Safety, Efficacy, PK, and PD Study of ALXN1820 in Adult Patients with Sickle Cell Disease
Research type
Research Study
Full title
A Phase 2a, Randomized, Open-Label Study to Evaluate Multiple Dosing Regimens of Subcutaneous ALXN1820 in Adult Patients with Sickle Cell Disease
IRAS ID
1005996
Contact name
Yang Dai
Contact email
Sponsor organisation
Alexion Pharmaceuticals, Inc
Eudract number
2022-001615-74
Research summary
ALXN1820-SCD-201 is a Phase 2a, randomised, open-label study with the purpose of assessing safety, tolerability, efficacy, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of multiple doses and dosing regimens of ALXN1820 SC in patients with Sickle Cell Disease (SCD).
The study will be conducted in adult participants with Sickle Cell Disease enrolled in up to 3 open-label cohorts (Cohorts 1, 2 and 3 [optional]) to receive multiple subcutaneous (SC) doses of open-label ALXN1820. The cohorts will enrol both participants with Sickle Cell Disease (SCD) who are being treated with a stable dose of hydroxyurea and participants with Sickle Cell Disease (SCD) who are not currently treated with hydroxyurea.The planned study duration is approximately 38 weeks for Cohorts 1 and 2: up to 56 days (8 weeks) for screening, 84 days (12 weeks) for the Treatment Period, and 126 days (18 week) for the Follow-up Period. Participants will attend the outpatient visit during the Treatment and Follow-up Period. The end of study for each individual participant is anticipated to be Day 211 (210 days).
The planned study duration is approximately 32 weeks for optional Cohort 3: up to 56 days (8 weeks) for screening, 42 days (6 weeks) for the Treatment Period, and 126 days (18 weeks) for the Follow-up Period. Patients will attend the outpatient visit during the Treatment and Follow-up Period with the option to stay at the inpatient facility. The end of study for each individual participant is anticipated to be Day 169 (168 days).
The study will include approximately 30 participants enrolled from around 24 - 30 centres globally. In the UK there will be 4 hospital sites with approximately 5 participants planned.
REC name
East of England - Cambridge South Research Ethics Committee
REC reference
22/EE/0280
Date of REC Opinion
23 Jun 2023
REC opinion
Further Information Favourable Opinion