Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Recombinant human heparan N-sulfatase · 2 trials · 2 indications
The BSID--III is a series of measurements to assess the motor (fine and gross), language (receptive and expressive), and cognitive development of infants and toddlers and consists of a series of developmental play tasks. The development quotient (DQ) is a means to express a neurodevelopmental/cognitive delay which was computed as a ratio and expressed as a percentage using the age equivalent score divided by the age at testing (\[age-equivalent score/chronological age\] × 100; range: 0, 100). The BSID--III DQ score is based on the cognitive domain. A positive value indicates improvement in health and cognition. Overall response was the maximum decline in the DQ of 10 points or less over 48 weeks. Number of participants with the overall response were reported here.
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 |
|---|---|---|
| 45 mg Q2W | ACTIVE_COMPARATOR | rhHNS 45 mg administered intrathecally Q2W (once every 2 weeks ie, every 14 days), for 48 weeks via the surgically implanted IDDD (or LP) |
| 45 mg Q4W | ACTIVE_COMPARATOR | rhHNS 45 mg administered intrathecally Q4W (once every 4 weeks ie, every 28 days), for 48 weeks via the surgically implanted IDDD (or LP) |
| Placebo | PLACEBO_COMPARATOR | The comparator group will receive no treatment with rhHNS. |
| 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] | DRUG | Recombinant human heparan N-sulfatase \[rhHNS\] |
| Recombinant human heparan N-sulfatase (rhHNS) | BIOLOGICAL | - |
Inclusion Criteria: Each patient must meet the following criteria to be enrolled in this study. 1. Documented MPS IIIA diagnosis 2. Age ≥12 months and ≤48 months 3. The patient has a DQ score ≥60% 4. The patient is medically stable, in the opinion of the Investigator, and able to accommodate the p...
Recombinant human heparan N-sulfatase is used for the treatment of Mucopolysaccharidosis (MPS) and Sanfilippo Syndrome, which are rare genetic disorders. It is being developed as an enzyme replacement therapy for these conditions.
Recombinant human heparan N-sulfatase is being developed by Takeda Pharmaceutical Company Limited, which is listed on the stock exchange under the ticker symbol TAK.
Recombinant human heparan N-sulfatase is in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. Clinical trials are ongoing to evaluate its safety and efficacy.
Recombinant human heparan N-sulfatase has been studied in clinical trials. One trial, NCT01155778, was a Phase 1 study in patients with Mucopolysaccharidosis. Another trial, NCT02060526, was a Phase 2 study in pediatric patients with Sanfilippo Syndrome. Both trials have been completed.
Recombinant human heparan N-sulfatase is a monoclonal antibody that targets the enzyme heparan N-sulfatase. It works by replacing the deficient enzyme in patients with Mucopolysaccharidosis and Sanfilippo Syndrome, helping to break down heparan sulfate and reduce its accumulation in cells.