Trial · NCT01741532

Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)

NCT01741532, Phase 3, Completed, ApoPharma; results first posted 2019-05-23

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN). Trials, record NCT01741532. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01741532

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Trial

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

Title: Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)
NCT number: NCT01741532
Status: Completed
Phases: Phase 3
Enrollment: 89 participants, actual
Lead sponsor: ApoPharma
Conditions: Pantothenate Kinase-Associated Neurodegeneration
Interventions: Deferiprone oral solution, Placebo
First posted: 2012-12-05
Last updated: 2019-07-05
Results first posted: 2019-05-23

Source ClinicalTrials.gov

Registered outcomes8

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

List	Outcome	Time frame	Description
Primary	Change in Score on Barry-Albright Dystonia Scale	Baseline to 18 Months	The Barry-Albright Dystonia (BAD) scale rates severity of dystonia (sustained muscle contractions causing twisting and repetitive movements or abnormal postures) in 8 body regions. The individual scores are summed to provide a total score ranging from 0 to 32, with higher scores indicating greater severity. The co-primary endpoint in this study was the change from baseline to Month 18 in BAD total score.
Primary	Score on Patient Global Impression of Improvement at End of Study	Month 18	The Patient Global Impression of Improvement (PGI-I) is a global index that assesses the response of a condition to a therapy by asking patients to rate their current state relative to their state at baseline. It consists of a 7-point rating scale, where 1=very much improved, 2= much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, and 7 = very much worse.
Secondary	Change in Score on Unified Parkinson's Disease Rating Scale	Baseline to 18 Months	The Unified Parkinson's Disease Rating Scale (UPDRS) is the major rating scale used to assess severity of symptoms of Parkinson's disease, some of which are similar to those of PKAN. The UPDRS subscales used in this study were Part I: Mentation, Behavior and Mood, scored from 0 (best) to 16 (worst); Part II: Activities of Daily Living, scored from 0 (best) to 52 (worst); Part III: Motor Examination, scored from 0 (best) to 108 (worst); and Part VI: Schwab and England Activities of Daily Living Scale, scored from 0% (worst) to 100% (best).
Secondary	Change in Score on Functional Independence Measure	Baseline to 18 Months	The Functional Independence Measure (FIM) scale is used to assess physical and cognitive disability in three areas of daily living: self-care, mobility, and cognition. Within each area, items are scored according to the level of assistance required to perform that activity of daily living. A score of 1-2 indicates that the patient is completely dependent on a helper to perform the task, a score of 3-5 indicates that the patient is moderately dependent, and a score of 6-7 indicates that no help is required. The individual scores are summed to provide a global score from 18 (worst) to 126 (best).
Secondary	Change in Score on WeeFIM	Baseline to 18 Months	The WeeFIM is the pediatric version of the Functional Independence Measure scale, and is used to assess physical and cognitive disability in three areas of daily living: self-care, mobility, and cognition. Within each area, items are scored according to the level of assistance required to perform that activity of daily living. A score of 1-2 indicates that the patient is completely dependent on a helper to perform the task, a score of 3-5 indicates that the patient is moderately dependent, and a score of 6-7 indicates that no help is required. The individual scores are summed to provide a global score from 18 (worst) to 126 (best).
Secondary	Change in Score on Pediatric Quality of Life	Baseline to 18 Months	The Pediatric Quality of Life (PedsQL) questionnaire is used to measure functional health and well-being from the patient's point of view. Separate versions of the questionnaire are available for children, young adults aged 18-25 years, and adults older than 25 years. Patients are asked to indicate how they have felt over the past month, and the scores of the 23 questions are used to generate an overall score that ranges from 0 (worst) to 100 (best).
Secondary	Change in Score on Pittsburgh Sleep Quality Index	Baseline to 18 Months	The Pittsburgh Sleep Quality Index (PSQI) is a self-rated questionnaire that assesses sleep quality and disturbances over a 1-month time interval. A total of 19 individual items are used to generate 7 "component" scores: subjective sleep quality, sleep latency, sleep duration, habitual sleep efficiency, sleep disturbances, use of sleeping medication, and daytime dysfunction, and a score is generated that ranges from 0 (best) to 21 (worst).
Secondary	Change in Level of Brain Iron	Baseline to 18 Months	Neurodegeneration in patients with PKAN is associated with localized brain iron accumulation, with the highest amount of accumulation seen in the globus pallidus, one of the main areas for motor control. MRI R2\* scans of this region were performed at baseline and Month 18 in a subset of patients who did not have a deep brain stimulation (DBS) device implanted, and for whom the use of anesthesia, if required, was deemed acceptable by the investigator.

