Trial Outcomes & Findings for Evaluation of Low-cost Techniques for Detecting Sickle Cell Disease and β-thalassemia in Nepal and Canada (NCT NCT05506358)

Last Updated: 2024-06-04

Results Overview

The following metrics will be determined for the low-cost tests to be evaluated as indicated below (where TP = true positive, TN = true negative, FP = false positive, FN = false negative): 1. Sensitivity = TP/(TP + FN) 2. Specificity = TN/(FP + TN) 3. Positive predictive value = TP/(TP + FP) 4. Negative predictive value = TN/(TN + FN) These metrics will be calculated for the low-cost technologies against the reference test, HPLC, for detecting the presence of sickle hemoglobin and β- thalassemia. The low-cost technologies include automated sickling test (standard sickling test enhanced using low-cost microscopy and machine learning), solubility test, HemoTypeSC, Sickle SCAN, and Gazelle Hb Variant test. The test results of the low-cost technologies will be compared with those of the reference test to get the values of TP, TN, FP and FN, which will then be used to calculate the metrics listed above.

Recruitment status

COMPLETED

Study phase

Target enrollment

145 participants

Primary outcome timeframe

baseline

Results posted on

2024-06-04

Participant Flow

Participant milestones

Participant milestones
Measure	HbSS HbSS: homozygous form of sickle cell disease	HbAS HbAS: heterozygous (carrier) form of sickle cell disease	HbS/β-thalassemia HbS/β-thalassemia: compound heterozygous form of sickle cell disease (with β-thalassemia)	HbA/β-thalassemia HbA/β-thalassemia: β-thalassemia trait (carrier form)	HbAA HbAA: participants without any known hemoglobin disorders, such as sickle cell disease, sickle cell trait, β-thalassemia, etc.
Overall Study STARTED	33	45	11	26	30
Overall Study COMPLETED	29	45	11	23	30
Overall Study NOT COMPLETED	4	0	0	3	0

Reasons for withdrawal

Reasons for withdrawal
Measure	HbSS HbSS: homozygous form of sickle cell disease	HbA/β-thalassemia HbA/β-thalassemia: β-thalassemia trait (carrier form)
Overall Study Sickle cell disease with transfusion within 3 months	2	0
Overall Study Sickle cell disease with Hereditary persistence of fetal hemoglobin (HPFH)	2	0
Overall Study Beta-thalassemia major with transfusion within 2 months	0	1
Overall Study Sample hemolysis observed under the microscope	0	1
Overall Study HbE trait	0	1

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	1) HbSS; 2) HbAS; 3) HbS/β-thalassemia; 4) HbA/β-thalassemia; 5) HbAA n=138 Participants Please note that the demographic information (age, sex) is reported for all different groups combined, and individual analysis of age and sex distribution per group is not performed due to the way the data was collected. In Canada, only averages and ranges for age and sex were collected rather than distribution per group. Number of participants considered in the study: 29 HbSS: homozygous form of sickle cell disease 45 HbAS: heterozygous (carrier) form of sickle cell disease 11 HbS/β-thalassemia: compound heterozygous form of sickle cell disease (with β-thalassemia) 23 HbA/β-thalassemia: β-thalassemia trait (carrier form) 30 HbAA: participants without any known hemoglobin disorders, such as sickle cell disease, sickle cell trait, β-thalassemia, etc.
Age, Categorical <=18 years	33 Participants n=138 Participants
Age, Categorical Between 18 and 65 years	103 Participants n=138 Participants
Age, Categorical >=65 years	2 Participants n=138 Participants
Sex: Female, Male Female	81 Participants n=138 Participants
Sex: Female, Male Male	57 Participants n=138 Participants
Region of Enrollment Canada	27 participants n=138 Participants
Region of Enrollment Nepal	111 participants n=138 Participants

PRIMARY outcome

Timeframe: baseline