Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as rhASA - Dose Level 1, HGT-1110
rhASA · 2 trials · 1 indication
An AE is any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in any phase of a clinical study, whether or not considered investigational drug product-related. TEAEs are defined as all AEs occurring or worsening at or after the first dose of investigational drug product in HGT-MLD-071 or ongoing from HGT-MLD-070 at the time of enrollment into HGT-MLD-071.
The presence of anti-HGT-1110 antibodies in CSF and serum will be assessed.
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 | Participants will receive 10 milligram (mg) of HGT-1110 (Recombinant human arylsulfatase A) intrathecal (IT) injection every-other-week (EOW). |
| Cohort 2 | EXPERIMENTAL | Participants will receive 30 mg of HGT-1110 IT injection EOW. |
| Cohort 3 | EXPERIMENTAL | Participants will receive 100 mg of HGT-1110 IT injection EOW. |
| Cohort 4 | EXPERIMENTAL | Participants will receive 100 mg of HGT-1110 IT injection once weekly for 12 weeks followed by 150 mg EOW. |
| Name | Type | Description |
|---|---|---|
| HGT-1110 | BIOLOGICAL | Participants will receive IT injection of HGT-1110. |
| 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. Participant has participated in Study HGT-MLD-070 (NCT01510028) through Week 40. 2. Participant must have no safety or medical issues that contraindicate participation. 3. The participant, participant's parent(s), or legally authorized representative(s) must provide written i...
rhASA is being developed for the treatment of metachromatic leukodystrophy (MLD), a rare inherited lysosomal storage disorder. It is an investigational therapy studied in patients with the late infantile form of MLD. The clinical program has focused on this rare disease indication.
rhASA is being developed by Takeda Pharmaceutical Company Limited, which trades under the ticker TAK. Takeda is the sponsor of the clinical program evaluating rhASA in metachromatic leukodystrophy.
rhASA is in Phase 1 clinical development. It has not been approved by the FDA and remains an investigational therapy. The Phase 1 trial has been completed, and no active trials are currently listed for rhASA.
rhASA has been evaluated in one completed Phase 1 trial, NCT00418561, titled Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD). The trial enrolled 13 patients in Denmark and studied patients aged 1 year and older.
Yes, rhASA is also known as Metazym, which is the name used in its clinical trial NCT00418561. The trial title refers to Metazym for the treatment of patients with late infantile metachromatic leukodystrophy. rhASA is also referred to as rhASA Dose Level 1.