Safety, Efficacy, PK and PD of CTAP101 (Calcifediol) ER Capsules for SHPT in HD Patients VDI

NCT03602261 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: CTAP101-CL-2010
• Study Type: interventional
• Target: 44
• Locations: 1 country
• Sites: 18

Brief Summary

Safety, Efficacy, PK and PD of CTAP101 (calcifediol) ER Capsules for SHPT in HD Patients VDI

Conditions

Secondary Hyperparathyroidism Due to Renal Causes; Chronic Kidney Diseases; Vitamin D Deficiency; Stage 5 Chronic Kidney Disease

Outcome Measures

Primary Outcomes (6)

• Number of Participants With Response During Efficacy Assessment Period

  To evaluate the efficacy of repeated dosing with 900 mcg per week of CTAP101 extended release (ER) Capsules versus placebo in raising mean serum total 25-hydroxyvitamin D (25D) to ≥50 ng/mL and in reducing mean plasma intact parathyroid hormone (iPTH) by at least 30% from pre-treatment baseline.

  26 weeks

• Total 25-hydroxyvitamin D Response Analysis During Efficacy Period

  Summary of participants with mean serum total 25-hydroxyvitamin D ≥50 ng/mL at the end of treatment

  26 weeks

• Number of Participants With Intact Parathyroid Hormone (iPTH) Response During Efficacy Assessment Period

  Summary of participants who experienced a reduction in mean plasma iPTH by greater than 30% from the pre-treatment baseline

  26 weeks

• Pharmacokinetic (PK) Profile (Cmax) of Serum Calcifediol

  To assess Cmax of serum calcifediol after a single dose of 900 mcg at the start of the study (Single Dose PK Period), and a repeat dose of 300 mcg at the end of the study (Repeat Dose PK Period)

  0, 4, 8, 12, 16, 20, 24, 30, 36, 42 and 48 hours post-dose (single and repeat dose)

• Pharmacokinetic (PK) Profile (Tmax) of Serum Calcifediol

  To assess Tmax of serum calcifediol after a single dose of 900 mcg at the start of the study (Single Dose PK Period), and a repeat dose of 300 mcg at the end of the study (Repeat Dose PK Period)

  0, 4, 8, 12, 16, 20, 24, 30, 36, 42 and 48 hours post-dose (single and repeat dose)

• Pharmacokinetic (PK) Profile (AUC0-t) of Serum Calcifediol

  To assess AUC0-t of serum calcifediol after a single dose of 900 mcg at the start of the study (Single Dose PK Period), and a repeat dose of 300 mcg at the end of the study (Repeat Dose PK Period)

  0, 4, 8, 12, 16, 20, 24, 30, 36, 42 and 48 hours post-dose (single and repeat dose)

Secondary Outcomes (1)

• Pharmacodynamic Analysis of 1,25-dihydroxyvitamin D

  26 weeks

Study Arms (2)

CTAP101 Capsules 900mcg/weekly

ACTIVE COMPARATOR

CTAP101 Oral Capsules/Calcifediol, calcidiol, 25-hydroxyvitamin D3, 900mcg/weekly for 26 weeks

