Trial · NCT03591575

Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children

NCT03591575, Phase 4, Completed, Chiesi Canada Corp; results first posted 2024-03-15

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children. Trials, record NCT03591575. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03591575

On this page

Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children
NCT number: NCT03591575
Status: Completed
Phases: Phase 4
Enrollment: 64 participants, actual
Lead sponsor: Chiesi Canada Corp
Conditions: Beta Thalassemia Major Anemia, Iron Overload
Interventions: Deferiprone oral solution, Placebo
First posted: 2018-07-19
Last updated: 2024-03-15
Results first posted: 2024-03-15

Source ClinicalTrials.gov

Registered outcomes2

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	The Percentage of Patients in Each Treatment Group Who Still Have a Serum Ferritin Level < 1000 Micrograms Per Liter (μg/L) at Month 12	12 months	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.
Secondary	Percentage of Patients With Serum Ferritin Still Below the Threshold at Different Time Points	4, 8, and 12 months	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 However, a safety feature of the study design was that patients who reached the serum ferritin threshold were withdrawn (i.e., so that they could begin standard chelation therapy).

Source ClinicalTrials.gov

Outcomes2

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	The Percentage of Patients in Each Treatment Group Who Still Have a Serum Ferritin Level < 1000 Micrograms Per Liter (μg/L) at Month 12	12 months	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.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Percentage of Patients With Serum Ferritin Still Below the Threshold at Different Time Points	4, 8, and 12 months	ITT population	Participants	COUNT_OF_PARTICIPANTS

Source ClinicalTrials.gov

Results by group8

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Units
The Percentage of Patients in Each Treatment Group Who Still Have a Serum Ferritin Level < 1000 Micrograms Per Liter (μg/L) at Month 12	Deferiprone		21	Participants
The Percentage of Patients in Each Treatment Group Who Still Have a Serum Ferritin Level < 1000 Micrograms Per Liter (μg/L) at Month 12	Placebo		12	Participants
Percentage of Patients With Serum Ferritin Still Below the Threshold at Different Time Points	Deferiprone	Number of patients still below threshold at Month 4	29	Participants
Percentage of Patients With Serum Ferritin Still Below the Threshold at Different Time Points	Placebo	Number of patients still below threshold at Month 4	28	Participants
Percentage of Patients With Serum Ferritin Still Below the Threshold at Different Time Points	Deferiprone	Number of patients still below threshold at Month 8	22	Participants
Percentage of Patients With Serum Ferritin Still Below the Threshold at Different Time Points	Placebo	Number of patients still below threshold at Month 8	20	Participants
Percentage of Patients With Serum Ferritin Still Below the Threshold at Different Time Points	Deferiprone	Number of patients still below threshold at Month 12	21	Participants
Percentage of Patients With Serum Ferritin Still Below the Threshold at Different Time Points	Placebo	Number of patients still below threshold at Month 12	12	Participants

Source ClinicalTrials.gov

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Deferiprone	32
Overall Study	STARTED	Placebo	32
Overall Study	COMPLETED	Deferiprone	26
Overall Study	COMPLETED	Placebo	31
Overall Study	NOT COMPLETED	Deferiprone	6
Overall Study	NOT COMPLETED	Placebo	1

Source ClinicalTrials.gov

Adverse events46

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Abdominal pain	Gastrointestinal disorders	Deferiprone	2	32	2
other	Abdominal pain	Gastrointestinal disorders	Placebo	3	32	3
other	Abdominal pain lower	Gastrointestinal disorders	Deferiprone	2	32	2
other	Abdominal pain lower	Gastrointestinal disorders	Placebo	0	32	0
other	Dental caries	Gastrointestinal disorders	Deferiprone	1	32	1
other	Dental caries	Gastrointestinal disorders	Placebo	2	32	2
other	Diarrhoea	Gastrointestinal disorders	Deferiprone	4	32	8
other	Diarrhoea	Gastrointestinal disorders	Placebo	5	32	5
other	Vomiting	Gastrointestinal disorders	Deferiprone	2	32	2
other	Vomiting	Gastrointestinal disorders	Placebo	3	32	3

1 to 10 of 46Source ClinicalTrials.gov

See all 46 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Deferiprone	0	32
Total, all-cause mortality	Placebo	0	32
Total, other adverse events	Deferiprone	29	32
Total, other adverse events	Placebo	29	32
Total, serious adverse events	Deferiprone	5	32
Total, serious adverse events	Placebo	2	32

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons3

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Deferiprone vs Placebo: The Percentage of Patients in Each Treatment Group Who Still Have a Serum Ferritin Level < 1000 Micrograms Per Liter (μg/L) at Month 12	Trial comparisons
Deferiprone vs Placebo: deaths from any cause	Trial comparisons
Deferiprone vs Placebo: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT03591575

GET https://conductscience.org/api/trials/v1/NCT03591575/events?page=2

API reference For AI assistants