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

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

Sponsors

Lead Sponsor: NKT Therapeutics

Source NKT Therapeutics
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.

Overall Status Completed
Start Date February 2013
Completion Date May 2015
Primary Completion Date May 2015
Phase Phase 1
Study Type Interventional
Primary Outcome
Measure Time Frame
Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs and (4) physical exam. 180 days
Secondary Outcome
Measure Time Frame
Pharmacokinetics assessed by plasma drug concentration levels 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 0, 0.25, 1, 2, 7, 14, 30, 60, 90, 120 days
Enrollment 21
Condition
Intervention

Intervention Type: Drug

Intervention Name: NKTT120

Arm Group Label: NKTT120

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

Gender: All

Minimum Age: 18 Years

Maximum Age: 60 Years

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Rosemary Mazanet, MD Study Director NKT Therapeutics
Location
Facility:
Children's Hospital & Research Center at Oakland | Oakland, California, 94609, United States
Johns Hopkins School of Medicine | Baltimore, Maryland, 21205, United States
Brigham and Women's Hospital | Boston, Massachusetts, 02115, United States
Washington University School of Medicine | St. Louis, Missouri, 63110, United States
UNC Sickle Cell Program | Chapel Hill, North Carolina, 27514, United States
Blood Center of Wisconsin | Milwaukee, Wisconsin, 53226, United States
Location Countries

United States

Verification Date

June 2015

Responsible Party

Type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 1
Arm Group

Label: NKTT120

Type: Experimental

Study Design Info

Allocation: N/A

Intervention Model: Single Group Assignment

Primary Purpose: Treatment

Masking: None (Open Label)

Source: ClinicalTrials.gov