Trial · NCT05174039

An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease

NCT05174039, Phase 1, Phase 2, Completed, Beyond Batten Disease Foundation; results first posted 2025-09-09

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease. Trials, record NCT05174039. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05174039

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Trial

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

Title: An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease
NCT number: NCT05174039
Status: Completed
Phases: Phase 1, Phase 2
Enrollment: 6 participants, actual
Lead sponsor: Beyond Batten Disease Foundation
Conditions: Batten Disease
Interventions: Miglustat 100 milligrams (mg) Oral Capsule
First posted: 2021-12-30
Last updated: 2025-09-09
Results first posted: 2025-09-09

Source ClinicalTrials.gov

Registered outcomes7

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

List	Outcome	Time frame	Description
Primary	Number of Treatment-emergent Adverse Events.	78 weeks	Number of treatment-emergent adverse events (TEAEs) assessed at all visits and phone calls, with severity classified according to CTCAE v5.0
Secondary	Miglustat Pharmacokinetic (PK) Parameter Cmax	8 weeks	Maximum plasma concentration Cmax
Secondary	Miglustat PK Parameter Tmax	8 weeks	Time of Maximum concentration observed T max
Secondary	Miglustat PK Parameter Area Under Curve (AUC)	8 weeks	Area under the plasma concentration versus time curve Area Under Curve from pre-administration to 8 hours post-administration (AUC0-8hour). Blood draws were taken at the following time points: pre-miglustat dose (0), 1, 2, 2.5, 3, 4, 6, and 8 hours after 8 weeks of treatment.
Secondary	Miglustat PK Parameter T1/2	8 weeks	Miglustat elimination half life T1/2
Secondary	Clinical Efficacy Based on Unified Batten Disease Rating Scale Subscores	78 weeks	Change from baseline of the modified Unified Batten Disease Rating Scale (UBDRS) Physical Assessment subscale (score from 0 to 112), specifically developed to assess motor symptoms in subjects with Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) disease. Higher scores indicate more severe disease.
Secondary	Clinical Efficacy With the Seizure Frequency	78 weeks	Seizure frequency were to be assessed using a seizure diary

Source ClinicalTrials.gov

Outcomes7

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Number of Treatment-emergent Adverse Events.	78 weeks		Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Miglustat Pharmacokinetic (PK) Parameter Cmax	8 weeks	Descriptive statistical analyses were performed for miglustat plasma concentration data.	ng/ml	GEOMETRIC_MEAN
SECONDARY	Miglustat PK Parameter Tmax	8 weeks	6	hour	GEOMETRIC_MEAN
SECONDARY	Miglustat PK Parameter Area Under Curve (AUC)	8 weeks	6	h*ng/ml	GEOMETRIC_MEAN
SECONDARY	Miglustat PK Parameter T1/2	8 weeks	6	hour	GEOMETRIC_MEAN
SECONDARY	Clinical Efficacy Based on Unified Batten Disease Rating Scale Subscores	78 weeks	6	units on a scale	MEAN
SECONDARY	Clinical Efficacy With the Seizure Frequency	78 weeks	6	number of seizures / 3 months	MEAN

Source ClinicalTrials.gov

Results by group7

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

Outcome	Group	Value	Spread	Units
Number of Treatment-emergent Adverse Events.	Oral Miglustat	6		Participants
Miglustat Pharmacokinetic (PK) Parameter Cmax	Oral Miglustat	2870	Geometric Coefficient of Variation 35.2	ng/ml
Miglustat PK Parameter Tmax	Oral Miglustat	3.09	Geometric Coefficient of Variation 44.1	hour
Miglustat PK Parameter Area Under Curve (AUC)	Oral Miglustat	19000	Geometric Coefficient of Variation 40.3	h*ng/ml
Miglustat PK Parameter T1/2	Oral Miglustat	7.59	Geometric Coefficient of Variation 19.0	hour
Clinical Efficacy Based on Unified Batten Disease Rating Scale Subscores	Oral Miglustat	1.76	Standard Deviation 4.96	units on a scale
Clinical Efficacy With the Seizure Frequency	Oral Miglustat	1.83	Standard Deviation 3.13	number of seizures / 3 months

Source ClinicalTrials.gov

Participant flow3

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Oral Miglustat	6
Overall Study	COMPLETED	Oral Miglustat	6
Overall Study	NOT COMPLETED	Oral Miglustat	0

Source ClinicalTrials.gov

Adverse events75

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	Diarrhea	Gastrointestinal disorders	Oral Miglustat	6	6	19
other	Weight decreased	Investigations	Oral Miglustat	6	6	17
other	Tremor	Nervous system disorders	Oral Miglustat	5	6	9
other	Influenza-like illness	General disorders	Oral Miglustat	5	6	6
other	Seizure	Nervous system disorders	Oral Miglustat	4	6	78
other	Anxiety	Psychiatric disorders	Oral Miglustat	4	6	4
other	Platelet count decreased	Investigations	Oral Miglustat	3	6	6
other	Upper respiratory tract infection	Infections and infestations	Oral Miglustat	3	6	5
other	Flatulence	Gastrointestinal disorders	Oral Miglustat	3	6	4
other	Anal incontinence	Gastrointestinal disorders	Oral Miglustat	3	6	4

1 to 10 of 75Source ClinicalTrials.gov

See all 75 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Oral Miglustat	0	6
Total, other adverse events	Oral Miglustat	6	6
Total, serious adverse events	Oral Miglustat	1	6

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/NCT05174039

GET https://conductscience.org/api/trials/v1/NCT05174039/events?page=2

API reference For AI assistants