A Study to Evaluate Efficacy, Safety, and PK of XEMBIFY®+Standard Medical Treatment (SMT) Compared to Placebo+SMT to Prevent Infections in Participants with HGG and Recurrent or Severe Infections Associated with B-cell Chronic Lymphocytic Leukemia, Multiple Myeloma, and Non-Hodgkin Lymphoma

Launched by GRIFOLS THERAPEUTICS LLC · Dec 1, 2022

Keywords

ClinConnect Summary

This clinical trial is studying a treatment called XEMBIFY® to see if it can help prevent serious bacterial infections in patients with certain types of blood cancers, including B-cell Chronic Lymphocytic Leukemia (CLL), Multiple Myeloma (MM), and Non-Hodgkin Lymphoma (NHL), who also have low levels of antibodies (a condition known as hypogammaglobulinemia). Participants will receive either XEMBIFY® along with standard medical treatment or a placebo (a treatment that looks like medicine but has no active ingredients) with standard medical treatment for one year. The goal is to find out if XEMBIFY® can reduce the number of major infections compared to the placebo.

To be eligible for the trial, participants must be at least 18 years old and have a confirmed diagnosis of one of the targeted blood cancers, along with a history of severe or recurrent infections. However, individuals who have recently had certain treatments or conditions, such as a stem cell transplant or active infections, may not qualify. If you decide to participate, you’ll have regular check-ups and monitoring throughout the year to assess your health and the effectiveness of the treatment. This trial is still looking for participants, and your involvement could contribute to important research that may improve care for patients with similar conditions.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Participants ≥18 years of age at screening visit
• * Participants with documented and confirmed diagnosis of any of the below diseases:
• • B-cell CLL according to International Workshop on CLL (iwCLL) criteria and RAI staging of intermediate (1 and 2) or high (3 and 4)
• • MM according to the International Myeloma Working Group criteria (IMWG), R-ISS stage II or, III; or
• • Histologically confirmed diagnosis of B-Cell NHL, Stage III or above (IV, Progressive/refractory, or recurrent/relapsed stage) according to the Lugano Classification.
• • Participants with HGG with IgG levels less than 5 g/L.
• • Participants with documented history of at least one severe bacterial infection (bacterial or viral) or recurrent bacterial/viral infections (that is., ≥ 3 infections) within 12 months before the screening visit. Severe bacterial/viral infections ≥ Grade 3 (as defined by Common Terminology Criteria for Adverse Events \[CTCAE\] Grades).
• Exclusion Criteria:
• • Participants with documented history of hematopoietic stem cell transplant.
• • Participants currently receiving immunoglobulin replacement therapy (IgRT) or have received IgG replacement treatment (i.e., prior immune globulin replacement therapy) within 6 months before the screening visit.
• • Participants with active infections at time of screening visit. Specific supportive anti-infective prophylactic defined in the CLL National Comprehensive Cancer Network (NCCN) or iwCLL guidelines and/or local/international guidelines for the CLL, and defined in local/international guidelines for MM and NHL are allowed, or recommended in the updated labelling of specific active target disease medicines used during the participation in the trial is also allowed.
• • Participants with active second malignancies.
• • Participants with known primary immunodeficiency (PI).
• • Participants with a life expectancy less than 1.5 years.
• • Participants with clinical evidence of any significant acute or chronic disease that, in the opinion of the investigator, may interfere with successful completion of the trial or place the subject at undue medical risk.
• • Participants have had a known serious adverse reaction (AR) to immunoglobulin or any anaphylactic reaction to blood or any blood-derived product.
• • Participants have a history of blistering skin disease, bleeding disorder, diffuse rash, recurrent skin infections, or other disorders where SC therapy would be contraindicated during the study based upon the Investigator's discretion.
• • Participants have known Selective Immunoglobulin A (IgA) Deficiency (with or without antibodies to IgA) (Note: exclusion is for the specific diagnostic entity. It does not exclude other forms of humoral primary immunodeficiency which have decreased IgA in addition to decreased IgG requiring IgG replacement).
• • Participants with severe known kidney disease \[as defined by estimated glomerular filtration rate \[eGFR\] less than (\<) 30 milliliter (mL)/min/1.73 square meter (m2)\] as determined by the Principal Investigator.
• • Participants that have liver enzyme levels (alanine aminotransferase \[ALT\], aspartate aminotransferase \[AST\], gammaglutamyl transferase \[GGT\], or lactate dehydrogenase \[LDH\]) greater than 3 times the upper limit of normal (ULN) at the Screening Visit as defined by the testing laboratory.
• • Participants have a history (either 1 episode within the year prior to the Screening Visit or 2 previous episodes over a lifetime) of or current diagnosis of thromboembolism (example, myocardial infarction, cerebrovascular accident, or transient ischemic attack) or deep venous thrombosis.
• • Participants are currently receiving anti-coagulation therapy which would make SC administration inadvisable (vitamin K antagonists, nonvitamin K antagonist oral anticoagulants \[example, dabigatran etexilate targeting Factor IIa, rivaroxaban, edoxaban, and apixaban targeting Factor Xa\], and parenteral anticoagulants \[example, fondaparinux\]).
• • Participants currently have a known hyperviscosity syndrome or hypercoagulable states.
• • Participants have a known previous infection or clinical signs and symptoms consistent with current hepatitis B virus or hepatitis C virus infection.
• • Participants with non-controlled arterial hypertension (systolic blood pressure \[SBP\] greater than 140 millimeters of mercury (mmHg) and/or diastolic blood pressure \[DBP\] greater than 90 mmHg), and/or a heart rate (HR) greater than100 bpm.
• • Participants with known substance or prescription drug abuse within 12 months before the Screening Visit.
• • Participants have participated in another clinical trial within 30 days prior to screening (observational studies without investigative treatments \[non-interventional\] are permitted).