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

Sickle Cell Disease Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01783691
• Other study ID #: 120-SCD1
• Status: Completed
• Phase: Phase 1
• First received: February 1, 2013
• Last updated: June 25, 2015
• Start date: February 2013
• Est. completion date: May 2015

Study information

• Verified date: June 2015
• Source: NKT Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 21
• Est. completion date: May 2015
• Est. primary completion date: May 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 60 Years

Eligibility

Inclusion Criteria:

• Age 18 to 50 years

• Subject has a confirmed diagnosis of HbSS or HbSß0thal

• 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

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

Drug:
• NKTT120

Locations

Country	Name	City	State
United States	Johns Hopkins School of Medicine	Baltimore	Maryland
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	Children's Hospital & Research Center at Oakland	Oakland	California
United States	Washington University School of Medicine	St. Louis	Missouri

Sponsors (1)

Lead Sponsor	Collaborator
NKT Therapeutics

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Pain scores as assessed by daily electronic diary		Daily for 120 days	No
Other	Quality of Life as assessed by two separate Quality of Life instruments		0, 30, 60, 90, 120 days	No
Other	Lung function as measured by pulmonary function tests		0; 30 minutes; 30, 60, 90, 120 days	No
Primary	Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs and (4) physical exam.		180 days	Yes
Secondary	Pharmacokinetics assessed by plasma drug concentration levels		0; 15, 30, 60 minutes; 3,6, hours; 1,2,3,7,14,30,60,90 days	No
Secondary	Pharmacodynamics as measured by assessments of peripheral blood lymphocyte subsets		0, 0.25, 1, 2, 7, 14, 30, 60, 90, 120 days	No