TrialPanorama/TrialPanorama-database huggingface dataset:
Primary endpoint: SF-36 Mental Health Summary Scale
Endpoint domain: Life impact
Endpoint subdomain: Emotional functioning/wellbeing
Primary endpoint: Health Services Utilization
Endpoint domain: Resource use
Endpoint subdomain: Other resource use
Primary endpoint: Diluted thrombin time (DTT)
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: anti FXa-activity
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Change From Baseline in Total Lesion Count at Week 12
Endpoint domain: Physiological/clinical
Endpoint subdomain: Skin and subcutaneous tissue outcomes
Primary endpoint: Change From Baseline in WOMAC Pain Subscale
Endpoint domain: Life impact
Endpoint subdomain: Pain
Primary endpoint: Change From Baseline in WOMAC Physical Function Subscale
Endpoint domain: Life impact
Endpoint subdomain: Physical functioning
Primary endpoint: Change From Baseline in Patient's Global Assessment of Osteoarthritis
Endpoint domain: Life impact
Endpoint subdomain: Perceived health status
Primary endpoint: Patient Discomfort VAS
Endpoint domain: Life impact
Endpoint subdomain: Emotional functioning/wellbeing
Primary endpoint: Maximum Self-reported Pain Score
Endpoint domain: Life impact
Endpoint subdomain: Emotional functioning/wellbeing
Primary endpoint: Maximum tolerated dose assessed by the number of subjects with dose-limiting toxicities (DLTs)
Endpoint domain: Adverse events/effects
Primary endpoint: Number of medication-related recommendations
Endpoint domain: Resource use
Endpoint subdomain: Need for further intervention
Primary endpoint: Continuous Complete Remission
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Patients discharged on day of surgery
Endpoint domain: Resource use
Endpoint subdomain: Hospital
Primary endpoint: Liver-related disease progression
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Retention of the Crown
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Gingival Status
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Parent's Esthetic Satisfaction
Endpoint domain: Life impact
Endpoint subdomain: Perceived health status
Primary endpoint: Frequency of BRCA1/2 mutations
Endpoint domain: Physiological/clinical
Endpoint subdomain: Neoplasms and related outcomes
Primary endpoint: Tumor treatment response
Endpoint domain: Physiological/clinical
Endpoint subdomain: Neoplasms and related outcomes
Primary endpoint: Judgement of learning
Endpoint domain: Life impact
Endpoint subdomain: Cognitive functioning
Primary endpoint: Clinical remission by a decline in Crohns disease activity index to <151
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Clinical improvement using inflammatory bowel disease quality of life questionnaire
Endpoint domain: Life impact
Endpoint subdomain: Global quality of life
Primary endpoint: Endoscopic improvement using Crohn's disease endoscopic activity index
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Complete clearance of all warts
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Severe local reaction during the treatment period
Endpoint domain: Adverse events/effects
Primary endpoint: Insulin Sensitivity Measure
Endpoint domain: Physiological/clinical
Endpoint subdomain: Metabolic and nutritional outcomes
Primary endpoint: change from Fugl-Meyer assessment
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: change from The Jebsen-Taylor
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: change from The Motor Activity Long (MAL)
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: change from The Box and Blocks test (TBB)
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Proportion of patients meeting inclusion criteria
Endpoint domain: Resource use
Endpoint subdomain: Other resource use
Primary endpoint: Proportion of patients who successfully schedule video visits
Endpoint domain: Resource use
Endpoint subdomain: Other resource use
Primary endpoint: Proportion of patients who successfully complete video visit
Endpoint domain: Resource use
Endpoint subdomain: Other resource use
Primary endpoint: Number of Patients Where TraceIT Tissue Marker Placement Achieved
Endpoint domain: Physiological/clinical
Endpoint subdomain: Surgical and medical procedures outcomes
Primary endpoint: Recurrence of pilonidal sinus
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: WOMAC 3.1 Index total
Endpoint domain: Life impact
Endpoint subdomain: Physical functioning
Primary endpoint: Pain assessment in non-communicating children postoperatively
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: rate of abdominal complications
Endpoint domain: Adverse events/effects
Primary endpoint: peri-implant plaque
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: peri-implant bleeding
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: peri-implant probing depth
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: overdenture retention
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Procedure pain
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Subjective units of distress (SUD)
Endpoint domain: Life impact
Endpoint subdomain: Emotional functioning/wellbeing
Primary endpoint: Number of Participants With a Clinically Significant Infection
Endpoint domain: Adverse events/effects
