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Trial record 17 of 22 for:    "Thalassemia" | "Deferiprone"

Pharmacokinetic Study of Deferiprone in Paediatric Patients (DEEP-1)

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ClinicalTrials.gov Identifier: NCT01740713
Recruitment Status : Completed
First Posted : December 4, 2012
Results First Posted : January 11, 2017
Last Update Posted : January 11, 2017
Sponsor:
Collaborator:
European Commission
Information provided by (Responsible Party):
Consorzio per Valutazioni Biologiche e Farmacologiche

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Single (Investigator)
Condition Chronic Iron Overload
Interventions Drug: Deferiprone, dose level 1
Drug: Deferiprone, dose level 2
Drug: Deferiprone, dose level 3
Enrollment 23
Recruitment Details A total of 23 children affected by transfusion-dependent haemoglobinopathies were enrolled in this study. Of these 23 children, 2 were screening failures and 3 early terminations.
Pre-assignment Details  
Arm/Group Title Deferiprone 25 mg/kg/Day Deferiprone 50 mg/kg/da Deferiprone 100 mg/kg/Day
Hide Arm/Group Description

Deferiprone will be administered at 25 mg/kg/day

Deferiprone, dose level 1: deferiprone liquid oral solution (80 mg/ml)

Deferiprone will be administered at 50 mg/kg/day

Deferiprone, dose level 2: deferiprone liquid oral solution (80 mg/ml)

Deferiprone will be administered at 100 mg/kg/day

Deferiprone, dose level 3: deferiprone liquid oral solution (80 mg/ml)

Period Title: Overall Study
Started 6 8 7
Completed 6 6 6
Not Completed 0 2 1
Arm/Group Title Deferiprone 25 mg/kg/Day Deferiprone 50 mg/kg/Day Deferiprone 100 mg/kg/Day Total
Hide Arm/Group Description

Deferiprone will be administered at 25 mg/kg/day

Deferiprone, dose level 1: deferiprone liquid oral solution (80 mg/ml)

Deferiprone will be administered at 50 mg/kg/day

Deferiprone, dose level 2: deferiprone liquid oral solution (80 mg/ml)

Deferiprone will be administered at 100 mg/kg/day

Deferiprone, dose level 3: deferiprone liquid oral solution (80 mg/ml)

Total of all reporting groups
Overall Number of Baseline Participants 6 8 7 21
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 8 participants 7 participants 21 participants
<=18 years
6
 100.0%
8
 100.0%
7
 100.0%
21
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Gender  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 8 participants 7 participants 21 participants
Female
3
  50.0%
4
  50.0%
4
  57.1%
11
  52.4%
Male
3
  50.0%
4
  50.0%
3
  42.9%
10
  47.6%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Italy Number Analyzed 6 participants 8 participants 7 participants 21 participants
6 8 7 21
1.Primary Outcome
Title CL/F
Hide Description 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.
Time Frame Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title PK Population
Hide Arm/Group Description:
Deferiprone liquid oral solution (80 mg/ml) has been administered at 25/50/100 mg/kg/day
Overall Number of Participants Analyzed 18
Mean (Standard Error)
Unit of Measure: litre/h
8.3  (0.569)
2.Primary Outcome
Title AUC (0-8h)
Hide Description 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.
Time Frame Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Deferiprone 25 mg/kg/Day Deferiprone 50 mg/kg/Day Deferiprone 100 mg/kg/Day
Hide Arm/Group Description:

Deferiprone will be administered at 25 mg/kg/day

Deferiprone, dose level 1: deferiprone liquid oral solution (80 mg/ml)

Deferiprone will be administered at 50 mg/kg/day

Deferiprone, dose level 2: deferiprone liquid oral solution (80 mg/ml)

Deferiprone will be administered at 100 mg/kg/day

Deferiprone, dose level 3: deferiprone liquid oral solution (80 mg/ml)

Overall Number of Participants Analyzed 6 6 6
Median (Inter-Quartile Range)
Unit of Measure: micromol*h/L
116.7
(90.6 to 129.0)
210.0
(173.1 to 266.6)
428.8
(291.4 to 547.8)
3.Primary Outcome
Title V/F
Hide Description 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
Time Frame Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title PK Population
Hide Arm/Group Description:
Deferiprone liquid oral solution (80 mg/ml) has been administered at 25/50/100 mg/kg/day
Overall Number of Participants Analyzed 18
Mean (Standard Error)
Unit of Measure: litres
18.7  (1.16)
4.Primary Outcome
Title Tmax
Hide Description 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.
Time Frame Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Deferiprone 25 mg/kg/Day Deferiprone 50 mg/kg/Day Deferiprone 100 mg/kg/Day
Hide Arm/Group Description:

Deferiprone will be administered at 25 mg/kg/day

Deferiprone, dose level 1: deferiprone liquid oral solution (80 mg/ml)

Deferiprone will be administered at 50 mg/kg/day

Deferiprone, dose level 2: deferiprone liquid oral solution (80 mg/ml)

Deferiprone will be administered at 100 mg/kg/day

Deferiprone, dose level 3: deferiprone liquid oral solution (80 mg/ml)

Overall Number of Participants Analyzed 6 6 6
Median (Inter-Quartile Range)
Unit of Measure: hour
0.33
(0.19 to 0.92)
0.33
(0.21 to 0.63)
0.37
(0.27 to 0.42)
5.Primary Outcome
Title Ka
Hide Description 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.
Time Frame Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title PK Population
Hide Arm/Group Description:
Deferiprone liquid oral solution (80 mg/ml) has been administered at 25/50/100 mg/kg/day
Overall Number of Participants Analyzed 18
Mean (Standard Error)
Unit of Measure: h^-1
9.13  (1.41)
6.Primary Outcome
Title Cmax
Hide Description 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.
Time Frame Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Deferiprone 25 mg/kg/Day Deferiprone 50 mg/kg/Day Deferiprone 100 mg/kg/Day
Hide Arm/Group Description:

