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

NCT01783691 | Completed Phase 1

• 1 other identifier: 120-SCD1
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 6

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.

Conditions

Sickle Cell Disease

Keywords

Sickle cell disease

Outcome Measures

Primary Outcomes (1)

• Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs and (4) physical exam.

  180 days

Secondary Outcomes (2)

• 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 Outcomes (3)

• 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

Study Arms (1)

NKTT120

EXPERIMENTAL

Drug: NKTT120

Interventions

NKTT120 DRUG

NKTT120

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• NKT Therapeutics: lead

Study Sites (6)

Children's Hospital & Research Center at Oakland

Oakland, California, 94609, United States

Location

Johns Hopkins School of Medicine

Baltimore, Maryland, 21205, United States

Location

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

UNC Sickle Cell Program

Chapel Hill, North Carolina, 27514, United States

Location

Blood Center of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Related Publications (1)

• 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.

  PMID: 28152086 DERIVED

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

NKTT120

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Rosemary Mazanet, MD

  NKT Therapeutics

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 1, 2013
• First Posted: February 5, 2013
• Study Start: February 1, 2013
• Primary Completion: May 1, 2015
• Study Completion: May 1, 2015
• Last Updated: June 26, 2015

Record last verified: 2015-06

Locations

US (6)