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

NCT ID: NCT01335165

Last Updated: 2014-05-22

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE1
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-04-30
• Study Completion Date: 2014-03-31

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.

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

TT30 (ALXN1102 Formulation)

IV: 0.1, 0.3, and 1.0 mg/kg

Group Type: EXPERIMENTAL

ALXN1102

Intervention Type: DRUG

Single Ascending Dose IV

TT30 (ALXN1103 Formulation)

IV: 3.0, 6.0, and 10.0 mg/kg

SC: 1.0 and 3.0 mg/kg

Group Type: EXPERIMENTAL

ALXN1103

Intervention Type: DRUG

Single Ascending Dose IV or SC

Interventions

ALXN1102

Single Ascending Dose IV

Intervention Type: DRUG

ALXN1103

Single Ascending Dose IV or SC

Intervention Type: DRUG

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Alexion Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Bill Lundberg, MD

Role: STUDY_DIRECTOR

Alexion Pharmaceuticals, Inc.

Locations

USC/Norris Comprehensive Cancer Center

Los Angeles, California, United States

Institute of Hematology and Blood Transfusion / Institut Hematologie a krevní transfuze

U Nemocnice 1, Prague 2, Czechia

Federico II University of Naples

Via Pansini 5, Naples, Italy

University Clinical Centre

Dębinki, Gdańsk, Poland

King's College Hospital NHS Foundation Trust

Denmark Hill, London, United Kingdom

Countries

United States; Czechia; Italy; Poland; United Kingdom

Other Identifiers

TT30-PNH-002

Identifier Type: -

Identifier Source: org_study_id