Phase 1
N=15
Study of ALXN1850 in Participants With Hypophosphatasia (HPP)
Hypophosphatasia
Bottom Line
View on ClinicalTrials.gov: NCT04980248 ↗Enrolled (actual)
15
Serious AEs
6.7%
Results posted
Dec 2024
Primary outcome: Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) — 3; 5; 4; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- ALXN1850 (Biological)
- Age
- Adult · 18+ yrs
- Sex
- All
- Sponsor
- Alexion Pharmaceuticals, Inc.
- Primary completion
- Aug 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) |
3; 5; 4; 0; 0; 1 | — |
| SECONDARY Maximum Observed Plasma Concentration (Cmax) of ALXN1850 Following Intravenous (IV) Dose, Subcutaneous (SC) Dose 1, SC Dose 2 and SC Dose 3 |
5.31; 13.5; 32.4; 0.871; 2.89; 8.60 | — |
| SECONDARY Area Under the Plasma Concentration Versus Time Curve From Time 0 Extrapolated to Infinity Following IV Dose of ALXN1850 |
407; 1010; 2450 | — |
| SECONDARY Area Under the Plasma Concentration Versus Time Curve From Time 0 to Dosing Interval (AUCtau) Following SC Dose 1, SC Dose 2 and SC Dose 3 |
115; 383; 1110; 111; 350; 872 | — |
| SECONDARY Area Under the Plasma Concentration Versus Time Curve From Time 0 to Dosing Interval (AUCtau) Values of the First SC Versus IV Administration of ALXN1850 |
0.286; 0.367; 0.368 | — |
| SECONDARY Absolute Change From Baseline in Plasma Concentration of Pyridoxal-5' Phosphate (PLP) at Week 1 |
-18.300; -7.600; -49.460; -97.950; -38.573; -28.055 | — |
| SECONDARY Absolute Change From Baseline in Plasma Concentration of Inorganic Pyrophosphate (PPi) at Week 1 |
-1.620; -1.930; -0.813; -1.505; -2.040 | — |
| SECONDARY Absolute Change From Baseline in Plasma Concentration of Pyridoxal Phosphate/ Pyridoxal (PLP/PL) Ratio at Week 1 |
-8.570; -16.070; -15.260; -7.620; -15.285; -8.368 | — |
| SECONDARY Percent Change From Baseline in Plasma Concentration of PLP at Week 1 |
-47.906; -17.512; -86.318; -33.895; -69.049; -70.515 | — |
| SECONDARY Percent Change From Baseline in Plasma Concentration of PPi at Week 1 |
-53.821; -70.956; -32.740; -55.328; -71.329 | — |
| SECONDARY Percent Change From Baseline in Plasma Concentration of PLP/PL Ratio Over Time at Week 1 |
-65.270; -92.944; -91.323; -35.104; -77.160; -60.816 | — |
| SECONDARY Number of Participants With Anti-drug Antibody (ADA) Positive and Neutralizing Antibody (NAb) Positive Status |
2; 0; 2; 0; 0; 0 | — |
Summary
This is an open-label, dose-escalating study to assess safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and immunogenicity of ALXN1850 when given intravenous (IV) and subcutaneous (SC) to adults with HPP.
Eligibility Criteria
Inclusion Criteria
- Confirmed clinical diagnosis of HPP
- Not anticipated to require further treatment with enzyme replacement therapy to treat participant's HPP after study completion
- Willing and able to follow protocol-specified contraception requirements
- Willing and able to give informed consent
Exclusion Criteria
- Primary or secondary hyperparathyroidism or hypoparathyroidism
- Fracture within 12 weeks of screening
- Current or relevant history of unstable physical or psychiatric illness
- Significant allergies
- Asfotase alfa use within 6 months and/or positive for asfotase alfa antidrug antibody/neutralizing antibodies
Data sourced from ClinicalTrials.gov (NCT04980248). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.