose level 1, Deferiprone, dose level 2 and Deferiprone, dose level 3 in people with Chronic Iron Overload, run by Consorzio per Valutazioni Biologiche e Farmacologiche at 7 sites in 3 countries.
Trial summary
Deferiprone (DFP) is the most extensively studied oral iron chelator to date. It has been authorised in Europe in 1999 for the treatment of iron overload in patients with beta-thalassaemia major when DFO is contraindicated or inadequate. Read more about this study
- Who is it for?
- Ages 1 month to 6 years
- Conditions the study is for
- Chronic Iron Overload
- What is being tested?
- Deferiprone, dose level 1, Deferiprone, dose level 2 and Deferiprone, dose level 3
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 23 participants
- Where
- 7 sitesin 3 countries
- Study dates
- Dec 2012to Feb 2014
- Phase
- Phase 2
- 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 1 month to 6 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patients in a chronic transfusional program who have received at least 150 ml/kg/year of packed red blood cells (corresponding approximately to 12 transfusions) and on current treatment with DFO, DFX, DFP; aged from 1 month to less than 6 years; or
- Patients naïve to any chelation treatment who have received not less than 150 ml/kg of packed red blood cells (corresponding to approximately 12 transfusions) and have ferritin levels > 800 ng/mL, aged from 1 month to less than 6 years; or
- Patients who meet the transfusion criteria (150 ml/kg/year corresponding approximately to 12 transfusions) and have known intolerance or contraindication to DFO
- And if all of the following criteria apply:
- Patients affected by any hereditary haemoglobinopathies requiring chronic transfusion therapy including but not limited to thalassaemia and sickle cell disease
- Written informed consent obtained from their legal guardian on the patient's behalf in accordance with the national legislations. According to his/her capability, patient's informed assent will be collected
Who cannot join
- Patient with known intolerance or contraindication to the trial treatment
- Patient with Hb levels less than 8g/dl (entry may be delayed until values return to normal)
- Patient with platelet count <100.000/mm3 or absolute neutrophil count <1.500/mm3 (entry may be delayed until values return to normal)
- Patient with evidence of abnormal liver function (ALT level >5 times the upper normal limit during six months preceding enrolment; entry may be delayed until values return to normal)
- iron overload from causes other than transfusional haemosiderosis
- severe heart dysfunction secondary to iron overload defined as the occurrence of heart failure or severe arrhythmia or as indicated by cardiac T2* lower than 10 ms, if recent MRI data is available,
Show all 15Show fewer
- Patient with serum creatinine level above the upper normal limit at screening; entry may be delayed until values return to normal.
- Serological evidence of chronic hepatitis B (presence of HBe Ag, HBsAg, HBcAb-IgM, in the absence of HBsAb).
- History of significant medical or psychiatric disorder that may impair compliance with the requirements of the protocol.
- The patient has received another investigational drug within 30 days prior to this study.
- Patient with a pre-existing condition or any other surgical or medical condition which might significantly interfere with normal gastrointestinal and hepatic function that could alter the absorption, metabolism, and/or excretion of the study drug.
- Patient with a known history of HIV seropositivity.
- Fever and other signs/symptoms of infection in the 10 days before drug administration(treatment day)
- Concomitant use of other iron chelators or trivalent cation-dependent medicinal products such as aluminium-based antacids.
- Patient with a chronic condition that does not allow suspension of related treatment from starting of washout until drug is administered.
Trial locations and contacts
7 sites in 3 countries.
7 sites in 3 countries
Italy5 sites
Azienda Ospedaliero-Universitaria Consorziale
Bari, Bari, 70124
Azienda Ospedaliera Antonio Cardarelli
Naples, Napoli, 80131
Azienda Ospedaliera Di Padova
Padova, Padova, 35127
Azienda Ospedaliera Ospedali Riuniti Villa Sofia - Cervello
Palermo, Palermo, 90146
Clinica Pediatrica Universita' - Asl 1
Sassari, Sassari, 07100
Cyprus1 site
Department of Medical and Public Health Services of the Ministry of Health
Nicosia
About this study
Official title: Multi-centre, Oral Single Dose Experimental and Modelling Study to Evaluate the Pharmacokinetics of Deferiprone in Patients Aged From 1 Month to Less Than 6 Years of Age Affected by Transfusion-dependent Haemoglobinopathies.
