Trial Outcomes & Findings for An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease (NCT NCT05174039)

NCT ID: NCT05174039

Last Updated: 2025-09-09

Results Overview

Number of treatment-emergent adverse events (TEAEs) assessed at all visits and phone calls, with severity classified according to CTCAE v5.0

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 6 participants
• Primary outcome timeframe: 78 weeks
• Results posted on: 2025-09-09

Participant Flow

Participants were recruited at hospital. The first participant was recruited on 10 March 2022 and the last participant was recruited on 08 September 2022.

Participants had their first enrolment visit to determine their eligibility, then a second visit within the next 42 days to confirm eligibility and perform baseline efficacy assessments. If eligibility was confirmed at the second visit, they started treatment within the next 15 days after this visit.

Participant milestones

Measure	Oral Miglustat The proposed dosing regimen was daily oral miglustat (MTD, up to 200 mg TID) Miglustat 100 mg Oral Capsule: participants initiated miglustat at Week 1 and dosing was to be escalated until 600mg/d. If a participant had not reached the maximum dose (600 mg/d) by Week 8, the Week 8 dose was to be participant's MTD.
Overall Study STARTED	6
Overall Study COMPLETED	6
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	Oral Miglustat n=6 Participants The proposed dosing regimen is daily oral miglustat (MTD, up to 200 mg TID) Miglustat 100Mg Oral Capsule: Participants initiated miglustat at Week 1 and dosing was to be escalated until 600mg/d. If a participant had not reached the maximum dose (600 mg/d) by Week 8, the Week 8 dose will be participant's MTD.
Age, Continuous	18.8 years STANDARD_DEVIATION 0.98 • n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	6 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	6 participants n=5 Participants
Body weight	70.45 kilogram STANDARD_DEVIATION 6.70 • n=5 Participants
Body Mass Index	23.63 kilogram/meter square STANDARD_DEVIATION 4.74 • n=5 Participants

PRIMARY outcome

Timeframe: 78 weeks

Number of treatment-emergent adverse events (TEAEs) assessed at all visits and phone calls, with severity classified according to CTCAE v5.0

Outcome measures

Measure	Oral Miglustat n=6 Participants The proposed dosing regimen is daily oral miglustat (MTD, up to 200 mg TID) Miglustat 100Mg Oral Capsule: Subjects will initiate miglustat at Week 1 and dosing will be escalated until 600mg/d. If a subject has not reached the maximum dose (600 mg/d) by Week 8, the Week 8 dose will be subject's MTD.
Number of Treatment-emergent Adverse Events.	6 Participants

SECONDARY outcome

Timeframe: 8 weeks

Population: Descriptive statistical analyses were performed for miglustat plasma concentration data.

Maximum plasma concentration Cmax

Outcome measures

Measure	Oral Miglustat n=6 Participants The proposed dosing regimen is daily oral miglustat (MTD, up to 200 mg TID) Miglustat 100Mg Oral Capsule: Subjects will initiate miglustat at Week 1 and dosing will be escalated until 600mg/d. If a subject has not reached the maximum dose (600 mg/d) by Week 8, the Week 8 dose will be subject's MTD.
Miglustat Pharmacokinetic (PK) Parameter Cmax	2870 ng/ml Geometric Coefficient of Variation 35.2

SECONDARY outcome

Timeframe: 8 weeks

Population: 6

Time of Maximum concentration observed T max

Outcome measures

Measure	Oral Miglustat n=6 Participants The proposed dosing regimen is daily oral miglustat (MTD, up to 200 mg TID) Miglustat 100Mg Oral Capsule: Subjects will initiate miglustat at Week 1 and dosing will be escalated until 600mg/d. If a subject has not reached the maximum dose (600 mg/d) by Week 8, the Week 8 dose will be subject's MTD.
Miglustat PK Parameter Tmax	3.09 hour Geometric Coefficient of Variation 44.1

SECONDARY outcome

Timeframe: 8 weeks

Population: 6

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.