Deferiprone will be administered at 25 mg/kg/day

Deferiprone, dose level 1: deferiprone liquid oral solution (80 mg/ml)

Deferiprone will be administered at 50 mg/kg/day

Deferiprone, dose level 2: deferiprone liquid oral solution (80 mg/ml)

Deferiprone will be administered at 100 mg/kg/day

Deferiprone, dose level 3: deferiprone liquid oral solution (80 mg/ml)

Overall Number of Participants Analyzed 6 6 6
Median (Inter-Quartile Range)
Unit of Measure: microM
61.7
(45.1 to 80.7)
119.8
(106 to 154)
229.5
(179.7 to 278.1)
7.Primary Outcome
Title Css
Hide Description 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.
Time Frame Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Deferiprone 25 mg/kg/Day Deferiprone 50 mg/kg/Day Deferiprone 100 mg/kg/Day
Hide Arm/Group Description:

Deferiprone will be administered at 25 mg/kg/day

Deferiprone, dose level 1: deferiprone liquid oral solution (80 mg/ml)

Deferiprone will be administered at 50 mg/kg/day

Deferiprone, dose level 2: deferiprone liquid oral solution (80 mg/ml)

Deferiprone will be administered at 100 mg/kg/day

Deferiprone, dose level 3: deferiprone liquid oral solution (80 mg/ml)

Overall Number of Participants Analyzed 6 6 6
Median (Inter-Quartile Range)
Unit of Measure: microM
2.1
(1.6 to 2.3)
3.7
(3.1 to 4.9)
7.7
(5.1 to 10)
8.Primary Outcome
Title Cmin
Hide Description Minimum plasma concentration. Secondary pharmacokinetic parameter derived on the basis of the individual predicted concentration vs. time profiles, through the pop-PK model.
Time Frame Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Deferiprone 25 mg/kg/Day Deferiprone 50 mg/kg/Day Deferiprone 100 mg/kg/Day
Hide Arm/Group Description:

Deferiprone will be administered at 25 mg/kg/day

Deferiprone, dose level 1: deferiprone liquid oral solution (80 mg/ml)

Deferiprone will be administered at 50 mg/kg/day

Deferiprone, dose level 2: deferiprone liquid oral solution (80 mg/ml)

Deferiprone will be administered at 100 mg/kg/day

Deferiprone, dose level 3: deferiprone liquid oral solution (80 mg/ml)

Overall Number of Participants Analyzed 6 6 6
Median (Inter-Quartile Range)
Unit of Measure: microM
1.5
(0.92 to 2.6)
1.9
(0.79 to 5.5)
6.8
(3.1 to 13.9)
9.Secondary Outcome
Title Adverse Events
Hide Description All the medical occurrences that started after the administration of the drug
Time Frame from drug administration up to 8 days post treatment
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Safety Population
Hide Arm/Group Description:
patients who may experience adverse events occurred before or after treatment
Overall Number of Participants Analyzed 21
Measure Type: Number
Unit of Measure: participants
3
Time Frame All the medical occurrences that started after the administration of the drug under investigation have been recorded as AEs till the follow -up visit at 8 days post dosing.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Deferiprone 25mg/kg/Day Deferiprone 50 mg/kg/Day Deferiprone 100 mg/kg/Day
Hide Arm/Group Description

Deferiprone will be administered at 25 mg/kg/day

Deferiprone, dose level 1: deferiprone liquid oral solution (80 mg/ml)

Deferiprone will be administered at 50 mg/kg/day

Deferiprone, dose level 2: deferiprone liquid oral solution (80 mg/ml)

Deferiprone will be administered at 100 mg/kg/day

Deferiprone, dose level 3: deferiprone liquid oral solution (80 mg/ml)

All-Cause Mortality
Deferiprone 25mg/kg/Day Deferiprone 50 mg/kg/Day Deferiprone 100 mg/kg/Day
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Deferiprone 25mg/kg/Day Deferiprone 50 mg/kg/Day Deferiprone 100 mg/kg/Day
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/6 (0.00%)      0/8 (0.00%)      0/7 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Deferiprone 25mg/kg/Day Deferiprone 50 mg/kg/Day Deferiprone 100 mg/kg/Day
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/6 (16.67%)      0/8 (0.00%)      2/7 (28.57%)    
Pregnancy, puerperium and perinatal conditions       
Infantile spitting up  1  0/6 (0.00%)  0 0/8 (0.00%)  0 2/7 (28.57%)  2
Skin and subcutaneous tissue disorders       
Rash trunk  1  1/6 (16.67%)  1 0/8 (0.00%)  0 0/7 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (Unspecified)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Clinical Research Coordinator
Organization: Consorzio per Valutazioni Biologiche e Farmacologiche
Phone: 0039 0382 25075
Responsible Party: Consorzio per Valutazioni Biologiche e Farmacologiche
ClinicalTrials.gov Identifier: NCT01740713     History of Changes
Other Study ID Numbers: DEEP-1
First Submitted: November 28, 2012
First Posted: December 4, 2012
Results First Submitted: March 4, 2016
Results First Posted: January 11, 2017
Last Update Posted: January 11, 2017