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Safety, Pharmacokinetic, and Pharmacodynamic Study of NKTT120 in Adult Patients With Stable Sickle Cell Disease (SCD)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01783691
Recruitment Status : Completed
First Posted : February 5, 2013
Last Update Posted : June 26, 2015
Sponsor:
Information provided by (Responsible Party):
NKT Therapeutics

Study Description
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Brief Summary:
The purpose of this study is to determine the safety, pharmacokinetics, and pharmacodynamics of NKTT120 in adult patients with stable sickle cell disease.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: NKTT120 Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 21 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Single Ascending Dose Phase 1 Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Biologic Activity of Intravenous NKTT120 in Adults With Stable Sickle Cell Disease
Study Start Date : February 2013
Actual Primary Completion Date : May 2015
Actual Study Completion Date : May 2015

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: NKTT120 Drug: NKTT120


Outcome Measures
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Primary Outcome Measures :
  1. Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs and (4) physical exam. [ Time Frame: 180 days ]

Secondary Outcome Measures :
  1. Pharmacokinetics assessed by plasma drug concentration levels [ Time Frame: 0; 15, 30, 60 minutes; 3,6, hours; 1,2,3,7,14,30,60,90 days ]
  2. Pharmacodynamics as measured by assessments of peripheral blood lymphocyte subsets [ Time Frame: 0, 0.25, 1, 2, 7, 14, 30, 60, 90, 120 days ]

Other Outcome Measures:
  1. Pain scores as assessed by daily electronic diary [ Time Frame: Daily for 120 days ]
  2. Quality of Life as assessed by two separate Quality of Life instruments [ Time Frame: 0, 30, 60, 90, 120 days ]
  3. Lung function as measured by pulmonary function tests [ Time Frame: 0; 30 minutes; 30, 60, 90, 120 days ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age 18 to 50 years
  • Subject has a confirmed diagnosis of HbSS or HbSβ0thal
  • Subject has stable SCD defined as not having acute VOC, ACS, or other major SCD associated event during the month prior to enrollment

Exclusion Criteria:

  • Subject had an SCD-related VOC or ACS that required hospitalization or treatment in acute care outpatient setting in the month prior to enrollment
  • Subject requires a program of prescheduled regularly administered packed red blood cell (pRBC) transfusions or Subject received a pRBC transfusion in the month prior to enrollment
  • Subject has evidence of latent or active tuberculosis
  • Subject has a major concurrent illness or medical condition
  • Subject is pregnant or nursing
Contacts and Locations
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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): NCT01783691


Locations
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United States, California
Children's Hospital & Research Center at Oakland
Oakland, California, United States, 94609
United States, Maryland
Johns Hopkins School of Medicine
Baltimore, Maryland, United States, 21205
United States, Massachusetts
Brigham and Women's Hospital
Boston, Massachusetts, United States, 02115
United States, Missouri
Washington University School of Medicine
St. Louis, Missouri, United States, 63110
United States, North Carolina
UNC Sickle Cell Program
Chapel Hill, North Carolina, United States, 27514
United States, Wisconsin
Blood Center of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Sponsors and Collaborators
NKT Therapeutics
Investigators
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Study Director: Rosemary Mazanet, MD NKT Therapeutics
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: NKT Therapeutics
ClinicalTrials.gov Identifier: NCT01783691    
Other Study ID Numbers: 120-SCD1
First Posted: February 5, 2013    Key Record Dates
Last Update Posted: June 26, 2015
Last Verified: June 2015
Keywords provided by NKT Therapeutics:
Sickle cell disease
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
 Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn