Clinical Trial: Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

Official Title: A Phase 1, Single Ascending Dose Study of the Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Brief Summary: The purpose of this research study is to understand the safety, pharmacokinetics and pharmacodynamics of a single dose of TT30 (ALXN1102 and ALXN1103 formulations) when given IV (through a vein) or SC (under the skin) to patients with PNH.

Detailed Summary:

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.


Sponsor: Alexion Pharmaceuticals

Current Primary Outcome: Assess the safety and tolerability of a single dose of TT30. [ Time Frame: 60 days ]

Safety and tolerability will be evaluated in all subjects by physical exam, vital signs, ECGs, laboratory changes over time, adverse events, and antibody development.


Original Primary Outcome: Assess the safety and tolerability of a single dose of TT30. [ Time Frame: One year ]

Safety and tolerability will be evaluated in all subjects by physical exam, vital signs, ECGs, laboratory changes over time, adverse events, and antibody development.


Current Secondary Outcome: Characterize PK, PD and immunogenicity of a single dose of TT30. [ Time Frame: 60 days ]

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.


Original Secondary Outcome: Characterize PK, PD and immunogenicity of a single dose of TT30. [ Time Frame: One year ]

Immunogenicity (PK, PD) will be assessed using standard measures for these parameters.


Information By: Alexion Pharmaceuticals

Dates:
Date Received: April 12, 2011
Date Started: April 2011
Date Completion:
Last Updated: May 21, 2014
Last Verified: May 2014