CompletedPhase 2

Long-Term Extension Study of KRN23 in Adult Subjects With X-Linked Hypophosphatemia (XLH)

United States20 participantsStarted 2015-01-30

Plain-language summary

The primary objectives of this study are to: * Assess the long-term safety of KRN23 subcutaneous (SC) administration in adult subjects with XLH * Assess the proportion of subjects achieving serum phosphorus levels in the normal range (2.5-4.5 mg/dL) with long-term administration of KRN23 * Assess long-term pharmacodynamics (PD) of KRN23 as measured by changes in the following: serum intact parathyroid hormone (iPTH); serum and urinary phosphorus; ratio of renal tubular maximum phosphate reabsorption rate to glomerular filtration rate (TmP/GFR) and tubular reabsorption of phosphate (TRP); serum 1,25-dihydroxy vitamin D (1,25\[OH\]2D); serum fibroblast growth factor 23 (FGF23); bone biomarkers: serum alkaline phosphatase (ALP), bone-specific ALP (BALP), carboxy terminal crosslinked telopeptide of type I collagen (CTx), and procollagen type 1 N-terminal propeptide (P1NP) * Assess long-term immunogenicity of KRN23 as measured by presence of anti-KRN23 antibody (ADA)

Who can participate

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Have participated in Kyowa Hakko Kirin Pharma, Inc.'s KRN23-INT-001 (NCT01340482) or KRN23-INT-002 (NCT01571596) studies (received at least 2 doses of KRN23)
✓. Estimated glomerular filtration rate (eGFR) ≥ 60 mL/min or eGFR of 45 to \< 60 mL/min at Screening with confirmation that the renal insufficiency was not due to nephrocalcinosis.
✓. Sexually active subjects must be willing to use an acceptable method of contraception (e.g., double barrier method) while participating in the study and for 30 days after receiving the last dose of KRN23.

Exclusion criteria

✕. Subject experienced a safety-related event in the KRN23-INT-001 or KRN23-INT-002 study that, in the opinion of the investigator and sponsor, precludes resuming KRN23 treatment.
✕. Presence of nephrocalcinosis on renal ultrasound that, in the opinion of the investigator and sponsor, precludes resuming KRN23 treatment.
✕. Pregnant or breastfeeding at Screening or planning to become pregnant (self or partner) at any time during the study.
✕. Participation in an investigational drug or device trial within 30 days of enrollment (other than KRN23-INT-001 or KRN23-INT-002).
✕. Use of a pharmacologic vitamin D metabolite or analog (e.g., calcitriol, doxercalciferol, and paricalcitol), phosphate, or aluminum hydroxide antacids (e.g., Maalox® and Mylanta®) within 21 days prior to Screening or during the study.

What they're measuring

Number of Participants With Adverse Events (AEs), Treatment Emergent AEs (TEAEs), Serious AEs (SAEs), and AEs Leading to Discontinuation or Death

Timeframe: Screening through the end of study plus 4-8 weeks. The mean duration of burosumab exposure was 165.6 weeks (range: 68-184 weeks).

Number of Participants With Clinically Significant Changes From Baseline in Vital Signs

Timeframe: Through Week 184

Number of Participants With Clinically Significant Changes From Baseline in Laboratory Values, by Category

Timeframe: Through Week 184

Number of Participants With Clinically Significant Changes From Baseline in Physical Exams, by Category

Timeframe: Through Week 184

Number of Participants With Clinically Significant Changes From Baseline in Echocardiogram (ECHO) Tests

Timeframe: Through Week 184

Number of Participants With Clinically Significant Changes From Baseline in ECGs

Timeframe: Through Week 184

Number of Participants With Clinically Significant Changes From Baseline in Renal Ultrasound, by Category

Timeframe: Through Week 184

Trial details

NCT IDNCT02312687

SponsorKyowa Kirin, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2018-11-30

Results submitted2019-11-26

View on ClinicalTrials.gov More X-Linked Hypophosphatemia trials

Plain-language summary

Who can participate

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Have participated in Kyowa Hakko Kirin Pharma, Inc.'s KRN23-INT-001 (NCT01340482) or KRN23-INT-002 (NCT01571596) studies (received at least 2 doses of KRN23)
✓. Estimated glomerular filtration rate (eGFR) ≥ 60 mL/min or eGFR of 45 to \< 60 mL/min at Screening with confirmation that the renal insufficiency was not due to nephrocalcinosis.
✓. Sexually active subjects must be willing to use an acceptable method of contraception (e.g., double barrier method) while participating in the study and for 30 days after receiving the last dose of KRN23.

Exclusion criteria

✕. Subject experienced a safety-related event in the KRN23-INT-001 or KRN23-INT-002 study that, in the opinion of the investigator and sponsor, precludes resuming KRN23 treatment.
✕. Presence of nephrocalcinosis on renal ultrasound that, in the opinion of the investigator and sponsor, precludes resuming KRN23 treatment.
✕. Pregnant or breastfeeding at Screening or planning to become pregnant (self or partner) at any time during the study.
✕. Participation in an investigational drug or device trial within 30 days of enrollment (other than KRN23-INT-001 or KRN23-INT-002).
✕. Use of a pharmacologic vitamin D metabolite or analog (e.g., calcitriol, doxercalciferol, and paricalcitol), phosphate, or aluminum hydroxide antacids (e.g., Maalox® and Mylanta®) within 21 days prior to Screening or during the study.

What they're measuring

Number of Participants With Adverse Events (AEs), Treatment Emergent AEs (TEAEs), Serious AEs (SAEs), and AEs Leading to Discontinuation or Death

Timeframe: Screening through the end of study plus 4-8 weeks. The mean duration of burosumab exposure was 165.6 weeks (range: 68-184 weeks).

Number of Participants With Clinically Significant Changes From Baseline in Vital Signs

Timeframe: Through Week 184

Number of Participants With Clinically Significant Changes From Baseline in Laboratory Values, by Category

Timeframe: Through Week 184

Number of Participants With Clinically Significant Changes From Baseline in Physical Exams, by Category

Timeframe: Through Week 184

Number of Participants With Clinically Significant Changes From Baseline in Echocardiogram (ECHO) Tests

Timeframe: Through Week 184

Number of Participants With Clinically Significant Changes From Baseline in ECGs

Timeframe: Through Week 184

Number of Participants With Clinically Significant Changes From Baseline in Renal Ultrasound, by Category

Timeframe: Through Week 184