Primary Immunodeficiency DiseasesNCT05758506
Developing a Screening Tool for Primary Immunodeficiency Disease (PID) in Pakistan
An observational study of Primary Immunodeficiency Diseases, run by PATH at 1 site in Pakistan.
Trial 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… Read more about this study
- Who is it for?
- Ages 3 months to 15 yearsHealthy volunteers can take part
- Conditions the study is for
- Primary Immunodeficiency Diseases
- What is being tested?
- Primary Immunodeficiency (PID) Rapid Diagnostic Test (RDT)
- Type of treatment
- Medical device
- How many people
- 200 participants
- Where
- 1 sitePakistan
- Study dates
- 1 Jul 2024to 15 Dec 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 months to 15 years
- All sexes
- Healthy volunteers accepted
- Child
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
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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.
- Conditions
- Primary Immunodeficiency Diseases
- Being tested
- Primary Immunodeficiency (PID) Rapid Diagnostic Test (RDT)
- Medical subject headings
- Primary Immunodeficiency Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Primary Immunodeficiency (PID) Rapid Diagnostic Test (RDT)Device
We are evaluating the accuracy of a new rapid diagnostic screening test. There is no medical intervention with the participants in this study.
| Study group | Role | What they receive |
|---|---|---|
| Pediatric PID patients (prospective) | Prospective, age-matched, healthy patients Receives: Device: Primary Immunodeficiency (PID) Rapid Diagnostic Test (RDT) | |
| Pediatric PID patients (recontacted from registry) | Not described Receives: Device: Primary Immunodeficiency (PID) Rapid Diagnostic Test (RDT) | |
| Prospective, age-matched, healthy patients | Not described Receives: Device: Primary Immunodeficiency (PID) Rapid Diagnostic Test (RDT) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Diagnostic accuracy
Estimates of diagnostic accuracy characteristics with 95% confidence intervals (sensitivity, specificity, PPV, NPV) of the PID screening test for relevant PIDs in a Pakistani population of both PID+ children and PID- age-matched healthy controls
Measured: 8 month
Determine usability, acceptability, feasibility of integrating PID test and digital reader into tertiary hospitals for improved patient care
Estimates of user effectiveness, efficacy, satisfaction, learnability, and feasibility among trained health care workers (HCWs) and laboratory technicians (LTs) will be evaluated using observation checklists and surveys. The observation checklist will evaluate the ability to administer the test, read the test results accurately after the appropriate amount of waiting time, record the result in the digital reader app, operate the digital app to read a photograph of the test result, and their ease of performing these tasks. This methodology adheres to ISO 9241-210 on human-centered design principles in usability testing.
Measured: 8 months
Secondary outcomes (5)
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.
Percent (%) agreement between each of the PID screening test results, digital app result, expert panel, and immunology panel.
Measured: 8 months
Effectiveness
Percent (%) agreement in PID screening test results between capillary whole blood samples and serum samples.
Measured: 8 months
Understandability of diagnostic instruction
Percent of trained end users who can accurately comprehend the material presented in the training course curriculum and the IFU.
Measured: 8 months
Usability of screening test result outputs
Percent of trained end users who can accurately interpret the investigational device result output.
Measured: 8 months
Usability for facilitators
Percent of stakeholders who would use the test, lists of barriers and facilitators cited for Pakistani context.
Measured: 8 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2023
- Study start1 Jul 2024
- Primary completion30 Sep 2024
- Study completion15 Dec 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Immunodeficiency DiseasesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 200 participants (actual)
Sponsor and investigators
- Lead sponsor
- PATH
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Megan Parker, PATH
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05758506
- Sponsor's protocol ID
- 1840225
- Status last verified
- Jul 2025
- First submitted
- 10 Nov 2022
- Last updated
- 31 Jul 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: The data generated by this study will inform decisions regarding product development and commercialization of the BioSure PID rapid screening test and digital reader. The data collected in this evaluation may be used to support regulatory approval of the test. The study will generate valuable data on the performance of this novel test in comparison to that of the Jeffrey Modell warning signs and the immunology panel enabling informed decision-making regarding recommendation of new highly sensitive POC tools for PID. Data will be shared with study partners and the test developers. This may include de-identified, individual-level data.
Study at a glance
From the registry record, last updated by the sponsor on 31 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Developing a Screening Tool for Primary Immunodeficiency Disease (PID) in Pakistan
- ClinicalTrials.gov ID
- NCT05758506
- Acronym / sponsor ID
- 1840225
- Conditions
- Primary Immunodeficiency Diseases
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 200 participants (actual)
- Lead sponsor
- PATHUniversity, hospital or other
- Study start
- 1 Jul 2024 (actual)
- Primary completion
- 30 Sep 2024 (actual)
- Study completion
- 15 Dec 2024 (actual)
- First posted
- 7 Mar 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Pediatric PID patients (prospective) | – | Device: Primary Immunodeficiency (PID) Rapid Diagnostic Test (RDT)Prospective, age-matched, healthy patients |
| Pediatric PID patients (recontacted from registry) | – | Device: Primary Immunodeficiency (PID) Rapid Diagnostic Test (RDT) |
| Prospective, age-matched, healthy patients | – | Device: Primary Immunodeficiency (PID) Rapid Diagnostic Test (RDT) |
Who may qualify
- Ages
- 3 months to 15 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
- 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
Key inclusion criteria (5 listed)
- Aged 3 months to 15 years
Key exclusion criteria (3 listed)
- Aged less than 3 months or greater than 15 years of age
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Primary Immunodeficiency (PID) Rapid Diagnostic Test (RDT)We are evaluating the accuracy of a new rapid diagnostic screening test. There is no medical intervention with the participants in this study. | Device | Pediatric PID patients (prospective), Pediatric PID patients (recontacted from registry), Prospective, age-matched, healthy patients |
Primary outcome
Diagnostic accuracy
Time frame: 8 month
Estimates of diagnostic accuracy characteristics with 95% confidence intervals (sensitivity, specificity, PPV, NPV) of the PID screening test for relevant PIDs in a Pakistani population of both PID+ children and PID- age-matched healthy controls
Determine usability, acceptability, feasibility of integrating PID test and digital reader into tertiary hospitals for improved patient care
Time frame: 8 months
Estimates of user effectiveness, efficacy, satisfaction, learnability, and feasibility among trained health care workers (HCWs) and laboratory technicians (LTs) will be evaluated using observation checklists and surveys. The observation checklist will evaluate the ability to administer the test, read the test results accurately after the appropriate amount of waiting time, record the result in the digital reader app, operate the digital app to read a photograph of the test result, and their ease of performing these tasks. This methodology adheres to ISO 9241-210 on human-centered design principles in usability testing.
5 secondary outcomes
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.
Time frame: 8 months
Effectiveness
Time frame: 8 months
Understandability of diagnostic instruction
Time frame: 8 months
Usability of screening test result outputs
Time frame: 8 months
Usability for facilitators
Time frame: 8 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jul 2025.
- Individual participant dataSharing planned: Yes. The data generated by this study will inform decisions regarding product development and commercialization of the BioSure PID rapid screening test and digital reader. The data collected in this evaluation may be used to support regulatory approval of the test. The study will generate valuable data on the performance of this novel test in comparison to that of the Jeffrey Modell warning signs and the immunology panel enabling informed decision-making regarding recommendation of new highly sensitive POC tools for PID. Data will be shared with study partners and the test developers. This may include de-identified, individual-level data.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Primary Immunodeficiency Diseases, run by PATH at 1 site in Pakistan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 31 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Pakistan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Diagnostic accuracy (time frame: 8 month). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Jul 2025.