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History of Changes for Study: NCT05543928
Phase 3 Safety and Efficacy Study of CTAP101 Extended-release Capsules in Children With Secondary Hyperparathyroidism
Latest version (submitted April 2, 2024) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 September 13, 2022 None (earliest Version on record)
2 November 14, 2022 Study Status
3 January 18, 2023 Study Status
4 February 22, 2023 Recruitment Status, Study Status, Contacts/Locations and Oversight
5 April 2, 2024 Study Status
Comparison Format:
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Changes (Side-by-Side) for Study: NCT05543928
September 13, 2022 (v1) -- February 22, 2023 (v4)

Changes in: Study Status, Oversight and Contacts/Locations
Open or close this module Study Identification
Unique Protocol ID: CTAP101-CL-3007 CTAP101-CL-3007
Brief Title: Phase 3 Safety and Efficacy Study of CTAP101 Extended-release Capsules in Children With Secondary Hyperparathyroidism Phase 3 Safety and Efficacy Study of CTAP101 Extended-release Capsules in Children With Secondary Hyperparathyroidism
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 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
Secondary IDs:
Open or close this module Study Status
Record Verification: September 2022 February 2023
Overall Status: Not yet recruitingRecruiting
Study Start: October 2022 January 31, 2023
Primary Completion: July 2025 [Anticipated] July 2025 [Anticipated]
Study Completion: July 2025 [Anticipated] July 2025 [Anticipated]
First Submitted: September 13, 2022 September 13, 2022
First Submitted that
Met QC Criteria:		 September 13, 2022 September 13, 2022
First Posted: September 16, 2022 [Actual] September 16, 2022 [Actual]
Last Update Submitted that
Met QC Criteria:		 September 13, 2022 February 22, 2023
Last Update Posted: September 16, 2022 [Actual] February 24, 2023 [Actual]
Open or close this module Sponsor/Collaborators
Sponsor: OPKO Health, Inc. OPKO Health, Inc.
Responsible Party: Sponsor Sponsor
Collaborators:
Open or close this module Oversight
U.S. FDA-regulated Drug: YesYes
U.S. FDA-regulated Device: NoNo
Data Monitoring: Yes Yes
Open or close this module Study Description
Brief 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. 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.
Detailed Description:
Open or close this module Conditions
Conditions: Chronic Kidney Disease stage3
Chronic Kidney Disease stage4
Vitamin d Deficiency
Secondary Hyperparathyroidism 		Chronic Kidney Disease stage3
Chronic Kidney Disease stage4
Vitamin d Deficiency
Secondary Hyperparathyroidism
Keywords:
Open or close this module Study Design
Study Type: InterventionalInterventional
Primary Purpose: TreatmentTreatment
Study Phase: Phase 3Phase 3
Interventional Study Model: Sequential Assignment Sequential Assignment
Number of Arms: 22
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Allocation: RandomizedRandomized
Enrollment: 108 [Anticipated] 108 [Anticipated]
Open or close this module Arms and Interventions
Arms Assigned Interventions
Placebo Comparator: Cohort 1 and Cohort 2 Placebo Drug: Placebo
Placebo
Experimental: Cohort 1 and Cohort 2 CTAP101 Capsule Drug: CTAP101
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.
Open or close this module Outcome Measures
Primary Outcome Measures:
1. 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. 		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.
2. 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. 		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.
3. Pharmacokinetic
[ Time Frame: 26 weeks ]

To assess the pharmacokinetic (PK) profile of 25-hydroxyvitamin D3 after repeated doses of CTAP101 Capsules in pediatric subjects 		Pharmacokinetic
[ Time Frame: 26 weeks ]

To assess the pharmacokinetic (PK) profile of 25-hydroxyvitamin D3 after repeated doses of CTAP101 Capsules in pediatric subjects
Secondary Outcome Measures:
1. 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 		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
2. 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 		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
3. 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 		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
4. 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 		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
Open or close this module Eligibility
Minimum Age: 8 Years 8 Years
Maximum Age: 17 Years 17 Years
Sex: All All
Gender Based:
Accepts Healthy Volunteers: NoNo
Criteria:

Inclusion Criteria:

