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

NKT Therapeutics

Status and phase: Completed; Phase 1

Conditions: Sickle Cell Disease

Treatments: Drug: NKTT120

Study type: Interventional

Funder types: Industry

Identifiers

NCT01783691

120-SCD1

Details and patient eligibility

About

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

Enrollment: 21 patients

Sex: All

Ages: 18 to 60 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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21 participants in 1 patient group

NKTT120

Experimental group

Treatment:

Drug: NKTT120