Trial Outcomes & Findings for A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants (NCT NCT04903288)

NCT ID: NCT04903288

Last Updated: 2026-04-24

Results Overview

HVA is a main metabolite of dopamine and HVA CSF levels are recognized as a proxy for dopamine levels in the brain.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE2

Target enrollment

13 participants

Primary outcome timeframe

Baseline (Day 1), Week 8

Results posted on

2026-04-24

Participant Flow

The study is ongoing. Only primary analysis results are reported. Final results will be reported after completion of study.

Participant milestones

Participant milestones
Measure	Eladocagene Exuparvovec Participants received eladocagene exuparvovec intraoperatively at 1.8×10\^11 vector genomes (vg) via SmartFlow® MR Compatible Ventricular Cannula in a single operative session. Participants received standard of care for their aromatic L-amino acid decarboxylase (AADC) deficiency during the study.
Trial Phase (8 Weeks) STARTED	13
Trial Phase (8 Weeks) Received at Least 1 Dose of Study Drug	13
Trial Phase (8 Weeks) COMPLETED	13
Trial Phase (8 Weeks) NOT COMPLETED	0
Extension Phase (48 Weeks) STARTED	13
Extension Phase (48 Weeks) COMPLETED	9
Extension Phase (48 Weeks) NOT COMPLETED	4
Long-Term Extension Phase (60 Months) STARTED	7
Long-Term Extension Phase (60 Months) COMPLETED	0
Long-Term Extension Phase (60 Months) NOT COMPLETED	7

Reasons for withdrawal

Reasons for withdrawal
Measure	Eladocagene Exuparvovec Participants received eladocagene exuparvovec intraoperatively at 1.8×10\^11 vector genomes (vg) via SmartFlow® MR Compatible Ventricular Cannula in a single operative session. Participants received standard of care for their aromatic L-amino acid decarboxylase (AADC) deficiency during the study.
Extension Phase (48 Weeks) Withdrawal by Subject	1
Extension Phase (48 Weeks) Ongoing in the Study	3
Long-Term Extension Phase (60 Months) Withdrawal by Subject	1
Long-Term Extension Phase (60 Months) Ongoing in the Study	6

Baseline Characteristics

A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Eladocagene Exuparvovec n=13 Participants Participants received eladocagene exuparvovec intraoperatively at 1.8×10\^11 vg via SmartFlow® MR Compatible Ventricular Cannula in a single operative session. Participants received standard of care for their AADC deficiency during the study.
Age, Continuous	45.2 months STANDARD_DEVIATION 29.48 • n=2 Participants
Sex: Female, Male Female	7 Participants n=2 Participants
Sex: Female, Male Male	6 Participants n=2 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=2 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	10 Participants n=2 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=2 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=2 Participants
Race (NIH/OMB) Asian	10 Participants n=2 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=2 Participants
Race (NIH/OMB) Black or African American	0 Participants n=2 Participants
Race (NIH/OMB) White	2 Participants n=2 Participants
Race (NIH/OMB) More than one race	0 Participants n=2 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=2 Participants
Homovanillic Acid (HVA) Metabolite Level	22.54 nanomoles (nmol)/liter (L) STANDARD_DEVIATION 32.34 • n=2 Participants

PRIMARY outcome

Timeframe: Baseline (Day 1), Week 8

Population: The safety population included all participants enrolled in the study who have received any amount of study drug. 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

HVA is a main metabolite of dopamine and HVA CSF levels are recognized as a proxy for dopamine levels in the brain.

Outcome measures

Outcome measures
Measure	Eladocagene Exuparvovec n=12 Participants Participants received eladocagene exuparvovec intraoperatively at 1.8×10\^11 vg via SmartFlow® MR Compatible Ventricular Cannula in a single operative session. Participants received standard of care for their AADC deficiency during the study.
Change From Baseline in HVA Metabolite Level at the End of the Trial Phase	29.53 nmol/L Standard Deviation 12.93

PRIMARY outcome

Timeframe: Baseline (Day 1) up to Week 8

Population: The safety population included all participants enrolled in the study who have received any amount of study drug.