  1. 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.
  2. 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.
  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, including:
    1. Serum albumin ≤ 3.0 g/dL;
    2. Serum transaminase (ALT or SGPT, AST or SGOT) > 2.5 times the upper limit of normal at screening; and,
    3. Urinary albumin excretion of >3000 mcg/mg creatinine.
  4. Exhibit during the initial or, if necessary, a screening visit after washout:
    1. Plasma iPTH >100 pg/mL (stage 3 CKD) or >160 pg/mL (stage 4 CKD)
    2. Serum calcium <9.8 mg/dL (corrected for albumin);
    3. Serum total 25-hydroxyvitamin D <30 ng/mL; and,
    4. Serum phosphorus >2.5 to ≤5.5 mg/dL (12 to <18 years) or ≤6.0 mg/dL (ages 8 to <12 years).
  5. 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.
  6. 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.
  7. If receiving ≤1,700 IU/day nutritional vitamin D (ergocalciferol or cholecalciferol) therapy, must agree to remain on a stable dose during the study.
  8. 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.
  9. 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.
  10. Willing and able to comply with study instructions and commit to all clinic visits for the duration of the study.
  11. Female subjects of childbearing potential must be neither pregnant nor lactating and must have a negative urine pregnancy test at the first screening visit.
  12. 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.
  13. Each subject or their legal representative must be able to read, understand and sign the informed consent form (ICF).

Exclusion Criteria:

  1. History of or planned kidney transplant or parathyroidectomy.
  2. History (prior three months) of serum calcium ≥9.8 mg/dL.
  3. Use of bisphosphonate therapy (denosumab) within six months prior to enrollment.
  4. 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.
  5. 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.
  6. Known or suspected hypersensitivity to any of the constituents of either investigational product.
  7. Currently participating in, or has participated in, an interventional/investigational study within 30 days prior to study screening.

Inclusion Criteria:

  1. 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.
  2. 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.
  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, including:
    1. Serum albumin ≤ 3.0 g/dL;
    2. Serum transaminase (ALT or SGPT, AST or SGOT) > 2.5 times the upper limit of normal at screening; and,
    3. Urinary albumin excretion of >3000 mcg/mg creatinine.
  4. Exhibit during the initial or, if necessary, a screening visit after washout:
    1. Plasma iPTH >100 pg/mL (stage 3 CKD) or >160 pg/mL (stage 4 CKD)
    2. Serum calcium <9.8 mg/dL (corrected for albumin);
    3. Serum total 25-hydroxyvitamin D <30 ng/mL; and,
    4. Serum phosphorus >2.5 to ≤5.5 mg/dL (12 to <18 years) or ≤6.0 mg/dL (ages 8 to <12 years).
  5. 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.
  6. 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.
  7. If receiving ≤1,700 IU/day nutritional vitamin D (ergocalciferol or cholecalciferol) therapy, must agree to remain on a stable dose during the study.
  8. 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.
  9. 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.
  10. Willing and able to comply with study instructions and commit to all clinic visits for the duration of the study.
  11. Female subjects of childbearing potential must be neither pregnant nor lactating and must have a negative urine pregnancy test at the first screening visit.
  12. 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.
  13. Each subject or their legal representative must be able to read, understand and sign the informed consent form (ICF).

Exclusion Criteria:

  1. History of or planned kidney transplant or parathyroidectomy.
  2. History (prior three months) of serum calcium ≥9.8 mg/dL.
  3. Use of bisphosphonate therapy (denosumab) within six months prior to enrollment.
  4. 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.
  5. 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.
  6. Known or suspected hypersensitivity to any of the constituents of either investigational product.
  7. Currently participating in, or has participated in, an interventional/investigational study within 30 days prior to study screening.
Open or close this module Contacts/Locations
Central Contact Person: Christina Gomes
Telephone: 747-888-3011
Email: cgomes@opko.com		Christina Gomes
Telephone: 747-888-3011
Email: cgomes@opko.com
Study Officials: Akhtar Ashfaq, MD
Study Director
OPKO Health 		Akhtar Ashfaq, MD
Study Director
OPKO Health
Locations: United States, Ohio
Nationwide Childrens Hospital
[Recruiting]
Columbus, Ohio, United States, 43205
Contact:Contact: John Mahan, MD 614-722-8535 john.mahan@nationwidechildrens.org
Contact:Principal Investigator: John Mahan, MD
Open or close this module IPDSharing
Plan to Share IPD: No No
Open or close this module References
Citations:
Links:
Available IPD/Information:
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U.S. National Library of Medicine | U.S. National Institutes of Health | U.S. Department of Health & Human Services