Primary endpoint: Cerebral gray matter volume
Endpoint domain: Physiological/clinical
Endpoint subdomain: Nervous system outcomes
Primary endpoint: Mean Score on the Scale for the Assessment of Negative Symptoms (SANS)
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Mean Change of Z-scores on the Brief Assessment of Cognition in Schizophrenia (BACS)
Endpoint domain: Life impact
Endpoint subdomain: Cognitive functioning
Primary endpoint: Nature of the mass
Endpoint domain: Physiological/clinical
Endpoint subdomain: Neoplasms and related outcomes
Primary endpoint: Caries status
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Cure of Head Lice
Endpoint domain: Physiological/clinical
Endpoint subdomain: Infections and infestations outcomes
Primary endpoint: Change From Baseline in Rhinoconjunctivitis Symptoms Score
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Change From Baseline in Rhinoconjunctivitis Medication Score
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Mean Change in Total CAPS-5 Score
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: 28-days mortality rate
Endpoint domain: Mortality/survival
Primary endpoint: Change in LENA Adult Word Count score
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Proficiency of laparoscopic suturing
Endpoint domain: Physiological/clinical
Endpoint subdomain: Surgical and medical procedures outcomes
Primary endpoint: Pain in preterm infants
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Best Overall Objective Response or Complete Response/Partial Response (CR/PR)
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Improvement in executive function deficits
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Target vessel failure (TVF) rate
Endpoint domain: Physiological/clinical
Endpoint subdomain: Cardiovascular outcomes
Primary endpoint: F3-F2 (Hz) accuracy of /r/ sounds
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Minimum effective concentration of bupivacaine
Endpoint domain: Physiological/clinical
Endpoint subdomain: Surgical and medical procedures outcomes
Primary endpoint: Size of Fibroids
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Proportion of participants achieving ACR20 response
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Volume of anesthetic drugs
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Pain release
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Breastfeeding Status
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Serotype distribution of invasive S. pneumoniae isolates
Endpoint domain: Physiological/clinical
Endpoint subdomain: Infections and infestations outcomes
Primary endpoint: Cmax of PF 04995274
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Cmax of PF 05082547
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Menorrhagia severity
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Proportion of Subjects Who Will Develop Mild Cognitive Impairment
Endpoint domain: Physiological/clinical
Endpoint subdomain: Nervous system outcomes
Primary endpoint: neonatal infection rate
Endpoint domain: Adverse events/effects
Primary endpoint: maternal infection rate
Endpoint domain: Adverse events/effects
Primary endpoint: Change in low back pain Disability
Endpoint domain: Life impact
Endpoint subdomain: Physical functioning
Primary endpoint: Patterns of disease progression
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: CHF composite response
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Change From Baseline in Bone Marrow Testosterone
Endpoint domain: Physiological/clinical
Endpoint subdomain: Endocrine outcomes
Primary endpoint: Change From Baseline in Bone Marrow Dihydrotestosterone
Endpoint domain: Physiological/clinical
Endpoint subdomain: Endocrine outcomes
Primary endpoint: Change in Baseline assessment of motor impairment
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Change from baseline assessment of Timed up and go
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Change from baseline assessment of 10 Meter Walk Test
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: self-reported 7-day point prevalence of abstinence
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: self-reported reduction of ≥ 50% in cigarette consumption
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Technical Success of tibial pedal arterial access
Endpoint domain: Physiological/clinical
Endpoint subdomain: Surgical and medical procedures outcomes
Primary endpoint: Change in Oxygenation (P/F Ratio)
Endpoint domain: Physiological/clinical
Endpoint subdomain: Respiratory outcomes
Primary endpoint: Absence of Prostate Cancer on Biopsy
Endpoint domain: Physiological/clinical
Endpoint subdomain: Neoplasms and related outcomes
Primary endpoint: Change in Vulvar Pain Scale
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Change in Dyspareunia Questionnaire
Endpoint domain: Life impact
Endpoint subdomain: Pain
Primary endpoint: Change in Number of Reversible Defects
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Polymyxin B plasma concentrations
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Proportion of Patients Who Achieve a Confirmed Response
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: CB1 protein expression
