Clinical trial NCT01783691: Safety, Pharmacokinetic, and Pharmacodynamic Study of NKTT120 in Adult Patients With Stable Sickle Cell Disease (SCD)
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Sickle Cell Disease
Keywords: Sickle cell disease
Lead sponsor: NKT Therapeutics
Sponsor class: INDUSTRY
First posted: 2013-02-05
Enrollment: 21
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs and (4) physical exam.
timeFrame: 180 days
Secondary outcomes:
measure: Pharmacokinetics assessed by plasma drug concentration levels
timeFrame: 0; 15, 30, 60 minutes; 3,6, hours; 1,2,3,7,14,30,60,90 days
measure: Pharmacodynamics as measured by assessments of peripheral blood lymphocyte subsets
timeFrame: 0, 0.25, 1, 2, 7, 14, 30, 60, 90, 120 days
Other outcomes:
measure: Pain scores as assessed by daily electronic diary
timeFrame: Daily for 120 days
measure: Quality of Life as assessed by two separate Quality of Life instruments
timeFrame: 0, 30, 60, 90, 120 days
measure: Lung function as measured by pulmonary function tests
timeFrame: 0; 30 minutes; 30, 60, 90, 120 days
Locations:
city: Oakland
country: United States
facility: Children's Hospital & Research Center at Oakland
state: California
city: Baltimore
country: United States
facility: Johns Hopkins School of Medicine
state: Maryland
city: Boston
country: United States
facility: Brigham and Women's Hospital
state: Massachusetts
city: St. Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Chapel Hill
country: United States
facility: UNC Sickle Cell Program
state: North Carolina
city: Milwaukee
country: United States
facility: Blood Center of Wisconsin
state: Wisconsin
Study officials:
affiliation: NKT Therapeutics
name: Rosemary Mazanet, MD
role: STUDY_DIRECTOR
References:
citation: Field JJ, Majerus E, Ataga KI, Vichinsky EP, Schaub R, Mashal R, Nathan DG. NNKTT120, an anti-iNKT cell monoclonal antibody, produces rapid and sustained iNKT cell depletion in adults with sickle cell disease. PLoS One. 2017 Feb 2;12(2):e0171067. doi: 10.1371/journal.pone.0171067. eCollection 2017.