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Also known as Recombinant human heparan N-sulfatase (rhHNS)
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 being developed for mucopolysaccharidosis (MPS) and Sanfilippo syndrome, also known as MPS IIIA. It is an enzyme replacement therapy given directly into the central nervous system to address the underlying enzyme deficiency in this rare inherited metabolic disorder.
Recombinant human heparan N-sulfatase targets F9 and is classified as an exogenous protein. It works by supplying a functional form of the heparan N-sulfatase enzyme, which is deficient in Sanfilippo syndrome, to help break down heparan sulfate that accumulates in cells.
Takeda Pharmaceutical Company Limited, which trades under the ticker TAK, is the developer of recombinant human heparan N-sulfatase. The company has sponsored clinical studies of the therapy in patients with Sanfilippo syndrome and related mucopolysaccharidosis conditions.
Recombinant human heparan N-sulfatase is in Phase 1 development and is investigational. It has not been approved for commercial use. Clinical work to date includes a completed Phase 1 study and a completed Phase 2 study in pediatric patients with early stage MPS IIIA disease.
Recombinant human heparan N-sulfatase has been studied in two completed trials. NCT01155778 was a Phase 1 safety, tolerability, and ascending dose study in MPS IIIA patients. NCT02060526 was a randomized, controlled, open-label Phase 2 study of rhHNS given via an intrathecal drug delivery device in pediatric patients with early stage MPS IIIA.
Yes. Recombinant human heparan N-sulfatase is also known as rhHNS, and the two names refer to the same investigational enzyme replacement therapy. The abbreviation rhHNS appears in the trial titles for the Sanfilippo syndrome and mucopolysaccharidosis studies.