Single-dose 21-valent pneumococcal conjugate vaccine for children and teens with sickle cell disease

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

This trial tests whether a single dose of the new PCV21 vaccine is safe and helps children and teens (ages 2–17) with sickle cell disease make protective antibodies compared with the licensed 20-valent pneumococcal vaccine.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 100 (estimated)
• Ages: 2 Years to 17 Years
• Sex: All
• Sponsor: Sanofi (Industry-sponsored)
• Drugs / interventions: chemotherapy, radiation
• Locations: 7 sites (Atlanta, Georgia and 6 other locations)
• Trial ID: NCT07247188 on ClinicalTrials.gov

What this trial studies

This phase 3 interventional study enrolls children and adolescents aged 2–17 years with documented sickle cell disease to receive a single dose of investigational 21-valent pneumococcal conjugate vaccine (PCV21) or the licensed 20-valent pneumococcal conjugate vaccine (20vPCV). Participants may have received prior pneumococcal conjugate or polysaccharide vaccines and will be followed for up to six months to monitor safety and antibody responses. The primary outcomes are safety events and immunogenicity measured by antibody levels after vaccination. The trial is sponsored by Sanofi and is being conducted at sites in Atlanta, GA and Omaha, NE.

Who should consider this trial

Good fit: Ideal candidates are children and adolescents aged 2–17 with a documented diagnosis of sickle cell disease who are not pregnant or breastfeeding and who can comply with study visits, with those of childbearing potential agreeing to required contraception and pregnancy testing.

Not a fit: Patients outside the 2–17 age range, those with a contraindication or known allergy to pneumococcal vaccine components, or those unable to follow study procedures are unlikely to receive benefit from participation.

Why it matters

Potential benefit: If successful, PCV21 could provide broader protection against pneumococcal strains and reduce invasive pneumococcal infections in children and adolescents with sickle cell disease.

How similar studies have performed: Licensed pneumococcal conjugate vaccines such as PCV13 and 20vPCV have demonstrated effectiveness in generating protective antibodies and preventing disease, so the 21-valent approach builds on an established strategy though the specific PCV21 formulation is investigational.

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.

Where this trial is running

Atlanta, Georgia and 6 other locations

• Site # 8400006 — Atlanta, Georgia, United States (Recruiting)
• Site # 8400002 — Atlanta, Georgia, United States (Recruiting)
• Site # 8400003 — Omaha, Nebraska, United States (Recruiting)
• Site # 8400001 — New Brunswick, New Jersey, United States (Recruiting)
• Site # 8400004 — Houston, Texas, United States (Recruiting)
• Site # 2140001 — Santo Domingo, Dominican Republic (Recruiting)
• Site # 2140003 — Santo Domingo, Dominican Republic (Recruiting)

Study contacts

• Study coordinator: Trial Transparency email recommended (Toll free number for US & Canada)

Conditions Sickle Cell Disease; Pneumococcal Vaccine; Pneumococcal Conjugate Vaccine