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

Paroxysmal Nocturnal Hemoglobinuria (PNH) Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01335165
• Other study ID #: TT30-PNH-002
• Status: Terminated
• Phase: Phase 1
• First received: April 12, 2011
• Last updated: May 21, 2014
• Start date: April 2011
• Est. completion date: March 2014

Study information

• Verified date: May 2014
• Source: Alexion Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationItaly: Instituto Superiore di SanitaPoland: Office for Registration of Medicinal Products, Medical Devices and Biological ProductsUnited Kingdom: Medicines and Healthcare Products Regulatory AgencyCzech Republic: State Institute for Drug Control
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 10
• Est. completion date: March 2014
• Est. primary completion date: March 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hemoglobinuria
• Hemoglobinuria, Paroxysmal
• Paroxysmal Nocturnal Hemoglobinuria (PNH)

Intervention

Drug:
• ALXN1102
Single Ascending Dose IV
• ALXN1103
Single Ascending Dose IV or SC

Locations

Country	Name	City	State
Czech Republic	Institute of Hematology and Blood Transfusion / Institut Hematologie a krevní transfuze	U nemocnice 1	Prague 2
Italy	Federico II University of Naples	Via Pansini 5	Naples
Poland	University Clinical Centre	Debinki 7	Gdansk
United Kingdom	King's College Hospital NHS Foundation Trust	Denmark Hill	London
United States	USC/Norris Comprehensive Cancer Center	Los Angeles	California

Sponsors (1)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals

Countries where clinical trial is conducted

United States,  Czech Republic,  Italy,  Poland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	60 days	Yes
Secondary	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.	60 days	No