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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01155778 | Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients | PHASE1 | COMPLETED | 12 | — | — | Jun 1, 2010 | Sep 1, 2012 | Jun 14, 2021 | 2 | Netherlands, United Kingdom |
An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered as a pharmaceutical product that did not necessarily have a causal relationship with this treatment. A serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent AEs (TEAEs) were defined as all adverse events (AEs) from the time of the surgery for IDDD implantation to the last follow up contact, 30 (±7) days after the end of study (EOS) procedures.
An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered as a pharmaceutical product that did not necessarily have a causal relationship with this treatment. A serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent AEs (TEAEs) were defined as all adverse events (AEs) from the time of the surgery for IDDD implantation to the last follow up contact, 30 (±7) days after the end of study (EOS) procedures.
Participants with positive, negative and missing status were reported.
Participants with positive, negative and missing status were reported.
Participants with IDDD failures were reported.
| Arm | Type | Description |
|---|---|---|
| 10 mg rhHNS | EXPERIMENTAL | 10 mg monthly via an IDDD (every 28 \[±7 days\]) for a total of 6 months |
| 45 mg rhHNS | EXPERIMENTAL | 45 mg monthly via an IDDD (every 28 \[±7 days\]) for a total of 6 months |
| 90 mg rhHNS | EXPERIMENTAL | Given IDDD as a 45 mg dose every 14 \[±2 days\] for a monthly total of 90 mg for 6 months |
| Name | Type | Description |
|---|---|---|
| Recombinant human heparan N-sulfatase (rhHNS) | BIOLOGICAL | - |
Inclusion Criteria Each patient had to meet the following criteria to be eligible for the study: 1. a.) Patients had a documented deficiency in sulfamidase enzyme activity of ≤10% of the lower limit of the normal range as measured in fibroblasts or leukocytes. AND either b or c b.) Patients ha...