- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05543928
Phase 3 Safety and Efficacy Study of CTAP101 Extended-release Capsules in Children With Secondary Hyperparathyroidism
April 2, 2024 updated by: OPKO Health, Inc.
A Multi-Center Study to Evaluate the Efficacy, Safety and Pharmacokinetics of CTAP101 Extended-release Capsules to Treat Secondary Hyperparathyroidism in Pediatric Subjects of Ages 8 to <18 Years With Stage 3 or 4 Chronic Kidney Disease and Vitamin D Insufficiency
This is a phase 3, multi-center, randomized, double-blind, placebo-controlled study in children with stage 3-4 chronic kidney disease (CKD), secondary hyperparathyroidism (SHPT) and vitamin D insufficiency.
Study Overview
Status
Recruiting
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Estimated)
108
Phase
- Phase 3
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
- Name: Christina Gomes
- Phone Number: 747-888-3011
- Email: cgomes@opko.com
Study Locations
-
-
Ohio
-
Columbus, Ohio, United States, 43205
- Recruiting
- Nationwide Childrens Hospital
-
Contact:
- John Mahan, MD
- Phone Number: 614-722-8535
- Email: john.mahan@nationwidechildrens.org
-
Principal Investigator:
- John Mahan, MD
-
-
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
8 years to 17 years (Child)
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
- Cohort 1: Be 12 to <18 years of age and have a body weight of ≥40 kg; Cohort 2: be 8 to <12 years of age and have a body weight of ≥20 kg.
- Be diagnosed with stage 3 to 4 CKD at least six months prior to the screening visit, and have an eGFR of ≥15 to <60 mL/min/1.73m2 at screening.
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, including:
- Serum albumin ≤ 3.0 g/dL;
- Serum transaminase (ALT or SGPT, AST or SGOT) > 2.5 times the upper limit of normal at screening; and,
- Urinary albumin excretion of >3000 mcg/mg creatinine.
Exhibit during the initial or, if necessary, a screening visit after washout:
- Plasma iPTH >100 pg/mL (stage 3 CKD) or >160 pg/mL (stage 4 CKD)
- Serum calcium <9.8 mg/dL (corrected for albumin);
- Serum total 25-hydroxyvitamin D <30 ng/mL; and,
- Serum phosphorus >2.5 to ≤5.5 mg/dL (12 to <18 years) or ≤6.0 mg/dL (ages 8 to <12 years).
- If taking calcitriol or other 1α-hydroxylated vitamin D analogs, or cinacalcet, be willing to forgo treatment with these agents for the duration of the study and complete an 8-week washout period prior to commencing treatment in the study.
- If taking >1,000 mg/day of elemental calcium, discontinue or reduce calcium use and/or use non-calcium based therapies for the duration of the study.
- If receiving ≤1,700 IU/day nutritional vitamin D (ergocalciferol or cholecalciferol) therapy, must agree to remain on a stable dose during the study.
- If taking >1,700 IU/day of nutritional vitamin D, must discontinue or decrease the dose to ≤1,700 IU/day, maintain that dose for the duration of the study, and complete an 8-week washout period prior to commencing treatment in the study provided that serum total 25-hydroxyvitamin D is ≥30 ng/mL. The washout period is not necessary if serum total 25-hydroxyvitami D is <30 ng/mL.
- If taking any bone modifying treatment that could interfere with study endpoints, must discontinue use of such agent(s) for the duration of the study.
- Willing and able to comply with study instructions and commit to all clinic visits for the duration of the study.
- Female subjects of childbearing potential must be neither pregnant nor lactating and must have a negative urine pregnancy test at the first screening visit.
- All female subjects of childbearing potential and male subjects 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.
- Each subject or their legal representative must be able to read, understand and sign the informed consent form (ICF).
Exclusion Criteria:
- History of or planned kidney transplant or parathyroidectomy.
- History (prior three months) of serum calcium ≥9.8 mg/dL.
- Use of bisphosphonate therapy (denosumab) within six months prior to enrollment.
- 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 physical condition that in the opinion of the investigator may worsen and/or interfere with participation in the study.
- History of neurological/psychiatric disorder, including psychotic disorder, or any reason which, in the opinion of the investigator makes adherence to a treatment or follow up schedule unlikely.
