Trial · NCT03041116

Efficacy and Safety Study of Fosmetpantotenate (RE-024) in PKAN Participants

NCT03041116, Phase 3, Terminated, Travere Therapeutics, Inc.; results first posted 2020-12-29

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Efficacy and Safety Study of Fosmetpantotenate (RE-024) in PKAN Participants. Trials, record NCT03041116. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03041116

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Trial

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

Title: Efficacy and Safety Study of Fosmetpantotenate (RE-024) in PKAN Participants
NCT number: NCT03041116
Status: Terminated
Phases: Phase 3
Enrollment: 84 participants, actual
Lead sponsor: Travere Therapeutics, Inc.
Conditions: Pantothenate Kinase-Associated Neurodegeneration
Interventions: Fosmetpantotenate, Placebo
First posted: 2017-02-02
Last updated: 2021-01-26
Results first posted: 2020-12-29
Limitations and caveats: The study was terminated since fosmetpantotenate did not show statistically significant effects or clinical benefits during the double-blind period.

Source ClinicalTrials.gov

Registered outcomes3

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

List	Outcome	Time frame	Description
Primary	Change From Baseline In The Pantothenate Kinase-Associated Neurodegeneration-Activities of Daily Living (PKAN-ADL) Total Score To The End Of The 24-week Double-blind Period	Baseline (Day -1), Week 24	Change from baseline to Week 24 activities of daily living was assessed on the PKAN-ADL scale based on the Unified Parkinson's Disease Rating Scale (UPDRS) Part II. The PKAN-ADL is a validated measure of the participant's ability to complete ADL that are impacted by the diffuse motor manifestations of PKAN. It consists of 12 items related to activities of daily living, including eating, dressing, and walking. Each item has responses ranging from 0-4, with a higher value indicating greater disability in the given activity. To compute the total score, responses are summed across the 12 items. The available range of total scores on the PKAN-ADL scale was from 0 (no ADL affected) to 48 (ADL highly affected). The reported least square mean (LSM) was adjusted for baseline score and age group from the Type III analysis. A decrease in score indicates improvement in symptoms.
Primary	Number Of Participants With At Least 1 (≥1) Treatment-emergent Adverse Event (TEAE) And Treatment-emergent Serious Adverse Event (TESAE) During The 24-week Double-blind Period	From Screening until end of Week 24	An adverse event (AE) is any untoward medical occurrence associated with the use of the investigational product (IP; active or placebo) in a participant, without regard to possibility of causal relationship with IP. A serious adverse event (SAE) is an AE resulting in any of the following outcomes: death; initial or prolonged inpatient hospitalization; life threatening experience (immediate risk of death from AE); persistent or significant disability/incapacity; congenital anomaly/birth defect; other medically important serious medical events. The TEAEs in the double-blind period are defined as AEs that are new or are a worsening of an existing condition that begins from day of first dose of IP until day after last dose for double-blind treatment period.
Secondary	Absolute Change From Baseline In The UPDRS Part 3 (UPDRS-III) Total Score To The End Of The 24-week Double-blind Period	Baseline (Day -1), Week 24	The UPDRS is a comprehensive assessment of the burden and severity of signs and symptoms of Parkinsonism captured via systematic interview and neurological examination. The UPDRS-III is a standardized neurological examination that evaluates the performance of movements commonly affected in Parkinson's disease, PKAN, and other movement disorders. Part III of the UPDRS consists of 27 items, which correspond to 14 domains related to motor abilities such as tremor, stability, and bradykinesia. Each item has responses ranging from 0-4. To compute the UPDRS-III total score, responses are summed across the 27 items, and accordingly, range from 0-108. For domain totals, responses are summed across all of the items in a given domain (when domain corresponds to multiple items). An increase in score indicates greater disability. The reported LSM was adjusted for baseline score and age group from the Type III analysis.

Source ClinicalTrials.gov

Outcomes3

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change From Baseline In The Pantothenate Kinase-Associated Neurodegeneration-Activities of Daily Living (PKAN-ADL) Total Score To The End Of The 24-week Double-blind Period	Baseline (Day -1), Week 24	Double-blind Full Analysis Set: any participant who received at least 1 dose of study drug.	units on a scale	LEAST_SQUARES_MEAN
PRIMARY	Number Of Participants With At Least 1 (≥1) Treatment-emergent Adverse Event (TEAE) And Treatment-emergent Serious Adverse Event (TESAE) During The 24-week Double-blind Period	From Screening until end of Week 24	Safety Population: any participant who received at least 1 dose of study drug during any period of the study.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Absolute Change From Baseline In The UPDRS Part 3 (UPDRS-III) Total Score To The End Of The 24-week Double-blind Period	Baseline (Day -1), Week 24	Double-blind Full Analysis Set: any participant who received at least 1 dose of study drug.	units on a scale	MEAN