An AE is any untoward medical occurrence associated with the use of a drug in humans, whether or not it is considered related to the drug. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease in a study participant who was administered gene therapy in this study. Number of participants with AEs related to the SmartFlow MR-compatible ventricular cannula used to administer eladocagene exuparvovec to pediatric participants at the end of Trial Phase (8 weeks after administration) are reported. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module."

Outcome measures

Outcome measures
Measure	Eladocagene Exuparvovec n=13 Participants Participants received eladocagene exuparvovec intraoperatively at 1.8×10\^11 vg via SmartFlow® MR Compatible Ventricular Cannula in a single operative session. Participants received standard of care for their AADC deficiency during the study.
Number of Participants With Adverse Events (AEs) Associated With the Surgical Administration of Eladocagene Exuparvovec Using the SmartFlow® MR-Compatible Ventricular Cannula	0 Participants

Serious events: 9 serious events

Other events: 13 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Eladocagene Exuparvovec n=13 participants at risk Participants received eladocagene exuparvovec intraoperatively at 1.8×10\^11 vg via SmartFlow® MR Compatible Ventricular Cannula in a single operative session. Participants received standard of care for their AADC deficiency during the study.
General disorders Pyrexia	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Infections and infestations Bacterial infection	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Infections and infestations COVID-19	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Infections and infestations Metapneumovirus pneumonia	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Infections and infestations Pneumonia	23.1% 3/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Infections and infestations Pneumonia viral	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Infections and infestations Urinary tract infection	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Nervous system disorders Dyskinesia	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Nervous system disorders Seizure	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Cardiac disorders Bradycardia	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Gastrointestinal disorders Vomiting	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Respiratory, thoracic and mediastinal disorders Bronchospasm	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Respiratory, thoracic and mediastinal disorders Respiratory arrest	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.