Deferiprone (DFP) is the most extensively studied oral iron chelator to date. It has been authorised in Europe in 1999 for the treatment of iron overload in patients with beta-thalassaemia major when DFO is contraindicated or inadequate. Despite a wide experience of DFP there are limited experimental data available on DFP in children and no pharmacokinetic data in children under 6 years of age. On the basis of the existing data in adults and adolescent, in the DEEP-1 trial a pharmacokinetic bridging model was developed to support the dose selection in children aged less than 6 years affected by transfusion dependent haemoglobinopathies. The study consisted of two phases, namely an experimental phase, during which patients received a single dose and a modeling phase, during which PK data obtained after single dose in patients < 6 years of age were analysed in conjunction with historical PK data in adults and older children and adolescents. The model-based analysis of the data obtained after single dose enabled the assessment of the dosing regimen required for the purpose of accurate pharmacokinetic bridging. The ratio between the predicted systemic exposure parameters (AUC and Cmax) in the target population and reference group were used as basis for recommendation of the dose in the target population.
Read the detailed description
Deferiprone (DFP) was investigated as therapy for children from 1 month to less than 6 years of age. The study was a multicenter randomised, single blind, and single dose PK study. The patients were randomised according to a stratification scheme in which three different dose levels were used.
Objectives: The primary objective of this study was to assess the pharmacokinetics of DFP in paediatric patients aged from 1 month to less than 6 years.
The secondary objectives of this study were:
- To identify dose levels yielding deferiprone exposures comparable to adults and define the dose rationale in children aged from 1 month to less than 6 years.
- To evaluate safety and tolerability of deferiprone after single dose administration in children aged from 1 month to less than 6 years.
- To evaluate the effect of demographic covariates on DFP disposition and estimate the clearance distribution across the population.
Endpoints: The primary endpoints of the study were pharmacokinetic and included:
- primary PK parameters: CL/F, Vd/F, Ka
- secondary PK parameters: AUC, Cmax, Tmax, Css and Cmin.
Secondary endpoints were assessment of clinical safety and tolerability .
Methods: Twenty-three patients were enrolled and 18 of those ( 9 males and 9 females) completed the study. The patients were administered at three dose levels ( 6 patients / each dose):
Dose level 1: 8.3 mg/kg as a single dose (every 8 h) for a total daily dose of 25mg/kg Dose level 2: 16.7 mg/kg as a single dose (every 8 h) for a total daily dose of 50 mg/kg Dose level 3: 33.3 mg/kg as a single dose (every 8 h) for a total daily dose of 100 mg/kg
Blood samples for PK analysis were taken at 6 sampling time intervals, different depending on the dose group: predose; in the range 10 -20 min; in the range 40-55min; in the range 1.05-1.15 h;in the range 1.25-5.30 h;in the range 6-8h after the first dose administration. The concentration of deferiprone was determined by a validated HPLC method. A population PK model approach was applied and the time course of deferiprone concentrations was analysed with Nonlinear mixed effects modelling in NONMEM, version 7.2.0. Model building included the assessment of the influence of relevant demographic covariates (i.e:body weight , age, height) on the disposition of deferiprone.
Using the pharmacokinetic model developed for the paediatric population in this study in conjunction with a model previously developed for 55 adult subjects , simulations were performed to evaluate drug exposure in children below 6 years of age and across a standard thalassaemic adult population.