Source ClinicalTrials.gov

Outcomes8

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

Type	Outcome	Time frame	Units	Measured as
PRIMARY	Change in Score on Barry-Albright Dystonia Scale	Baseline to 18 Months	score on a scale	LEAST_SQUARES_MEAN
PRIMARY	Score on Patient Global Impression of Improvement at End of Study	Month 18	score on a scale	LEAST_SQUARES_MEAN
SECONDARY	Change in Score on Unified Parkinson's Disease Rating Scale	Baseline to 18 Months	score on a scale	LEAST_SQUARES_MEAN
SECONDARY	Change in Score on Functional Independence Measure	Baseline to 18 Months	score on a scale	LEAST_SQUARES_MEAN
SECONDARY	Change in Score on WeeFIM	Baseline to 18 Months	score on a scale	LEAST_SQUARES_MEAN
SECONDARY	Change in Score on Pediatric Quality of Life	Baseline to 18 Months	score on a scale	LEAST_SQUARES_MEAN
SECONDARY	Change in Score on Pittsburgh Sleep Quality Index	Baseline to 18 Months	score on a scale	LEAST_SQUARES_MEAN
SECONDARY	Change in Level of Brain Iron	Baseline to 18 Months	Hz	LEAST_SQUARES_MEAN

Source ClinicalTrials.gov

Results by group24

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

Outcome	Group	Category	Value	Spread	Units
Change in Score on Barry-Albright Dystonia Scale	Deferiprone		2.48	Standard Error 0.63	score on a scale
Change in Score on Barry-Albright Dystonia Scale	Placebo		3.99	Standard Error 0.82	score on a scale
Score on Patient Global Impression of Improvement at End of Study	Deferiprone		4.55	Standard Error 0.30	score on a scale
Score on Patient Global Impression of Improvement at End of Study	Placebo		4.66	Standard Error 0.38	score on a scale
Change in Score on Unified Parkinson's Disease Rating Scale	Deferiprone	UPDRS Part I	-0.25	Standard Error 0.43	score on a scale
Change in Score on Unified Parkinson's Disease Rating Scale	Placebo	UPDRS Part I	-0.07	Standard Error 0.55	score on a scale
Change in Score on Unified Parkinson's Disease Rating Scale	Deferiprone	UPDRS Part II	1.09	Standard Error 1.19	score on a scale
Change in Score on Unified Parkinson's Disease Rating Scale	Placebo	UPDRS Part II	2.36	Standard Error 1.52	score on a scale
Change in Score on Unified Parkinson's Disease Rating Scale	Deferiprone	UPDRS Part III	5.38	Standard Error 2.20	score on a scale
Change in Score on Unified Parkinson's Disease Rating Scale	Placebo	UPDRS Part III	2.06	Standard Error 2.79	score on a scale

1 to 10 of 24Source ClinicalTrials.gov

See all 24 results by group

Participant flow10

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Deferiprone	59
Overall Study	STARTED	Placebo	30
Overall Study	Exposed	Deferiprone	58
Overall Study	Exposed	Placebo	30
Overall Study	Provided Post-baseline Efficacy Data	Deferiprone	58
Overall Study	Provided Post-baseline Efficacy Data	Placebo	28
Overall Study	COMPLETED	Deferiprone	49
Overall Study	COMPLETED	Placebo	27
Overall Study	NOT COMPLETED	Deferiprone	10
Overall Study	NOT COMPLETED	Placebo	3

Source ClinicalTrials.gov

Adverse events196

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	Anaemia	Blood and lymphatic system disorders	Deferiprone	12	58	23
other	Anaemia	Blood and lymphatic system disorders	Placebo	0	30	0
other	Leukocytosis	Blood and lymphatic system disorders	Deferiprone	2	58	3
other	Leukocytosis	Blood and lymphatic system disorders	Placebo	2	30	2
other	Ear pain	Ear and labyrinth disorders	Deferiprone	1	58	1
other	Ear pain	Ear and labyrinth disorders	Placebo	3	30	3
other	Abdominal pain upper	Gastrointestinal disorders	Deferiprone	4	58	6
other	Abdominal pain upper	Gastrointestinal disorders	Placebo	5	30	7
other	Constipation	Gastrointestinal disorders	Deferiprone	2	58	3
other	Constipation	Gastrointestinal disorders	Placebo	4	30	4

1 to 10 of 196Source ClinicalTrials.gov

See all 196 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Deferiprone	0	58
Total, all-cause mortality	Placebo	0	30
Total, other adverse events	Deferiprone	57	58
Total, other adverse events	Placebo	30	30
Total, serious adverse events	Deferiprone	18	58
Total, serious adverse events	Placebo	10	30

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
31202468	DERIVED

Source PubMed

Grants1

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.

Project	Title	Principal investigators	Organization	Funder	Fiscal years
R01FD004103	Phase 2 Study of Deferiprone in the Treatment of NBIA	VICHINSKY, ELLIOTT P	CHILDREN'S HOSPITAL & RES CTR AT OAKLAND	FOOD AND DRUG ADMINISTRATION	2012 to 2015

Source NIH RePORTER

Trial comparisons2

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

Comparison	Source
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/NCT01741532

GET https://conductscience.org/api/trials/v1/NCT01741532/results?page=2

API reference For AI assistants