A Phase 1 interventional study of ALXN1102 and ALXN1103 in Paroxysmal Nocturnal Hemoglobinuria (PNH), sponsored by Alexion Pharmaceuticals, Inc.. Terminated at 5 sites in 5 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2014-05-22.
Sponsored by Alexion Pharmaceuticals, Inc. · Phase 1, Interventional, and Treatment
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.
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.
107 studies on the registry are indexed under Hemoglobinuria; 10 are open to participants now.
This study's enrollment of 10 is below the median of 27 across 89 interventional studies indexed under Hemoglobinuria.
Browse Hemoglobinuria studies →Alexion Pharmaceuticals, Inc. is the lead sponsor of 249 studies on the registry; 25 are open to participants now.
Of its 98 completed or terminated interventional studies of FDA-regulated products, 70 (71%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
IV: 0.1, 0.3, and 1.0 mg/kg
Drug: ALXN1102
IV: 3.0, 6.0, and 10.0 mg/kg SC: 1.0 and 3.0 mg/kg
Drug: ALXN1103
Single Ascending Dose IV
Single Ascending Dose IV or SC
Assess the safety and tolerability of a single dose of TT30.
Safety and tolerability will be evaluated in all subjects by physical exam, vital signs, ECGs, laboratory changes over time, adverse events, and antibody development.
Time frame: 60 days
Characterize PK, PD and immunogenicity of a single dose of TT30.
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.
Time frame: 60 days
This study is terminated, as verified in May 2014. You cannot join it, but the record below documents what was studied.
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Alexion Pharmaceuticals, Inc.