Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
rhASA- Dose Level 1 · 1 trial · 1 indication
An adverse event (AE) is any untoward, undesired, unplanned clinical event in the form of signs, symptoms, disease, or laboratory or physiological observations occurring in a participant, participating in a clinical study with study drug, regardless of causal relationship. TEAEs were AEs occurred after study drug administration that were absent before treatment or that worsened relative to pre-treatment state, up to Week 28 until evaluation (when last cohort had 26-week evaluation and data management performed within 4 weeks) completed.
GMFM was measured using GMFM-88 item scores and summed to calculate a total GMFM-88 score. For each GMFM-88 item, the score was between 0 (minimal) to 3 (maximum). The total GMFM-88 score was between 0 (minimal) and 264 (maximum). The decrease in GMFM score over time indicates worsening of disease over time. Relative change from baseline at Week 26 was calculated as percentage change from baseline divided by the age-difference in months between first and last visit. Adjusted mean and 95 percent (%) confidence intervals were reported.
Relative change from baseline at Week 26 was calculated as percentage change from baseline divided by the age-difference in months between first and last visit. Adjusted mean and 95 percent (%) confidence intervals were reported.
Number of participants with shifts between negative (value=0) and positive (value=1) values in urine sulfatide levels from baseline at Week 26 is reported.
Mullen's Scales of Early Learning is used to assess performance and learning ability in young children. The scale consisted of 144 items that had specific scoring criteria for each item. The scores were converted to T-scores with a decrease in score indicating worsening of disease. Relative change from baseline at Week 26 was calculated as percentage change from baseline divided by the age-difference in months between first and last visit. Adjusted mean and 95 percent (%) confidence intervals were reported.
| Arm | Type | Description |
|---|---|---|
| Cohort 1 | EXPERIMENTAL | Metazym (Recombinant human arylsulfatase A (rhASA)): 25 U/kg as a single dose - hereafter 50 U/kg |
| Cohort 2 | EXPERIMENTAL | 100 U/kg Metazym (Recombinant human arylsulfatase A (rhASA)) |
| Cohort 3 | EXPERIMENTAL | 200 U/kg Metazym (Recombinant human arylsulfatase A (rhASA)) |
| Name | Type | Description |
|---|---|---|
| rhASA - Dose Level 1 | BIOLOGICAL | Intravenous infusion 25 U/kg as a single dose - hereafter 50 U/kg every other week for 26 weeks |
| rhASA - Dose Level 2 | BIOLOGICAL | Intravenous infusion 100 U/kg every other week for 26 weeks |
| rhASA - Dose Level 3 | BIOLOGICAL | Intravenous infusion 200 U/kg every other week for 26 weeks |
Inclusion Criteria: 1. Subject's legally authorized guardian(s) must provide signed, informed consent prior to performing any study-related activities (Trial-related activities are any procedures that would not have been performed during normal management of the subject). 2. The patient must have a...
rhASA- Dose Level 1 is an investigational therapy being studied for the treatment of Metachromatic Leukodystrophy (MLD), a rare genetic disorder. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
rhASA- Dose Level 1 is being developed by Takeda Pharmaceutical Company Limited, which trades under the ticker TAK. The company is conducting clinical research on this investigational therapy for Metachromatic Leukodystrophy.
rhASA- Dose Level 1 is in Phase 1 clinical development. It is an investigational therapy and has not been approved for commercial use. The Phase 1 trial has been completed, and the drug remains under clinical investigation.
rhASA- Dose Level 1 was studied in a completed Phase 1 clinical trial with the identifier NCT00418561. The trial, titled 'Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD),' enrolled 13 participants in Denmark.
rhASA- Dose Level 1 is associated with the trial name Metazym, as indicated in the clinical trial title. The investigational therapy is being studied for Metachromatic Leukodystrophy, and the trial was conducted in Denmark.