Trial Outcomes & Findings for Phase 3 Safety and Efficacy Study of CTAP101 Extended-release Capsules in Children With Secondary Hyperparathyroidism (NCT NCT05543928)

NCT ID: NCT05543928

Last Updated: 2025-07-25

Results Overview

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.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

2 participants

Primary outcome timeframe

26 weeks

Results posted on

2025-07-25

Participant Flow

Participant milestones

Participant milestones
Measure
Cohort 1 and Cohort 2 Placebo
Placebo: Placebo
Cohort 1 and Cohort 2 CTAP101 Capsule
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.
Overall Study
STARTED
0
0
Overall Study
COMPLETED
0
0
Overall Study
NOT COMPLETED
0
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline data not reported

PRIMARY outcome

Timeframe: 26 weeks

Population: The study was terminated before randomization

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.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: 26 weeks

Population: The study was terminated before randomization

Safety and tolerability will be evaluated in the safety population by AEs, PEs, VS, hematology and laboratory evaluations, and ECGs.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: 26 weeks

Population: The study was terminated before randomization

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

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 26 weeks

Population: The study was terminated before randomization

To evaluate the efficacy of repeated dosing with CTAP101 Capsules versus placebo in raising serum total 25-hydroxyvitamin D to ≥30 ng/mL

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 26 weeks

Population: The study was terminated before randomization

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

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 26 weeks

Population: The study was terminated before randomization

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

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 26 weeks

Population: The study was terminated before randomization

To evaluate the safety of CTAP101 Capsules versus placebo with regard to the incidence of hypercalcemia and hyperphosphatemia

Outcome measures

Outcome data not reported

Adverse Events

Cohort 1 and Cohort 2 Placebo

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Cohort 1 and Cohort 2 CTAP101 Capsule

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

OPKO Health Inc

OPKO Health Inc

Phone: 3055754100

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place