ALXN1850-HPP-301- Hickory-ALXN1850 vs Placebo in Adolescent & Adult Asfotase Alfa Naive Pts with HPP
Research type
Research Study
Full title
A Phase 3, Randomized, Double-blinded, Placebo-controlled, Multicenter Study to Evaluate Efficacy and Safety of ALXN1850 (Recombinant Alkaline Phosphatase) Administered Subcutaneously in Adolescent (12 to < 18 years of age) and Adult Participants with Hypophosphatasia Who Have Not Previously Been Treated with Asfotase Alfa.
IRAS ID
1008899
Contact name
Zara Butt
Contact email
Sponsor organisation
Alexion Pharmaceuticals, Inc.
Clinicaltrials.gov Identifier
Research summary
The purpose of the study is to assess if the study drug (ALXN1850) works better than placebo when given to adolescent and adult participants with hypophosphatasia (HPP) who have not received Asfotase Alfa (Strensiq®) before. To evaluate how well ALXN1850 works, functional outcomes & radiographic changes will be measured. The study also aims to assess, but not limited to, the side effects of ALXN1850 and how often they happen, and the severity of side effects compared to placebo, as well as evaluating if the participant's health quality of life outcomes improve.
Strensiq® is the only approved treatment for HPP for paediatric onset patients. ALXN1850 has been developed to address the unmet needs of Strensiq®, which are to increase drug exposure & make enzyme activity more efficient, thereby enabling study drug dosing at lower volumes and less frequently. Another reason for ALXN1850 is to reach a broader population of patients as Strensiq® is currently only approved for paediatric onset patients.
The study consists of 4 phases; Screening, Randomised Evaluation Period (REP), Open Label Extension (OLE) & Safety Follow Up (SFU). Participants who meet the study eligibility criteria will have a 2/3 and 1/3 chance of receiving ALXN1850 or placebo respectively. During REP, participants will be randomly assigned to receive study intervention (ALXN1850 or placebo) starting on Day 1 and thereafter for a total of 24 weeks. The ALXN1850 group will receive a body weight-dose every 2 weeks via subcutaneous (SC) injection. The placebo group will receive placebo every 2 weeks via SC injection. All participants will be offered a dose of ALXN1850 every 2 weeks in the OLE phase. The OLE phase lasts about 2.5 years (132 weeks) and is divided into 2 parts (A & B). Participants will enter the SFU 4 weeks after the last study drug dose. The total study duration is approximately 164 weeks (approximately 3 years). About 114 participants from 23 countries will participate in this study.REC name
East of England - Cambridge Central Research Ethics Committee
REC reference
23/EE/0257
Date of REC Opinion
15 Jan 2024
REC opinion
Further Information Favourable Opinion