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: CB2 protein expression
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Gene Expression of the endocannabinoid system components
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: thoracoabdominal volume
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Sperm concentration per milliliter of seminal fluid
Endpoint domain: Physiological/clinical
Endpoint subdomain: Reproductive system and breast outcomes
Primary endpoint: Proportion of Sperm motility
Endpoint domain: Physiological/clinical
Endpoint subdomain: Reproductive system and breast outcomes
Primary endpoint: Proportion of Sperm morphology
Endpoint domain: Physiological/clinical
Endpoint subdomain: Reproductive system and breast outcomes
Primary endpoint: change in functional network connectivity
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Attention and concentration test
Endpoint domain: Physiological/clinical
Endpoint subdomain: Nervous system outcomes
Primary endpoint: Sympathetic activation
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Area Under the Curve From 0-48 (AUC0-48) of Aprepitant
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Maximum Plasma Concentration (Cmax) of Aprepitant
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Clinical Scoring System for Vulvar Lichen Sclerosus
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Follow-up adherence
Endpoint domain: Resource use
Endpoint subdomain: Need for further intervention
Primary endpoint: Change in Vegetable Intake
Endpoint domain: Physiological/clinical
Endpoint subdomain: Metabolic and nutritional outcomes
Primary endpoint: Change in Total Food Intake
Endpoint domain: Physiological/clinical
Endpoint subdomain: Metabolic and nutritional outcomes
Primary endpoint: Change in Meal Energy Density
Endpoint domain: Physiological/clinical
Endpoint subdomain: Metabolic and nutritional outcomes
Primary endpoint: Incidence of HCV reinfection
Endpoint domain: Physiological/clinical
Endpoint subdomain: Infections and infestations outcomes
Primary endpoint: Detection rate of invasive breast cancers
Endpoint domain: Physiological/clinical
Endpoint subdomain: Neoplasms and related outcomes
Primary endpoint: Cumulative 24 months incidence of interval cancers
Endpoint domain: Physiological/clinical
Endpoint subdomain: Neoplasms and related outcomes
Primary endpoint: successful identification of the SLN
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: accuracy of SLN identification
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: proportion of false negative SLN results
Endpoint domain: Adverse events/effects
Primary endpoint: Development of Ventilator Acquired Pneumonia
Endpoint domain: Adverse events/effects
Primary endpoint: Number of Subjects Healed at Day 14
Endpoint domain: Mortality/survival
Primary endpoint: Proportion of Subjects Healed at Day 21
Endpoint domain: Mortality/survival
Primary endpoint: Platelet Reactions Units
Endpoint domain: Physiological/clinical
Endpoint subdomain: Blood and lymphatic system outcomes
Primary endpoint: Morbidity Outcomes
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Symptoms of Mental Health Disorders
Endpoint domain: Life impact
Endpoint subdomain: Emotional functioning/wellbeing
Primary endpoint: Treatment Group Responder Rate
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Pharmacokinetics Profile
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: MMRC Dyspnea score
Endpoint domain: Physiological/clinical
Endpoint subdomain: Respiratory outcomes
Primary endpoint: COPD Assessment Test CAT
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: ICIQ-SF (International Urinary Incontinence Consultation) Survey
Endpoint domain: Life impact
Endpoint subdomain: Perceived health status
Primary endpoint: Real-time polymerase chain reaction (PCR)
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Prosodic test
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: 45-day mortality after corticosteroid treatment
Endpoint domain: Mortality/survival
Primary endpoint: Number of enrolled subjects who have completed the study
Endpoint domain: Resource use
Endpoint subdomain: Need for further intervention
Primary endpoint: Cumulative Chest Tube Drainage
Endpoint domain: Physiological/clinical
Endpoint subdomain: Surgical and medical procedures outcomes
Primary endpoint: 31P-MRS Redox Ratio
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Disease free patients
Endpoint domain: Mortality/survival
Primary endpoint: Change From Baseline for Clinical Global Impressions of Improvement (CGI-I)
Endpoint domain: Life impact
Endpoint subdomain: Perceived health status
Primary endpoint: Change From Baseline in the Participant's Assessment for Quality of Life for Alzheimer's Dementia (QoL-AD)
Endpoint domain: Life impact
Endpoint subdomain: Global quality of life
Primary endpoint: Overall response rate as antitumor effect
Endpoint domain: Physiological/clinical
Endpoint subdomain: Neoplasms and related outcomes
Primary endpoint: lymphoedema-related quality of life
Endpoint domain: Life impact
Endpoint subdomain: Global quality of life
Primary endpoint: cancer quality of life
Endpoint domain: Life impact
Endpoint subdomain: Global quality of life
Primary endpoint: limb circumference measurement
