A Phase 1, Randomized, Double Blind, Placebo-Controlled, Single Ascending Dose Study in Healthy Participants Followed by a Phase 2a Open Label Study in Participants with PNH and Residual Anemia to Evaluate the Safety, Tolerability, PK and PD of ADX-038
Status: Recruiting
Location: See all (4) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY
The first-in-human Phase 1/Phase 2a study described herein will evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ADX-038 in both healthy participants (HP) and in patients with paroxysmal nocturnal hemoglobinuria (PNH).
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: t
View:
• 18 to 55 years of age
• Participants who are healthy as determined by medical evaluation
• History of recent meningococcal, pneumococcal and Haemophilus influenzae type B vaccinations or willing to be vaccinated
• Screening tests negative for illicit drug, nicotine, and alcohol use
• at least 18 years of age
• Diagnosis of paroxysmal nocturnal hemoglobinuria based on documented clone size
• Hemoglobin concentration of less than 12 gram per deciliter
• History of recent meningococcal, pneumococcal and Haemophilus influenzae type b vaccinations or willing to be vaccinated
• On a stable anti-C5 regimen for greater than or equal to 12 weeks prior to Day 1
Locations
Other Locations
Australia
Nucleus Network Brisbane
RECRUITING
Brisbane
Peter MacCallum Cancer Centre
NOT_YET_RECRUITING
Melbourne
Royal Melbourne Hospital
NOT_YET_RECRUITING
Parkville
United Kingdom
Richmond Pharmacology Ltd
RECRUITING
London
Contact Information
Primary
Richard Friend, MD
r.friend@nucleusnetwork.com.au
+61 403 415 925
Time Frame
Start Date:2023-08-07
Estimated Completion Date:2026-09-30
Participants
Target number of participants:50
Treatments
Experimental: Phase 1 - Active ADX-038 administered to HP
For each cohort in Phase 1 (SAD), 8 participants will be randomized in a 3:1 ratio; 6 participants to active (ADX-038): 2 participants to control (matched placebo). Randomization will be on Day 1. Initially, 2 sentinel participants (1 active and 1 placebo) will be randomized and dosed. The sentinel participants will be evaluated for safety. The investigator's assessment and the independent medical monitor will decide upon the randomization and dosing of the 6 remaining participants (5 active and 1 placebo) according to the randomization schedule.
Placebo_comparator: Phase 1- Placebo administered to HP
For each cohort in Phase 1 (SAD), 8 participants will be randomized in a 3:1 ratio; 6 participants to active (ADX-038): 2 participants to control (matched placebo). Randomization will be on Day 1. Initially, 2 sentinel participants (1 active and 1 placebo) will be randomized and dosed. The sentinel participants will be evaluated for safety. The investigator's assessment and the independent medical monitor will decide upon the randomization and dosing of the 6 remaining participants (5 active and 1 placebo) according to the randomization schedule.
Experimental: Phase 2a - ADX-038 administered to PNH participants
This will be initiated at the dose level determined by the Safety Review Committee from SAD in HPs. The treatment of PNH participants is an open-label study.