Actively Recruiting
Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
Led by ADARx Pharmaceuticals, Inc. · Updated on 2026-04-22
50
Participants Needed
4
Research Sites
260 weeks
Total Duration
On this page
Sponsors
A
ADARx Pharmaceuticals, Inc.
Lead Sponsor
A
ADARx Australia Pty Ltd
Collaborating Sponsor
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official Title
Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Phase 1 participants must be 18 to 55 years old
- Phase 1 participants must be healthy based on medical evaluation
- Participants must have recent meningococcal, pneumococcal, and Haemophilus influenzae type B vaccinations or be willing to be vaccinated
- Screening tests must be negative for illicit drug, nicotine, and alcohol use
- Phase 2a participants must be at least 18 years old
- Phase 2a participants must have a diagnosis of paroxysmal nocturnal hemoglobinuria based on documented clone size
- Phase 2a participants must have hemoglobin concentration less than 12 g/dL
- Phase 2a participants must be on a stable anti-C5 regimen for at least 12 weeks before Day 1
You will not qualify if you...
- History of significant medical conditions except excised non-melanoma skin cancer or low grade cervical intraepithelial neoplasia without recent recurrence
- Any viral, bacterial, parasitic, or fungal infection within 30 days for Phase 1 or 14 days for Phase 2a
- Frequent respiratory, nasopharyngeal, or ear infections (more than 5 per year)
- History of environmental exposure increasing risk of certain infections
- Complement deficiency or immunodeficiency syndrome
- Major surgery or significant traumatic injury within 3 months
- History of anaphylaxis or hypersensitivity reactions
- History of penicillin allergy
- History of splenectomy
- History of alcohol abuse or illicit drug use
- Recent blood or plasma donation within specified timeframes
- Abnormal screening laboratory or vital sign results
- Clinically significant ECG findings
- Pregnant or lactating females
- Recent use of prescription or over-the-counter medications except specified exceptions
- Use of investigational therapeutics within 30 days or expected washout period
- Unwilling or unable to adhere to study-related prophylactic antibiotics
- History of HIV, active hepatitis B or C infection
- History of meningococcal or tuberculosis infection
- History of malignancy in past 5 years except as specified
- History of hematopoietic stem cell transplantation
- Inflammatory bowel disease, systemic lupus erythematosus, rheumatoid arthritis, or chronic liver disease
- Clinically significant and uncontrolled medical conditions such as thromboembolic disease, acute coronary syndrome, and diabetes
- Alcohol consumption restrictions before dosing in Phase 2a
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 4 locations
1
Nucleus Network Brisbane
Brisbane, Queensland, Australia, 4006
Active, Not Recruiting
2
Peter MacCallum Cancer Centre
Melbourne, Victoria, Australia, 3000
Not Yet Recruiting
3
Royal Melbourne Hospital
Parkville, Victoria, Australia, 3052
Actively Recruiting
4
Richmond Pharmacology Ltd
London, United Kingdom, SE1 1YR
Active, Not Recruiting
Research Team
L
Lyle Gee
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
TRIPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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