Trial Outcomes & Findings for Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children (NCT NCT03591575)
NCT ID: NCT03591575
Last Updated: 2024-03-15
Results Overview
Current treatment guidelines are that standard iron chelation therapy should begin after a serum ferritin level of 1000 μg/L has been reached; thus, patients who were at this level at two consecutive visits were removed from the study so that they could start on this therapy.
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
64 participants
Primary outcome timeframe
12 months
Results posted on
2024-03-15
Participant Flow
Participant milestones
| Measure |
Deferiprone
Subjects in this group will receive deferiprone oral solution at a dosage up to 75 milligrams per kilogram of body weight (mg/kg) per day, divided into 3 equal doses
Deferiprone oral solution: Liquid formulation of deferiprone, with a concentration of 80 mg/mL
|
Placebo
Subjects in this group will receive placebo solution at a volume equal to what they would receive if they were in the active arm, divided into 3 equal doses
Placebo: Liquid solution that matches deferiprone oral solution in appearance and taste
|
|---|---|---|
|
Overall Study
STARTED
|
32
|
32
|
|
Overall Study
COMPLETED
|
26
|
31
|
|
Overall Study
NOT COMPLETED
|
6
|
1
|
Reasons for withdrawal
| Measure |
Deferiprone
Subjects in this group will receive deferiprone oral solution at a dosage up to 75 milligrams per kilogram of body weight (mg/kg) per day, divided into 3 equal doses
Deferiprone oral solution: Liquid formulation of deferiprone, with a concentration of 80 mg/mL
|
Placebo
Subjects in this group will receive placebo solution at a volume equal to what they would receive if they were in the active arm, divided into 3 equal doses
Placebo: Liquid solution that matches deferiprone oral solution in appearance and taste
|
|---|---|---|
|
Overall Study
Adverse Event
|
2
|
0
|
|
Overall Study
Withdrawal by Subject
|
4
|
1
|
Baseline Characteristics
Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children
Baseline characteristics by cohort
| Measure |
Deferiprone
n=32 Participants
Subjects in this group will receive deferiprone oral solution at a dosage up to 75 milligrams per kilogram of body weight (mg/kg) per day, divided into 3 equal doses
Deferiprone oral solution: Liquid formulation of deferiprone, with a concentration of 80 mg/mL
|
Placebo
n=32 Participants
Subjects in this group will receive placebo solution at a volume equal to what they would receive if they were in the active arm, divided into 3 equal doses
Placebo: Liquid solution that matches deferiprone oral solution in appearance and taste
|
Total
n=64 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
3.03 Years
STANDARD_DEVIATION 2.42 • n=99 Participants
|
2.63 Years
STANDARD_DEVIATION 1.70 • n=107 Participants
|
2.83 Years
STANDARD_DEVIATION 2.08 • n=206 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=99 Participants
|
11 Participants
n=107 Participants
|
23 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
20 Participants
n=99 Participants
|
21 Participants
n=107 Participants
|
41 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
32 Participants
n=99 Participants
|
31 Participants
n=107 Participants
|
63 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
6 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
9 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
26 Participants
n=99 Participants
|
29 Participants
n=107 Participants
|
55 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Region of Enrollment
Egypt
|
26 Participants
n=99 Participants
|
29 Participants
n=107 Participants
|
55 Participants
n=206 Participants
|
|
Region of Enrollment
Indonesia
|
6 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
9 Participants
n=206 Participants
|
|
Serum ferritin (µg/L)
|
513.84 micrograms per liter
STANDARD_DEVIATION 236.06 • n=99 Participants
|
451.69 micrograms per liter
STANDARD_DEVIATION 196.43 • n=107 Participants
|
482.76 micrograms per liter
STANDARD_DEVIATION 217.68 • n=206 Participants
|
PRIMARY outcome
Timeframe: 12 monthsPopulation: Intent-to-Treat (ITT) population. For patients who withdrew early, imputed data were used to estimate the serum ferritin values that would likely have been seen at Month 12 had they continued.
Current treatment guidelines are that standard iron chelation therapy should begin after a serum ferritin level of 1000 μg/L has been reached; thus, patients who were at this level at two consecutive visits were removed from the study so that they could start on this therapy.
Outcome measures
| Measure |
Deferiprone
n=32 Participants
Subjects in this group will receive deferiprone oral solution at a dosage up to 75 milligrams per kilogram of body weight (mg/kg) per day, divided into 3 equal doses
Deferiprone oral solution: Liquid formulation of deferiprone, with a concentration of 80 mg/mL
|
Placebo
n=32 Participants
Subjects in this group will receive placebo solution at a volume equal to what they would receive if they were in the active arm, divided into 3 equal doses
Placebo: Liquid solution that matches deferiprone oral solution in appearance and taste
|
|---|---|---|
|
The Percentage of Patients in Each Treatment Group Who Still Have a Serum Ferritin Level < 1000 Micrograms Per Liter (μg/L) at Month 12
|
21 Participants
|
12 Participants
|
SECONDARY outcome
Timeframe: 4, 8, and 12 monthsPopulation: ITT population
The outcome measure was the percentage of patients in each group still below the serum ferritin threshold at Months 4, 8, and 12. Without adequate chelation therapy, most patients receiving red blood cell transfusions are likely to exceed this level within a few months. However, a safety feature of the study design was that patients who reached the serum ferritin threshold were withdrawn (i.e., so that they could begin standard chelation therapy).
