A Repeated Study of KRN23 in Adults With X-Linked Hypophosphatemia

Kyowa Kirin

Status and phase: Completed; Phase 2; Phase 1

Conditions: X-linked Hypophosphatemia

Treatments: Drug: KRN23

Study type: Interventional

Funder types: Industry

Identifiers

NCT01340482

KRN23-INT-001

Details and patient eligibility

About

The primary purpose of this study is to assess the safety and efficacy of repeated subcutaneous (SC) injections of KRN23 in adult subjects with X-Linked Hypophosphatemia (XLH). A Bone Substudy will evaluate the effects of single-blind KRN23 versus Placebo on bone mineral density and bone quality.

Enrollment: 29 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Main Inclusion Criteria:

1. 18 years or older
2. Clinical diagnosis of XLH
3. TmP/GFR < 2.0 mg/dL
4. GFR ≥60 mL/min
5. Corrected Ca <10.8 mg/dL
6. A willingness to utilize adequate contraception and not become pregnant[or to have their partner(s) become pregnant] during the study
7. Additional inclusion criteria apply

Main Exclusion Criteria:

1. Have any sign of active infectious disease or have had an infection requiring treatment with antibiotics within 3-weeks of Screening
2. History of known immunodeficiency
3. Pregnant or lactating females subjects or female planning to be become pregnant during the study
4. Receipt of a live (attenuated) vaccine (except for influenza vaccines) within 3-months of Screening, and/or during the study
5. Use of pharmacologic vitamin D metabolite or its analog, phosphate, calcimimetics, and ingestion of aluminum hydroxide antacids within 10-days prior to Screening and/or dosing
6. Use of any supplement containing phosphate, calcium and/or vitamin D within 10-days prior to Screening and/or dosing
7. Condition which could present a concern for either the subject's safety or difficulty with data interpretation
8. Additional exclusion criteria apply

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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29 participants in 1 patient group

KRN23

Experimental group

Description:

Escalating doses of KRN23 (0.05, 0.10, 0.30 and 0.60 mg/kg) will be administered SC every 28 days (up to 4 doses)

Treatment:

Drug: KRN23