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

June 25, 2015 updated by: NKT Therapeutics

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

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

21

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Oakland, California, United States, 94609
        • Children'S Hospital & Research Center At Oakland
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • Johns Hopkins School of Medicine
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Brigham and Women's Hospital
    • Missouri
      • St. Louis, Missouri, United States, 63110
        • Washington University School of Medicine
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27514
        • UNC Sickle Cell Program
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Blood Center of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 60 years (Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: NKTT120
Drug: NKTT120

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Time Frame
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
0; 15, 30, 60 minutes; 3,6, hours; 1,2,3,7,14,30,60,90 days
Pharmacodynamics as measured by assessments of peripheral blood lymphocyte subsets
Time Frame: 0, 0.25, 1, 2, 7, 14, 30, 60, 90, 120 days
0, 0.25, 1, 2, 7, 14, 30, 60, 90, 120 days

Other Outcome Measures

Outcome Measure
Time Frame
Pain scores as assessed by daily electronic diary
Time Frame: Daily for 120 days
Daily for 120 days
Quality of Life as assessed by two separate Quality of Life instruments
Time Frame: 0, 30, 60, 90, 120 days
0, 30, 60, 90, 120 days
Lung function as measured by pulmonary function tests
Time Frame: 0; 30 minutes; 30, 60, 90, 120 days
0; 30 minutes; 30, 60, 90, 120 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

NKT Therapeutics

Investigators

  • Study Director: Rosemary Mazanet, MD, NKT Therapeutics

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

February 1, 2013

Primary Completion (Actual)

May 1, 2015

Study Completion (Actual)

May 1, 2015

Study Registration Dates

First Submitted

February 1, 2013

First Submitted That Met QC Criteria

February 4, 2013

First Posted (Estimate)

February 5, 2013

Study Record Updates

Last Update Posted (Estimate)

June 26, 2015

Last Update Submitted That Met QC Criteria

June 25, 2015

Last Verified

June 1, 2015

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 120-SCD1

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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