Chronic Kidney Disease stage3Phase 3NCT05543928
Phase 3 Safety and Efficacy Study of CTAP101 Extended-release Capsules in Children With Secondary Hyperparathyroidism
A Phase 3 study testing CTAP101 in people with Chronic Kidney Disease stage3 and Chronic Kidney Disease stage4, run by OPKO Health, Inc. at 2 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 8 to 17 years
- Conditions the study is for
- Chronic Kidney Disease stage3, Chronic Kidney Disease stage4, Vitamin d Deficiency
- What is being tested?
- CTAP101
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 2 participants
- Where
- 2 sitesUnited States
- Study dates
- 31 Jan 2023to 19 Jun 2024
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
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- 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-hydroxyvitamin 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).
Who cannot join
- 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.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
OPKO Study Site
Columbus, Ohio, 43205
OPKO Study Site
Greenville, South Carolina, 29615
About this study
Official title: 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.
- Conditions
- Chronic Kidney Disease stage3, Chronic Kidney Disease stage4, Vitamin d Deficiency, Secondary Hyperparathyroidism
- Being tested
- CTAP101, Placebo
- Medical subject headings
- Vitamin D Deficiency, Hyperparathyroidism, Secondary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CTAP101Drug
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.
PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 and Cohort 2 Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
| Cohort 1 and Cohort 2 CTAP101 Capsule | Experimental | Not described Receives: Drug: CTAP101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Numbers of Subjects Who Attained Mean Decrease in Plasma iPTH of 30% From Baseline
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.
Measured: 26 weeks
Safety and Tolerability
Safety and tolerability will be evaluated in the safety population by AEs, PEs, VS, hematology and laboratory evaluations, and ECGs.
Measured: 26 weeks
Pharmacokinetic
To assess the pharmacokinetic (PK) profile of 25-hydroxyvitamin D3 after repeated doses of CTAP101 Capsules in pediatric subjects
Measured: 26 weeks
Secondary outcomes (4)
Level of Serum Total 25-hydroxyvitamin D at ≥30 ng/mL Compared to Placebo
To evaluate the efficacy of repeated dosing with CTAP101 Capsules versus placebo in raising serum total 25-hydroxyvitamin D to ≥30 ng/mL
Measured: 26 weeks
Plasma iPTH Mean Absolute Changes and Serum Total 25-hydroxyvitamin D
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
Measured: 26 weeks
Pharmacodynamic Effects of Repeated Doses of CTAP101 Capsules
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-times-phosphorus (CaxP) product, and the change in mean urine calcium:creatinine ratio
Measured: 26 weeks
Incidence of Hypercalcemia and Hyperphosphatemia
To evaluate the safety of CTAP101 Capsules versus placebo with regard to the incidence of hypercalcemia and hyperphosphatemia
Measured: 26 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2022
- Study start31 Jan 2023
- Primary completion19 Jun 2024
- Study completion19 Jun 2024
Why it stopped: FDA changed the post-marketing requirement necessitating a new study design and protocol.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Chronic Kidney Disease stage3Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- OPKO Health, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Akhtar Ashfaq, MD, Study director, OPKO Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05543928
- Sponsor's protocol ID
- CTAP101-CL-3007
- Status last verified
- Jul 2025
- First submitted
- 13 Sep 2022
- Last updated
- 25 Jul 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05543928
- Acronym / sponsor ID
- CTAP101-CL-3007
- Conditions
- Chronic Kidney Disease stage3, Chronic Kidney Disease stage4, Vitamin d Deficiency, Secondary Hyperparathyroidism
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: FDA changed the post-marketing requirement necessitating a new study design and protocol.
- Enrolment
- 2 participants (actual)
- Lead sponsor
- OPKO Health, Inc.Industry (pharma, biotech, devices)
- Study start
- 31 Jan 2023 (actual)
- Primary completion
- 19 Jun 2024 (actual)
- Study completion
- 19 Jun 2024 (actual)
- First posted
- 16 Sep 2022 (actual)
- Results
- Posted 25 Jul 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 and Cohort 2 Placebo | Placebo comparator | Drug: Placebo |
| Cohort 1 and Cohort 2 CTAP101 Capsule | Experimental | Drug: CTAP101 |
Who may qualify
- Ages
- 8 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (20 listed)
- 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:
- Exhibit during the initial or, if necessary, a screening visit after washout:
- 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.
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CTAP101CTAP101 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. | Drug | Cohort 1 and Cohort 2 CTAP101 Capsule |
| PlaceboPlacebo | Drug | Cohort 1 and Cohort 2 Placebo |
Primary outcome
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.
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.
Pharmacokinetic
Time frame: 26 weeks
To assess the pharmacokinetic (PK) profile of 25-hydroxyvitamin D3 after repeated doses of CTAP101 Capsules in pediatric subjects
4 secondary outcomes
Level of Serum Total 25-hydroxyvitamin D at ≥30 ng/mL Compared to Placebo
Time frame: 26 weeks
Plasma iPTH Mean Absolute Changes and Serum Total 25-hydroxyvitamin D
Time frame: 26 weeks
Pharmacodynamic Effects of Repeated Doses of CTAP101 Capsules
Time frame: 26 weeks
Incidence of Hypercalcemia and Hyperphosphatemia
Time frame: 26 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jul 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Akhtar Ashfaq, MD study director
OPKO Health
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing CTAP101 in people with Chronic Kidney Disease stage3 and Chronic Kidney Disease stage4, run by OPKO Health, Inc. at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 20 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 25 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 25 Jul 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Numbers of Subjects Who Attained Mean Decrease in Plasma iPTH of 30% From Baseline (time frame: 26 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Jul 2025.