Descriptive statistics were used to summarise adverse events, vital signs and clinical lab data (haematology, biochemistry and virology)
Results: A one-compartment model with first-order absorption was found to best describe the disposition of deferiprone. The choice of three dose levels enable to assess linearity of pharmacokinetic across the dose range. The disposition parameters estimated through the pop-PK model included CL/F , V/F. In addition to the final model parameter estimates, the secondary pharmacokinetic parameters were derived based on the individual predicted concentration vs. time profiles and were summarised per dose level.
Body weight was found to be a good predictor of inter-individual differences in the population under investigation.
As expected from the known safety profile of the drug, no Serious Adverse Events were observed during the DEEP-1 PK Study.
Based on the simulation analysis performed, a similar exposure is achieved in adults and children in terms of AUC and Css when receiving the current dosing regimen both at 75 and 100 mg/kg/day, while a considerable increase in Cmax was observed in children when compared to the adult population. However, exposure is the parameter related to clinical response rather than Cmax. Indeed, simulations suggested that a dosing regimen of 25 mg/kg t.i.d. (75 mg/kg/day) is recommended for children aged from 1 month to < 6 years, with the possibility of titration up to 33.3 mg/kg t.i.d. (100 mg/kg/day), if necessary.
- Conditions
- Chronic Iron Overload
- Being tested
- Deferiprone, dose level 1, Deferiprone, dose level 2, Deferiprone, dose level 3
- Keywords
- chronic iron overload, hereditary haemoglobinopathy, beta thalassaemia major
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Deferiprone, dose level 1Drug
Also called DFP
a solution at 80 mg/mL will be administered orally
Deferiprone, dose level 2Drug
Also called DFP
a solution at 80 mg/mL will be administered orally
Deferiprone, dose level 3Drug
Also called DFP
a solution at 80 mg/mL will be administered orally
| Study group | Role | What they receive |
|---|---|---|
| Deferiprone, dose level 1 | Experimental | single dose level of 8.3 mg/kg every 8 hours for a corresponding total daily dose of 25 mg/kg/day. Receives: Drug: Deferiprone, dose level 1 |
| Deferiprone, dose level 2 | Experimental | single dose level of 16.7 mg/kg every 8 hours for a corresponding total daily dose of 50 mg/kg/day. Receives: Drug: Deferiprone, dose level 2 |
| Deferiprone, dose level 3 | Experimental | single dose level of 33.3 mg/kg every 8 hours for a corresponding total daily dose of 100 mg/kg/day. Receives: Drug: Deferiprone, dose level 3 |
What is being measured
The primary outcome is the main question the study is designed to answer.
CL/F
Plasma clearance after oral administration. The parameter was estimated through a population pharmacokinetic model, during which concentration data obtained after single oral dose ( at 3 dose levels) of DFP in patients aged from 1 month to less than 6 years of age.
Measured: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)
AUC (0-8h)
Area under concentration versus time curve from 0 to 8 h post dosing. Secondary pharmacokinetic parameter derived on the basis of the individual predicted concentration vs. time profiles, through the pop-PK model.
Measured: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)
V/F
volume of distribution after oral administration. The parameter was estimated through a population pharmacokinetic model, during which concentration data obtained after single oral dose ( at 3 dose levels) of DFP in patients aged from 1 month to less than 6 years of age
Measured: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)
Tmax
Time at which the maximum concentration (Cmax) is reached. Secondary pharmacokinetic parameters such as Cmax, Min, Tmax, Css and AUC (0-8h) were derived based on the individual predicted concentration vs. time profiles.
Measured: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)
Ka
Absorption rate constant. The parameter was estimated through a population pharmacokinetic model, during which concentration data obtained after single oral dose ( at 3 dose levels) of DFP in patients aged from 1 month to less than 6 years of age.
Measured: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)
Cmax
Maximum concentration reached in plasma. Secondary pharmacokinetic parameter derived on the basis of the individual predicted concentration vs. time profiles, through the pop-PK model.
Measured: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)
Css
Plasma concentration reached at steady state. Secondary pharmacokinetic parameter derived on the basis of the individual predicted concentration vs. time profiles, through the pop-PK model.
