Rickets

Researchers are studying vitamin D levels in hospitalized individuals with neurological disorders that cause motor disabilities. The goal is to gather important data to help create guidelines for vitamin D use in these populations, including people with stroke, spinal cord injury, and traumatic brain injury. The study also aims to evaluate how vitamin D supplements affect bone density, body composition, strength, walking speed, physical performance, and pain. The study is a two-year randomized, controlled trial enrolling 120 participants, including 30 controls. Participants with low vitamin D levels will receive vitamin D supplements either as 50,000 I.U. weekly for 8 weeks or 25,000 I.U. for 4 weeks followed by 10,000 I.U. drops weekly for another 4 weeks. Controls have vitamin D levels above 20 ng/ml and do not receive supplementation. Measurements include serum vitamin D and related biochemical markers, assessed at the start and end of the study. Participants will undergo various assessments such as bone density scans using DXA, strength tests with a dynamometer, walking speed tests, physical performance evaluations with the SPPB test, and pain scoring. Demographic data and lifestyle factors like exercise and smoking will also be recorded. Safety and tolerance of vitamin D supplementation will be monitored. The primary outcome is the number of participants with neurological disease and low vitamin D levels, with secondary outcomes including improvements in structural and functional parameters due to supplementation.

Researchers are evaluating the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of KK8123 in adults with X-linked hypophosphatemia (XLH) through a Phase 1/2, multicenter, open-label, dose-escalation study. This first-in-human study aims to better understand how KK8123 behaves in the body and its effects on patients with this condition. The study is sponsored by Kyowa Kirin Co., Ltd. and includes an optional safety extension period. The study includes a Screening Period of up to 28 days, followed by Part 1, which is a Dose Escalation Period consisting of a planned Treatment Period and an Observation Period lasting 32 to 44 weeks. Part 2 is an optional Extension Period for additional safety evaluation. Participants receive subcutaneous doses of KK8123 at different levels, including low dose (single dose), mild dose (multiple doses), and high dose (multiple doses), with dosing confirmed for later cohorts. The study groups include several cohorts receiving escalating doses and an extension group. Participants will undergo multiple assessments during the study, including laboratory tests for hematology and clinical chemistry, measurement of serum phosphorus levels, echocardiograms, renal ultrasounds, and monitoring of vital signs such as heart rate and blood pressure. Researchers will track treatment-emergent adverse events (TEAEs) and measure KK8123 drug concentrations over time. Follow-up periods last up to 44 weeks in Part 1 and up to 52 weeks in Part 2. Participants are expected to adhere to study visit schedules and complete all assessments throughout their involvement.

Researchers are following participants with pediatric-onset hypophosphatasia (HPP), a rare bone disease that begins in childhood, to understand how the treatment asfotase alfa works over time. This observational sub-study will track patients at various sites in the United States and possibly other countries for at least five years, focusing on how the body responds to the treatment and any immune-related issues that may affect its effectiveness. All participants will receive asfotase alfa given under standard care, typically as subcutaneous injections. Younger children under 2 years old are recommended to have clinic visits about every three months until they turn two, then about every six months afterward. The study monitors biochemical, clinical, imaging (if needed), and quality of life outcomes related to HPP to understand the long-term effects of treatment. Participants will be regularly evaluated over a minimum five-year period, including assessments by their treating physicians to detect any loss of treatment effectiveness or serious immune-related side effects. Data collection will include clinical exams, laboratory tests, functional assessments, and quality of life questionnaires. The study aims to gather detailed information about treatment outcomes and safety in real-world settings over time.

Researchers are evaluating methods to accurately restore the joint line position during Total Knee Arthroplasty (TKA) in patients with severe varus deformity due to Type M gonarthrosis, a form of advanced knee osteoarthritis. This is important because proper joint line restoration affects knee function, pain, implant wear, and overall patient satisfaction. The study compares patients with two types of gonarthrosis deformities to find the best technique for determining the joint line using preoperative and intraoperative measurements, focusing particularly on the adductor ratio method. The study involves patients undergoing cemented bicondylar posterior cruciate ligament (PCL) substituting total knee replacement. Two patient groups include those with intraarticular varus deformity and those with metaphyseal varus deformity, both having severe osteoarthritis. Researchers will observe and measure anatomical landmarks during surgery and on radiographs before and after the procedure. This includes measuring the distance between specific bone landmarks and the joint line to assess which method most reliably determines the joint line position. Participants will undergo preoperative radiological assessments and intraoperative caliper measurements followed by postoperative X-rays over two years. Functional outcomes will be evaluated using the Hospital for Special Surgery (HSS) knee score before surgery and at one year after. The primary measurement is the accuracy of joint line determination by the adductor ratio compared to other techniques. This observational study aims to improve surgical planning and outcomes in complex knee replacement cases with severe deformity.

