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

May 21, 2014 updated by: Alexion Pharmaceuticals

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

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

10

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Czech Republic
    • Prague 2
      • U nemocnice 1, Prague 2, Czech Republic, 128 08
        • Institute of Hematology and Blood Transfusion / Institut Hematologie a krevní transfuze
  • Italy
    • Naples
      • Via Pansini 5, Naples, Italy, 80131
        • Federico II University of Naples
  • Poland
    • Gdańsk
      • Dębinki 7, Gdańsk, Poland, 80-952
        • University Clinical Centre
  • United Kingdom
    • London
      • Denmark Hill, London, United Kingdom, SE5 9RS UK
        • King's College Hospital NHS Foundation Trust
  • United States
    • California
      • Los Angeles, California, United States, 90033
        • USC/Norris Comprehensive Cancer Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: TT30 (ALXN1102 Formulation)
IV: 0.1, 0.3, and 1.0 mg/kg
Drug: ALXN1102
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
Drug: ALXN1103
Single Ascending Dose IV or SC

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
60 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
60 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alexion Pharmaceuticals

Investigators

  • Study Director: Bill Lundberg, MD, Alexion Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

April 1, 2011

Primary Completion (Actual)

March 1, 2014

Study Completion (Actual)

March 1, 2014

Study Registration Dates

First Submitted

April 12, 2011

First Submitted That Met QC Criteria

April 13, 2011

First Posted (Estimate)

April 14, 2011

Study Record Updates

Last Update Posted (Estimate)

May 22, 2014

Last Update Submitted That Met QC Criteria

May 21, 2014

Last Verified

May 1, 2014

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • TT30-PNH-002

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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