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

ADARx Pharmaceuticals, Inc.8 个研究点分布在 2 个国家目标入组 50 人2023年8月7日

概览

简要总结

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).

详细描述

The clinical study described in this protocol is a Phase 1/Phase 2a study evaluating safety, tolerability, PK, and PD of ADX-038. The study consists of 2 parts: 1. Phase 1 - Randomized, double-blind, placebo-controlled, parallel group, single ascending dose (SAD) in HP with up to 5 dose cohorts. 2. Phase 2a - Open label, single-arm (ADX-038), 2 dose study in participants with paroxysmal nocturnal hemoglobinuria (PNH) and residual anemia on a standard-of-care (SOC) anti-C5 regimen of ravulizumab or eculizumab.

注册库

clinicaltrials.gov

开始日期

2023年8月7日

结束日期

2028年7月31日

最后更新

10天前

研究类型

Interventional

研究设计

Parallel

性别

All

研究者

发起方

ADARx Pharmaceuticals, Inc.

责任方

Sponsor

入排标准

入选标准

•Phase 1 Key Inclusion Criteria
•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
•Phase 1 Key

排除标准

•History of any significant medical conditions, except for completely excised non-melanoma skin cancer or low grade cervical intraepithelial neoplasia without evidence of recurrence within the prior 3 months
•Any viral, bacterial, parasitic, or fungal infection within the prior 30 days
•Frequent respiratory, nasopharyngeal or ear infections (more than 5 infections per year)
•History of environmental exposure or sick contact that increase the risk of meningococcal, pneumococcal and/or Haemophilus influenza type B infections
•Complement deficiency or immunodeficiency syndrome
•Major surgery or significant traumatic injury within the prior 3 months
•History of anaphylaxis or hypersensitivity reactions
•History of penicillin allergy
•History of splenectomy
•History of alcohol abuse or illicit drug use

研究组 & 干预措施

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.

干预措施: ADX-038

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.

干预措施: Placebo

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.

干预措施: ADX-038