Measured: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)
Cmin
Minimum plasma concentration. Secondary pharmacokinetic parameter derived on the basis of the individual predicted concentration vs. time profiles, through the pop-PK model.
Measured: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)
Secondary outcomes (1)
Adverse Events
All the medical occurrences that started after the administration of the drug
Measured: from drug administration up to 8 days post treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Dec 2012 (estimated)
- Study startDec 2012
- Primary completionDec 2013
- Study completionFeb 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.
- Other trials for Chronic Iron OverloadStudies 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 2
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Investigator
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- Consorzio per Valutazioni Biologiche e Farmacologiche
- Sponsor type
- University, hospital or other
- Collaborators
- European Commission
- Responsible party
- Sponsor
- Investigators
- Oscar Della Pasqua, Study chair, Universiteit Leiden, The NetherlandsGiovanni Carlo Del Vecchio, Principal investigator, Azienda Ospedaliero-Universitaria Consorziale Policlinico di Bari, Italy
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01740713
- Sponsor's protocol ID
- DEEP-1
- Acronym
- DEEP-1
- Status last verified
- Nov 2016
- First submitted
- 28 Nov 2012
- Last updated
- 11 Jan 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 11 Jan 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multi-centre, Oral Single Dose Experimental and Modelling Study to Evaluate the Pharmacokinetics of Deferiprone in Patients Aged From 1 Month to Less Than 6 Years of Age Affected by Transfusion-dependent Haemoglobinopathies.
- ClinicalTrials.gov ID
- NCT01740713
- Acronym / sponsor ID
- DEEP-1 / DEEP-1
- Conditions
- Chronic Iron Overload
- Keywords
- chronic iron overload, hereditary haemoglobinopathy, beta thalassaemia major
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 23 participants (actual)
- Lead sponsor
- Consorzio per Valutazioni Biologiche e FarmacologicheUniversity, hospital or other
- Collaborators
- European Commission
- Study start
- Dec 2012
- Primary completion
- Dec 2013 (actual)
- Study completion
- Feb 2014 (actual)
- First posted
- 4 Dec 2012 (estimated)
- Results
- Posted 11 Jan 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Deferiprone (DFP) is the most extensively studied oral iron chelator to date. It has been authorised in Europe in 1999 for the treatment of iron overload in patients with beta-thalassaemia major when DFO is contraindicated or inadequate. Despite a wide experience of DFP there are limited experimental data available on DFP in children and no pharmacokinetic data in children under 6 years of age. On the basis of the existing data in adults and adolescent, in the DEEP-1 trial a pharmacokinetic bridging model was developed to support the dose selection in children aged less than 6 years affected by transfusion dependent haemoglobinopathies. The study consisted of two phases, namely an experimental phase, during which patients received a single dose and a modeling phase, during which PK data obtained after single dose in patients < 6 years of age were analysed in conjunction with historical PK data in adults and older children and adolescents. The model-based analysis of the data obtained after single dose enabled the assessment of the dosing regimen required for the purpose of accurate pharmacokinetic bridging. The ratio between the predicted systemic exposure parameters (AUC and Cmax) in the target population and reference group were used as basis for recommendation of the dose in the target population.
Detailed description
Deferiprone (DFP) was investigated as therapy for children from 1 month to less than 6 years of age. The study was a multicenter randomised, single blind, and single dose PK study. The patients were randomised according to a stratification scheme in which three different dose levels were used.
Objectives: The primary objective of this study was to assess the pharmacokinetics of DFP in paediatric patients aged from 1 month to less than 6 years.
The secondary objectives of this study were:
- To identify dose levels yielding deferiprone exposures comparable to adults and define the dose rationale in children aged from 1 month to less than 6 years.
- To evaluate safety and tolerability of deferiprone after single dose administration in children aged from 1 month to less than 6 years.
- To evaluate the effect of demographic covariates on DFP disposition and estimate the clearance distribution across the population.