Outcome measures
| Measure |
Deferiprone
n=32 Participants
Subjects in this group will receive deferiprone oral solution at a dosage up to 75 milligrams per kilogram of body weight (mg/kg) per day, divided into 3 equal doses
Deferiprone oral solution: Liquid formulation of deferiprone, with a concentration of 80 mg/mL
|
Placebo
n=32 Participants
Subjects in this group will receive placebo solution at a volume equal to what they would receive if they were in the active arm, divided into 3 equal doses
Placebo: Liquid solution that matches deferiprone oral solution in appearance and taste
|
|---|---|---|
|
Percentage of Patients With Serum Ferritin Still Below the Threshold at Different Time Points
Number of patients still below threshold at Month 4
|
29 Participants
|
28 Participants
|
|
Percentage of Patients With Serum Ferritin Still Below the Threshold at Different Time Points
Number of patients still below threshold at Month 8
|
22 Participants
|
20 Participants
|
|
Percentage of Patients With Serum Ferritin Still Below the Threshold at Different Time Points
Number of patients still below threshold at Month 12
|
21 Participants
|
12 Participants
|
Adverse Events
Deferiprone
Serious events: 5 serious events
Other events: 29 other events
Deaths: 0 deaths
Placebo
Serious events: 2 serious events
Other events: 29 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Deferiprone
n=32 participants at risk
Subjects in this group will receive deferiprone oral solution at a dosage up to 75 milligrams per kilogram of body weight (mg/kg) per day, divided into 3 equal doses
Deferiprone oral solution: Liquid formulation of deferiprone, with a concentration of 80 mg/mL
|
Placebo
n=32 participants at risk
Subjects in this group will receive placebo solution at a volume equal to what they would receive if they were in the active arm, divided into 3 equal doses
Placebo: Liquid solution that matches deferiprone oral solution in appearance and taste
|
|---|---|---|
|
Blood and lymphatic system disorders
Agranulocytosis
|
3.1%
1/32 • Number of events 1 • 12 months
|
0.00%
0/32 • 12 months
|
|
Blood and lymphatic system disorders
Autoimmune haemolytic anaemia
|
0.00%
0/32 • 12 months
|
3.1%
1/32 • Number of events 1 • 12 months
|
|
Blood and lymphatic system disorders
Neutropenia
|
12.5%
4/32 • Number of events 4 • 12 months
|
3.1%
1/32 • Number of events 1 • 12 months
|
|
Infections and infestations
Dengue fever
|
3.1%
1/32 • Number of events 1 • 12 months
|
0.00%
0/32 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Bronchitis chronic
|
0.00%
0/32 • 12 months
|
3.1%
1/32 • Number of events 1 • 12 months
|
Other adverse events
| Measure |
Deferiprone
n=32 participants at risk
Subjects in this group will receive deferiprone oral solution at a dosage up to 75 milligrams per kilogram of body weight (mg/kg) per day, divided into 3 equal doses
Deferiprone oral solution: Liquid formulation of deferiprone, with a concentration of 80 mg/mL
|
Placebo
n=32 participants at risk
Subjects in this group will receive placebo solution at a volume equal to what they would receive if they were in the active arm, divided into 3 equal doses
Placebo: Liquid solution that matches deferiprone oral solution in appearance and taste
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
6.2%
2/32 • Number of events 2 • 12 months
|
9.4%
3/32 • Number of events 3 • 12 months
|
|
Gastrointestinal disorders
Abdominal pain lower
|
6.2%
2/32 • Number of events 2 • 12 months
|
0.00%
0/32 • 12 months
|
|
Gastrointestinal disorders
Dental caries
|
3.1%
1/32 • Number of events 1 • 12 months
|
6.2%
2/32 • Number of events 2 • 12 months
|
|
Gastrointestinal disorders
Diarrhoea
|
12.5%
4/32 • Number of events 8 • 12 months
|
15.6%
5/32 • Number of events 5 • 12 months
|
|
Gastrointestinal disorders
Vomiting
|
6.2%
2/32 • Number of events 2 • 12 months
|
9.4%
3/32 • Number of events 3 • 12 months
|
|
General disorders
Pyrexia
|
62.5%
20/32 • Number of events 60 • 12 months
|
56.2%
18/32 • Number of events 55 • 12 months
|
|
Infections and infestations
Bronchitis
|
9.4%
3/32 • Number of events 3 • 12 months
|
18.8%
6/32 • Number of events 7 • 12 months
|
|
Infections and infestations
Gastroenteritis
|
15.6%
5/32 • Number of events 5 • 12 months
|
12.5%
4/32 • Number of events 5 • 12 months
|
|
Infections and infestations
Nasopharyngitis
|
25.0%
8/32 • Number of events 10 • 12 months
|
25.0%
8/32 • Number of events 14 • 12 months
|
|
Infections and infestations
Pharyngitis
|
12.5%
4/32 • Number of events 5 • 12 months
|
3.1%
1/32 • Number of events 1 • 12 months
|
|
Infections and infestations
Tonsillitis
|
6.2%
2/32 • Number of events 3 • 12 months
|
12.5%
4/32 • Number of events 4 • 12 months
|
|
Infections and infestations
Upper respiratory tract infection
|
12.5%
4/32 • Number of events 18 • 12 months
|
15.6%
5/32 • Number of events 8 • 12 months
|
|
Injury, poisoning and procedural complications
Thermal burn
|
0.00%
0/32 • 12 months
|
6.2%
2/32 • Number of events 2 • 12 months
|
|
Investigations
Neutrophil count decreased
|
28.1%
9/32 • Number of events 19 • 12 months
|
28.1%
9/32 • Number of events 10 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/32 • 12 months
|
6.2%
2/32 • Number of events 2 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
12.5%
4/32 • Number of events 5 • 12 months
|
18.8%
6/32 • Number of events 8 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
6.2%
2/32 • Number of events 2 • 12 months
|
3.1%
1/32 • Number of events 1 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
6.2%
2/32 • Number of events 2 • 12 months
|
21.9%
7/32 • Number of events 9 • 12 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60