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

Primary Purpose

Sickle Cell Disease

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
NKTT120
Sponsored by
NKT Therapeutics
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Sickle Cell Disease focused on measuring Sickle cell disease

Eligibility Criteria

18 Years - 60 Years (Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Children's Hospital & Research Center at Oakland
  • Johns Hopkins School of Medicine
  • Brigham and Women's Hospital
  • Washington University School of Medicine
  • UNC Sickle Cell Program
  • Blood Center of Wisconsin

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

NKTT120

Arm Description

Outcomes

Primary Outcome Measures

Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs and (4) physical exam.

Secondary Outcome Measures

Pharmacokinetics assessed by plasma drug concentration levels
Pharmacodynamics as measured by assessments of peripheral blood lymphocyte subsets

Full Information

First Posted
February 1, 2013
Last Updated
June 25, 2015
Sponsor
NKT Therapeutics
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1. Study Identification

Unique Protocol Identification Number
NCT01783691
Brief Title
Safety, Pharmacokinetic, and Pharmacodynamic Study of NKTT120 in Adult Patients With Stable Sickle Cell Disease (SCD)
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 Type
Interventional

2. Study Status

Record Verification Date
June 2015
Overall Recruitment Status
Completed
Study Start Date
February 2013 (undefined)
Primary Completion Date
May 2015 (Actual)
Study Completion Date
May 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
NKT Therapeutics

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease
Keywords
Sickle cell disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
21 (Actual)

8. Arms, Groups, and Interventions

Arm Title
NKTT120
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
NKTT120
Primary Outcome Measure Information:
Title
Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs and (4) physical exam.
Time Frame
180 days
Secondary Outcome Measure Information:
Title
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
Title
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 Pre-specified Outcome Measures:
Title
Pain scores as assessed by daily electronic diary
Time Frame
Daily for 120 days
Title
Quality of Life as assessed by two separate Quality of Life instruments
Time Frame
0, 30, 60, 90, 120 days
Title
Lung function as measured by pulmonary function tests
Time Frame
0; 30 minutes; 30, 60, 90, 120 days

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Rosemary Mazanet, MD
Organizational Affiliation
NKT Therapeutics
Official's Role
Study Director
Facility Information:
Facility Name
Children's Hospital & Research Center at Oakland
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Facility Name
Johns Hopkins School of Medicine
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
Brigham and Women's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Washington University School of Medicine
City
St. Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
UNC Sickle Cell Program
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27514
Country
United States
Facility Name
Blood Center of Wisconsin
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
28152086
Citation
Field JJ, Majerus E, Ataga KI, Vichinsky EP, Schaub R, Mashal R, Nathan DG. NNKTT120, an anti-iNKT cell monoclonal antibody, produces rapid and sustained iNKT cell depletion in adults with sickle cell disease. PLoS One. 2017 Feb 2;12(2):e0171067. doi: 10.1371/journal.pone.0171067. eCollection 2017.
Results Reference
derived

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

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