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Completed

NCT Number: NCT01783691

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

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

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Key information

Conditions

Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies

Age range

18 year–60 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

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

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

NKTT120

Drug

Primary outcomes

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

    Time frame: 180 days

Secondary outcomes

  1. Pharmacokinetics assessed by plasma drug concentration levels

    Time frame: 0; 15, 30, 60 minutes; 3,6, hours; 1,2,3,7,14,30,60,90 days

  2. Pharmacodynamics as measured by assessments of peripheral blood lymphocyte subsets

    Time frame: 0, 0.25, 1, 2, 7, 14, 30, 60, 90, 120 days

Other outcomes

  1. Pain scores as assessed by daily electronic diary

    Time frame: Daily for 120 days

  2. Quality of Life as assessed by two separate Quality of Life instruments

    Time frame: 0, 30, 60, 90, 120 days

  3. Lung function as measured by pulmonary function tests

    Time frame: 0; 30 minutes; 30, 60, 90, 120 days

Sponsors and collaborators

Lead sponsor

NKT Therapeutics

Industry

Registry information

Official study 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

Important dates

Study start
2013
Primary completion
2015
Study completion
2015
First posted
Feb 5, 2013
Registry last updated
Jun 26, 2015

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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