Drug: Calcifediol Oral Capsule

Placebo Capsules weekly

PLACEBO COMPARATOR

Placebo Oral Capsules/weekly for 26 weeks

Drug: Placebo oral capsule

Interventions

Calcifediol Oral Capsule DRUG

Capsule, weekly

Also known as: CTAP101

CTAP101 Capsules 900mcg/weekly

Placebo oral capsule DRUG

Capsule, weekly

Placebo Capsules weekly

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Each subject must meet the following criteria to be enrolled in this study:
• Be at least 18 years of age.
• Be diagnosed with CKD requiring in-center HD tiw for the preceding 6 months, as confirmed by medical history.
• 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; and,
• Serum transaminase (alanine transaminase \[ALT\], glutamic pyruvic transaminase \[SGPT\], aspartate aminotransferase \[AST\] or glutamic oxaloacetic transaminase \[SGOT\]) \> 2.5 times the upper limit of normal at screening.
• Be receiving calcimimetic therapy (either etelcalcetide or cinacalcet) and/or calcitriol or other 1α-hydroxylated vitamin D analog (paricalcitol or doxercalciferol) for at least 1 month at the time of screening for enrollment. Approximately 50% of enrolled subjects will have been receiving calcimimetic therapy.
• Exhibit during the initial screening visit:
• Plasma immunoreactive parathyroid hormone (iPTH) ≥150 pg/mL and \<600 pg/mL if receiving etelcalcetide, cinacalcet, calcitriol or other 1α-hydroxylated vitamin D analog (paricalcitol or doxercalciferol); or
• Plasma iPTH ≥300 pg/mL and \<900 pg/mL if not receiving etelcalcetide, cinacalcet, calcitriol or other 1α-hydroxylated vitamin D analog; and,
• Serum total 25-hydroxyvitamin D \<30 ng/mL if not receiving vitamin D supplementation.
• When otherwise confirmed eligible at Visit 1, must forgo any further treatment with etelcalcetide and cinacalcet for the duration of the study and undergo an 8-week washout period.
• When otherwise confirmed eligible at Visit 1, must forgo any further treatment with calcitriol or other 1α-hydroxylated vitamin D analogs or vitamin D supplements for the duration of the study and undergo an 8-week washout period.
• Exhibit after the 8-week washout period (if required due to prior use of etelcalcetide, cinacalcet, calcitriol or other 1α-hydroxylated vitamin D analogs, or vitamin D supplementation):
• Plasma iPTH increased by at least 50%;

You may not qualify if:

• Subjects who meet any of the following criteria will be excluded from the study:
• Scheduled kidney transplant or parathyroidectomy.
• History (prior 2 months) of corrected serum calcium ≥9.8 mg/dL or serum phosphorus
• ≥6.5 mg/dL if not receiving calcitriol or other 1α-hydroxylated vitamin D analog.
• Receipt of bisphosphonate therapy or other bone modifying treatment (eg, denosumab) within 6 months prior to enrollment.
• Known previous or concomitant serious illness or medical condition, such as malignancy, human immunodeficiency virus, significant gastrointestinal or hepatic disease, intestinal malabsorption disorder, hepatitis or cardiovascular event that in the opinion of the investigator may worsen or reduce life expectancy, and/or interfere with participation in the study.
• History of neurological/psychiatric disorder, including psychotic disorder or dementia, 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 the study drugs.
• 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 (18)

AKDHC Medical Research Services

Peoria, Arizona, 85381, United States

Location

AKDHC Medical Research Services

Phoenix, Arizona, 85027, United States

Location

AKDHC Medical Research Services

Phoenix, Arizona, 85035, United States

Location

AKDHC Medical Research Services

Phoenix, Arizona, 85258, United States

Location

WCCT Global, Inc.

Cypress, California, 90630, United States

Location

Hacienda Dialysis Center

Hacienda Heights, California, 91745, United States

Location

California Institute of Renal Research CKD/Dialysis & Transplant Division

La Mesa, California, 91942, United States

Location

Long Beach Quest Dialysis Center

Long Beach, California, 90807, United States

Location

Ontario Dialysis Center

Ontario, California, 91762, United States

Location

North America Research Institute, Inc.

San Dimas, California, 91773, United States

Location

Laurel Canyon Dialysis, LLC

Sun Valley, California, 91352, United States

Location

University of Colorado Denver Anschutz Medical Campus

Aurora, Colorado, 80045, United States

Location

Research by Design, LLC

Chicago, Illinois, 60643, United States

Location

Northshore University Health

Evanston, Illinois, 60201, United States

Location

Renal and Transplant Associates of New England

Springfield, Massachusetts, 01107, United States

Location

Southwest MS Nephrology

McComb, Mississippi, 39601, United States

Location

Southwest Houston Research LTD

Houston, Texas, 77099, United States

Location

Kidney & Hypertension Specialists

San Antonio, Texas, 78207, United States

Location

MeSH Terms

Conditions

Renal Insufficiency, Chronic; Vitamin D Deficiency

Interventions

Calcifediol

Condition Hierarchy (Ancestors)

Renal Insufficiency; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Avitaminosis; Deficiency Diseases; Malnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Hydroxycholecalciferols; Cholecalciferol; Cholestenes; Cholestanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Sterols; Vitamin D; Secosteroids; Membrane Lipids; Lipids

Results Point of Contact

• Title: Akhtar Ashfaq
• Organization: OPKO Health

AAshfaq@opko.com

305-575-423

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 13, 2018
• First Posted: July 26, 2018
• Study Start: July 9, 2018
• Primary Completion: February 24, 2021
• Study Completion: February 24, 2021
• Last Updated: March 30, 2025
• Results First Posted: March 30, 2025

Record last verified: 2025-03

Locations

US (18)