Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01887938 | An Efficacy and Safety Study of HGT-1110 in Participants With Metachromatic Leukodystrophy | PHASE1 | COMPLETED | 24 | — | — | May 23, 2013 | Feb 13, 2026 | Apr 21, 2026 | 15 | Australia, Brazil +5 |
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.
| 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. |
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...