Sickle Cell Disease
Conditions
Keywords
Sickle cell disease
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.
Interventions
DRUGNKTT120
Sponsors
NKT Therapeutics
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 60 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs and (4) physical exam. | 180 days |
Secondary
| 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 |
Other
| Measure | Time frame |
|---|---|
| Pain scores as assessed by daily electronic diary | Daily for 120 days |
| Quality of Life as assessed by two separate Quality of Life instruments | 0, 30, 60, 90, 120 days |
| Lung function as measured by pulmonary function tests | 0; 30 minutes; 30, 60, 90, 120 days |
Countries
United States
Outcome results
None listed