Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase 1, Single Ascending Dose Study of the Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)|
- Assess the safety and tolerability of a single dose of TT30. [ Time Frame: 60 days ] [ Designated as safety issue: Yes ]Safety and tolerability will be evaluated in all subjects by physical exam, vital signs, ECGs, laboratory changes over time, adverse events, and antibody development.
- Characterize PK, PD and immunogenicity of a single dose of TT30. [ Time Frame: 60 days ] [ Designated as safety issue: No ]Immunogenicity will be will be assessed using standard measures for these parameters. PK and PD will be assessed by using standard measures, including reticulocyte count and lactate dehydrogenase (LDH) levels.
|Study Start Date:||April 2011|
|Study Completion Date:||March 2014|
|Primary Completion Date:||March 2014 (Final data collection date for primary outcome measure)|
Experimental: TT30 (ALXN1102 Formulation)
IV: 0.1, 0.3, and 1.0 mg/kg
Single Ascending Dose IV
Experimental: TT30 (ALXN1103 Formulation)
IV: 3.0, 6.0, and 10.0 mg/kg
SC: 1.0 and 3.0 mg/kg
Single Ascending Dose IV or SC
This is an open-label, single-dose, dose-escalation study to assess the safety, tolerability, PK, PD, and immunogenicity of TT30 given as an IV infusion and as a SC injection in subjects with PNH or evidence of circulating PNH cells.
Eligible subjects with PNH will be vaccinated with meningococcal vaccine at least two weeks prior to dosing (if not previously vaccinated or if revaccination is required).
On study day 1, the subject will receive the single dose of TT30 as an IV infusion over 60 minutes or as a SC injection, and be followed with additional study evaluations over the next 59 days.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01335165
|United States, California|
|USC/Norris Comprehensive Cancer Center|
|Los Angeles, California, United States, 90033|
|Institute of Hematology and Blood Transfusion / Institut Hematologie a krevní transfuze|
|U nemocnice 1, Prague 2, Czech Republic, 128 08|
|Federico II University of Naples|
|Via Pansini 5, Naples, Italy, 80131|
|University Clinical Centre|
|Dębinki 7, Gdańsk, Poland, 80-952|
|King's College Hospital NHS Foundation Trust|
|Denmark Hill, London, United Kingdom, SE5 9RS UK|
|Study Director:||Bill Lundberg, MD||Alexion Pharmaceuticals|