Microsampling Assays for Immunosuppressive Drugs in Children

NCT ID: NCT04989686

Last Updated: 2025-07-14

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

66 participants

Study Classification

OBSERVATIONAL

Study Start Date

2023-06-08

Study Completion Date

2025-05-01

Brief Summary

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Immunosuppressive therapy is used to treat and manage solid organ and bone marrow/stem cell transplants in children. However, it can be harmful if too little or too much is given. Monitoring immunosuppressive drug (cyclosporine A, tacrolimus, and sirolimus) concentrations in the blood is important to ensure that the drug is given safely and effectively, but current approaches for collecting blood from a vein are painful and often difficult in children. Investigators seek to compare a new approach for monitoring immunosuppressive drug concentrations using a novel small volume blood sampling device, called Tasso-M20, to the traditional way of collecting blood from a vein. Additionally Investigators are interested in assessing patient and family perceptions of the Tasso-M20 device being used for immunosuppressive therapy and their comfortability using the device outside of a clinical setting.

The primary objective of this project is to identify the relationship between cyclosporine A (CYA), tacrolimus (TAC), and sirolimus (SIR) concentrations in the venous blood (gold-standard) and capillary whole blood obtained using the microsampling device Tasso-M20.

The secondary objective of this study is to investigate the stability of CYA, TAC, and SIR in blood samples collected using the Tasso-M20 device under the conditions of shipping and storage.

The sub-study objective is to thematically compare subjects' and families' perceptions of blood collection via the Tasso-M20 device and standard venous blood collection.

Detailed Description

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Immunosuppressive therapy is used to treat and manage solid organ and bone marrow/stem cell transplants; however, suboptimal dosing can lead to organ rejection and graft failure. Immunosuppressant drugs require therapeutic drug monitoring (TDM) to ensure dosing is adequate and therapeutic concentrations are achieved and maintained. The optimal blood concentrations of these drugs are critical to minimize toxicity and simultaneously prevent allograft rejection in an individual transplant patient. Often life-long TDM is required necessitating hospital or laboratory visits for routine venous blood sampling by phlebotomy. Immunosuppressive drugs, cyclosporine A (CYA), tacrolimus (TAC), and sirolimus (SIR) require routine TDM. This entails immunocompromised people leaving their homes for laboratory visits, potentially increasing their risk of acquiring infections.

TDM for CYA, TAC, and SIR are required due to their narrow therapeutic targets: CYA 150-400 ng/mL, TAC 5-12 ng/mL, and SIR 4-12 ng/mL. CYA, TAC, and SIR are primarily distributed in erythrocytes and should be quantified in whole blood. Immunoassays and the liquid chromatography-tandem mass spectrometry (LC-MS/MS) assays are the two commonly used methods of TDM for CYA, TAC, and SIR. While immunoassays provide an accurate measurement of concentrations, they often have some limitations on specificity. LC-MS/MS assays are very specific and efficient since they can quantify multiple analytes with a single method. At the Children's Hospital of Philadelphia (CHOP), the clinical TDM immunoassays for CYA, TAC, and SIR require 0.5-1.0 mL of blood. TDM immunoassays require blood collection by a trained practitioner.

Volumetric absorptive microsampling (VAMS) with an FDA-approved Tasso-M20 device allows for the accurate and precise collection of a fixed volume of blood from a capillary needle without the need for phlebotomy. The Tasso-M20 (FDA Class 1 exempt device) consists of a sample head with a lancet that is activated with the push of a button to accurately and painlessly collect blood samples from the deltoid (or similar) muscle (capillary sampling) of the subjects on all four tips (17.5 µL each). An LC-MS/MS assay with 20 µL blood, as obtained by the Tasso device, was shown to provide the required test range for TDM of trough values. This microsampling technique could be utilized clinically to promote the provision of TDM in children but has not been studied for immunosuppressive drugs (CYA, TAC, and SIR) in children.

Children and their parents/guardians who consent to the optional sub-study will be administered a brief survey after one study visit, to ask about their perceptions of the two methods of blood collection that they experienced at the visit.

Conditions

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Immunosuppression

Study Design

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Observational Model Type

OTHER

Study Time Perspective

PROSPECTIVE

Study Groups

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Cyclosporine A

1. Males and females \<18 years of age
2. Weight greater than 5 kg
3. Receiving cyclosporine A as the standard of care
4. Has scheduled/anticipated blood draw to quantify the concentration of cyclosporine A for clinical indications
5. Parental/guardian permission (informed consent), and subject's assent if applicable.

Microsampling

Intervention Type OTHER

Volumetric absorptive microsampling (VAMS) with Tasso-M20 devices allows for the accurate and precise collection of a fixed small volume of blood from a capillary needle without the need for phlebotomy.

Tacrolimus

1. Males and females \<18 years of age
2. Weight greater than 5 kg
3. Receiving tacrolimus as the standard of care
4. Has scheduled/anticipated blood draw to quantify the concentration of tacrolimus for clinical indications
5. Parental/guardian permission (informed consent), and subject's assent if applicable.

Microsampling

Intervention Type OTHER

Volumetric absorptive microsampling (VAMS) with Tasso-M20 devices allows for the accurate and precise collection of a fixed small volume of blood from a capillary needle without the need for phlebotomy.

Sirolimus

1. Males and females \<18 years of age
2. Weight greater than 5 kg
3. Receiving sirolimus as the standard of care
4. Has scheduled/anticipated blood draw to quantify the concentration of sirolimus for clinical indications
5. Parental/guardian permission (informed consent), and subject's assent if applicable.

Microsampling

Intervention Type OTHER

Volumetric absorptive microsampling (VAMS) with Tasso-M20 devices allows for the accurate and precise collection of a fixed small volume of blood from a capillary needle without the need for phlebotomy.

Interventions

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Microsampling

Volumetric absorptive microsampling (VAMS) with Tasso-M20 devices allows for the accurate and precise collection of a fixed small volume of blood from a capillary needle without the need for phlebotomy.

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

1. Males and females \<18 years of age
2. Weight greater than 5 kg
3. Receiving CYA, TAC, and/or SIR as standard of care
4. Has scheduled/anticipated blood draw to quantify the concentration of CYA, TAC, and SIR\* for clinical indications
5. Parental/guardian permission (informed consent), and subject's assent if applicable.

Exclusion Criteria

1\) Unable to provide blood samples.

\* Potential subjects and their parents/guardians may be approached prior to having a blood draw scheduled if they meet all other eligibility criteria.

Sub-Study Criteria:

Sub-study criteria for child participants will not differ from the main study. Adult participants are required to be a parent/legal guardian of a study subject.
Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

NIH

Sponsor Role collaborator

Children's Hospital of Philadelphia

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Children's Hospital of Phildelphia

Philadelphia, Pennsylvania, United States

Site Status

Countries

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United States

References

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Other Identifiers

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5R21HD106103-02

Identifier Type: NIH

Identifier Source: secondary_id

View Link

22-020410

Identifier Type: -

Identifier Source: org_study_id

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