Endpoints: The primary endpoints of the study were pharmacokinetic and included:
- primary PK parameters: CL/F, Vd/F, Ka
- secondary PK parameters: AUC, Cmax, Tmax, Css and Cmin.
Secondary endpoints were assessment of clinical safety and tolerability .
Methods: Twenty-three patients were enrolled and 18 of those ( 9 males and 9 females) completed the study. The patients were administered at three dose levels ( 6 patients / each dose):
Dose level 1: 8.3 mg/kg as a single dose (every 8 h) for a total daily dose of 25mg/kg Dose level 2: 16.7 mg/kg as a single dose (every 8 h) for a total daily dose of 50 mg/kg Dose level 3: 33.3 mg/kg as a single dose (every 8 h) for a total daily dose of 100 mg/kg
Blood samples for PK analysis were taken at 6 sampling time intervals, different depending on the dose group: predose; in the range 10 -20 min; in the range 40-55min; in the range 1.05-1.15 h;in the range 1.25-5.30 h;in the range 6-8h after the first dose administration. The concentration of deferiprone was determined by a validated HPLC method. A population PK model approach was applied and the time course of deferiprone concentrations was analysed with Nonlinear mixed effects modelling in NONMEM, version 7.2.0. Model building included the assessment of the influence of relevant demographic covariates (i.e:body weight , age, height) on the disposition of deferiprone.
Using the pharmacokinetic model developed for the paediatric population in this study in conjunction with a model previously developed for 55 adult subjects , simulations were performed to evaluate drug exposure in children below 6 years of age and across a standard thalassaemic adult population.
Descriptive statistics were used to summarise adverse events, vital signs and clinical lab data (haematology, biochemistry and virology)
Results: A one-compartment model with first-order absorption was found to best describe the disposition of deferiprone. The choice of three dose levels enable to assess linearity of pharmacokinetic across the dose range. The disposition parameters estimated through the pop-PK model included CL/F , V/F. In addition to the final model parameter estimates, the secondary pharmacokinetic parameters were derived based on the individual predicted concentration vs. time profiles and were summarised per dose level.
Body weight was found to be a good predictor of inter-individual differences in the population under investigation.
As expected from the known safety profile of the drug, no Serious Adverse Events were observed during the DEEP-1 PK Study.
Based on the simulation analysis performed, a similar exposure is achieved in adults and children in terms of AUC and Css when receiving the current dosing regimen both at 75 and 100 mg/kg/day, while a considerable increase in Cmax was observed in children when compared to the adult population. However, exposure is the parameter related to clinical response rather than Cmax. Indeed, simulations suggested that a dosing regimen of 25 mg/kg t.i.d. (75 mg/kg/day) is recommended for children aged from 1 month to < 6 years, with the possibility of titration up to 33.3 mg/kg t.i.d. (100 mg/kg/day), if necessary.