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Response achievement
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: false negative rate to diagnose a pathological complete response
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Intermittent catheterization acceptance
Endpoint domain: Life impact
Endpoint subdomain: Perceived health status
Primary endpoint: Somatosensory evoked potential
Endpoint domain: Physiological/clinical
Endpoint subdomain: Nervous system outcomes
Primary endpoint: Methylation status at NRC3C1 and FKBP5 gene
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: time to reconstructive surgery
Endpoint domain: Physiological/clinical
Endpoint subdomain: Surgical and medical procedures outcomes
Primary endpoint: maximum tolerated dose of topotecan
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Percentage of Participants Achieving Predefined Antibody Level ≥0.1 IU/mL for Tetanus Toxoid
Endpoint domain: Physiological/clinical
Endpoint subdomain: Immune system outcomes
Primary endpoint: Percentage of Participants Achieving Predefined Antibody Level ≥1:8 for Poliovirus
Endpoint domain: Physiological/clinical
Endpoint subdomain: Immune system outcomes
Primary endpoint: Percentage of Participants Achieving Predefined Antibody Level ≥10.0 mIU/mL for Hepatitis B
Endpoint domain: Physiological/clinical
Endpoint subdomain: Immune system outcomes
Primary endpoint: 6 Minutes Walking Distance Test (6MWD)
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Forced expiratory volume in one second, FEV1
Endpoint domain: Physiological/clinical
Endpoint subdomain: Respiratory outcomes
Primary endpoint: variation of performance on naming subtest of the LEXIS
Endpoint domain: Physiological/clinical
Endpoint subdomain: Cognitive functioning
Primary endpoint: variation of performance on designation subtest of the LEXIS
Endpoint domain: Physiological/clinical
Endpoint subdomain: Cognitive functioning
Primary endpoint: variation of performance on semantic matching subtest of the LEXIS
Endpoint domain: Physiological/clinical
Endpoint subdomain: Cognitive functioning
Primary endpoint: Rise in D-dimer blood levels
Endpoint domain: Physiological/clinical
Endpoint subdomain: Blood and lymphatic system outcomes
Primary endpoint: Fall in Fibrinogen blood levels
Endpoint domain: Physiological/clinical
Endpoint subdomain: Blood and lymphatic system outcomes
Primary endpoint: Incidence of postoperative lymphocele formation
Endpoint domain: Physiological/clinical
Endpoint subdomain: Lymphatic system outcomes
Primary endpoint: Changes of free urinary flow rate
Endpoint domain: Physiological/clinical
Endpoint subdomain: Renal and urinary outcomes
Primary endpoint: Changes of post void residual urine
Endpoint domain: Physiological/clinical
Endpoint subdomain: Renal and urinary outcomes
Primary endpoint: Carotid Intima-Media Thickness
Endpoint domain: Physiological/clinical
Endpoint subdomain: Cardiovascular outcomes
Primary endpoint: Percentage of Participants With HIV-1 RNA ≥50 Copies/mL at Week 48
Endpoint domain: Physiological/clinical
Endpoint subdomain: Infections and infestations outcomes
Primary endpoint: Percentage of Participants Who Discontinued Study Intervention up to Week 48
Endpoint domain: Resource use
Endpoint subdomain: Need for further intervention
Primary endpoint: Number of Patients With at Least a 100-point Decrease in CDAI Score
Endpoint domain: Physiological/clinical
Endpoint subdomain: Gastrointestinal outcomes
Primary endpoint: Pain Scale Evaluation
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Number of Participants With Any Treatment-emergent Adverse Event (TEAE) Occurring in ≥5% of Participants in Any Treatment Group
Endpoint domain: Adverse events/effects
Primary endpoint: Mean Change From the Pre-randomization Baseline of the Core Study to the End of Treatment in Body Mass Index (BMI)
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Mean Subject/Caregiver Global Impression of Change (S/CGIC) Score
Endpoint domain: Life impact
Endpoint subdomain: Perceived health status
Primary endpoint: Differences in Immune Cells
Endpoint domain: Physiological/clinical
Endpoint subdomain: Immune system outcomes
Primary endpoint: Pharmacokinetics of Oseltamivir
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: All cause postoperative morbidity
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes
Primary endpoint: Mean max change (ml) from baseline in FEV1 over a period of 24 h
Endpoint domain: Physiological/clinical
Endpoint subdomain: Respiratory outcomes
Primary endpoint: Mean % change from baseline in FEV1 over a period of 24 h
Endpoint domain: Physiological/clinical
Endpoint subdomain: Respiratory outcomes
Primary endpoint: Mean max change (ml) from baseline in FVC over a period of 24 h
Endpoint domain: Physiological/clinical
Endpoint subdomain: Respiratory outcomes
Primary endpoint: Mean % change from baseline in FVC over a period of 24 h
Endpoint domain: Physiological/clinical
Endpoint subdomain: Respiratory outcomes
Primary endpoint: Stabilisation or Regression of Tanner Pubertal Stage
Endpoint domain: Physiological/clinical
Endpoint subdomain: Endocrine outcomes
Primary endpoint: Change in Upper Limb function
Endpoint domain: Physiological/clinical
Endpoint subdomain: General outcomes