- Known or suspected hypersensitivity to any of the constituents of either investigational product.
- Currently participating in, or has participated in, an interventional/investigational study within 30 days prior to study screening.
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: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Placebo Comparator: Cohort 1 and Cohort 2 Placebo
|
Placebo
|
Experimental: Cohort 1 and Cohort 2 CTAP101 Capsule
|
CTAP101 Capsules is an extended-release (ER) oral formulation of calcifediol which was approved as Rayaldee® ER Capsules in June 2016 by the United States (US) Food and Drug Administration (FDA) for the treatment of secondary hyperparathyroidism (SHPT) in adult patients with stage 3 or 4 chronic kidney disease (CKD) and vitamin D insufficiency (VDI), defined as serum total 25-hydroxyvitamin D levels less than 30 ng/mL.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Numbers of subjects who attained mean decrease in plasma iPTH of 30% from baseline
Time Frame: 26 weeks
|
The primary efficacy endpoint is the proportion of subjects in the intent-to- treat (ITT) population (age 8 to <18 years) attaining a mean decrease in plasma iPTH of at least 30% from pre-treatment baseline compared to placebo during the EAP.
|
26 weeks
|
Safety and tolerability
Time Frame: 26 weeks
|
Safety and tolerability will be evaluated in the safety population by AEs, PEs, VS, hematology and laboratory evaluations, and ECGs.
|
26 weeks
|
Pharmacokinetic
Time Frame: 26 weeks
|
To assess the pharmacokinetic (PK) profile of 25-hydroxyvitamin D3 after repeated doses of CTAP101 Capsules in pediatric subjects
|
26 weeks
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Level of serum total 25-hydroxyvitamin D at ≥30 ng/mL compared to placebo
Time Frame: 26 weeks
|
To evaluate the efficacy of repeated dosing with CTAP101 Capsules versus placebo in raising serum total 25-hydroxyvitamin D to ≥30 ng/mL
|
26 weeks
|
Plasma iPTH mean absolute changes and serum total 25-hydroxyvitamin D
Time Frame: 26 weeks
|
To determine the time courses of mean absolute changes from pre-treatment baseline in serum total 25-hydroxyvitamin D and plasma iPTH during administration of repeated doses of CTAP101 Capsules
|
26 weeks
|
Pharmacodynamic effects of repeated doses of CTAP101 Capsules
Time Frame: 26 weeks
|
To assess the PD effects of repeated doses of CTAP101 Capsules versus placebo on mean serum calcium (corrected for albumin), serum phosphorus and serum calcium-timesphosphorus (CaxP) product, and the change in mean urine calcium:creatinine ratio
|
26 weeks
|
Incidence of hypercalcemia and hyperphosphatemia
Time Frame: 26 weeks
|
To evaluate the safety of CTAP101 Capsules versus placebo with regard to the incidence of hypercalcemia and hyperphosphatemia
|
26 weeks
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Akhtar Ashfaq, MD, OPKO Health
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 (Actual)
January 31, 2023
Primary Completion (Estimated)
July 1, 2025
Study Completion (Estimated)
July 1, 2025
Study Registration Dates
First Submitted
September 13, 2022
First Submitted That Met QC Criteria
September 13, 2022
First Posted (Actual)
September 16, 2022
Study Record Updates
Last Update Posted (Actual)
April 3, 2024
Last Update Submitted That Met QC Criteria
April 2, 2024
Last Verified
April 1, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Neoplasms
- Urologic Diseases
- Endocrine System Diseases
- Disease Attributes
- Renal Insufficiency
- Nutrition Disorders
- Parathyroid Diseases
- Neoplastic Processes
- Avitaminosis
- Deficiency Diseases
- Malnutrition
- Chronic Disease
- Female Urogenital Diseases
- Female Urogenital Diseases and Pregnancy Complications
- Urogenital Diseases
- Male Urogenital Diseases
- Kidney Diseases
- Renal Insufficiency, Chronic
- Hyperparathyroidism
- Neoplasm Metastasis
- Vitamin D Deficiency
- Hyperparathyroidism, Secondary
Other Study ID Numbers
- CTAP101-CL-3007
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
product manufactured in and exported from the U.S.
Yes
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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