Study design
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Deferiprone, dose level 1 | Experimental | Drug: Deferiprone, dose level 1single dose level of 8.3 mg/kg every 8 hours for a corresponding total daily dose of 25 mg/kg/day. |
| Deferiprone, dose level 2 | Experimental | Drug: Deferiprone, dose level 2single dose level of 16.7 mg/kg every 8 hours for a corresponding total daily dose of 50 mg/kg/day. |
| Deferiprone, dose level 3 | Experimental | Drug: Deferiprone, dose level 3single dose level of 33.3 mg/kg every 8 hours for a corresponding total daily dose of 100 mg/kg/day. |
Who may qualify
- Ages
- 1 month to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Patients in a chronic transfusional program who have received at least 150 ml/kg/year of packed red blood cells (corresponding approximately to 12 transfusions) and on current treatment with DFO, DFX, DFP; aged from 1 month to less than 6 years; or
- Patients naïve to any chelation treatment who have received not less than 150 ml/kg of packed red blood cells (corresponding to approximately 12 transfusions) and have ferritin levels > 800 ng/mL, aged from 1 month to less than 6 years; or
- Patients who meet the transfusion criteria (150 ml/kg/year corresponding approximately to 12 transfusions) and have known intolerance or contraindication to DFO
- Patients affected by any hereditary haemoglobinopathies requiring chronic transfusion therapy including but not limited to thalassaemia and sickle cell disease
- Written informed consent obtained from their legal guardian on the patient's behalf in accordance with the national legislations. According to his/her capability, patient's informed assent will be collected
Key exclusion criteria (15 listed)
- Patient with known intolerance or contraindication to the trial treatment
- Patient with Hb levels less than 8g/dl (entry may be delayed until values return to normal)
- Patient with platelet count <100.000/mm3 or absolute neutrophil count <1.500/mm3 (entry may be delayed until values return to normal)
- Patient with evidence of abnormal liver function (ALT level >5 times the upper normal limit during six months preceding enrolment; entry may be delayed until values return to normal)
- iron overload from causes other than transfusional haemosiderosis
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 |
|---|---|---|
| Deferiprone, dose level 1Also: DFPa solution at 80 mg/mL will be administered orally | Drug | Deferiprone, dose level 1 |
| Deferiprone, dose level 2Also: DFPa solution at 80 mg/mL will be administered orally | Drug | Deferiprone, dose level 2 |
| Deferiprone, dose level 3Also: DFPa solution at 80 mg/mL will be administered orally | Drug | Deferiprone, dose level 3 |
Primary outcome
CL/F
Time frame: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)
Plasma clearance after oral administration. The parameter was estimated through a population pharmacokinetic model, during which concentration data obtained after single oral dose ( at 3 dose levels) of DFP in patients aged from 1 month to less than 6 years of age.
AUC (0-8h)
Time frame: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)
Area under concentration versus time curve from 0 to 8 h post dosing. Secondary pharmacokinetic parameter derived on the basis of the individual predicted concentration vs. time profiles, through the pop-PK model.
V/F
Time frame: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)
volume of distribution after oral administration. The parameter was estimated through a population pharmacokinetic model, during which concentration data obtained after single oral dose ( at 3 dose levels) of DFP in patients aged from 1 month to less than 6 years of age
Tmax
Time frame: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)
Time at which the maximum concentration (Cmax) is reached. Secondary pharmacokinetic parameters such as Cmax, Min, Tmax, Css and AUC (0-8h) were derived based on the individual predicted concentration vs. time profiles.
Ka
Time frame: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)
Absorption rate constant. The parameter was estimated through a population pharmacokinetic model, during which concentration data obtained after single oral dose ( at 3 dose levels) of DFP in patients aged from 1 month to less than 6 years of age.
Cmax
Time frame: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)
Maximum concentration reached in plasma. Secondary pharmacokinetic parameter derived on the basis of the individual predicted concentration vs. time profiles, through the pop-PK model.
Css
Time frame: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)
Plasma concentration reached at steady state. Secondary pharmacokinetic parameter derived on the basis of the individual predicted concentration vs. time profiles, through the pop-PK model.
Cmin
Time frame: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)
Minimum plasma concentration. Secondary pharmacokinetic parameter derived on the basis of the individual predicted concentration vs. time profiles, through the pop-PK model.
1 secondary outcome
Adverse Events
Time frame: from drug administration up to 8 days post treatment
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jan 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Undecided
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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.
Oscar Della Pasqua study chair
Universiteit Leiden, The Netherlands
Giovanni Carlo Del Vecchio principal investigator
Azienda Ospedaliero-Universitaria Consorziale Policlinico di Bari, Italy
7 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Deferiprone, dose level 1, Deferiprone, dose level 2 and Deferiprone, dose level 3 in people with Chronic Iron Overload, run by Consorzio per Valutazioni Biologiche e Farmacologiche at 7 sites in 3 countries.
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 6 inclusion and 15 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 11 Jan 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Italy, Cyprus, Egypt. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 11 Jan 2017). 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?
CL/F (time frame: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 11 Jan 2017.