Other adverse events

Other adverse events
Measure	Eladocagene Exuparvovec n=13 participants at risk Participants received eladocagene exuparvovec intraoperatively at 1.8×10\^11 vg via SmartFlow® MR Compatible Ventricular Cannula in a single operative session. Participants received standard of care for their AADC deficiency during the study.
Investigations SARS-CoV-2 test positive	15.4% 2/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Investigations Viral test positive	15.4% 2/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Investigations White blood cell count increased	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Metabolism and nutrition disorders Decreased appetite	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Metabolism and nutrition disorders Hyperglycaemia	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Metabolism and nutrition disorders Hypocalcaemia	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Metabolism and nutrition disorders Hypoglycaemia	15.4% 2/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Metabolism and nutrition disorders Hypokalaemia	23.1% 3/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Metabolism and nutrition disorders Hypomagnesaemia	15.4% 2/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Metabolism and nutrition disorders Hypophosphataemia	23.1% 3/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Metabolism and nutrition disorders Polydipsia	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Nervous system disorders Dyskinesia	76.9% 10/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Nervous system disorders Myoclonus	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Psychiatric disorders Insomnia	23.1% 3/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Gastrointestinal disorders Gastrooesophageal reflux disease	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Gastrointestinal disorders Haematemesis	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Gastrointestinal disorders Salivary hypersecretion	23.1% 3/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Gastrointestinal disorders Stomatitis	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Gastrointestinal disorders Upper gastrointestinal haemorrhage	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Gastrointestinal disorders Vomiting	30.8% 4/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
General disorders Face oedema	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
General disorders Infusion site bruising	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
General disorders Injury associated with device	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
General disorders Puncture site pain	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
General disorders Pyrexia	76.9% 10/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Infections and infestations COVID-19	15.4% 2/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Infections and infestations Clostridium difficile infection	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Infections and infestations Conjunctivitis bacterial	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Infections and infestations Ear infection	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Infections and infestations Gastroenteritis viral	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Infections and infestations Impetigo	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Infections and infestations Influenza	23.1% 3/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Infections and infestations Nasopharyngitis	30.8% 4/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Infections and infestations Otitis media	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Infections and infestations Otitis media acute	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Infections and infestations Pneumonia	15.4% 2/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Infections and infestations Pneumonia aspiration	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Investigations Lymphocyte count increased	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Gastrointestinal disorders Gastrointestinal haemorrhage	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Blood and lymphatic system disorders Anaemia	30.8% 4/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Blood and lymphatic system disorders Lymphadenopathy	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Cardiac disorders Tachycardia	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Eye disorders Dry eye	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Eye disorders Eye swelling	15.4% 2/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Eye disorders Ocular hyperaemia	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Eye disorders Oculogyric crisis	15.4% 2/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Eye disorders Periorbital oedema	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Gastrointestinal disorders Anal fissure	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Gastrointestinal disorders Constipation	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Gastrointestinal disorders Diarrhoea	61.5% 8/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Gastrointestinal disorders Discoloured vomit	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Gastrointestinal disorders Dysphagia	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Gastrointestinal disorders Flatulence	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Infections and infestations Post viral fatigue syndrome	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Infections and infestations Rhinovirus infection	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Infections and infestations Tinea cruris	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Infections and infestations Upper respiratory tract infection	23.1% 3/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Infections and infestations Viral upper respiratory tract infection	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Injury, poisoning and procedural complications Contusion	15.4% 2/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Injury, poisoning and procedural complications Fall	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Injury, poisoning and procedural complications Forearm fracture	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Injury, poisoning and procedural complications Incision site haemorrhage	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Injury, poisoning and procedural complications Post procedural hypotension	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Injury, poisoning and procedural complications Scratch	15.4% 2/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Injury, poisoning and procedural complications Skin abrasion	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Injury, poisoning and procedural complications Skin pressure mark	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Injury, poisoning and procedural complications Stoma site discharge	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Investigations Alanine aminotransferase increased	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Investigations Aspartate aminotransferase increased	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Investigations Blood alkaline phosphatase increased	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Investigations Blood bicarbonate decreased	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Investigations Blood creatinine decreased	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Investigations Blood potassium decreased	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Investigations Eosinophil count increased	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Investigations Heart rate increased	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Respiratory, thoracic and mediastinal disorders Atelectasis	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Respiratory, thoracic and mediastinal disorders Cough	38.5% 5/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Respiratory, thoracic and mediastinal disorders Dyspnoea	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Respiratory, thoracic and mediastinal disorders Epistaxis	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Respiratory, thoracic and mediastinal disorders Hypoxia	15.4% 2/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Respiratory, thoracic and mediastinal disorders Increased upper airway secretion	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Respiratory, thoracic and mediastinal disorders Lung hyperinflation	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Respiratory, thoracic and mediastinal disorders Nasal congestion	23.1% 3/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Respiratory, thoracic and mediastinal disorders Rhinitis allergic	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	23.1% 3/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Respiratory, thoracic and mediastinal disorders Sinus congestion	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Respiratory, thoracic and mediastinal disorders Tachypnoea	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Respiratory, thoracic and mediastinal disorders Upper respiratory tract congestion	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Skin and subcutaneous tissue disorders Dermatitis atopic	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Skin and subcutaneous tissue disorders Dermatitis diaper	38.5% 5/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Skin and subcutaneous tissue disorders Eczema	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Skin and subcutaneous tissue disorders Rash	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Skin and subcutaneous tissue disorders Skin lesion inflammation	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Vascular disorders Cyanosis	7.7% 1/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.
Vascular disorders Hypotension	30.8% 4/13 • Baseline (Day 1) up to the interim data cut-off date (up to 897 days) The safety population included all participants enrolled in the study who have received any amount of study drug.

Additional Information

Patient Advocacy

PTC Therapeutics, Inc.

Phone: 1-866-562-4620

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the sponsor for review. The sponsor may consult with the PI to require changes to the communication or extend the embargo.
Publication restrictions are in place

Restriction type: OTHER