Trial · NCT02174848

Long-term Deferiprone Treatment in Patients With Pantothenate Kinase-Associated Neurodegeneration

NCT02174848, Phase 3, Completed, ApoPharma; results first posted 2019-07-17

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Long-term Deferiprone Treatment in Patients With Pantothenate Kinase-Associated Neurodegeneration. Trials, record NCT02174848. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02174848

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Trial

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

Title: Long-term Deferiprone Treatment in Patients With Pantothenate Kinase-Associated Neurodegeneration
NCT number: NCT02174848
Status: Completed
Phases: Phase 3
Enrollment: 68 participants, actual
Lead sponsor: ApoPharma
Conditions: Pantothenate Kinase-Associated Neurodegeneration
Interventions: Deferiprone oral solution
First posted: 2014-06-26
Last updated: 2020-08-25
Other registries' IDs: eudract:2012-000845-11
Results first posted: 2019-07-17

Source ClinicalTrials.gov

Registered outcomes6

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

List	Outcome	Time frame	Description
Primary	Number of Participants With Adverse Events	18 months	Safety and tolerability were assessed based on changes in: frequency of adverse events (AEs), frequency of serious adverse events (SAEs), and discontinuation due to AEs. No statistical comparison between the groups was conducted as all participants received the same study product.
Secondary	Change in Score on the BAD Scale -- Comparison of Treatment Groups Over Each Study	Baseline and Month 18 of each study	The Barry-Albright Dystonia (BAD) scale is an instrument for rating the severity of dystonia in eight body regions. The individual scores are summed to provide a total score that ranges from 0 to 32; the higher the score, the more severe the dystonia. Patients were assessed for the change in total BAD score over the course of both the initial study (during which one group received placebo and the other received deferiprone) and the extension study (during which both groups received deferiprone).
Secondary	Change in Score on the BAD Scale -- Comparison of Placebo-DFP Patients Across Studies	Baseline and Month 18 of each study	The Barry-Albright Dystonia (BAD) scale is an instrument for rating the severity of dystonia in eight body regions. The individual scores are summed to provide a total score that ranges from 0 to 32; the higher the score, the more severe the dystonia. Patients were assessed for the change in total BAD score over the course of each study.
Secondary	Change in Score on the BAD Scale -- Comparison of DFP-DFP Patients Across Studies	Baseline and Month 18 of each study	The Barry-Albright Dystonia (BAD) scale is an instrument for rating the severity of dystonia in eight body regions. The individual scores are summed to provide a total score that ranges from 0 to 32; the higher the score, the more severe the dystonia. Patients were assessed for the change in total BAD score over the course of the study.
Secondary	Proportion of Patients With Improved or Unchanged BAD Score	Month 18 of each study	Patients were deemed to be responders if their BAD total score either improved or remained unchanged from baseline, with baseline being the start of each study for the placebo-DFP group and the start of the initial study for the DFP-DFP group
Secondary	Patient Global Impression of Improvement (PGI-I) Comparison of Placebo-DFP Patients Across Studies	Month 18 of each study	The Patient Global Impression of Improvement (PGI-I) is a global index used to rate the response of a condition to a therapy. Patients were asked at each post-baseline visit to rate their overall condition since the start of the extension study on a 7-point rating scale: 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, and 7 = very much worse.

Source ClinicalTrials.gov

Outcomes6

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Number of Participants With Adverse Events	18 months	Safety population	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Change in Score on the BAD Scale -- Comparison of Treatment Groups Over Each Study	Baseline and Month 18 of each study		score on a scale	MEAN
SECONDARY	Change in Score on the BAD Scale -- Comparison of Placebo-DFP Patients Across Studies	Baseline and Month 18 of each study		score on a scale	MEAN
SECONDARY	Change in Score on the BAD Scale -- Comparison of DFP-DFP Patients Across Studies	Baseline and Month 18 of each study		score on a scale	MEAN
SECONDARY	Proportion of Patients With Improved or Unchanged BAD Score	Month 18 of each study		Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Patient Global Impression of Improvement (PGI-I) Comparison of Placebo-DFP Patients Across Studies	Month 18 of each study		score on a scale	MEAN

Source ClinicalTrials.gov

Results by group20

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

Outcome	Group	Category	Value	Spread	Units
Number of Participants With Adverse Events	Placebo-DFP	Number of patients with at least one AE	22		Participants
Number of Participants With Adverse Events	DFP-DFP	Number of patients with at least one AE	42		Participants
Number of Participants With Adverse Events	Placebo-DFP	Number of patients with at least one SAE	12		Participants
Number of Participants With Adverse Events	DFP-DFP	Number of patients with at least one SAE	14		Participants
Number of Participants With Adverse Events	Placebo-DFP	Number of patients who withdrew due to an AE	2		Participants
Number of Participants With Adverse Events	DFP-DFP	Number of patients who withdrew due to an AE	1		Participants
Change in Score on the BAD Scale -- Comparison of Treatment Groups Over Each Study	Placebo-DFP	Change in BAD score over initial study	4.4	Standard Deviation 4.8	score on a scale
Change in Score on the BAD Scale -- Comparison of Treatment Groups Over Each Study	DFP-DFP	Change in BAD score over initial study	1.9	Standard Deviation 3.2	score on a scale
Change in Score on the BAD Scale -- Comparison of Treatment Groups Over Each Study	Placebo-DFP	Change in BAD score over extension study	1.4	Standard Deviation 3.7	score on a scale
Change in Score on the BAD Scale -- Comparison of Treatment Groups Over Each Study	DFP-DFP	Change in BAD score over extension study	1.4	Standard Deviation 2.4	score on a scale

1 to 10 of 20Source ClinicalTrials.gov

See all 20 results by group

Participant flow8

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Placebo-DFP	24
Overall Study	STARTED	DFP-DFP	44
Overall Study	Provided Post-baseline Efficacy Data	Placebo-DFP	19
Overall Study	Provided Post-baseline Efficacy Data	DFP-DFP	43
Overall Study	COMPLETED	Placebo-DFP	17
Overall Study	COMPLETED	DFP-DFP	38
Overall Study	NOT COMPLETED	Placebo-DFP	7
Overall Study	NOT COMPLETED	DFP-DFP	6

Source ClinicalTrials.gov

Adverse events104

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	Anemias	Blood and lymphatic system disorders	All Patients	12	68	27
other	Abdominal pain upper	Gastrointestinal disorders	All Patients	8	68	16
other	Diarrhoea	Gastrointestinal disorders	All Patients	4	68	6
other	Dysphagia	Gastrointestinal disorders	All Patients	8	68	8
other	Nausea	Gastrointestinal disorders	All Patients	5	68	5
other	Vomiting	Gastrointestinal disorders	All Patients	10	68	13
other	Condition aggravated	General disorders	All Patients	17	68	25
other	Pain	General disorders	All Patients	5	68	5
other	Pyrexia	General disorders	All Patients	20	68	64
other	Bronchitis	Infections and infestations	All Patients	6	68	30

1 to 10 of 104Source ClinicalTrials.gov

See all 104 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	All Patients	2	68
Total, other adverse events	All Patients	66	68
Total, serious adverse events	All Patients	33	68

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 comparisons0

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

None in this release.

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/NCT02174848

GET https://conductscience.org/api/trials/v1/NCT02174848/events?page=2

API reference For AI assistants