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| ID | Type | Description | Link |
|---|---|---|---|
| ALXN1850-HPP-303 | Other Identifier | Alexion Pharmaceuticals, Inc. |
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The primary purpose of this study is to assess the safety and tolerability of ALXN1850 versus asfotase alfa in pediatric participants with HPP previously treated with asfotase alfa.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ALXN1850 | Experimental | Starting at Day 1 of the Randomized Evaluation Period participants will receive ALXN1850 for a total of 24 weeks. Participants will receive bodyweight dependent doses of either 20mg, 35mg or 50mg of ALXN1850 once q2w via SC injection. During Part A of the OLE Period, participants will have frequent visits over the first 24 weeks; Part B of the OLE Period participants will have visits every 9 months for up to approximately 108 weeks. |
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| asfotase alfa | Experimental | Starting at Day 1 of the Randomized Evaluation Period, participants will receive asfotase alfa for a total of 24 weeks. Participants will receive 6 mg/kg/week of asfotase alfa via SC injection as either 2 mg/kg 3 times per week or 1 mg/kg 6 times per week. Part A of the OLE Period participants will have frequent visits over the first 24 weeks; Part B will have visits every 9 months for up to approximately 108 weeks. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ALXN1850 | Drug | ALXN1850 will be administered via subcutaneous (SC) injection. |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants with Treatment-emergent Adverse Events (TEAEs) | Baseline Through Day 169 |
| Measure | Description | Time Frame |
|---|---|---|
| Radiographic Global Impression of Change (RGI-C) Score at the end of the Randomized Evaluation Period (Day 169) | Baseline, Day 169 | |
| Change from Baseline in Rickets Severity Score (RSS) at the end of the Randomized Evaluation Period (Day 169) | Baseline, Day 169 |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site | Hartford | Connecticut | 06106 | United States | ||
| Research Site |
Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
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| asfotase alfa |
| Drug |
Asfotase alfa will be administered via SC injection. |
|
| Change from Baseline in 6-Minute Walk Test (6MWT) at the end of the Randomized Evaluation Period (Day 169) | Baseline, Day 169 |
| Change from Baseline in Percent Predicted 6MWT at the end of the Randomized Evaluation Period (Day 169) | Baseline, Day 169 |
| Change from Baseline in Bruininks Oseretsky Test of Motor Proficiency, Second Edition (BOT2) Score at the end of the Randomized Evaluation Period (Day 169) | Baseline, Day 169 |
| Change from Baseline in Peabody Developmental Motor Scales, Third Edition (PDMS-3) Score at the end of the Randomized Evaluation Period (Day 169) | Baseline, Day 169 |
| Baltimore |
| Maryland |
| 21287 |
| United States |
| Research Site | Minneapolis | Minnesota | 55455 | United States |
| Research Site | Kansas City | Missouri | 64108 | United States |
| Research Site | Durham | North Carolina | 27705 | United States |
| Research Site | Nashville | Tennessee | 37212 | United States |
| Research Site | Mar del Plata | B7600 | Argentina |
| Research Site | South Brisbane | 4101 | Australia |
| Research Site | Ottawa | Ontario | K1H 8L1 | Canada |
| Research Site | Bunkyō City | 113-8431 | Japan |
| Research Site | Minatoku | 105-8471 | Japan |
| Research Site | Suita-shi | 565-0871 | Japan |
| Research Site | Yonago-shi | 683-8504 | Japan |
| Research Site | Altındağ | 06230 | Turkey (Türkiye) |
| Research Site | Ankara | 06560 | Turkey (Türkiye) |
| Research Site | Edirne | 22030 | Turkey (Türkiye) |
| Research Site | Erzurum | 25240 | Turkey (Türkiye) |
| Research Site | Istanbul | 34899 | Turkey (Türkiye) |
| Research Site | Birmingham | B4 6NH | United Kingdom |
| Research Site | Manchester | United Kingdom |
| Research Site | Sheffield | S10 2TH | United Kingdom |
| ID | Term |
|---|---|
| D007014 | Hypophosphatasia |
| ID | Term |
|---|---|
| D008664 | Metal Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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| ID | Term |
|---|---|
| C570710 | asfotase alfa |
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