Overview
Safety, Tolerability, PK and PD of ADX-038 in HV and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
Status:
Not yet recruiting
Not yet recruiting
Trial end date:
2025-06-30
2025-06-30
Target enrollment:
0
0
Participant gender:
All
All
Summary
The first-in-human Phase 1 study described herein will evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ADX-038 in both healthy volunteers (HV) and in patients with paroxysmal nocturnal hemoglobinuria (PNH).Phase:
Phase 1Accepts Healthy Volunteers?
Accepts Healthy VolunteersDetails
Lead Sponsor:
ADARx Pharmaceuticals, Inc.Collaborator:
ADARx Australia Pty Ltd
Criteria
Inclusion Criteria1. Male and female adults 18 to 55 years old
2. Body mass index (BMI) between 18 and 32 kg/m2
3. Contraception use by men or women should be consistent with local regulations
regarding the methods of contraception for those participating in clinical studies
4. Willing and able to provide informed consent and comply with all study visits and
procedures
5. Negative urine drug, nicotine/tobacco, and breath alcohol test result
6. Neisseria meningitis vaccination
7. Pneumococcus vaccination
8. Hemophilus influenzae vaccination Exclusion Criteria
9. Any significant medical history
10. Active malignancy and/or history of malignancy in the past 5 years
11. History of liver disease, Gilbert's syndrome, or abnormal liver function test
12. Estimated creatinine clearance <60 mL/min using the Cockcroft-Gault formula
13. Any active infection or acute illness
14. History of meningococcal infection or frequent respiratory, nasopharyngeal or ear
infections
15. History of previous or current tuberculosis infection.
16. Prior splenectomy
17. Major surgery or significant traumatic injury occurring within 3 months
18. Have any other conditions that, in the opinion of the Investigator or Sponsor, would
make the participant unsuitable for inclusion
19. Positive serology tests for human immunodeficiency virus hepatitis B surface antigen
or hepatitis C virus
20. Use of any prescription, vaccines, supplements/vitamins, or over-the counter
medication
21. Treatment with another investigational product within 30 days
22. Known any clinically significant allergic reactions
23. Known hypersensitivity to any of the study drug ingredients or penicillin.
24. History or presence of alcohol
25. Blood donation
26. Pregnancy
27. May have a higher risk to be exposed to infected individuals, for example active
healthcare employees.
Criteria (Part B) Inclusion Criteria
28. Male and female adults 18-65 years old
29. Confirmed diagnosis of PNH based on documented clone size of PNH blood cells by flow
cytometry.
30. Serum LDH levels are at least 1.25-fold above the ULN for non-treated participants
31. Liver function test values are less than 2x ULN
32. Mean hemoglobin (Hb) <12 g/dL.
33. A history of red blood cell transfusion within at least 3 months
34. Body mass index (BMI) between 18 and 32 kg/m2, inclusive, at screening.
35. Contraception use by men or women should be consistent with local regulations
regarding the methods of contraception for those participating in clinical studies.
36. Willing and able to provide informed consent and comply with all study visits and
procedures
37. Neisseria meningitis vaccination
38. Pneumococcus vaccination
39. Hemophilus influenzae vaccination
Exclusion Criteria
40. Known or suspected hereditary or acquired complement deficiency
41. History of clinically significant arterial or venous thrombosis
42. History of hematopoietic stem cell transplantation
43. History of meningococcal infection
44. Any significant medical history
45. Active malignancy and/or history of malignancy in the past 5 years
46. Any active viral, bacterial, parasitic, or fungal infection or acute illness
47. Any evidence of sero-positive autoimmune connective tissue diseases
48. Any evidence of active inflammatory conditions
49. History of previous or current tuberculosis infection.
50. Prior splenectomy
51. Major surgery or significant traumatic injury occurring within 3 months
52. Have any other conditions that, in the opinion of the Investigator or Sponsor, would
make the participant unsuitable for inclusion
53. Inadequate organ function
54. Positive serology tests for human immunodeficiency virus hepatitis B surface antigen
or hepatitis C virus
55. Willing to continue after enrollment with their current treatment with a complement
inhibitor.
56. Use of vaccines, or changes in any prescription, supplements/vitamins, or over-the
counter medication
57. Treatment with another investigational product or biologic agent within 30 days
58. Blood donation within 30 days
59. Pregnancy