Outcome measures

Measure	Oral Miglustat n=6 Participants The proposed dosing regimen is daily oral miglustat (MTD, up to 200 mg TID) Miglustat 100Mg Oral Capsule: Subjects will initiate miglustat at Week 1 and dosing will be escalated until 600mg/d. If a subject has not reached the maximum dose (600 mg/d) by Week 8, the Week 8 dose will be subject's MTD.
Miglustat PK Parameter Area Under Curve (AUC)	19000 h*ng/ml Geometric Coefficient of Variation 40.3

SECONDARY outcome

Timeframe: 8 weeks

Population: 6

Miglustat elimination half life T1/2

Outcome measures

Measure	Oral Miglustat n=6 Participants The proposed dosing regimen is daily oral miglustat (MTD, up to 200 mg TID) Miglustat 100Mg Oral Capsule: Subjects will initiate miglustat at Week 1 and dosing will be escalated until 600mg/d. If a subject has not reached the maximum dose (600 mg/d) by Week 8, the Week 8 dose will be subject's MTD.
Miglustat PK Parameter T1/2	7.59 hour Geometric Coefficient of Variation 19.0

SECONDARY outcome

Timeframe: 78 weeks

Population: 6

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.

Outcome measures

Measure	Oral Miglustat n=6 Participants The proposed dosing regimen is daily oral miglustat (MTD, up to 200 mg TID) Miglustat 100Mg Oral Capsule: Subjects will initiate miglustat at Week 1 and dosing will be escalated until 600mg/d. If a subject has not reached the maximum dose (600 mg/d) by Week 8, the Week 8 dose will be subject's MTD.
Clinical Efficacy Based on Unified Batten Disease Rating Scale Subscores	1.76 units on a scale Standard Deviation 4.96

SECONDARY outcome

Timeframe: 78 weeks

Population: 6

Seizure frequency were to be assessed using a seizure diary

Outcome measures

Measure	Oral Miglustat n=6 Participants The proposed dosing regimen is daily oral miglustat (MTD, up to 200 mg TID) Miglustat 100Mg Oral Capsule: Subjects will initiate miglustat at Week 1 and dosing will be escalated until 600mg/d. If a subject has not reached the maximum dose (600 mg/d) by Week 8, the Week 8 dose will be subject's MTD.
Clinical Efficacy With the Seizure Frequency	1.83 number of seizures / 3 months Standard Deviation 3.13

Adverse Events

Oral Miglustat

Serious events: 1 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Measure	Oral Miglustat n=6 participants at risk The proposed dosing regimen is daily oral miglustat (MTD, up to 200 mg TID) Miglustat 100Mg Oral Capsule: Subjects will initiate miglustat at Week 1 and dosing will be escalated until 600mg/d. If a subject has not reached the maximum dose (600 mg/d) by Week 8, the Week 8 dose will be subject's MTD.
Gastrointestinal disorders Dysphagia	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Infections and infestations Medical device site cellulitis	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.

