ClinicalTrials.gg
CompletedNCT01704079Updated Jan 13, 2025Results posted

Safety and Efficacy of CTAP101 to Treat Secondary Hyperparathyroidism in Stage 3 or 4 CKD and Vitamin D Insufficiency

A Phase 3 interventional study of CTAP101 30 μg capsules and Sugar pill to CTAP101 30 μg capsules in Chronic Kidney Disease, Hyperparathyroidism, Secondary and Vitamin D Deficiency, sponsored by OPKO IP Holdings II, Inc.. Completed at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-01-13.

Sponsored by OPKO IP Holdings II, Inc. (part of OPKO Health, Inc.) · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
216
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study summary

This study will evaluate the efficacy of CTAP101 Capsules versus placebo in reducing intact parathyroid hormone (iPTH) by at least 30% from pretreatment baseline; safety and tolerability of CTAP101 will also be evaluated

Read the detailed description

This is a phase 3, multi-center, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of CTAP101 Capsules administered for 26 weeks to treat SHPT in subjects ≥18 years of age with stage 3 or 4 CKD and vitamin D insufficiency. The study will be conducted at approximately 40 sites within the United States (US). Approximately 550 subjects will be screened to randomize approximately 210 eligible subjects, stratified by CKD stage (approximately 105 subjects in each stage) in a 2:1 ratio to receive either CTAP101 Capsules or matching placebo. An Interactive Voice Response System (IVRS) will provide study treatment group assignments using the computer-generated randomization code provided by the IVRS vendor. Subjects will receive an initial daily dose of 1 capsule (CTAP101 Capsules, 30 µg, or matching placebo) for the first 12 weeks. After l2 weeks (visit 8) and per pre-defined criteria, the dose may be increased in a blinded fashion by the IVRS with oversight by an independent medical monitor.

02

Conditions studied

  • Chronic Kidney Disease
  • Hyperparathyroidism, Secondary
  • Vitamin D Deficiency

Keywords

  • Parathyroid Diseases
  • Renal Insufficiency
  • Kidney Failure, Chronic
  • Hyperparathyroidism, Secondary
  • Vitamin D
  • Hyperparathyroidism
  • Kidney Diseases
  • Kidney Failure
  • Renal Insufficiency, Chronic
03

In context

Renal Insufficiency, Chronic

3,090 studies on the registry are indexed under Renal Insufficiency, Chronic; 698 are open to participants now.

This study's enrollment of 216 is above the median of 73 across 2,157 interventional studies indexed under Renal Insufficiency, Chronic.

Browse Renal Insufficiency, Chronic studies →

Lead sponsor

OPKO IP Holdings II, Inc. is the lead sponsor of 5 studies on the registry; none are open to participants now.

First submitted before OPKO IP Holdings II, Inc. became part of OPKO Health, Inc..

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  1. Urinary albumin excretion ≤ 3000 mcg/mg of creatinine
  2. Stage 3 or 4 CKD
  3. Plasma iPTH: ≥ 85 pg/mL and \< 500 pg/mL
  4. Serum Ca: ≥ 8.4 mg/dL and \< 9.8 mg/dL
  5. Serum P: ≥ 2.0 mg/dL and \< 5.0 mg/dL
  6. Serum 25-hydroxyvitamin D: ≥ 10 ng/mL and \< 30 ng/mL.
  7. Stable dose of Vitamin D therapy ≤ 1600 IU/day and receiving same dose for at least 2 months

Exclusion criteria

Exclusion Criteria:

  1. History of kidney transplant or parathyroidectomy
  2. Spot urine calcium:creatinine ratio > 0.2 (>200 mg/g Cr)
  3. Current serious illness, such as , , liver disease, cardiovascular event or hepatitis
  4. Currently on dialysis
  5. Use of pharmacological dose of ergocalciferol or cholecalciferol (≥ 50,000 IU mcg per month) during the study
Terms in these criteria, in plain language(2)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Cancer (malignancy)

Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.

Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.

HIV

The human immunodeficiency virus, which weakens the immune system.

Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.

05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Double (Participant, Investigator)
Enrollment
216 participants (actual)

Study arms

  • Active comparator
    CTAP101 30 μg capsules

    1 CTAP101 30 μg capsule daily at bedtime for 12 weeks, with a possible increase to 2 CTAP101 30 μg capsules daily at bedtime for study duration, if needed (26 weeks total). Subjects not requiring a dose increase after 12 weeks would receive 1 CTAP101 30 μg capsule and one sugar pill (to CTAP101 30 μg capsule) for weeks 13-26.

