Trial · NCT07882290
Registered outcomes24
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 20 of 24
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Best Corrected Visual Acuity (BCVA) | BCVA will be measured at baseline, 26 weeks, 52 weeks and in case of premature treatment discontinuation. | Visual Acuity will be measured in each eye by an experienced Visual Acuity Examiner (VAE, ophthalmologist, orthoptist, optometrist, or trained ophthalmic technician) using either: * The Early Treatment Diabetic Retinopathy Study Logarithm of the Minimum Angle of Resolution chart in subjects evaluable by this method. Each line of the LogMAR chart comprises the same number of test letters (effectively standardizing the test across letter size); letter size from line to line varies logarithmically. Adapted tests (LEA symbol tests) will be used for pre-school age subjects (below 6 years of age), * If the subject is unable to read the chart at any distance, visual acuity will be tested one foot distance, using qualitative assessment (counting fingers, hand motion, light/no light perception). Each of these conditions corresponds to a LogMAR value to be used for efficacy analysis. |
| Secondary | Change from baseline in Unified Batten Disease Rating Scale (UBDRS) subscales | UBDRS subscales will be evaluated at baseline, 26 weeks, 52 weeks and in case of premature treatment discontinuation | * Unified Batten Disease Rating Scale subscale - Clinical Global Impression - Overall severity (min: 0=none, max: 5=severe) * Unified Batten Disease Rating Scale - modified Capability subscale 4 items (school, chores, play, Activities of Daily Living, min:0=normal, max: 12=most severe) * Unified Batten Disease Rating Scale modified - Physical Assessment subscale 2 items (speech clarity and gait, min: 0=normal, max: 8=most severe) * Unified Batten Disease Rating Scale - Clinical Global Impression severity subscores (seizures, cognitive function, behavior, mood, and motor, min: 1=no impairment, max: 5=severely impaired for each subscore). |
| Secondary | Hamburg Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) | JNCL Scores will be measured at baseline, 26 weeks, 52 weeks and in case of premature treatment discontinuation | Modified Hamburg Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) scores: (motor function, max: 3=normal, min: 0=immobile) |
| Other | General Health Status | General Health Status questionnaire will be evaluated at baseline, Week 26, Week 52 and in case of premature treatment discontinuation | HEALTH UTILITIES INDEX® Mark 2 and Mark 3 40-Item Questionnaire (HUI23P2USEn.40Q). The maximal score is 1.0, corresponding to a perfect health status, the minimal score represents death. |
| Other | The 23-item PedsQL 4.0 Generic Core Scales | The 23-item PedsQL 4.0 Generic Core Scales is assessed at baseline, Week 26, Week 52 and in case of premature treatment discontinuation. | The 23-item PedsQL 4.0 Generic Core Scales is a a parent proxy-report that assesses parents' perceptions of their child's Health Related Quality Of Life. Each item is evaluated with a 5-point response scale (0=never a problem, 4=almost always a problem). |
| Other | Safety assessment | Safety assessment will be performed at each of the 4 on-site visits, during the 5 phone calls or the 13 televisits, and in case or premature treatment discontinuation. | Number of participants with treatment-emergent adverse events (TEAEs), TEAEs leading to permanent discontinuation and serious adverse events (SAEs), adverse events of special interest, with abnormal laboratory tests results, abnormal 12-lead electrocardiogram (ECG) findings, abnormal slit lamp evaluation, abnormal vital signs as well as abnormal physical, neurological and mental state examinations findings |
| Other | PK Assessment Cmax | Pk assessment Cmax will be conducted at Day 1 and Week 26. | PK Assessment Maximum Plasma Concentration Cmax in ng/mL |
| Other | PK Assessment Tmax | Pk assessment Tmax will be conducted at Day 1 And Week 26. | PK Assessment Time to Maximum Plasma Concentration Cmax in hour |
| Other | PK Assessment AUC 0-t, AUC 0-∞ | Pk assessment AUC will be conducted at Day 1 and Week 26. | Area under the concentration-time curve calculated to the last observable concentration at time t (AUC 0-t), area under the concentration-time curve extrapolated to infinity (AUC 0-∞) in h\*ng/mL |
| Other | PK Assessment AUC 0-8hr (equivalent to AUC 0-Tau) | Pk assessment Tmax will be conducted at Week 26. | Area Under Curve (0-8hr equivalent to AUC 0-Tau) in h\*ng/mL |
| Other | PK Assessment T1/2 | Pk assessment T1/2 will be conducted at Day 1 | Elimination half-life in hour |
| Other | Body weight | Body weight will be measured at screening, baseline, on a weekly basis from Week 2 to Week 8, and on Weeks 9,11, 13, 17, 21, 26, 30, 34, 38, 42, 48 and 52, and in case of premature treatment discontinuation. | Body weight will be measured in kilograms |
| Other | Height | Height will be measured at baseline, Week 26, Week 52 and in case of premature treatment discontinuation | Height will be measured in centimeters |
| Other | ECG heart rate | ECG will be measured at baseline, Week 26, Week 52 and in case of premature treatment discontinuation | 12-lead electrocardiogram heart rate in beats / minute |
| Other | ECG QT interval | ECG will be measured at baseline, Week 26, Week 52 and in case of premature treatment discontinuation | 12-lead electrocardiogram QT interval (msec) |
| Other | ECG PR interval | ECG will be measured at baseline, Week 26, Week 52 and in case of premature treatment discontinuation | 12-lead electrocardiogram PR interval (msec) |
| Other | ECG QRS interval | ECG will be measured at baseline, Week 26, Week 52 and in case of premature treatment discontinuation | 12-lead electrocardiogram QRS interval (msec) |
| Other | Lab tests haematology | Hemoglobin will be measured at baseline, Week 26, Week 52 and in case of premature treatment discontinuation | Hemoglobin (g/L) |
| Other | Lab tests haematology | Hematocrit and differential white blood cell count will be measured at baseline, Week 26, Week 52 and in case of premature treatment discontinuation | Hematocrit (%) Differential white blood cell count (%) |
| Other | Lab tests haematology | Erythrocytes, leucocytes and thrombocytes will be measured at baseline, Week 26, Week 52 and in case of premature treatment discontinuation | Erythrocytes (Red Blood Cells Count (RBC): x 10¹²/L) Leukocytes (White Blood Cell Count (WBC): × 10⁹/L) Thrombocytes (Platelets: × 10⁹/L) |
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.
For software
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