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Safety of Single Ascending Doses of CSL889 in Adult Patients With Stable Sickle Cell Disease

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ClinicalTrials.gov Identifier: NCT04285827
Recruitment Status : Not yet recruiting
First Posted : February 26, 2020
Last Update Posted : April 23, 2021
Sponsor:
Information provided by (Responsible Party):
CSL Behring

Tracking Information
First Submitted Date  ICMJE February 24, 2020
First Posted Date  ICMJE February 26, 2020
Last Update Posted Date April 23, 2021
Estimated Study Start Date  ICMJE May 2021
Estimated Primary Completion Date July 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 24, 2020)
  • Percentage of subjects with treatment-emergent adverse events (TEAEs) by CSL889 dose level [ Time Frame: Up to 32 days after start of CSL889 infusion ]
  • Percentage of subjects with TEAEs by severity by CSL889 dose level [ Time Frame: Up to 32 days after start of CSL889 infusion ]
  • Percentage of subjects with TEAEs by causality by CSL889 dose level [ Time Frame: Up to 32 days after start of CSL889 infusion ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: February 24, 2020)
  • Maximum observed serum concentration (Cmax) of CSL889 by CSL889 dose level [ Time Frame: Up to 32 days after CSL889 infusion ]
  • Area under CSL889 serum concentration-time curve (AUC) from time 0 to time t (AUC0-t) by CSL889 dose level [ Time Frame: Up to 32 days after CSL889 infusion ]
  • Maximum observed serum concentration (Cmax) of CSL889 by CSL889 dose level AUC extrapolated to infinity (AUC0-inf) by CSL889 dose level [ Time Frame: Up to 32 days after CSL889 infusion ]
  • Time of Cmax (tmax) by CSL889 dose level [ Time Frame: Up to 32 days after CSL889 infusion ]
  • Terminal half-life (t1/2) by CSL889 dose level [ Time Frame: Up to 32 days after CSL889 infusion ]
  • Clearance (CL) of CSL889 by CSL889 dose level [ Time Frame: Up to 32 days after CSL889 infusion ]
  • Volume of distribution (Vz) of CSL889 by CSL889 dose level [ Time Frame: Up to 32 days after CSL889 infusion ]
  • Percentage of subjects with detectable antibodies to CSL889 by dose level [ Time Frame: Up to 32 days after CSL889 infusion ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety of Single Ascending Doses of CSL889 in Adult Patients With Stable Sickle Cell Disease
Official Title  ICMJE A Phase 1, Multi-Center, Open Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of CSL889 in Adult Patients With Stable Sickle Cell Disease
Brief Summary This is a phase 1, first-in-human, multi-center, open-label, single ascending dose (SAD) 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 stable sickle cell disease (SCD). The study involves sequential dose escalation of cohorts with between-group assessments of key safety and PK variables.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Sickle Cell Disease
Intervention  ICMJE Biological: CSL889
Administered as an IV infusion
Study Arms  ICMJE
  • Experimental: CSL889 Cohort 1 (Dose 1)
    CSL889 administered as a single IV infusion
    Intervention: Biological: CSL889
  • Experimental: CSL889 Cohort 2 (Dose 2)
    CSL889 administered as a single IV infusion
    Intervention: Biological: CSL889
  • Experimental: CSL889 Cohort 3 (Dose 3)
    CSL889 administered as a single IV infusion
    Intervention: Biological: CSL889
  • Experimental: CSL889 Cohort 4 (Dose 4)
    CSL889 administered as a single IV infusion
    Intervention: Biological: CSL889
  • Experimental: CSL889 Cohort 5 (Dose 5)
    CSL889 administered as a single IV infusion
    Intervention: Biological: CSL889
  • Experimental: CSL889 Cohort 6 (Dose 6)
    CSL889 administered as a single IV infusion
    Intervention: Biological: CSL889
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Not yet recruiting
Estimated Enrollment  ICMJE
 (submitted: February 24, 2020)
24
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE July 2022
Estimated Primary Completion Date July 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Diagnosis of SCD characterized by HbSS or SCD characterized by the compound heterozygous state of the βS mutation with β0 thalassemia mutations (HbSβ0)
  • Aged 18 to 60 years, inclusive
  • Stable SCD for at least 30 days before Day 1
  • Subject is either not taking hydroxyurea and / or L-glutamine, or subject has been taking hydroxyurea and / or L-glutamine for at least 30 days before Day 1 on a stable, well tolerated regimen that is planned to continue without change throughout the study

Exclusion Criteria:

  • Hospitalization for vaso-occlusive crisis (VOC) or treated with parenteral pain medications in other medical settings such as the emergency department or day hospital for VOC during the past 30 days before Day 1
  • Blood transfusion within the 90 days before Day 1, or expecting blood transfusion during the study
  • 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.
  • 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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 60 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Trial Registration Coordinator 610-878-4000 clinicaltrials@cslbehring.com
Listed Location Countries  ICMJE Netherlands,   United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04285827
Other Study ID Numbers  ICMJE CSL889_1001
2019-001870-27 ( EudraCT Number )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
Plan Description:

CSL will consider requests to share Individual Patient Data (IPD) from systematic review groups or bona-fide researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.

Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD.

If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.

Supporting Materials: Study Protocol
Supporting Materials: Statistical Analysis Plan (SAP)
Time Frame: IPD requests may be submitted to CSL no earlier than 12 months after publication of the results of this study via an article made available on a public website.
Access Criteria:

Requests may only be made by systematic review groups or bona-fide researchers whose proposed use of the IPD is non-commercial in nature and has been approved by an internal review committee.

An IPD request will not be considered by CSL unless the proposed research question seeks to answer a significant and unknown medical science or patient care question as determined by CSL's internal review committee.

The requesting party must execute an appropriate data sharing agreement before IPD will be made available.

Responsible Party CSL Behring
Study Sponsor  ICMJE CSL Behring
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Study Director CSL Behring
PRS Account CSL Behring
Verification Date April 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP