Study to Assess PXL770 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)
NCT05146284 · Status: WITHDRAWN · Phase: PHASE2 · Type: INTERVENTIONAL
Last updated 2024-12-16
Summary
A randomized open-label Phase 2a study to assess the pharmacokinetics and pharmacodynamic parameters of PXL770 after 12 weeks of treatment in male subjects with adrenomyeloneuropathy (AMN).
Conditions
- Adrenomyeloneuropathy
Interventions
- DRUG
-
PXL770
Tablet
Sponsors & Collaborators
-
Poxel SA
lead INDUSTRY
Study Design
- Allocation
- RANDOMIZED
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- PARALLEL
Eligibility
- Min Age
- 18 Years
- Max Age
- 65 Years
- Sex
- MALE
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2023-09-30
- Primary Completion
- 2024-09-30
- Completion
- 2024-09-30
- FDA Drug
- Yes
More Related Trials
-
Pediatric Patients Aged 1 to 6 Years With APDS
NCT05693129 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3
-
Anabolic Steroids for Nutritional Rehabilitation of Critically Ill Patients
NCT00242463 ·Status: TERMINATED ·Phase: PHASE2
-
Effects and Safety of Infusion of Low-Doses of Methylprednisolone in Early ALI and ARDS in Children
NCT01757899 ·Status: WITHDRAWN ·Phase: PHASE2
-
Dose Escalation, MTD, Safety and PK Study of a Single Dose SC Injection of TransCon PEG Treprostinil in Healthy Male Volunteers
NCT02149095 ·Status: WITHDRAWN ·Phase: PHASE1
-
Pediatric Patients Aged 4 to 11 Years With APDS
NCT05438407 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3
-
Intraumbilical Amino Acids and Glucose Supplementation Via Port by Severe IUGR in Human Fetuses
NCT02596594 ·Status: COMPLETED ·Phase: NA
-
Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy
NCT00004418 ·Status: TERMINATED ·Phase: PHASE2
-
Study to Find the Highest Safe Dose of Soluble Guanylate Cyclase (sGC) Activator, BAY 1211163 Administered as Multiple Doses by Inhalation to Patients Who Cannot Breathe by Their Own and Suffer From a Type of Lung Failure That Causes Fluid to Build up in the Lungs Making Breathing Difficult (ARDS)
NCT04609943 ·Status: TERMINATED ·Phase: PHASE1
-
L-citrulline for Prevention of Sequelae of Acute Lung Injury in Pediatrics Undergoing Cardiopulmonary Bypass for Heart Defects
NCT02891837 ·Status: COMPLETED ·Phase: PHASE3
-
ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome
NCT04304482 ·Status: COMPLETED ·Phase: PHASE2/PHASE3
-
Investigation of the Efficacy and Safety of Drotrecogin Alfa (Activated) in Pediatric Severe Sepsis
NCT00049764 ·Status: COMPLETED ·Phase: PHASE3
-
Phase 2c Dose Comparison Study of MP4OX in Trauma
NCT01973504 ·Status: WITHDRAWN ·Phase: PHASE2
-
Staccato Zaleplon Single Dose Pharmacokinetics
NCT00620620 ·Status: COMPLETED ·Phase: PHASE1
-
Ascending Single Dose Study of Rhu-pGelsolin in Patients With Decreased Gelsolin Levels
NCT00671307 ·Status: COMPLETED ·Phase: PHASE1/PHASE2
-
Methoxsalen and Extracorporeal Photopheresis (ECP) for the Treatment of Pediatric Participants With Steroid Refractory Acute Graft Versus Host Disease
NCT02524847 ·Status: TERMINATED ·Phase: PHASE3
-
Study to Evaluate Safety and Dosimetry of Lutathera in Adolescent Patients With GEP-NETs and PPGLs
NCT04711135 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE2
-
Safety and Tolerance Study of Recombinant Human Albumin Injection in Healthy Subjects.
NCT04641364 ·Status: COMPLETED ·Phase: PHASE1
-
Therapeutic Use of Oral Sodium Phosphate (Z-521) in Primary Hypophosphatemic Rickets
NCT01237288 ·Status: COMPLETED ·Phase: PHASE3
-
Comparative Phase 3 Trial to Assess the Safety and Efficacy of Triple-chamber Parenteral Nutrition Formulation
NCT01579097 ·Status: COMPLETED ·Phase: PHASE3
-
Systematic Early Use of Neuromuscular Blocking Agents in ARDS Patients
NCT00299650 ·Status: COMPLETED ·Phase: PHASE4
-
Plasma Exchange for Amanita Toxin-induced Acute Liver Failure
NCT06187220 ·Status: COMPLETED
-
Alpha Lipoic Acid as an Adjuvant Treatment in Acute Phosphide Poisoning
NCT02760823 ·Status: UNKNOWN ·Phase: PHASE2
-
Infusion of Prostacyclin vs Placebo for 72-hours in Trauma Patients With Haemorrhagic Shock Suffering From Organ Failure
NCT03903939 ·Status: COMPLETED ·Phase: PHASE2
-
Safety and Efficacy Study of Acthar in Subjects With ARDS
NCT02113735 ·Status: WITHDRAWN ·Phase: PHASE2
-
Efficacy and Safety of Venetoclax Combined With Dexamethasone and Etoposide in HLH
NCT05546060 ·Status: UNKNOWN ·Phase: PHASE1