    Drug: CTAP101 30 μg capsules · Other: Sugar pill to CTAP101 30 μg capsules

  • Placebo comparator
    Sugar pill to CTAP101 30 μg

    1 sugar pill to CTAP101 30 μg capsule daily at bedtime for 12 weeks, with an increase to 2 sugar pills to CTAP101 30 μg capsules daily at bedtime for weeks 13-26.

    Other: Sugar pill to CTAP101 30 μg capsules

Interventions

  • CTAP101 30 μg capsule taken daily at bedtime

    Also known as: Calcifediol

  • OtherSugar pill to CTAP101 30 μg capsules

    Sugar pill capsule (placebo to CTAP101 30 μg capsule) taken daily at bedtime.

    Also known as: Placebo

06

What researchers measure

Primary outcomes

  1. Number of Participants in the Intent to Treat Population With Decrease in Plasma Intact Parathyroid Hormone (iPTH) of ≥30% From Pre-treatment Baseline

    Number of subjects in the intent to treat population attaining mean decrease in plasma intact Parathyroid Hormone (iPTH) of ≥30% from P\\pre-treatment baseline in the efficacy assessment phase (EAP) referred to as responders

    Time frame: Approximately 6 months

Secondary outcomes

  1. Number of Participants in the Per Protocol Population With Decrease in Plasma Intact Parathyroid Hormone (iPTH) of ≥30% From Pre-treatment Baseline

    Number of subjects in the per protocol population attaining a mean decrease in plasma intact parathyroid hormone (iPTH) of ≥30% from pre-treatment baseline in the efficacy assessment phase (EAP), referred to as responders

    Time frame: Approximately 6 months

  2. Subjects in the Intent to Treat Population With Normal Serum Total 25-hydroxyvitamin D

    Subjects in the intent to treat population with normal serum total 25-hydroxyvitamin D (\>/= 30 ng/dL)

    Time frame: Approximately 6 months

  3. Subjects in the Per Protocol Population With Normal Serum Total 25-hydroxyvitamin D

    Subjects in the per protocol population with normal serum total 25-hydroxyvitamin D (\>/= 30 ng/mL)

    Time frame: Approximately 6 months

07

Results

Posted Aug 17, 2016

Participant flow

This multicenter study was conducted at 45 investigational sites within the US. Subjects were enrolled and treated from 04 February 2013 through 13 August 2014.

Participant flow — Overall Study
MilestoneSugar Pill to CTAP101 30 μgCTAP101 30 μg Capsules
Started72144
Entered extension study53102
Completed58121
Not completed1423
Withdrew: Withdrawal by subject57
Withdrew: Lost to follow-up25
Withdrew: Adverse event47
Withdrew: Protocol violation10
Withdrew: Subject noncompliance23
Withdrew: Serum calcium >/= 11.0 mg/dl, repeat con01

Outcome measures

PrimaryNumber of Participants in the Intent to Treat Population With Decrease in Plasma Intact Parathyroid Hormone (iPTH) of ≥30% From Pre-treatment Baseline

Number of subjects in the intent to treat population attaining mean decrease in plasma intact Parathyroid Hormone (iPTH) of ≥30% from P\\pre-treatment baseline in the efficacy assessment phase (EAP) referred to as responders

Time frame:
Approximately 6 months
Reported as:
Number · participants
Number of Participants in the Intent to Treat Population With Decrease in Plasma Intact Parathyroid Hormone (iPTH) of ≥30% From Pre-treatment Baseline
participantsSugar Pill to CTAP101 30 μgCTAP101 30 μg Capsules
Number of Participants in the Intent to Treat Population With Decrease in Plasma Intact Parathyroid Hormone (iPTH) of ≥30% From Pre-treatment Baseline549
Statistical analysis
  • Sugar Pill to CTAP101 30 μg vs CTAP101 30 μg Capsules · Cochran-Mantel-Haenszel · p = <0.0001
SecondaryNumber of Participants in the Per Protocol Population With Decrease in Plasma Intact Parathyroid Hormone (iPTH) of ≥30% From Pre-treatment Baseline

Number of subjects in the per protocol population attaining a mean decrease in plasma intact parathyroid hormone (iPTH) of ≥30% from pre-treatment baseline in the efficacy assessment phase (EAP), referred to as responders

Time frame:
Approximately 6 months
Reported as:
Number · participants
Number of Participants in the Per Protocol Population With Decrease in Plasma Intact Parathyroid Hormone (iPTH) of ≥30% From Pre-treatment Baseline
participantsSugar Pill to CTAP101 30 μgCTAP101 30 μg Capsules
Number of Participants in the Per Protocol Population With Decrease in Plasma Intact Parathyroid Hormone (iPTH) of ≥30% From Pre-treatment Baseline547
Statistical analysis
  • Sugar Pill to CTAP101 30 μg · Cochran-Mantel-Haenszel · p = <0.0001
SecondarySubjects in the Intent to Treat Population With Normal Serum Total 25-hydroxyvitamin D

