Trial · NCT04088734
Registered outcomes22
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 20 of 22
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Incidence, Type and Severity of Related Treatment-Emergent Adverse Events (TEAEs) by Time Frame | From the first dose of study drug to <30 days postdose, Day 30, 60, 90, 180 and Month 12 | An adverse event (AE) is any untoward medical occurrence or unintended change from the time informed consent form (ICF) is signed, including inter-current illness that occurs during the course of a clinical trial after treatment has started, whether considered related to treatment or not. TEAEs are those that occurred after the start of study drug. Related adverse events were categorized as possible, probable, or definitely. |
| Primary | Incidence, Type and Severity of Serious Adverse Events (SAEs) by Time Frame | From signing of informed consent through Day 60, 90, 180 and up to Day 454 (> 12 months) | An SAE is defined as any untoward medical occurrence that, at any dose: 1. Results in death 2. Is life threatening 3. Requires inpatient hospitalization or prolongation of existing hospitalization 4. Results in persistent disability/incapacity 5. Is a congenital anomaly/birth defect 6. Other situations such as important medical events that may not be immediately life threatening or result in death or hospitalization but may jeopardize the participant or may require medical or surgical intervention to prevent one of the other outcomes listed in the above definition. Relationship to study drug was defined as unrelated, unlikely, possible, probable, or definitely. |
| Primary | Change From Baseline (BL) in Multiples of Normal of Liver and Spleen Volumes After Treatment | Baseline, Day 30, 180, Month 12 | As Measured by Magnetic Resonance Imaging (MRI). Baseline value of multiple of normal is calculated using the baseline values of the Liver volume/Spleen Volume/Height and Weight. * Body Surface Area (BSA) (m2)=( Height(cm) \* Weight (kg)/3600)1/2. * Normal Liver Volume=(689.9 \* BSA (m)) - 24.7. * Normal Spleen Volume (mL)=(4.6 \* Weight (kg)) + 0.7. * Liver Volume (multiples of normal)=Subject Liver Volume (mL)/Normal Liver Volume (mL). * Spleen Volume (multiples of normal)=Subject Spleen Volume (mL)/Normal Spleen Volume (mL). |
| Primary | Change From BL in Cerebrospinal Fluid (CSF) Heparan Sulfate Levels After Treatment | Baseline, Day 30, Day 180, Month 12 | Change from baseline in CSF heparan sulfate levels after treatment |
| Secondary | Change From Baseline in Plasma Heparan Sulfate After Treatment | Baseline, Day 30, Day 180, Month 12 | |
| Secondary | Change From Baseline in Urine Glycosaminoglycans After Treatment | Baseline, Day 30, Day 180, Month 12 | |
| Secondary | Change From Baseline in Urine Heparan Sulfate After Treatment | Baseline, Day 30, Day 180, Month 12 | |
| Secondary | Change From Baseline in CSF N-Sulfoglucosamine Sulfohydrolase (SGSH) Enzyme Activity Levels After Treatment | Baseline, Day 30, Day 180, and Month 12 | |
| Secondary | Change From Baseline in Heparan N-Sulfatase (Type A) After Treatment | Baseline, Day 30, Day 180, Month 12 | |
| Secondary | Change From Baseline in Plasma SGSH After Treatment | Baseline, Day 30, Day 180, Month 12 | |
| Secondary | Change From Baseline in Brain Volumes After Treatment | Baseline, 12 months | As measured by MRI |
| Secondary | Change From Baseline in Brain Volumes After Treatment: Average Total Cortical Thickness | Baseline, 12 months | As measured by MRI |
| Secondary | Change From Baseline in Sleep Pattern as Measured by the Modified Children's Sleep Habits Questionnaire (CSHQ) Subscore Total After Treatment | Baseline, Day 180, Month 12 | CSHQ is a caregiver-completed, 35-item questionnaire that assesses the frequency of behaviors associated with common pediatric sleep difficulties. Eight domains of sleep, including Bedtime Resistance, Sleep Onset Delay, Sleep Duration, Sleep Anxiety, Night Awakenings, Parasomnias, Sleep Disordered Breathing, and Daytime Sleepiness are assessed, producing eight individual subdomain scores and an overall CSHQ subscore total. CSHQ total score is calculated by adding all the 8 subscores, and ranges from 36 to 108. A higher score is indicative of more disturbed sleep. |
| Secondary | Change From Baseline in Pediatric Quality of Life Inventory (PedsQL™) Core Generic Scales Total Score | Baseline, Day 180, Month 12 | PedsQL is a brief measure of health-related quality of life in children. The Peds QL Generic Core Scales was used in the study, consisting of 23 items applicable for healthy school and community populations, as well as pediatric populations with acute and chronic conditions. The four scales include Physical Functioning, Emotional Functioning, Social Functioning, and School Functioning. Item scores are added together and averaged to produce a Core total score, where higher scores on a scale of 0-100 indicate better Health-related Quality of Life (HRQOL). |
| Secondary | Change From Baseline in Parent Quality of Life, Using the Parenting Stress Index, 4th Edition (PSI-4) Total Stress Raw Score | Baseline, Day 180, Month 12 | The Parenting Stress Index, 4th Edition evaluates the magnitude and type of stress in a parent/child relationship. The short form version was used in the study, consisting of 36 items divided into three domains: Parental Distress (PD), Parent-Child Dysfunctional Interaction (P-CDI), and Difficult Child (DC), which combine together to form a Total Stress raw score. Total Stress raw scores can range from 36 - 180, with higher raw scores indicating higher levels of stress. |
| Secondary | Change From Baseline in Gastrointestinal Symptoms Using the PedsQL™ Gastrointestinal (GI) Symptoms Scales Score | Baseline, Day 180, Month 12 | The PedsQL Gastrointestinal Symptoms Scale is a specific module of the PedsQL that measures gastrointestinal symptoms in patients with acute and chronic health conditions as well as healthy school and community populations. The Parent Report version was used on the study, consisting of 58 items across 10 dimensions, assessing parents' perceptions of their child's GI-specific symptoms. Item scores are added together and averaged to produce a GI symptoms scales score, where higher scores on a scale of 0-100 indicate better GI specific QOL. |
| Secondary | Change From Baseline in Parent Global Impression (PGI) Total Score | Baseline, Day 180, Month 12 | The Parent Global Impression scale evaluates all aspects of a patients' health and assesses if there has been an improvement or decline in clinical status, as reported by the parent/caregiver. This study used a modified version with symptoms relevant to the patient population in the trial. Nine symptoms were scored at each visit, using a 7-point rating scale where 3 = much better, 2 = better, 1 = slightly better, 0 = same, -1 = slightly worse, -2 = worse, and -3 = much worse. The nine symptom scores are added together to produce a PGI total score, ranging from -27 to 27. |
| Secondary | Clinical Global Impression Improvement Scale at Day 180 and Month 12 | Day 180, Month 12 | The Clinical Global Impression of Improvement scale is a brief, stand-alone assessment of the clinician's view of the patient's global functioning prior to and after initiating a study medication, specifically looking at whether the patient has demonstrated improvement or not. Assessment of improvement was scored at each visit, using a 7-point rating scale where 0 = not assessed, 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, and 7 = very much worse. |
| Secondary | Change From Baseline in Parent Symptoms Score Questionnaire | Baseline, Day 180, Month 12 | The Parent Symptoms Score Questionnaire contains 29 symptoms with an indicator of whether the symptom was present or absent. |
| Secondary | Percent Change From Baseline in Body Mass Index After Treatment | Baseline, Day 30, Day 180, Month 12 |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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