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

Phase 1

Terminated

• Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH)
• Interventions: Drug: ALXN1102; Drug: ALXN1103

• Registration Number: NCT01335165
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

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 Description

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.

Study Timeline

• Study Start: 2011-04
• Study First Submitted: 2011-04-12
• Study First Submitted QC: 2011-04-13
• Study First Posted: 2011-04-14
• Primary Completion: 2014-03
• Study Completion: 2014-03
• Status Verified: 2014-05
• Last Update Submitted: 2014-05-21
• Last Update Posted: 2014-05-22

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

• Individuals at least 18 years of age with a diagnosis of PNH & vaccination against meningococcus.

Exclusion Criteria

• Abnormal renal or liver function
• History of meningococcal disease
• History of Guillain-Barre syndrome
• Known infection with HIV or Hepatitis B or C
• History of thrombotic events

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
TT30 (ALXN1102 Formulation)	ALXN1102	IV: 0.1, 0.3, and 1.0 mg/kg
TT30 (ALXN1103 Formulation)	ALXN1103	IV: 3.0, 6.0, and 10.0 mg/kg SC: 1.0 and 3.0 mg/kg

Primary Outcome Measures

Name	Time	Method
Assess the safety and tolerability of a single dose of TT30.	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.

Secondary Outcome Measures

Name	Time	Method
Characterize PK, PD and immunogenicity of a single dose of TT30.	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.

Trial Locations

Locations (5)

University Clinical Centre; 🇵🇱 Dębinki 7, Gdańsk, Poland

Institute of Hematology and Blood Transfusion / Institut Hematologie a krevní transfuze; 🇨🇿 U nemocnice 1, Prague 2, Czech Republic

Federico II University of Naples; 🇮🇹 Via Pansini 5, Naples, Italy

USC/Norris Comprehensive Cancer Center; 🇺🇸 Los Angeles, California, United States

King's College Hospital NHS Foundation Trust; 🇬🇧 Denmark Hill, London, United Kingdom