Clinical trial NCT05758506: Developing a Screening Tool for Primary Immunodeficiency Disease (PID) in Pakistan
Official title: Developing a Screening Tool for Primary Immunodeficiency Disease (PID) in Pakistan
Study type: OBSERVATIONAL
Overall status: NOT_YET_RECRUITING
Conditions: Primary Immunodeficiency Diseases
Lead sponsor: PATH
Sponsor class: OTHER
First posted: 2023-03-07
Enrollment: 230
Sex: ALL
Minimum age (years): 3
Maximum age (years): 15
Standard ages: CHILD
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: - 130 potential PID patients with (5 relevant PIDs: PADs) within Sindh and Punjab states V1.0, 6 Jan 2022

• 100 healthy, age-matched children within Sindh and Punjab state

Brief summary: Case-control diagnostic accuracy study with 130 potential pediatric PID+ (primary immunodeficiency) patients, and 100 age-matched, healthy controls (PID-). The potential PID+ participants will be recruited prospectively through 9 hospitals in Sindh and Punjab states or contacted via the PID surveillance registry developed by AKU Hospital's Polio Excretion in PID study to identify children with primary antibody deficiency (PAD+: a type of PID+); healthy, age-matched PID-participants will be recruited by snowball sampling. At the point of care, health care workers (HCWs) will collect capillary blood samples (0.1mL) to run the PID rapid screening test and reader on potential PID+ participants (identified by exhibiting \>2 of the Jeffrey Modell warning signs) and healthy, age-matched controls. All pediatric study participants will be sent to the hospital lab to have a confirmatory immunology panel (see 4.4.1 Diagnosing PID for the battery of tests) run on a serum/plasma sample to confirm their PID diagnosis (PID+/PAD-, PID+/PAD+, PID-); a 1.5uL aliquot of serum/plasma will simultaneously be used to run a PID rapid screening test by a laboratory technician (LT). HCWs and LTs will be blinded to true PID status. Blood and serum PID rapid screening test results will be compared to the confirmatory immunology panel to determine diagnostic accuracy. All clinical management of study participants will follow the standard of care for PID in Pakistan and will be based upon the immunology panel result. The HCWs and LTs administering the tests will be trained prior to the diagnostic accuracy test (see Objectives below) and will provide feedback on the tool post-training and post-use to assess usability, acceptability, and feasibility of integrating the test and digital reader into tertiary hospitals for the purpose of improved national PID surveillance, improved PID patient care, and polio eradication in Pakistan.


Eligibility criteria: Inclusion Criteria:

* Aged 3 months to 15 years

  * Presents for care at 1 of 9tertiary hospitals participating in the study
  * Potential PID+ (Case): Child is identified by HCW to have \>2 Jeffrey Model warning signs of PID. \[Note: Upon immunology panel testing, this group will be separated into three categories: PID-, PID+, and PID+/PAD+ meaning they have 1 of 5 relevant primary antibody deficiencies (PAD+)\].
  * PID-(Control): Child is identified (by snowball sampling) as being in good health, and having the same age (within 12 months) as another child who produced a PID+ result.
  * Caregiver consents to participate in the study and provides informed consent. If their children are 10 years of age or older, that child must also give assent.

Exclusion Criteria:

* Aged less than 3 months or greater than 15 years of age

  * Caregiver does not consent to participate, or child \>10years does not give assent to participate.
  * Having received IVIG in the past 90 days

Primary outcomes:
measure: Diagnostic accuracy
timeFrame: 8 month
measure: Determine usability, acceptability, feasibility of integrating PID test and digital reader into tertiary hospitals for improved patient care
timeFrame: 8 months
Secondary outcomes:
measure: Effectiveness - agreement between the PID screening test readouts (#1, #2) with each other, the digital reading, a PATH expert panel, and the local PID standard of care immunology tests.
timeFrame: 8 months
measure: Effectiveness
timeFrame: 8 months
measure: Understandability of diagnostic instruction
timeFrame: 8 months
measure: Usability of screening test result outputs
timeFrame: 8 months
measure: Usability for facilitators
timeFrame: 8 months
Other outcomes:
Locations:
city: Karachi
country: Pakistan
facility: Aga Khan University Hospital
Study officials:
References: