Trial Outcomes & Findings for MTX110 by Convection-Enhanced Delivery in Treating Participants With Newly-Diagnosed Diffuse Intrinsic Pontine Glioma (NCT NCT03566199)

Last Updated: 2022-02-25

Results Overview

Adverse events and clinically significant laboratory abnormalities which meet Grade 3, 4, or 5 criteria according to Common Terminology Criteria for Adverse Events (CTCAE) classified by investigators and treating physicians as related to study treatment (probable, possible, and definite) will be summarized by maximum intensity/grade. Adverse events will be graded according to CTCAE version 4.0.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

7 participants

Primary outcome timeframe

Up to 12 Months

Results posted on

2022-02-25

Participant Flow

All participants were only enrolled into Phase 1 of the clinical trial. The phase 2 expansion cohort was not activated for enrollment at behest of pharmaceutical supplier.

Each participant was permitted to have a dose-escalation assigned to subsequent rounds of CED treatment upon review of safety data at each round. A dose-escalation during treatment in a previous participant, determined the starting dosage assigned to the next participant. The first three participants enrolled received escalated doses in the second or third round of CED treatment.

Participant milestones

Participant milestones
Measure	Phase 1: Starting Dose Level 1 (MTX110) Participant received starting dose of single CED of 30 micrometer of MTX110 on 1 day (day 1); Total volume 3 mL. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Panobinostat Nanoparticle Formulation MTX110: Given IT Convection-Enhanced Delivery (CED): Undergo CED	Phase 1: Starting Dose Level 3 (MTX110) Participant received starting dose of repeated CED of 30 micrometer of MTX110 on n 2 consecutive days (days 1, 2); Total volume 8 mL (4 mL on each day). Panobinostat Nanoparticle Formulation MTX110: Given IT Convection-Enhanced Delivery (CED): Undergo CED	Phase 1: Starting Dose Level 5 (MTX110) Participant received starting dose of repeated CED of 30 micrometer of MTX110 on n 2 consecutive days (days 1, 2); Total volume 12 mL (6 mL on each day). Panobinostat Nanoparticle Formulation MTX110: Given IT Convection-Enhanced Delivery (CED): Undergo CED	Phase 1: Starting Dose Level 7 (MTX110) Participant received starting dose of repeated CED of 90 micrometer of MTX110 on n 2 consecutive days (days 1, 2); Total volume 12 mL (6 mL on each day). Panobinostat Nanoparticle Formulation MTX110: Given IT Convection-Enhanced Delivery (CED): Undergo CED
Overall Study STARTED	1	1	1	4
Overall Study COMPLETED	1	1	1	4
Overall Study NOT COMPLETED	0	0	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

MTX110 by Convection-Enhanced Delivery in Treating Participants With Newly-Diagnosed Diffuse Intrinsic Pontine Glioma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Phase 1: Treatment (MTX110) - All Participants n=7 Participants Participants receive panobinostat nanoparticle formulation MTX110 IT by CED infusion on day 1 or days 1 and 2 as determined by dose level. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Panobinostat Nanoparticle Formulation MTX110: Given IT Convection-Enhanced Delivery (CED): Undergo CED
Age, Customized	8 years n=99 Participants
Sex: Female, Male Female	1 Participants n=99 Participants
Sex: Female, Male Male	6 Participants n=99 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=99 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	5 Participants n=99 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	2 Participants n=99 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants
Race (NIH/OMB) Asian	1 Participants n=99 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants
Race (NIH/OMB) Black or African American	0 Participants n=99 Participants
Race (NIH/OMB) White	4 Participants n=99 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=99 Participants
Region of Enrollment United States	7 participants n=99 Participants