Subjects in the intent to treat population with normal serum total 25-hydroxyvitamin D (\>/= 30 ng/dL)

Time frame:
Approximately 6 months
Reported as:
Number · participants
Subjects in the Intent to Treat Population With Normal Serum Total 25-hydroxyvitamin D
participantsSugar Pill to CTAP101 30 μgCTAP101 30 μg Capsules
Subjects in the Intent to Treat Population With Normal Serum Total 25-hydroxyvitamin D5120
Statistical analysis
  • Sugar Pill to CTAP101 30 μg vs CTAP101 30 μg Capsules · Cochran-Mantel-Haenszel · p = <0.0001
SecondarySubjects in the Per Protocol Population With Normal Serum Total 25-hydroxyvitamin D

Subjects in the per protocol population with normal serum total 25-hydroxyvitamin D (\>/= 30 ng/mL)

Time frame:
Approximately 6 months
Reported as:
Number · participants
Subjects in the Per Protocol Population With Normal Serum Total 25-hydroxyvitamin D
participantsSugar Pill to CTAP101 30 μgCTAP101 30 μg Capsules
Subjects in the Per Protocol Population With Normal Serum Total 25-hydroxyvitamin D5116
Statistical analysis
  • Sugar Pill to CTAP101 30 μg vs CTAP101 30 μg Capsules · Cochran-Mantel-Haenszel · p = <0.0001

Adverse events

Collected over 6 months from randomization to end of therapy. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Sugar Pill to CTAP101 30 μg—11/72 (15.3%)18/72 (25%)
CTAP101 30 μg Capsules—22/144 (15.3%)32/144 (22.2%)
Most frequent serious events
Showing 10 of 41
Most frequent serious events
EventSugar Pill to CTAP101 30 μgCTAP101 30 μg Capsules
SepsisInfections and infestations2/720/144
DehydrationMetabolism and nutrition disorders2/721/144
Cardiac failure congestiveCardiac disorders0/723/144
PneumoniaInfections and infestations1/723/144
Blood creatinine increasedInvestigations0/723/144
AnaemiaBlood and lymphatic system disorders0/722/144
Acute myocardial infarctionCardiac disorders1/721/144
Cardiac failureCardiac disorders1/721/144
AstheniaGeneral disorders1/720/144
Chest painGeneral disorders1/721/144
Most frequent other events
Most frequent other events
EventSugar Pill to CTAP101 30 μgCTAP101 30 μg Capsules
HypertensionVascular disorders6/728/144
GoutMetabolism and nutrition disorders5/726/144
Oedema peripheralGeneral disorders3/729/144
ConstipationGastrointestinal disorders4/725/144
NasopharyngitisInfections and infestations1/728/144
Metabolic acidosisMetabolism and nutrition disorders4/721/144
Pain in extremityMusculoskeletal and connective tissue disorders4/723/144

Baseline characteristics

Age, Continuous
Age, Continuous(years)Sugar Pill to CTAP101 30 μgCTAP101 30 μg CapsulesTotal
Mean65.3 ± 10.0666.8 ± 10.9066.3 ± 10.63
Sex: Female, Male
Sex: Female, Male(Participants)Sugar Pill to CTAP101 30 μgCTAP101 30 μg CapsulesTotal
Female3971110
Male3373106
Region of Enrollment
Region of Enrollment(participants)Sugar Pill to CTAP101 30 μgCTAP101 30 μg CapsulesTotal
United States72144216
08

Study locations

1 site
  • OPKO Renal
    Bannockburn, Illinois 60015, United States
09

Timeline

Tracking since Sep 25, 2026
Not marked actual or estimatedDates not marked actual or estimated

The record gives a start of Nov 2012, a primary completion of Aug 2014 and a completion of Sep 2014. It does not say whether these dates are actual or estimated.

Results were first posted in Aug 2016, 2 years after primary completion. A results delay was requested in Oct 2015.

How these figures are worked out, and which studies are left out of them.

Registered
Oct 6, 2012
First posted
Oct 11, 2012
Start
Nov 2012
Primary completionActual
Aug 2014
CompletionActual
Sep 2014
Results delay requested
Oct 22, 2015
Results first posted
Aug 17, 2016
Last update
Jan 13, 2025
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Jan 13, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT01704079
Responsible party
Sponsor
First posted
Oct 11, 2012
Start date
Nov 2012
Primary completion
Aug 2014
Completion
Sep 2014
Results posted
Aug 17, 2016
Last update
Jan 13, 2025

Study contacts

Joel Melnick, MD
study director · OPKO Renal

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

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Studies of the drug tested here: Modified-release Calcifediol Capsule

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