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	Spread	Units
Change From Baseline In The Pantothenate Kinase-Associated Neurodegeneration-Activities of Daily Living (PKAN-ADL) Total Score To The End Of The 24-week Double-blind Period	Fosmetpantotenate		-1.3	Standard Error 0.56	units on a scale
Change From Baseline In The Pantothenate Kinase-Associated Neurodegeneration-Activities of Daily Living (PKAN-ADL) Total Score To The End Of The 24-week Double-blind Period	Placebo		-1.3	Standard Error 0.58	units on a scale
Number Of Participants With At Least 1 (≥1) Treatment-emergent Adverse Event (TEAE) And Treatment-emergent Serious Adverse Event (TESAE) During The 24-week Double-blind Period	Fosmetpantotenate	≥1 TEAE	34		Participants
Number Of Participants With At Least 1 (≥1) Treatment-emergent Adverse Event (TEAE) And Treatment-emergent Serious Adverse Event (TESAE) During The 24-week Double-blind Period	Placebo	≥1 TEAE	35		Participants
Number Of Participants With At Least 1 (≥1) Treatment-emergent Adverse Event (TEAE) And Treatment-emergent Serious Adverse Event (TESAE) During The 24-week Double-blind Period	Fosmetpantotenate	≥1 TESAE	8		Participants
Number Of Participants With At Least 1 (≥1) Treatment-emergent Adverse Event (TEAE) And Treatment-emergent Serious Adverse Event (TESAE) During The 24-week Double-blind Period	Placebo	≥1 TESAE	6		Participants
Absolute Change From Baseline In The UPDRS Part 3 (UPDRS-III) Total Score To The End Of The 24-week Double-blind Period	Fosmetpantotenate		0.7	Standard Deviation 5.25	units on a scale
Absolute Change From Baseline In The UPDRS Part 3 (UPDRS-III) Total Score To The End Of The 24-week Double-blind Period	Placebo		-1.0	Standard Deviation 6.81	units on a scale

Source ClinicalTrials.gov

Participant flow16

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

Period	Milestone	Group	Participants
Double-blind	STARTED	Fosmetpantotenate	41
Double-blind	STARTED	Placebo	43
Double-blind	Received at Least 1 Dose of Study Drug	Fosmetpantotenate	41
Double-blind	Received at Least 1 Dose of Study Drug	Placebo	43
Double-blind	COMPLETED	Fosmetpantotenate	41
Double-blind	COMPLETED	Placebo	37
Double-blind	NOT COMPLETED	Fosmetpantotenate	0
Double-blind	NOT COMPLETED	Placebo	6
Open-label	STARTED	Fosmetpantotenate	41
Open-label	STARTED	Placebo	37

1 to 10 of 16Source ClinicalTrials.gov

See all 16 participant flow

Adverse events1,374

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	Hypersensitivity	Immune system disorders	Fosmetpantotenate	1	41	1
other	Hypersensitivity	Immune system disorders	Placebo	0	37	0
other	Hypersensitivity	Immune system disorders	Fosmetpantotenate During Double-blind Period	0	41	0
other	Hypersensitivity	Immune system disorders	Placebo During Double-blind Period	0	43	0
other	Hypersensitivity	Immune system disorders	Fosmetpantotenate During Open-label Period	1	41	1
other	Hypersensitivity	Immune system disorders	Placebo During Open-label Period	0	37	0
other	Drug hypersensitivity	Immune system disorders	Fosmetpantotenate	0	41	0
other	Drug hypersensitivity	Immune system disorders	Placebo	0	37	0
other	Drug hypersensitivity	Immune system disorders	Fosmetpantotenate During Double-blind Period	0	41	0
other	Drug hypersensitivity	Immune system disorders	Placebo During Double-blind Period	1	43	1

1 to 10 of 1,374Source ClinicalTrials.gov

See all 1,374 adverse events

Adverse event totals18

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Fosmetpantotenate	1	41
Total, all-cause mortality	Placebo	2	37
Total, all-cause mortality	Fosmetpantotenate During Double-blind Period	0	41
Total, all-cause mortality	Placebo During Double-blind Period	2	43
Total, all-cause mortality	Fosmetpantotenate During Open-label Period	1	41
Total, all-cause mortality	Placebo During Open-label Period	0	37
Total, other adverse events	Fosmetpantotenate	35	41
Total, other adverse events	Placebo	25	37
Total, other adverse events	Fosmetpantotenate During Double-blind Period	34	41
Total, other adverse events	Placebo During Double-blind Period	35	43

1 to 10 of 18Source ClinicalTrials.gov

See all 18 adverse event totals

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
31055958	RESULT

Source PubMed

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/NCT03041116

GET https://conductscience.org/api/trials/v1/NCT03041116/events?page=2

API reference For AI assistants