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

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ClinicalTrials.gov Identifier: NCT01335165
Recruitment Status : Terminated (Inability to reach enrollment as planned)
First Posted : April 14, 2011
Last Update Posted : May 22, 2014
Information provided by (Responsible Party):
Alexion Pharmaceuticals

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.

Condition or disease Intervention/treatment Phase
Paroxysmal Nocturnal Hemoglobinuria (PNH) Drug: ALXN1102 Drug: ALXN1103 Phase 1

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  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (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)
Study Start Date : April 2011
Primary Completion Date : March 2014
Study Completion Date : March 2014

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

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. 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.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01335165

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
Federico II University of Naples
Via Pansini 5, Naples, Italy, 80131
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
Study Director: Bill Lundberg, MD Alexion Pharmaceuticals

Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01335165     History of Changes
Other Study ID Numbers: TT30-PNH-002
First Posted: April 14, 2011    Key Record Dates
Last Update Posted: May 22, 2014
Last Verified: May 2014

Keywords provided by Alexion Pharmaceuticals:

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