Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

A Safety and Tolerability Study of BIVV020 in Adults With Cold Agglutinin 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: NCT04269551
Recruitment Status : Recruiting
First Posted : February 17, 2020
Last Update Posted : April 7, 2020
Sponsor:
Information provided by (Responsible Party):
Sanofi

Brief Summary:

Primary Objective:

To assess the safety and tolerability in participants with cold agglutinin disease (CAD), after a single dose of intravenous (IV) BIVV020

Secondary Objectives:

To assess, in participants with cold agglutinin disease, after a single dose of intravenous (IV) BIVV020:

  • The effect of BIVV020 on complement mediated hemolysis
  • The pharmacodynamics (PD) of BIVV020 relating to complement inhibition
  • The pharmacokinetics (PK) of BIVV020
  • The immunogenicity of BIVV020

Condition or disease Intervention/treatment Phase
Autoimmune Haemolytic Anaemia Drug: BIVV020 Phase 1

Detailed Description:
Up to 23 weeks (screening period up to 8 weeks, treatment period 15 weeks)

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter, Phase 1b, Open Label, Nonrandomized, Single Dose Study Evaluating the Safety, Tolerability and Activity of BIVV020 in Adults With Cold Agglutinin Disease
Actual Study Start Date : February 6, 2020
Estimated Primary Completion Date : November 2021
Estimated Study Completion Date : November 2021


Arm Intervention/treatment
Experimental: BIVV020 IV
Single administration dose 1, plus two optional doses of BIVV020 administered intravenously.
Drug: BIVV020
Pharmaceutical form:solution for injection Route of administration: intravenous




Primary Outcome Measures :
  1. Assessment of adverse events (AEs) [ Time Frame: Screening to Day 106 ]
    Number of participants with adverse events (AEs)


Secondary Outcome Measures :
  1. Mean change from baseline in bilirubin over time [ Time Frame: Day 1 to Day 106 ]
    Assessment of total bilirubin

  2. Mean change from baseline in hemoglobin over time [ Time Frame: Day 1 to Day 106 ]
    Assessment of hemoglobin

  3. Complement System Classical Pathway Levels as Measured by WIESLAB Assay [ Time Frame: Day 1 to Day 106 ]
    Inhibition by BIVV020 of the complement system classical pathway measured by the WIESLAB assay

  4. Complement System Alternative Pathway Levels as Measured by WIESLAB Assay [ Time Frame: Day 1 to Day 106 ]
    Effect of BIVV0020 on the complement system alternative pathway measured by the WIESLAB assay

  5. Total Complement (CH50) Levels [ Time Frame: Day 1 to Day 106 ]
    Complement CH50 is a blood test that helps us determine whether protein abnormalities and deficiencies in the complement system are responsible for any increase in autoimmune activity. It will be assessed using complement assays.

  6. Total Complement Factor C4 Levels [ Time Frame: Day 1 to Day 106 ]
    Total C4 Levels will be assessed in plasma using complement assays

  7. PK parameter: Cmax [ Time Frame: Day 1 to Day 106 ]
    Observed maximum plasma concentration

  8. PK parameter: tmax [ Time Frame: Day 1 to Day 106 ]
    Observed first time to reach Cmax

  9. PK parameter: AUClast [ Time Frame: Day 1 to Day 106 ]
    Area under the plasma concentration versus time curve calculated using the trapezoidal method from time zero to the real time tlast

  10. PK parameter: AUC0-∞ [ Time Frame: Day 1 to Day 106 ]
    Calculated area under the plasma concentration versus time curve extrapolated to infinity

  11. Number of participants with anti-BIVV antibodies [ Time Frame: Day 1 to Day 106 ]
    Observed number of participants with BIVV020 antibodies



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion criteria :

  • Male and/or female patients, ≥ 18 years of age with cold agglutinin disease as defined by:

    1. Chronic hemolysis per Investigator's judgement,
    2. Polyspecific direct antiglobulin test (DAT) positive,
    3. Monospecific DAT strongly positive for C3d,
    4. Cold agglutinin (CAg) titer ≥ 64 at 4 C; and,
    5. IgG DAT ≤1+.
  • A hemoglobin level ≤11 mg/dL.
  • A total bilirubin level above the normal reference range that is thought to be due to hemolysis.
  • Documented vaccinations against encapsulated bacterial pathogens (Neisseria meningitidis, including serogroup B meningococcus and Streptococcus pneumoniae) within five years of screening or willing to complete protocol specified vaccinations.
  • Having given written informed consent prior to undertaking any study-related procedure.

Exclusion criteria:

  • Cold agglutinin syndrome secondary to infection, rheumatologic disease, or known high grade hematologic malignancy, or known solid organ tumor.
  • Clinically relevant infection of any kind within one month preceding screening.
  • Treatment with anti-CD20 monotherapy within three months or anti CD20 combination therapies within six months prior to screening.
  • Concurrent treatment with systemic immunosuppressive agents targeting B- or T-cell function and/or cytotoxic agents within 3 months prior to screening. Concurrent treatment with other systemic immunosuppressants within 5.5 half-lives of the drug prior to screening.
  • Any specific complement system inhibitor within three months prior to screening.
  • Concurrent treatment with systemic corticosteroids other than a stable daily dose equivalent to ≤10 mg/day prednisone within three months prior to screening.
  • If female, pregnant or lactating.

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.


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): NCT04269551


Contacts
Layout table for location contacts
Contact: Trial Transparency email recommended (Toll free number for US & Canada) 800-633-1610 ext option 6 Contact-US@sanofi.com

Locations
Layout table for location information
United States, Georgia
Investigational Site Number 8400002 Recruiting
Fayetteville, Georgia, United States, 30214
Sponsors and Collaborators
Sanofi
Investigators
Layout table for investigator information
Study Director: Clinical Sciences & Operations Sanofi

Layout table for additonal information
Responsible Party: Sanofi
ClinicalTrials.gov Identifier: NCT04269551    
Other Study ID Numbers: PDY16370
2019-001844-22 ( EudraCT Number )
U1111-1233-0831 ( Other Identifier: UTN )
First Posted: February 17, 2020    Key Record Dates
Last Update Posted: April 7, 2020
Last Verified: April 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://www.clinicalstudydatarequest.com/

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
Anemia, Hemolytic, Autoimmune
Anemia, Hemolytic
Anemia
Hematologic Diseases
Autoimmune Diseases
Immune System Diseases