Phase 3 Safety and Efficacy Study of CTAP101 Extended-release Capsules in Children With Secondary Hyperparathyroidism

NCT05543928 | Terminated Phase 3 Results DMC FDA Drug

• 1 other identifier: CTAP101-CL-3007
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 2

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.

Conditions

Chronic Kidney Disease stage3; Chronic Kidney Disease stage4; Vitamin d Deficiency; Secondary Hyperparathyroidism

Outcome Measures

Primary Outcomes (3)

• 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.

  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.

  26 weeks

• Pharmacokinetic

  To assess the pharmacokinetic (PK) profile of 25-hydroxyvitamin D3 after repeated doses of CTAP101 Capsules in pediatric subjects

  26 weeks

Secondary Outcomes (4)

• Level of Serum Total 25-hydroxyvitamin D at ≥30 ng/mL Compared to Placebo

  26 weeks

• Plasma iPTH Mean Absolute Changes and Serum Total 25-hydroxyvitamin D

  26 weeks

• Pharmacodynamic Effects of Repeated Doses of CTAP101 Capsules

  26 weeks

• Incidence of Hypercalcemia and Hyperphosphatemia

  26 weeks

Study Arms (2)

Cohort 1 and Cohort 2 Placebo

PLACEBO COMPARATOR

Drug: Placebo

Cohort 1 and Cohort 2 CTAP101 Capsule

EXPERIMENTAL

Drug: CTAP101

Interventions

CTAP101 DRUG

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.

Cohort 1 and Cohort 2 CTAP101 Capsule

Placebo DRUG

Placebo

Cohort 1 and Cohort 2 Placebo

Eligibility Criteria

• Age: 8 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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-hydroxyvitamin D is \<30 ng/mL.

You may not qualify if:

• 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.

Sponsors & Collaborators

• OPKO Health, Inc.: lead

Study Sites (2)

OPKO Study Site

Columbus, Ohio, 43205, United States

Location

OPKO Study Site

Greenville, South Carolina, 29615, United States

Location

MeSH Terms

Conditions

Vitamin D Deficiency; Hyperparathyroidism, Secondary

Condition Hierarchy (Ancestors)

Avitaminosis; Deficiency Diseases; Malnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases; Hyperparathyroidism; Parathyroid Diseases; Endocrine System Diseases

Results Point of Contact

• Title: OPKO Health Inc
• Organization: OPKO Health Inc

contact@opko.com

3055754100

Study Officials

• Akhtar Ashfaq, MD

  OPKO Health

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 13, 2022
• First Posted: September 16, 2022
• Study Start: January 31, 2023
• Primary Completion: June 19, 2024
• Study Completion: June 19, 2024
• Last Updated: July 25, 2025
• Results First Posted: July 25, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)