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

This study has been terminated.
(Inability to reach enrollment as planned)
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01335165
First received: April 12, 2011
Last updated: May 21, 2014
Last verified: May 2014
  Purpose

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.


Condition Intervention Phase
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Drug: ALXN1102
Drug: ALXN1103
Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase 1, Single Ascending Dose Study of the Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Resource links provided by NLM:


Further study details as provided by Alexion Pharmaceuticals:

Primary Outcome Measures:
  • Assess the safety and tolerability of a single dose of TT30. [ Time Frame: 60 days ] [ Designated as safety issue: Yes ]
    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:
  • Characterize PK, PD and immunogenicity of a single dose of TT30. [ Time Frame: 60 days ] [ Designated as safety issue: No ]
    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.


Enrollment: 10
Study Start Date: April 2011
Study Completion Date: March 2014
Primary Completion Date: March 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01335165

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

No publications provided

Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01335165     History of Changes
Other Study ID Numbers: TT30-PNH-002
Study First Received: April 12, 2011
Last Updated: May 21, 2014
Health Authority: United States: Food and Drug Administration
Italy: Instituto Superiore di Sanita
Poland: Office for Registration of Medicinal Products, Medical Devices and Biological Products
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Czech Republic: State Institute for Drug Control

Keywords provided by Alexion Pharmaceuticals:
PNH

Additional relevant MeSH terms:
Hemoglobinuria
Hemoglobinuria, Paroxysmal
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms
Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases

ClinicalTrials.gov processed this record on August 28, 2014