PRIMARY outcome

Timeframe: Up to 12 Months

Population: While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study

Outcome measures

Outcome measures
Measure	Phase 1: Treatment (MTX110) n=7 Participants Participants receive panobinostat nanoparticle formulation MTX110 IT by CED infusion on day 1 or days 1 and 2 as determined by dose level. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Panobinostat Nanoparticle Formulation MTX110: Given IT Convection-Enhanced Delivery (CED): Undergo CED
Proportion of Participants With Grade 3 or Higher, Treatment-related, Adverse Events Muscle weakness right-sided (Grade 3)	0.142 proportion of participants
Proportion of Participants With Grade 3 or Higher, Treatment-related, Adverse Events Stridor (Grade 3)	0.142 proportion of participants
Proportion of Participants With Grade 3 or Higher, Treatment-related, Adverse Events Vagus nerve disorder (Grade 3)	0.142 proportion of participants
Proportion of Participants With Grade 3 or Higher, Treatment-related, Adverse Events Gait disturbance (Grade 3)	0.142 proportion of participants
Proportion of Participants With Grade 3 or Higher, Treatment-related, Adverse Events Neutrophil count decreased (Grade 3)	0.142 proportion of participants

SECONDARY outcome

Timeframe: Up to 12 Months

Population: While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study

Overall survival is defined as the percentage of participants alive from time of diagnosis up to 12 months.

Outcome measures

Outcome measures
Measure	Phase 1: Treatment (MTX110) n=7 Participants Participants receive panobinostat nanoparticle formulation MTX110 IT by CED infusion on day 1 or days 1 and 2 as determined by dose level. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Panobinostat Nanoparticle Formulation MTX110: Given IT Convection-Enhanced Delivery (CED): Undergo CED
Overall Survival Rate (OS) at 12 Months	85.7 percentage of participants Interval 63.3 to 100.0

Adverse Events

Phase 1: Treatment (MTX110)

Serious events: 1 serious events

Other events: 7 other events

Deaths: 7 deaths

Serious adverse events

Serious adverse events
Measure	Phase 1: Treatment (MTX110) n=7 participants at risk Participants receive panobinostat nanoparticle formulation MTX110 IT by CED infusion on day 1 or days 1 and 2 as determined by dose level. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Panobinostat Nanoparticle Formulation MTX110: Given IT Convection-Enhanced Delivery (CED): Undergo CED
Musculoskeletal and connective tissue disorders Muscle weakness right-sided	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Nervous system disorders Vagus nerve disorder	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.

