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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04285827
Recruitment Status : Not yet recruiting
First Posted : February 26, 2020
Last Update Posted : April 3, 2020
Sponsor:
Information provided by (Responsible Party):
CSL Behring

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.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Biological: CSL889 Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Estimated Study Start Date : September 2020
Estimated Primary Completion Date : March 2021
Estimated Study Completion Date : March 2021

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: CSL889 Cohort 1 (Dose 1)
CSL889 administered as a single IV infusion
Biological: CSL889
Administered as an IV infusion

Experimental: CSL889 Cohort 2 (Dose 2)
CSL889 administered as a single IV infusion
Biological: CSL889
Administered as an IV infusion

Experimental: CSL889 Cohort 3 (Dose 3)
CSL889 administered as a single IV infusion
Biological: CSL889
Administered as an IV infusion

Experimental: CSL889 Cohort 4 (Dose 4)
CSL889 administered as a single IV infusion
Biological: CSL889
Administered as an IV infusion

Experimental: CSL889 Cohort 5 (Dose 5)
CSL889 administered as a single IV infusion
Biological: CSL889
Administered as an IV infusion

Experimental: CSL889 Cohort 6 (Dose 6)
CSL889 administered as a single IV infusion
Biological: CSL889
Administered as an IV infusion




Primary Outcome Measures :
  1. Percentage of subjects with treatment-emergent adverse events (TEAEs) by CSL889 dose level [ Time Frame: Up to 32 days after start of CSL889 infusion ]
  2. Percentage of subjects with TEAEs by severity by CSL889 dose level [ Time Frame: Up to 32 days after start of CSL889 infusion ]
  3. Percentage of subjects with TEAEs by causality by CSL889 dose level [ Time Frame: Up to 32 days after start of CSL889 infusion ]

Secondary Outcome Measures :
  1. Maximum observed serum concentration (Cmax) of CSL889 by CSL889 dose level [ Time Frame: Up to 32 days after CSL889 infusion ]
  2. 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 ]
  3. 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 ]
  4. Time of Cmax (tmax) by CSL889 dose level [ Time Frame: Up to 32 days after CSL889 infusion ]
  5. Terminal half-life (t1/2) by CSL889 dose level [ Time Frame: Up to 32 days after CSL889 infusion ]
  6. Clearance (CL) of CSL889 by CSL889 dose level [ Time Frame: Up to 32 days after CSL889 infusion ]
  7. Volume of distribution (Vz) of CSL889 by CSL889 dose level [ Time Frame: Up to 32 days after CSL889 infusion ]
  8. Percentage of subjects with detectable antibodies to CSL889 by dose level [ Time Frame: Up to 32 days after CSL889 infusion ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

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): NCT04285827


Contacts
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Contact: Trial Registration Coordinator 610-878-4000 clinicaltrials@cslbehring.com

Sponsors and Collaborators
CSL Behring
Investigators
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Study Director: Study Director CSL Behring
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Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT04285827    
Other Study ID Numbers: CSL889_1001
2019‐001870‐27 ( EudraCT Number )
First Posted: February 26, 2020    Key Record Dates
Last Update Posted: April 3, 2020
Last Verified: April 2020
Individual Participant Data (IPD) Sharing Statement:
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
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.


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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn