Trial record 8 of 39 for:    "Paroxysmal nocturnal hemoglobinuria"

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

This study is currently recruiting participants.
Verified August 2012 by Alexion Pharmaceuticals
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01335165
First received: April 12, 2011
Last updated: November 5, 2012
Last verified: August 2012
  Purpose

The present study represents the first time in human (FTIH) trial for TT30 and is being performed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamic (PD) [ex vivo CAP and CCP activities] and immunogenicity of single IV or SC doses of TT30 in subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH) or evidence of circulating PNH cells.


Condition Intervention Phase
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Drug: TT30 IV
Drug: TT30 SC
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: One year ] [ 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: One year ] [ Designated as safety issue: No ]
    Immunogenicity (PK, PD) will be assessed using standard measures for these parameters.


Estimated Enrollment: 24
Study Start Date: December 2010
Estimated Study Completion Date: February 2013
Estimated Primary Completion Date: December 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Cohort 1
0.1. mg/kg
Drug: TT30 IV
single-ascending dose
Experimental: Cohort 2
0.3 mg/kg
Drug: TT30 IV
single-ascending dose
Experimental: Cohort 3
1.0 mg/kg
Drug: TT30 IV
single-ascending dose
Experimental: Cohort 4
3.0 mg/kg
Drug: TT30 IV
single-ascending dose
Experimental: Cohort 5
5.0 mg/kg
Drug: TT30 IV
single-ascending dose
Experimental: Cohort 1-SC
1.0 mg/kg
Drug: TT30 SC
single-ascending dose
Experimental: Cohort 2-SC
3.0 mg/kg
Drug: TT30 SC
single-ascending dose

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.

Any cohort may be expanded by up to 3 subjects for purposes of safety evaluation based on specific criteria defined below, and a further 6 subjects may be added to a cohort to obtain additional safety and PK data. All subjects will sign informed consent and be screened for eligibility within 28 days of receiving the single dose of TT30. Once a subject is determined to be eligible for the study, the subject will be vaccinated with meningococcal vaccine at least two weeks prior to dosing (if not previously vaccinated; if revaccination is recommended per the manufacturer's guideline, or if vaccinated greater than 3 years ago; or if subjects have been previously vaccinated, but there is no adequate documentation to verify prior vaccination).

On Study day 1, the day of TT30 dosing, the subject will come to the GCRC or clinic for the required testing prior to dosing. Once the testing is complete the subject will receive the single dose of TT30 as an IV infusion over 60 minutes or as an SC injection. The subject will be observed for any untoward effects and the required study evaluations will be completed for the remainder of Study day 1. Subjects may leave the clinic after all required evaluations are complete and they have been observed for at least 8 hours following the completion of the IV infusion of TT30.

Subjects will return for the required 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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01335165

Locations
United States, California
USC/Norris Comprehensive Cancer Center Recruiting
Los Angeles, California, United States, 90033
Contact: Laurie Hornor, RN    323-864-3978    Laurie.Hornor@med.usc.edu   
Principal Investigator: Ilene Weitz, MD         
Czech Republic
Recruiting
Prague, Czech Republic
Contact: Alexion Pharmaceuticals       burkel@alxn.com   
Italy
Recruiting
Naples, Italy
Contact: Alexion Pharmaceuticals       burkel@alxn.com   
Poland
Recruiting
Gdansk, Poland
Contact: Alexion Pharmaceuticals       burkel@alxn.com   
United Kingdom
Recruiting
London, United Kingdom
Contact: Alexion Pharmaceuticals       burkel@alxn.com   
Sponsors and Collaborators
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: November 5, 2012
Health Authority: United States: Food and Drug Administration

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 April 17, 2014