Clinical Trials Logo
  1. Home
  2. Other
  3. NCT05876312

Safety, Tolerability, PK and PD of ADX-038 in HV and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients — PNH  

Paroxysmal Nocturnal Hemoglobinuria (PNH) Clinical Trial

Official title:
A Phase 1, Randomized, Placebo-Controlled, Double Blind Single Ascending Dose Study in Healthy Volunteers Followed by Open Label Treatment in Patients With PNH to Evaluate the Safety, Tolerability, PK and PD of ADX-038

Clinical Trial 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).

Clinical Trial Description

The clinical study described in this protocol is a Phase 1, single-center study evaluating safety, tolerability, PK, and PD of ADX-038. The study consists of 2 parts: 1. Part A - Randomized, double-blind, placebo-controlled, parallel group, single ascending dose (SAD) in HV with up to 5 dose cohorts. 2. Part B - Expansion cohort in participants with paroxysmal nocturnal hemoglobinuria (PNH) at selected dose from Part A and will be open label. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05876312
Study type Interventional
Source ADARx Pharmaceuticals, Inc.
Contact Richard Friend, MD
Phone +61 403 415 925
Email r.friend@nucleusnetwork.com.au
Status Recruiting
Phase Phase 1
Start date July 3, 2023
Completion date June 30, 2025
View Details
See also
  Status Clinical Trial Phase
Completed NCT00004143 - Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes Phase 2
Terminated NCT05116787 - BCX9930 for the Treatment of PNH in Subjects Not Receiving Other Complement Inhibitor Therapy Phase 2
Recruiting NCT06294301 - A Study of Single and Multiple Doses of LP-005 in Healthy Adult Participants Phase 1
Completed NCT03472885 - Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab Phase 2
Completed NCT03946748 - Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Phase 2
Enrolling by invitation NCT03427060 - Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms Phase 2
Completed NCT03078582 - Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Phase 2
Completed NCT03053102 - Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Phase 2
Not yet recruiting NCT06312644 - Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
Completed NCT03588026 - Treating Paroxysmal Nocturnal Haemoglobinuria Patients With rVA576 Phase 3
Completed NCT03056040 - ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab Phase 3
Completed NCT00145613 - Haploidentical Stem Cell Transplant for Treatment Refractory Hematological Malignancies Phase 2
Completed NCT00587054 - Trial of Allogeneic Stem Cell Transplants From HLA Compatible, Related and Unrelated Donors After a Myeloablative Preparative Regimen With Hyperfractionated TBI, Thiotepa and Fludarabine For Adult Patients With Lymphohematopoietic Disorders Phase 2
Completed NCT04820530 - Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy Phase 3
Terminated NCT03225287 - Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical Study Phase 2
Completed NCT04558918 - Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment Phase 3
Completed NCT02352493 - A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNH Phase 1/Phase 2
Recruiting NCT04901936 - A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Phase 2
Completed NCT02264639 - A Phase I Study to Assess the Safety of Pegcetacoplan (APL-2) as an Add-On to Standard of Care in Subjects With PNH Phase 1
Recruiting NCT03520647 - Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis Phase 2