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

Phase 1

Completed

• Conditions: Sickle Cell Disease
• Interventions: Drug: NKTT120

• Registration Number: NCT01783691
• Lead Sponsor: 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2013-02
• Study First Submitted: 2013-02-01
• Study First Submitted QC: 2013-02-04
• Study First Posted: 2013-02-05
• Primary Completion: 2015-05
• Study Completion: 2015-05
• Status Verified: 2015-06
• Last Update Submitted: 2015-06-25
• Last Update Posted: 2015-06-26

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 21

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
NKTT120	NKTT120	-

Primary Outcome Measures

Name	Time	Method
Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs and (4) physical exam.	180 days

Secondary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (6)

Johns Hopkins School of Medicine; 🇺🇸 Baltimore, Maryland, United States

Children's Hospital & Research Center at Oakland; 🇺🇸 Oakland, California, United States

Washington University School of Medicine; 🇺🇸 St. Louis, Missouri, United States

Brigham and Women's Hospital; 🇺🇸 Boston, Massachusetts, United States

UNC Sickle Cell Program; 🇺🇸 Chapel Hill, North Carolina, United States

Blood Center of Wisconsin; 🇺🇸 Milwaukee, Wisconsin, United States