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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06079359 | Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP | PHASE3 | ACTIVE NOT_RECRUITING | 30 | — | — | May 14, 2024 | Aug 24, 2028 | May 28, 2026 | 32 | United States, Australia +14 |
| NCT06079372 | Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa | PHASE3 | ACTIVE NOT_RECRUITING | 43 | — | — | Apr 2, 2024 | Feb 29, 2028 | May 5, 2026 | 21 | United States, Argentina +5 |
| NCT06079281 | Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa | PHASE3 | ACTIVE NOT_RECRUITING | 124 | — | — | Jan 3, 2024 | Mar 29, 2028 | May 28, 2026 | 65 | United States, Argentina +15 |
| NCT04980248 | Study of ALXN1850 in Participants With Hypophosphatasia (HPP) | PHASE1 | COMPLETED | 15 | — | — | Sep 28, 2021 | Aug 24, 2022 | Dec 27, 2024 | 4 | United States |
TEAEs were defined as any adverse events (AEs) that began or worsened on or after the first dose of treatment until the final follow-up visit. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. TESAEs were defined as any serious AEs that began or worsened on or after the first dose of treatment until the final follow-up visit. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
| Arm | Type | Description |
|---|---|---|
| ALXN1850 | EXPERIMENTAL | Starting at Day 1 of the Randomized Evaluation Period, the ALXN1850 group will receive bodyweight dependent doses of either 20mg, 35mg or 50mg of ALXN1850 once every 2 weeks (q2w) via SC injection, for 24 weeks. Participants will enter the OLE Period and continue q2w dosing with ALXN1850 for up to 132 weeks. |
| Placebo | PLACEBO_COMPARATOR | Starting at Day 1 during the Randomized Evaluation Period, participants will receive placebo q2w for a total of 24 weeks. Participants will enter the OLE Period and continue q2w dosing with ALXN1850 for up to 132 weeks. |
| 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. |
| Placebo Group | PLACEBO_COMPARATOR | During the Randomized Evaluation Period, the placebo group will receive placebo on Day 1, followed by once every 2 weeks (q2w) via SC injection for 24 weeks. Participants will enter the OLE Period and receive bodyweight dependent doses of either 20mg, 35mg, or 50mg of ALXN1850 and continue q2w dosing with ALXN1850 for up to 132 weeks. |
| ALXN1850 Group | EXPERIMENTAL | Starting at Day 1 of the Randomized Evaluation Period, the ALXN1850 group will receive bodyweight dependent doses of either 20mg, 35mg or 50mg of ALXN1850 once q2w via SC injection, for 24 weeks. Participants will enter the OLE Period and continue q2w dosing with ALXN1850 for up to 132 weeks. |
| Name | Type | Description |
|---|---|---|
| ALXN1850 | DRUG | Participants will receive a ALXN1850 via subcutaneous (SC) injection. |
| Placebo | DRUG | Participants will receive placebo via SC injection. |
| asfotase alfa | DRUG | Asfotase alfa will be administered via SC injection. |
Inclusion Criteria: * Diagnosis of HPP documented in the medical records, and the following criteria fulfilled without other probable cause than HPP: 1. Presence of HPP-related rickets on skeletal X-rays during the Screening Period, with a minimum Rickets Severity Score (RSS) of 1.0 AND 2. Ser...