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X-linked Hypophosphatemia clinical trials

View clinical trials related to X-linked Hypophosphatemia.

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NCT ID: NCT06067932 Completed - Clinical trials for X-linked Hypophosphatemia

Foot Disorders in X-linked Hypophosphatemia

Start date: July 1, 2020
Phase:
Study type: Observational [Patient Registry]

This study aimed to characterize foot pathologies using X-rays and clinical examination and assess related outcome scores in adolescents and adults with X-linked Hypophophatemia

NCT ID: NCT05181839 Completed - Clinical trials for X-Linked Hypophosphatemia

A Study to Describe the Lived Experience of XLH for Adolescents at End of Skeletal Growth

Start date: November 24, 2021
Phase:
Study type: Observational [Patient Registry]

An observational, prospective, mixed-methods study involving the integration of quantitative and qualitative data exploring the lived experience of burosumab-treated adolescents with XLH at the end of skeletal growth.

NCT ID: NCT04695860 Completed - Clinical trials for X-linked Hypophosphatemia

Anti-FGF23 (Burosumab) in Adult Patients With XLH

BurGER
Start date: January 7, 2021
Phase: Phase 3
Study type: Interventional

X-linked hypophosphatemia (XLH) rare genetic disorder due by inactivating mutations in the PHEX gene leading to increased levels in FGF-23. Elevated FGF-23 reduces renal phosphate reabsorbtion and and limits 1-alpha hydroxylase driven Vitamin D activation, eventually leading to phosphate wasting, defective bone mineralization and additional health issues. Burosumab is a recombinant fully human IgG1 monoclonal antibody developed to treat XLH by binding FGF23, thereby restoring normal phosphate homeostasis. BUR03 is a Phase 3b open-label, single-arm, single-center study to confirm the efficacy and safety of Burosumab treatment in adult (age ≥18 years) XLH patients without upper age limit and irrespective of baseline pain level and to further evaluate the efficacy in this cohort and the assocaited effect of treatment on physical functioning, mobility and activity. The study aims at enrolling and treating 34 subjects with a confirmed diagnosis of XLH with q4w s.c. injection of Burosumab 1mg/kg body weight over 48 weeks. Primary objective is to attiain normal serum phosphorus levels, secondary objectives include key parameters of physical function and activity, mobility and mineral homeostasis.

NCT ID: NCT04419363 Recruiting - Rare Diseases Clinical Trials

Burosumab in Children and Adolescents With X-linked Hypophosphatemia

Start date: March 18, 2018
Phase: Phase 4
Study type: Interventional

In this prospective longitudinal cohort study we studied the efficacy and safety of burosumab in real-clinical practice for <13- and >13-years old children affected with X-linked hypophosphatemia. 57 children with XLH were switched from conventional treatment to burosumab. After 12 months we assessed the efficacy and safety of treatment with burosumab on the whole cohort and separately on the cohort of >13-years old adolescents.

NCT ID: NCT04273490 Completed - Clinical trials for X-linked Hypophosphatemia

Characterising Pain, QoL, Body Composition, Arterial Stiffness, Muscles and Bones in Adult Persons With XLH and Healthy Controls

Start date: February 18, 2020
Phase:
Study type: Observational

Hereditary hypophosphatemia (XLH) is a rare, inherited disease. Loss-of-function mutation in the phosphate regulating gene with homologies to endopeptidases on the X-chromosome (PHEX) results in excess fibroblast growth factor 23 (FGF23) production and manifests as rickets in children and osteomalacia in adults. This study aims to characterize and measure pain, quality of life, muscle function, body composition, arterial stiffness, bone mineral density, geometry and microarchitecture in patients with XLH compared to age and gender-matched controls.

NCT ID: NCT04146935 Completed - Clinical trials for X-linked Hypophosphatemia

Examining the Effect of Burosumab on Muscle Function

Start date: November 13, 2019
Phase: Phase 4
Study type: Interventional

Patients with X-linked hypophosphatemia (XLH) often report symptoms of fatigue and weakness particularly after exertion, in addition to their skeletal complaints. In previous trials using KRN23 (same drug as burosumab/Crysvita®), patients report these symptoms improve. The investigators wish to test this hypothesis directly by measuring muscle energy when patients begin treatment with Crysvita® for the first time.

NCT ID: NCT03920072 Completed - Clinical trials for X-linked Hypophosphatemia

Study of the Anti-FGF23 Antibody, Burosumab, in Adults With XLH

Start date: March 7, 2019
Phase: Phase 3
Study type: Interventional

This is phase 3b open-label, international, multicenter study to continue to monitor the long-term safety and efficacy of burosumab in adult patients with XLH that participated in previous clinical trials with burosumab (UX023-CL303 / UX023-CL304).

NCT ID: NCT03879915 Recruiting - Clinical trials for X-linked Hypophosphatemia

Dental Implants in Patients With X-linked Hypophosphatemia

IMPLANTS-XLH
Start date: September 27, 2019
Phase:
Study type: Observational

X-linked hypophosphatemia (XLH) is a rare genetic skeletal disease where increased phosphate wasting in the kidney leads to hypophosphatemia and prevents normal mineralization of bone and dentin, with osteomalacia as a principal manifestation. In previous works, the investigators have shown that adults with XLH present with more frequent and severe periodontitis than in the general population, and that vitamin D and phosphate supplementation improves their periodontal health, as it does for the osteomalacia. Their medical records also reveal that early implant failure is dramatically increased in these patients, when no supplementation is implemented, and standard surgical protocols followed. In contrast, the investigator's preliminary data showed that successful osseointegration was achieved with supplementation prior and after implant placement and extended healing time. Here, the investigators propose to assess the current recommendations for implant therapy in XLH patients, with 24 implants placed. The current recommendations consist of: 1) supplementation with vitamin D and phosphate for 3 months prior to implant placement and 6 months after; 2) implant healing time extended to 6 months. If osseointegration is achieved, prosthesis will be fabricated. Radiographic and clinical examination at 6, 12, 18 and 24 months after placement of the definitive restoration will evaluate the implant osseointegration, crestal bone level and peri-implant tissues health.

NCT ID: NCT03820518 Recruiting - Clinical trials for X-linked Hypophosphatemia

Using Different Doses of Active Vitamin D Combined With Neutral Phosphate in Children With X-linked Hypophosphatemia

Start date: January 1, 2017
Phase: Phase 4
Study type: Interventional

X-linked hypophosphatemia (XLH) is the most common form of heritable rickets. Current treatments include active vitamin D metabolites (e.g. calcitriol) and phosphate salts. There is no consistent weight-based dosing of calcitriol and phosphate now. The primary objective of this study is to establish the efficacy of different dose of calcitriol combined with neutral phosphate in children with XLH.

NCT ID: NCT03775187 Available - Clinical trials for X-linked Hypophosphatemia

Expanded Access to Burosumab

Start date: n/a
Phase:
Study type: Expanded Access

Individual patient expanded access requests may be considered for patients who have no other treatment options