Study of a Single Dose of a 21-valent Pneumococcal Conjugate Vaccine in Children and Adolescents With Sickle Cell Disease

Official title

A Phase 3, Randomized, Modified Double-blind, Active-controlled, Parallel-group, 2-arm Study to Investigate the Safety and Immunogenicity of a Single Dose of a 21-valent Pneumococcal Conjugate Vaccine in Children and Adolescents With Sickle Cell Disease

Overview

The purpose of this study is to measure whether PCV21 vaccine (investigational pneumococcal vaccine) is safe and can help the body to develop germ-fighting agents called "antibodies" (immunogenicity) compared with 20vPCV (licensed pneumococcal vaccine) when given as a single dose to children aged 2 to 17 years with sickle cell disease who had received or not a previous vaccination with pneumococcal conjugate or pneumococcal polysaccharide vaccine.

Detailed description

The study duration per participant will be up to 6 months.

Interventions

• Biological PCV21
  Investigational pneumococcal conjugate vaccine
• Biological 20vPCV
  20-valent pneumococcal conjugate vaccine

Primary outcome measures

• Number of participants reporting immediate adverse events (AEs) [Time frame: Within 30 minutes post-vaccination]
• Number of participants reporting solicited injection site and solicited systemic reactions [Time frame: Up to Day 7 post-vaccination]
• Number of participants reporting unsolicited (spontaneously reported) injection site reactions and unsolicited systemic AEs after injection of a pneumococcal vaccine [Time frame: Within 30 days post-vaccination]
• Number of participants reporting serious adverse events (SAEs) and adverse events of special interest (AESIs) [Time frame: From day 0 to day 181]
• Number of participants with serotype-specific immunoglobulin G (IgG) concentrations for all serotypes included in PCV21 [Time frame: On day 30 post-vaccination]

Secondary outcome measures (3)

• Number of participants with serotype-specific IgG concentrations for all serotypes included in PCV21 [Time frame: Pre-vaccination and at 30 days post-vaccination]
• Number of participants with serotype specific opsonophagocytic activity (OPA) titers for all serotypes included in PCV21 [Time frame: On day 30]
• Number of participants with serotype specific opsonophagocytic activity (OPA) titers for all serotypes included in PCV21 [Time frame: Pre-vaccination and at 30 days post-vaccination]

Eligibility criteria

Inclusion criteria

AGE

• Aged 2 to 17 years on the day of inclusion.

TYPE OF PARTICIPANT AND DISEASE CHARACTERISTICS

• Participants who have a documented diagnosis of sickle cell disease (SCD) in their medical record.

SEX, CONTRACEPTIVE/BARRIER METHOD AND PREGNANCY TESTING REQUIREMENTS

• A participant is eligible to participate if the participant is not pregnant or breastfeeding and one of the following conditions applies:

• Is of non-childbearing potential. To be considered of non-childbearing potential, a female must be pre-menarchal or surgically sterile. OR
• Is of childbearing potential and agrees to use an effective contraceptive method or abstinence from at least 4 weeks prior to study intervention administration until at least 4 weeks after study intervention administration. A participant of childbearing potential must have a negative highly sensitive pregnancy test (urine or serum as required by local regulation) within 25 hours before the study intervention.

INFORMED CONSENT

• Assent form has been signed and dated by the participant (based on local regulations), and, if applicable, informed consent form has been signed and dated by the parent(s) or another legally acceptable representative (LAR) and by an independent witness, if required by local regulations.

OTHER INCLUSIONS

• Participant and parent(s)/LAR are able to attend all scheduled visits and to comply with all study procedures.

Exclusion criteria

MEDICAL CONDITIONS

• Known or suspected congenital or acquired immunodeficiency; or receipt of immunosuppressive therapy, such as anti-cancer chemotherapy or radiation therapy, within the preceding 6 months; or long-term systemic corticosteroid therapy.
• History of microbiologically confirmed S. pneumoniae infection or disease.
• History of seizure or significant stable or progressive neurological disorders such as inflammatory nervous system diseases, encephalopathy, and cerebral palsy.
• Known systemic hypersensitivity to any of the study interventions components, or history of a life-threatening reaction to the study interventions used in the study or to a product containing any of the same substances.
• Laboratory-confirmed thrombocytopenia, or known thrombocytopenia, as reported by the parent/LAR, contraindicating intramuscular (IM) injection.
• Bleeding disorder, or receipt of anticoagulants in the 3 weeks preceding inclusion, contraindicating IM injection.
• Chronic illness that, in the opinion of the investigator, is at a stage where it might interfere with study conduct or completion.
• Moderate or severe acute illness/infection (according to investigator judgment) or febrile illness (temperature ≥ 38.0°C \[≥ 100.4°F\]) on the day of study intervention administration. A prospective participant should not be included in the study until the condition has resolved or the febrile event has subsided.

For children/adolescents (6 to 17 YoA) only

• Alcohol, prescription drug, or substance abuse that, in the opinion of the Investigator, might interfere with the study conduct or completion.

PRIOR/CONCOMITANT THERAPY

• Receipt of at least one dose of 20vPCV.
• For children aged < 6 years: receipt of < 3 doses of pneumococcal conjugate vaccine or any dose of 23-valent pneumococcal polysaccharide vaccine (PPSV23).
• For children and adolescents aged ≥ 6 years: receipt of PPSV23 < 5 years before study vaccination or last PCV dose < 8 weeks before study vaccination.
• Receipt of any vaccine in the 4 weeks preceding the study intervention administration or planned receipt of any vaccine in the 4 weeks following the study intervention administration, except for US licensed influenza vaccination, which may be received at least 2 weeks before or 2 weeks after study vaccination. This exception includes monovalent pandemic influenza vaccines and multivalent influenza vaccines, as applicable per local recommendations.
• Receipt of immune globulins, blood or blood-derived products in the past 3 months.
• Receipt of an oral or injectable antibiotic therapy for any acute illness within 72 hours prior to the first blood draw.

PRIOR/CONCURRENT CLINICAL STUDY EXPERIENCE

• Participation at the time of study enrollment (or in the 6 weeks preceding the first study intervention administration) or planned participation during the present study period in another clinical study investigating a vaccine, drug, medical device, or medical procedure.

OTHER EXCLUSIONS For children (2 to 5 YoA) only

• Being in an emergency setting.
• Identified as a natural or adopted child of the Investigator or employee with direct involvement in the proposed study.

For children/adolescents (6 to 17 YoA) only

• Deprived of freedom by an administrative or court order, or in an emergency setting, or hospitalized involuntarily.
• Identified as an Investigator or employee of the Investigator or study center with direct involvement in the proposed study, or identified as an immediate family member (ie, parent, spouse, natural or adopted child) of the Investigator or employee with direct involvement in the proposed study.

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Parallel assignment

Masking

Single blind

Primary purpose

Prevention

Study locations

United States · 5 centers

• Site # 8400006 — Atlanta
• Site # 8400002 — Atlanta
• Site # 8400003 — Omaha
• Site # 8400001 — New Brunswick
• Site # 8400004 — Houston

Dominican Republic · 2 centers

• Site # 2140001 — Santo Domingo
• Site # 2140003 — Santo Domingo

Identifiers

NCT: NCT07247188 · PSK00026 · U1111-1319-0153

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