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A Phase Ib Study of NVX-508 in Sickle Cell Disease

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ClinicalTrials.gov Identifier: NCT03013426
Recruitment Status : Withdrawn (NuvOx Pharma suspended the clinical program related to this study.)
First Posted : January 6, 2017
Last Update Posted : July 16, 2018
Sponsor:
Collaborators:
NuvOx Pharma Pty Ltd
Noguchi Memorial Institute for Medical Research
Information provided by (Responsible Party):
Amma Owusu-Ansah, MD, University of Pittsburgh

Brief Summary:
This Phase 1b study in adults with sickle cell disease (SCD) in steady-state (non-acutely ill) aims to evaluate safety and toxicity of NVX-508 in a multi-dosing paradigm as well as to determine the maximum tolerated dose (MTD) in this population. The information gained from this study will be used in making decisions about the appropriate dose(s) and dosing schedule in future multicenter studies of the efficacy of NVX-508 in the treatment of vaso-occlusive episodes (VOE).

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: NVX-508 Phase 1

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase Ib, Dose-finding and Pharmacodynamic Study of NVX-508 in Sickle Cell Disease Patients
Estimated Study Start Date : July 2018
Estimated Primary Completion Date : December 2018
Estimated Study Completion Date : December 2018

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: NVX-508
Administration of 1, 2 or 4 doses of NVX-508 at three dose levels; 0.05ml/kg. 0.1ml/kg and 0.17ml/kg in a 3 + 3 design.
Drug: NVX-508
Administration of NVX-508 emulsion intravenously




Primary Outcome Measures :
  1. Maximum tolerated dose of NVX-508 [ Time Frame: 1 year ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Clinical diagnosis of SCD without acute VOE and/or ACS.
  • Age 18 years and older
  • Adequate hematologic, renal and hepatic function, defined by:

    1. Absolute neutrophil count (ANC) ≥ 1.5 x109/L
    2. Platelet count ≥ 100 x 109/L,
    3. Hemoglobin ≥ 60 g/L
    4. International normalized ratio (INR) < 1.5 x upper limit of normal (ULN)
    5. Activated partial thromboplastin time (APTT) < 1.5 x ULN
    6. Plasma creatinine < 1.5 x ULN
    7. Total bilirubin < 2.5 x ULN (in the presence of Gilbert's syndrome or indirect hyperbilirubinemia caused by hemolysis)
    8. Aspartate transaminase (AST) < 2.5 x ULN
    9. Alanine transaminase (ALT) < 2.5 x ULN
  • Ability of the prospective subject to understand and willingness to sign written informed consent document

Exclusion Criteria:

  • Patients who have received any other investigational agent within 4 weeks before enrollment.
  • Patients who have had a VOE/ACS in the previous 4 weeks before enrollment.
  • Stroke or transient ischemic attack within 6 months before enrollment.
  • Myocardial infarction within 6 months before enrollment, unstable angina, New York Heart Association class II or greater congestive heart failure, or uncontrolled hypertension (systolic BP > 160 mmHg and/or diastolic BP > 100 mmHg).
  • Congenital long QT syndrome, or corrected QT interval ( QTc) > 450 milliseconds (msec) in males and > 470 mSec in females on EKG.
  • Uncontrolled arrhythmia or any history of clinically significant arrhythmia in the past 6 months
  • Clinically-significant chronic obstructive pulmonary disease or asthma that is not controlled by medication.
  • A history of other malignancies, except adequately treated non-melanoma skin cancer, curatively treated in-situ cancer or other solid tumors curatively treated with no evidence of disease for ≥ 2 years.
  • Current anticoagulant or antiplatelet therapy, except for prophylactic doses of low molecular weight heparins or low-dose aspirin.
  • History of allergic reactions attributed to compounds of similar chemical composition to NVX- 508.
  • Women who are pregnant or breastfeeding.
  • Inability to comply with study procedures.
  • History or evidence of any other clinically-significant condition that, in the opinion of the investigator, would pose a risk to subject safety or interfere with study procedures, evaluation or completion.
  • Patients with active VOE or ACS or other significant current acute complication of SCD.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03013426


Sponsors and Collaborators
Amma Owusu-Ansah, MD
NuvOx Pharma Pty Ltd
Noguchi Memorial Institute for Medical Research
Investigators
Principal Investigator: Amma T Owusu-Ansah, MD University of Pittsburgh

Publications:

Responsible Party: Amma Owusu-Ansah, MD, Assistant Professor of Medicine and Clinical Director, Center for Translational and International Hematology, University of Pittsburgh
ClinicalTrials.gov Identifier: NCT03013426     History of Changes
Other Study ID Numbers: PRO16110118
First Posted: January 6, 2017    Key Record Dates
Last Update Posted: July 16, 2018
Last Verified: July 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn