A Phase 1, Randomized, Double-Blind, Two-Arm, Placebo-Controlled, Single and Multiple Dose Escalation Study to Assess the Safety and Tolerability of LJPC-401 in Healthy Adults
Completed
- Conditions
- hereditary haemochromatosisthalassemiasickle cell diseasemyelodysplasia (MDS)
- Registration Number
- ACTRN12617000689370
- Lead Sponsor
- a Jolla Pharmaceutical Australia Pty Ltd, an affiliate of La Jolla Pharmaceutical Company
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- All
- Target Recruitment
- 36
Inclusion Criteria
Not provided
Exclusion Criteria
Not provided
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Related Research Topics
Explore scientific publications, clinical data analysis, treatment approaches, and expert-compiled information related to the mechanisms and outcomes of this trial. Click any topic for comprehensive research insights.
What molecular mechanisms does LJPC-401 target in the treatment of hereditary haemochromatosis and other iron overload disorders?
How does the safety profile of LJPC-401 compare to standard-of-care treatments for thalassemia and sickle cell disease?
Are there specific biomarkers that can predict patient response to LJPC-401 in iron metabolism-related conditions?
What are the potential adverse events associated with LJPC-401 and how do they compare to other iron chelators like deferoxamine or deferiprone?
How does LJPC-401's mechanism of action differ from existing therapies for myelodysplastic syndromes and what combination approaches might enhance its efficacy?