Sickle Cell Disease

Safety of Single Doses of CSL889 in Adult Patients With Sickle Cell Disease
NCT04285827 | Phase 1 | Interventional

This is a phase 1, first-in-human, multi-center, open-label, single dose cohort study to evaluate the safety and tolerability, pharmacokinetics (PK), exploratory pharmacodynamics (PD), and biomarkers of target engagement of CSL889 following single intravenous (IV) doses in subjects with sickle cell disease (SCD). The study involves sequential dose escalation of cohorts with between-group assessments of key safety and PK variables.

Trial Information
14 Sites
32 Participants
Recruiting
18 Years to 60 Years

If interested, contact clinicaltrials@cslbehring.com for more information

SUBMIT
University of Illinois Hospital and Health Science Systems
Chicago,Illinois,United States,60612
The Johns Hopkins Hospital
Baltimore,Maryland,United States,21287
University of Minnesota
Minneapolis,Minnesota,United States,55455
Brody School of Medicine at East Carolina University
Greenville,North Carolina,United States,27834
Ohio State University
Columbus,Ohio,United States,43201
UPMC Hillman Cancer Center
Pittsburgh,Pennsylvania,United States,15232
Medical University of South Carolina
Charleston,South Carolina,United States,29425
Amsterdam UMC Academic Medical Center
Amsterdam,Netherlands,M13 9WL
Erasmus University Medical Center
Rotterdam,Netherlands
Liverpool University Hospital
Liverpool,United Kingdom
Guys and St. Thomas
London,United Kingdom
University College London Hospital
London,United Kingdom
Manchester University Hospitals NHS Foundation Trust Manchester Royal Infirmary
Manchester,United Kingdom
Early Phase Unit
Manchester,United Kingdom

Study Eligibility Criteria

  • Diagnosis of SCD as documented in the subject's medical record
  • Aged 18 to 60 years, inclusive
  • Stable SCD for at least 30 days before Day 1. Stable SCD is defined as the subject being at his or her medical baseline, with no evidence of worsening of disease over the last 30 days (including VOC, recent major surgery, hospitalization, serious infection, significant bleeding, cerebrovascular accident, seizures, or IV opioids)(Part A)
  • Uncomplicated VOC requiring parenteral opioid treatment and admission to hospital for management. Uncomplicated VOC is defined as sickle cell pain without the following associated clinical features (Part B):
  • Fever (> 38.5 °C)
  • Hypotension (< 90/60 mmHg)
  • Hypoxia (< 90% oxygen saturation on room air, or requiring oxygen therapy to maintain oxygen saturation above 90%)
  • New neurological signs and / or symptoms clinically suggestive of stroke or transient ischemic attack
  • Signs and / or symptoms of Acute Chest Syndrome, accompanied by any new pulmonary infiltrate on chest radiography (chest X-ray to be performed if clinically indicated and according to local clinical guidelines)
  • Subject is either not taking one of the study permitted SCD therapies (hydroxyurea, L-glutamine, L-glutaminecrizanlizumab, and/or voxelotor) or subject has been taking one or more of those for at least 30 days before Day 1 and is on a stable, well tolerated regimen that is planned to continue without change throughout the study
  • History of primary hemorrhagic stroke
  • History or evidence of inherited bleeding diathesis or significant coagulopathy at risk for bleeding
  • Weight >110 kg (242 lbs)
  • Surgery within 30 days before Day 1 or any preplanned surgeries during the study (minor surgeries may be permitted under local anesthesia before screening, with permission of the medical monitor)
  • Female subjects who are pregnant or breastfeeding
  • Female subject of childbearing potential or fertile male subject either not using or not willing to use an acceptable method of contraception to avoid pregnancy during the study and for 30 days after receipt of CSL889.
  • Treatment with any other drug / biologic that is newly approved for SCD during the conduct of this study within 90 days before Day 1. Exceptions: crizanlizumab [Adakveo®] and voxelotor [Oxbryta®] ] are permitted (where prescribed).
  • Treatment with another investigational product within 30 days or within 5 half-lives of the product (whichever is greater) before Day 1
  • Vaccination within 30 days before Day 1, or planned vaccination during the study
  • Body-mass index < 16 kg/m2 or weight < 50 kg (110 lbs)
  • History of anaphylactic-type reactions, transfusion related reaction, asthma, or autoimmune disease

Additional Studies

Additional studies can be found at ClinicalTrials.gov