Other adverse events

Measure	Oral Miglustat n=6 participants at risk The proposed dosing regimen is daily oral miglustat (MTD, up to 200 mg TID) Miglustat 100Mg Oral Capsule: Subjects will initiate miglustat at Week 1 and dosing will be escalated until 600mg/d. If a subject has not reached the maximum dose (600 mg/d) by Week 8, the Week 8 dose will be subject's MTD.
Gastrointestinal disorders Diarrhea	100.0% 6/6 • Number of events 19 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Investigations Weight decreased	100.0% 6/6 • Number of events 17 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Nervous system disorders Tremor	83.3% 5/6 • Number of events 9 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
General disorders Influenza-like illness	83.3% 5/6 • Number of events 6 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Nervous system disorders Seizure	66.7% 4/6 • Number of events 78 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Psychiatric disorders Anxiety	66.7% 4/6 • Number of events 4 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Investigations Platelet count decreased	50.0% 3/6 • Number of events 6 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Infections and infestations Upper respiratory tract infection	50.0% 3/6 • Number of events 5 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Gastrointestinal disorders Flatulence	50.0% 3/6 • Number of events 4 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Gastrointestinal disorders Anal incontinence	50.0% 3/6 • Number of events 4 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Musculoskeletal and connective tissue disorders Muscular weakness	50.0% 3/6 • Number of events 3 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Nervous system disorders Lethargy	50.0% 3/6 • Number of events 3 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Metabolism and nutrition disorders Decreased appetite	33.3% 2/6 • Number of events 5 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Renal and urinary disorders Urinary incontinence	33.3% 2/6 • Number of events 4 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Nervous system disorders Drooling	33.3% 2/6 • Number of events 3 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Nervous system disorders Myoclonus	33.3% 2/6 • Number of events 2 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
General disorders Fatigue	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Infections and infestations Pneumonia	33.3% 2/6 • Number of events 2 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Musculoskeletal and connective tissue disorders arthralgia	33.3% 2/6 • Number of events 2 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Musculoskeletal and connective tissue disorders Joint range of motion decreased	33.3% 2/6 • Number of events 2 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Nervous system disorders Generalized tonic-clonic seizures	16.7% 1/6 • Number of events 4 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Nervous system disorders Nervous system disorder	16.7% 1/6 • Number of events 4 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Gastrointestinal disorders Dysphagia	16.7% 1/6 • Number of events 3 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Gastrointestinal disorders Abdominal pain	16.7% 1/6 • Number of events 3 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Gastrointestinal disorders Constipation	16.7% 1/6 • Number of events 2 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Gastrointestinal disorders Vomiting	16.7% 1/6 • Number of events 2 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Investigations Blood creatinine phosphokinase increased	16.7% 1/6 • Number of events 2 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Nervous system disorders Dysarthria	16.7% 1/6 • Number of events 2 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Respiratory, thoracic and mediastinal disorders Epistaxis	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Gastrointestinal disorders Intestinal dilatation	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Gastrointestinal disorders Abdominal pain upper	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Gastrointestinal disorders Gastrointestinal haemorrage	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Gastrointestinal disorders Haematochezia	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Investigations Weight increase	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Investigations Electrocardiogram P Wave abnormal	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Investigations Vitamine B12 increased	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Nervous system disorders Hemiparesis	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Nervous system disorders Loss of proprioception	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Nervous system disorders Somnolence	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Nervous system disorders Partial seizures	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Infections and infestations Ear infection	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Infections and infestations Infected bite	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Infections and infestations Nail infection	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Infections and infestations Otitis media	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Infections and infestations Rhinitis	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Infections and infestations COVID-19	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Infections and infestations Viral infection	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Musculoskeletal and connective tissue disorders Back pain	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Musculoskeletal and connective tissue disorders Weight bearing difficulty	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Musculoskeletal and connective tissue disorders Scoliosis	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Psychiatric disorders Confusional state	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Psychiatric disorders Irritability	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Respiratory, thoracic and mediastinal disorders Cough	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Respiratory, thoracic and mediastinal disorders Dyspnoea	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Respiratory, thoracic and mediastinal disorders Aphonia	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Musculoskeletal and connective tissue disorders Lung opacity	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Respiratory, thoracic and mediastinal disorders Atelectasis	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Cardiac disorders Conduction disorder	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Cardiac disorders Left ventricular hypertrophy	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Cardiac disorders Pericardial effusion	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Ear and labyrinth disorders Ear pain	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Ear and labyrinth disorders Hypoacousis	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Ear and labyrinth disorders Vestibular disorder	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Injury, poisoning and procedural complications Fall	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Injury, poisoning and procedural complications Wound	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Metabolism and nutrition disorders Hyperkalaemia	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Metabolism and nutrition disorders Hypokalaemia	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Skin and subcutaneous tissue disorders Vancomycin infusion reaction	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Skin and subcutaneous tissue disorders Night sweats	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Eye disorders Cataract	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.
Eye disorders Eye disorder	16.7% 1/6 • Number of events 1 • Total treatment duration ranged between 510 days and 804 days, during which adverse events were collected until participants could switch to an expanded access program. The adverse event reporting focus on treatment emergent adverse events.

Additional Information

Chief Medical Officer

Theranexus

Phone: 0688941976

Email: marie.sebille@theranexus.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place