Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS)

Recruiting

• Conditions: Pulmonary Arterial Hypertension; Urinary Tract Infections in Children; Hemophilia; Menorrhagia; Attention Deficit Hyperactivity Disorder; Multisystem Inflammatory Syndrome in Children (MIS-C); Coronavirus Infection (COVID-19); Pain; Hyperphosphatemia; Edema

• Registration Number: NCT04278404
• Lead Sponsor: Duke University

Brief Summary

The study investigators are interested in learning more about how drugs, that are given to children by their health care provider, act in the bodies of children and young adults in hopes to find the most safe and effective dose for children. The primary objective of this study is to evaluate the PK of understudied drugs currently being administered to children per SOC as prescribed by their treating provider.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-02-18
• Study First Submitted QC: 2020-02-18
• Study First Posted: 2020-02-20
• Study Start: 2020-03-05
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-11
• Last Update Posted: 2025-03-12
• Primary Completion: 2026-09
• Study Completion: 2027-09

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 5000

Inclusion Criteria

1. Participant is < 21 years of age
2. Parent/ Legal Guardian/ Adult Participant can understand the consent process and is willing to provide informed consent/HIPAA:
3. (a) Participant is receiving one or more of the study drugs of interest at the time of enrollment or (b) Participant is NOT receiving one or more of the study drugs of interest but is SARS-COV-2 positive within 60 days prior to enrollment

Exclusion Criteria

1. Participant has a known pregnancy

   Below exclusion criteria apply only to:

   Participants receiving one or more of the study drugs of interest at the time of enrollment, DOI administration or PK sampling: (Refer to DOI specific appendices for details on enrollment cohort specifications and additional eligibility criteria)

2. Has had intermittent dialysis within previous 24 hours

3. Has had a kidney transplant within previous 30 days

4. Has had a liver transplant within previous 1 year

5. Has had a stem cell transplant within previous 1 year

6. Has had therapeutic hypothermia within previous 24 hours

7. Has had plasmapheresis within the previous 24 hours

8. Has a Ventricular Assist Device

9. Has any condition which would make the participant, in the opinion of the investigator, unsuitable for the study

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Elimination rate constant (ke) as measured by PK sampling	Data will be collected up to 90 days from the time of consent. For participants with Down Syndrome enrolling at sites designated as Down Syndrome sites, participants will be in the study for up to 210 days.
Half-life (t1/2) as measured by PK sampling	Data will be collected up to 90 days from the time of consent. For participants with Down Syndrome enrolling at sites designated as Down Syndrome sites, participants will be in the study for up to 210 days.
Volume of distribution (V) or apparent oral volume of distribution (V/F) as measured by PK sampling	Data will be collected up to 90 days from the time of consent. For participants with Down Syndrome enrolling at sites designated as Down Syndrome sites, participants will be in the study for up to 210 days.
Clearance (CL) or apparent oral clearance (CL/F) as measured by PK sampling	Data will be collected up to 90 days from the time of consent. For participants with Down Syndrome enrolling at sites designated as Down Syndrome sites, participants will be in the study for up to 210 days.
Time to achieve maximum concentration (Tmax) as measured by PK sampling	Data will be collected up to 90 days from the time of consent. For participants with Down Syndrome enrolling at sites designated as Down Syndrome sites, participants will be in the study for up to 210 days.
Maximum concentration (Cmax) as measured by PK sampling	Data will be collected up to 90 days from the time of consent. For participants with Down Syndrome enrolling at sites designated as Down Syndrome sites, participants will be in the study for up to 210 days.
Absorption rate constant (ka) as measured by PK sampling	Data will be collected up to 90 days from the time of consent. For participants with Down Syndrome enrolling at sites designated as Down Syndrome sites, participants will be in the study for up to 210 days.
AUC (area under the curve) as measured by PK sampling	Data will be collected up to 90 days from the time of consent. For participants with Down Syndrome enrolling at sites designated as Down Syndrome sites, participants will be in the study for up to 210 days.

Trial Locations

Locations (49)

Phoenix Children's Hospital; 🇺🇸 Phoenix, Arizona, United States

Arkansas Children's Hospital Research Institute; 🇺🇸 Little Rock, Arkansas, United States

University of California, Los Angeles Medical Center; 🇺🇸 Los Angeles, California, United States

Lucile Packard Children's Hospital; 🇺🇸 Palo Alto, California, United States

Colorado University Denver; 🇺🇸 Aurora, Colorado, United States

The Children's Hospital Colorado; 🇺🇸 Aurora, Colorado, United States

Alfred I. DuPont Hospital for Children; 🇺🇸 Wilmington, Delaware, United States

University of Florida Jacksonville Shands Medical Center; 🇺🇸 Jacksonville, Florida, United States

Kapiolani Womens and Childrens Medical Center; 🇺🇸 Honolulu, Hawaii, United States

Ann and Robert H. Lurie Childrens Hospital of Chicago; 🇺🇸 Chicago, Illinois, United States

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