Pharmacokinetics, Pharmacodynamics, Safety and Tolerability Evaluation of CTAP101 Extended-release Capsules in Pediatric Participants

March 25, 2026 updated by: OPKO Health, Inc.

An Open-Label, Randomized, Multiple-Dose, Multi-Center Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of CTAP101 Extended-release Capsules in Pediatric Participants <18 Years of Age With Secondary Hyperparathyroidism, Stage 3 or 4 Chronic Kidney Disease and Vitamin D Insufficiency

The goal of this clinical trial is to evaluate safety, tolerability, and how CTAP101 Capsules work to treat Pediatric Participants with Secondary Hyperparathyroidism, Stage 3 or 4 Chronic Kidney Disease and Vitamin D Insufficiency. The main questions it aims to answer are:

  1. To assess the pharmacokinetics (PK) and Pharmacodynamics (PD) of CTAP101 Capsules
  2. To investigate the safety and tolerability of CTAP101 Capsules

Participants will take a daily dosage of CTAP101 Capsules at bedtime for 8 weeks.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

40

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • OPKO Site
        • Contact:
    • Ohio
      • Columbus, Ohio, United States, 43205
        • OPKO Site
        • Contact:
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • OPKO Site
        • Contact:
    • South Carolina
      • Greenville, South Carolina, United States, 29615
        • OPKO Site
        • Contact:
    • Wisconsin
      • Madison, Wisconsin, United States, 53562
        • OPKO Site
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

Each participant must meet the following criteria to be enrolled in Cohort 1 of this study:

  1. Be 8 to <18 years of age with a body weight of ≥40 kg.
  2. Be diagnosed with stage 3 or 4 CKD at least six months prior to the screening visit (Visit 1) and have an eGFR of ≥15 to <60 mL/min/1.73m2 at screening.
  3. Be without any disease state or physical condition that might impair evaluation of safety or which, in the investigator's opinion, would interfere with study participation.

  4. Exhibit during the initial or, if necessary, a screening visit after washout:

    1. Plasma iPTH >85 pg/mL;
    2. Serum Ca <9.8 mg/dL;
    3. Serum P ≤5.5 mg/dL; and
    4. Serum 25D <30 ng/mL.
  5. If taking calcitriol or other 1α-hydroxylated vitamin D therapy, be willing to forgo treatment with these agents for the duration of the study and complete a 2-week washout period prior to commencing treatment in the study.
  6. If receiving ≤2,000 IU/day or 50,000 IU/month (1,250 mcg) vitamin D supplementation (ergocalciferol or cholecalciferol) therapy, must agree to remain on a stable dose during the study.
  7. If taking >2,000 IU/day of vitamin D supplementation, must discontinue or decrease the dose to ≤2,000 IU/day, maintain that dose for the duration of the study and complete a 2-week washout period prior to commencing treatment in the study.
  8. If taking >1000 mg/day of elemental Ca through Ca-based phosphate binder or antacid therapy, must discontinue or decrease the dose to ≤ 1000 mg/day, maintain that dose for the duration of the study, and complete a 2-week washout period prior to commencing treatment in the study.
  9. If taking any bone metabolism therapy that could interfere with study endpoints, must discontinue use of such agent(s) for the duration of the study.
  10. Willing and able to comply with study procedures and instructions and commit to all study visits for the duration of the study.
  11. Willing and able to safely swallow the CTAP101 capsules whole.
  12. Female participants of childbearing potential must be neither pregnant nor lactating and must have a negative urine pregnancy test at the first screening visit.
  13. All female participants of childbearing potential and male participants with female partners of childbearing potential must agree to use effective contraception (eg, implants, injectables, combined oral contraceptives, intrauterine device, sexual abstinence, vasectomy or vasectomized partner) for the duration of the study.
  14. Each participant or their legal representative must be able to read, understand and sign the ICF.

Exclusion Criteria:

Participants who meet any of the following criteria will be excluded from the study:

  1. Kidney transplant within the 26 weeks preceding screening or kidney transplant rejection within the 26 weeks preceding screening.
  2. No history of parathyroidectomy or planned parathyroidectomy.
  3. History (prior three months) of serum Ca ≥9.8 mg/dL.
  4. Use of calcimimetic or calcifediol therapy within three months prior to consent.
  5. Use of bisphosphonate or denosumab therapy within six months prior to consent.
  6. Known or suspected hypersensitivity to any of the constituents in CTAP101 Capsules.
  7. Known previous or concomitant serious illness or medical condition, such as malignancy, human immunodeficiency virus, significant gastrointestinal or hepatic disease or cardiovascular event or hepatitis, or abnormal ECG or physical condition that in the opinion of the investigator may worsen and/or interfere with participation in the study.
  8. History of neurological/psychiatric disorder, including psychotic disorder, or any reason which, in the opinion of the investigator makes adherence to a treatment or FU schedule unlikely.
  9. Currently participating in, or has participated in, an interventional/investigational study within 30 days prior to study screening that in the opinion of the investigator and/or sponsor would interfere with the safety, assessments, and results of this study.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort 1 - Group A
30 mcg CTAP101 Capsules
Drug: CTAP101
Capsules
Experimental: Cohort 1 - Group B
30 mcg or 60 mcg CTAP101 Capsules
Drug: CTAP101
Capsules

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of treatment emergent adverse events (TEAE)
Time Frame: Up to 14 weeks
TEAEs will be graded as mild, moderate, severe.
Up to 14 weeks
Apparent clearance (CL/F)
Time Frame: Up to 14 weeks
To determine the CL/F of CTAP101 Capsules during and after repeated dosing
Up to 14 weeks
Volume of distribution (Vd/F)
Time Frame: Up to 14 weeks
To determine the Vd/F of CTAP101 Capsules during and after repeated dosing
Up to 14 weeks
Terminal elimination half-life (t1/2)
Time Frame: Up to 14 weeks
To determine the t1/2 of CTAP101 Capsules during and after repeated dosing
Up to 14 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

OPKO Health, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

June 1, 2026

Primary Completion (Estimated)

October 1, 2027

Study Completion (Estimated)

August 1, 2028

Study Registration Dates

First Submitted

March 20, 2026

First Submitted That Met QC Criteria

March 20, 2026

First Posted (Actual)

March 27, 2026

Study Record Updates

Last Update Posted (Actual)

March 30, 2026

Last Update Submitted That Met QC Criteria

March 25, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CTAP101-CL-2015

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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