Study Stopped
Inability to reach enrollment as planned
Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
A Phase 1, Single Ascending Dose Study of the Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
1 other identifier
interventional
10
5 countries
5
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_1
Started Apr 2011
Typical duration for phase_1
5 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
April 1, 2011
CompletedFirst Submitted
Initial submission to the registry
April 12, 2011
CompletedFirst Posted
Study publicly available on registry
April 14, 2011
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2014
CompletedStudy Completion
Last participant's last visit for all outcomes
March 1, 2014
CompletedMay 22, 2014
May 1, 2014
2.9 years
April 12, 2011
May 21, 2014
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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
Secondary Outcomes (1)
Characterize PK, PD and immunogenicity of a single dose of TT30.
60 days
Study Arms (2)
TT30 (ALXN1102 Formulation)
EXPERIMENTALIV: 0.1, 0.3, and 1.0 mg/kg
TT30 (ALXN1103 Formulation)
EXPERIMENTALIV: 3.0, 6.0, and 10.0 mg/kg SC: 1.0 and 3.0 mg/kg
Interventions
Eligibility Criteria
You may qualify if:
- Individuals at least 18 years of age with a diagnosis of PNH \& vaccination against meningococcus.
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (5)
USC/Norris Comprehensive Cancer Center
Los Angeles, California, 90033, United States
Institute of Hematology and Blood Transfusion / Institut Hematologie a krevní transfuze
U Nemocnice 1, Prague 2, 128 08, Czechia
Federico II University of Naples
Via Pansini 5, Naples, 80131, Italy
University Clinical Centre
Dębinki, Gdańsk, 80-952, Poland
King's College Hospital NHS Foundation Trust
Denmark Hill, London, SE5 9RS UK, United Kingdom
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Bill Lundberg, MD
Alexion Pharmaceuticals, Inc.
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 12, 2011
First Posted
April 14, 2011
Study Start
April 1, 2011
Primary Completion
March 1, 2014
Study Completion
March 1, 2014
Last Updated
May 22, 2014
Record last verified: 2014-05