Other adverse events

Other adverse events
Measure	Phase 1: Treatment (MTX110) n=7 participants at risk Participants receive panobinostat nanoparticle formulation MTX110 IT by CED infusion on day 1 or days 1 and 2 as determined by dose level. Courses repeat every 4-8 weeks for up to 24 months in the absence of disease progression or unacceptable toxicity. Panobinostat Nanoparticle Formulation MTX110: Given IT Convection-Enhanced Delivery (CED): Undergo CED
General disorders Fatigue	57.1% 4/7 • Number of events 5 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
General disorders Gait disturbance	42.9% 3/7 • Number of events 5 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
General disorders Pain	42.9% 3/7 • Number of events 8 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
General disorders Irritability	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Nervous system disorders Headache	71.4% 5/7 • Number of events 8 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Nervous system disorders Ataxia	57.1% 4/7 • Number of events 17 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Nervous system disorders Dizziness	57.1% 4/7 • Number of events 5 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Nervous system disorders Abducens nerve disorder	42.9% 3/7 • Number of events 4 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Nervous system disorders Dysarthria	28.6% 2/7 • Number of events 7 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Nervous system disorders Facial nerve disorder	28.6% 2/7 • Number of events 2 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Nervous system disorders Nervous system disorders - Other	28.6% 2/7 • Number of events 4 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Nervous system disorders Paresthesia	28.6% 2/7 • Number of events 2 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Nervous system disorders Trigeminal nerve disorder	28.6% 2/7 • Number of events 2 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Nervous system disorders Vagus nerve disorder	28.6% 2/7 • Number of events 2 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Nervous system disorders Accessory nerve disorder	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Nervous system disorders Aphonia	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Nervous system disorders Dysgeusia	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Nervous system disorders Dysphasia	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Nervous system disorders Glossopharyngeal nerve disorder	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Nervous system disorders Tremor	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Investigations Electrocardiogram QT corrected interval prolonged	42.9% 3/7 • Number of events 4 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Investigations Lymphocyte count decreased	42.9% 3/7 • Number of events 9 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Investigations White blood cell decreased	42.9% 3/7 • Number of events 8 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Investigations Alanine aminotransferase increased	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Investigations Aspartate aminotransferase increased	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Investigations Blood bilirubin increased	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Investigations Hemoglobin increased	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Investigations Neutrophil count decreased	14.3% 1/7 • Number of events 5 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Investigations Weight loss	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Metabolism and nutrition disorders Hypokalemia	42.9% 3/7 • Number of events 5 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Metabolism and nutrition disorders Hypomagnesemia	28.6% 2/7 • Number of events 2 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Metabolism and nutrition disorders Hyperglycemia	14.3% 1/7 • Number of events 2 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Metabolism and nutrition disorders Hyperkalemia	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Metabolism and nutrition disorders Hypoglycemia	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Musculoskeletal and connective tissue disorders Muscle weakness right-sided	57.1% 4/7 • Number of events 7 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Musculoskeletal and connective tissue disorders Muscle weakness left-sided	28.6% 2/7 • Number of events 3 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Musculoskeletal and connective tissue disorders Neck pain	28.6% 2/7 • Number of events 2 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Gastrointestinal disorders Constipation	28.6% 2/7 • Number of events 2 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Gastrointestinal disorders Dysphagia	28.6% 2/7 • Number of events 3 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Gastrointestinal disorders Diarrhea	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Gastrointestinal disorders Vomiting	14.3% 1/7 • Number of events 2 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Respiratory, thoracic and mediastinal disorders Cough	42.9% 3/7 • Number of events 6 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Respiratory, thoracic and mediastinal disorders Nasal congestion	42.9% 3/7 • Number of events 5 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Respiratory, thoracic and mediastinal disorders Epistaxis	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Respiratory, thoracic and mediastinal disorders Hiccups	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Respiratory, thoracic and mediastinal disorders Stridor	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Skin and subcutaneous tissue disorders Skin ulceration	14.3% 1/7 • Number of events 2 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Skin and subcutaneous tissue disorders Urticaria	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders - Other	28.6% 2/7 • Number of events 3 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Vascular disorders Hematoma	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Vascular disorders Hypertension	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Vascular disorders Hypotension	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Cardiac disorders Sinus bradycardia	14.3% 1/7 • Number of events 3 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Cardiac disorders Sinus tachycardia	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Ear and labyrinth disorders External ear inflammation	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Ear and labyrinth disorders External ear pain	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Ear and labyrinth disorders Hearing impaired	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Eye disorders Blurred vision	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Eye disorders Corneal ulcer	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Eye disorders Eye disorders - Other	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Psychiatric disorders Agitation	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Psychiatric disorders Insomnia	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Psychiatric disorders Psychiatric disorders - Other	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Blood and lymphatic system disorders Anemia	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Immune system disorders Allergic reaction	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Infections and infestations Otitis externa	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Injury, poisoning and procedural complications Fall	14.3% 1/7 • Number of events 2 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Injury, poisoning and procedural complications Wound dehiscence	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Renal and urinary disorders Urinary incontinence	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Renal and urinary disorders Urinary tract pain	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.
Renal and urinary disorders Urinary urgency	14.3% 1/7 • Number of events 1 • Up to 24 months Additional adverse event and mortality data was collected for participants for up to 24 months following initial diagnosis. While individual participants were assigned to receive separate doses, participants are presented in one Arm/Group due to confidentiality concerns for this rare disease study.

Additional Information

Dr. Sabine Mueller, MD, PhD.

University of California, San Francisco

Phone: (415) 502-7301

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place