Researchers are studying men with metastatic castration-sensitive prostate cancer to see if taking oral acetate can increase the amount of a gut bacterium called Akkermansia muciniphila. The trial aims to confirm this increase compared to standard care and to assess how well patients tolerate oral acetate. The study also explores whether higher levels of this bacterium relate to better metabolic health and bone strength. This is an early phase 1 clinical trial sponsored by Western University. Participants are randomly assigned to one of two groups. One group takes an apple cider vinegar caplet daily, containing 143 mg with 36% acetic acid, for three months. The other group receives standard care without supplementation. The study includes follow-up periods lasting up to six months to monitor changes and side effects. Throughout the study, stool samples will be collected at multiple time points to measure Akkermansia muciniphila levels. Researchers will also evaluate various metabolic markers such as blood sugar, cholesterol, and insulin resistance, plus bone health indicators. Side effects and tolerability will be checked after three months. Participants are monitored over six months to assess both primary and secondary outcomes related to metabolism and bone health.

Researchers are investigating the effects of sweet almonds on healing femur and tibia shaft fractures compared to routine vitamin D3 supplements. This study addresses the need for an affordable and safe alternative to bone-strengthening drugs, which often cause side effects and are costly. The trial focuses on patients with vitamin D insufficiency or deficiency, assessing the potential of almonds to improve bone healing and vitamin D levels in the blood. The trial is a single-center, open-label, crossover randomized controlled study involving 136 participants aged 18 to 55 with trauma-related femur and tibia shaft fractures. Participants are divided into two groups based on their vitamin D levels and receive treatments including daily whole almonds (5 grams/day) and weekly oral vitamin D3 (200,000 units) in various sequences with washout periods. Group 1 includes vitamin D insufficient patients receiving almonds and vitamin D3 in a crossover design, while Group 2 includes vitamin D deficient patients receiving vitamin D3 alone, almonds alone, or a combination followed by crossover treatments. Participants undergo follow-up visits at 4 and 13 weeks post-treatment, where fracture healing is monitored through routine X-rays and Radiological Union Score for Tibial fractures (RUST). Safety is closely assessed, with adverse events recorded and managed according to international guidelines. The study also tracks treatment adherence with daily diaries and compares costs and changes in blood vitamin D levels. Patients may withdraw at any time without losing their rights, and the study will be completed within 24 months including a close-out period.

Researchers are evaluating the link between vitamin D deficiency, postural stability, and neuromuscular control in adults aged 20 to 40 years. This observational study compares individuals with vitamin D deficiency to healthy controls to better understand how vitamin D status affects neuromuscular responses and balance. Participants are divided into two groups based on routine clinical test results for vitamin D levels. Those with vitamin D deficiency and those with normal vitamin D status both undergo a single session of non-invasive assessments. These include surface electromyography to measure muscle activity and tests of postural stability during various standing tasks, including single-leg and double-leg stance with and without vibration stimulation. During the single assessment session, researchers will record muscle activation and postural stability parameters under different conditions. They will analyze neuromuscular reflexes and how loading and vibration influence balance. The study aims to provide insights for future rehabilitation and balance training strategies. Participation involves no invasive procedures, medications, or additional blood tests, and results from prior routine labs are used to determine vitamin D status.

This research aims to understand the relationship between vitamin D levels, bone metabolism, and obesity in children aged 3 to 15 years who have experienced low-energy trauma. The study compares children with confirmed fractures to those with similar trauma but no fractures to explore factors that may contribute to bone fragility and fracture risk. Participants are divided into two groups: children with radiologically confirmed low-energy fractures and children with low-energy trauma but no fractures. Blood samples are collected within seven days of trauma to measure markers such as 25-hydroxyvitamin D, parathyroid hormone, alkaline phosphatase, calcium, and phosphate. Dietary calcium intake and body measurements are also recorded to evaluate nutritional and metabolic status. Children will undergo baseline assessments including body mass index measurement and a dietary questionnaire. Researchers analyze blood markers and calculate a composite bone risk score to identify factors linked to fracture risk. The study uses statistical methods to compare groups and assess the association between bone markers and fractures, aiming to improve understanding of pediatric bone health and inform prevention strategies.

Researchers are conducting a non-interventional, prospective, multi-center study to explore the occurrence of hypophosphatasia (HPP) among adult patients who visit rheumatology departments. The study aims to better understand the characteristics of HPP in this population and to develop a diagnostic algorithm to help identify the condition. This investigation involves collecting and analyzing data without altering patient care. Participants will undergo diagnostic tests, including a second alkaline phosphatase (ALP) measurement taken 1 to 4 weeks after the initial test. Patients are grouped based on whether they have persistent low ALP levels indicating HPP or transient low ALP levels as a control group without HPP. This approach helps researchers assess the prevalence and patterns of HPP in rheumatology patients. During the study, participants will provide informed consent and be monitored over a 36-month period to determine the prevalence of HPP. The study involves routine clinical assessments and laboratory tests, focusing on ALP levels. Data collected will assist in establishing an algorithm to improve diagnosis of HPP in adults. The study started in August 2024 and is expected to continue until September 2027.

Researchers are studying adults with hypovitaminosis D, a condition characterized by low vitamin D levels in the blood. This randomized clinical trial compares three different ways of taking vitamin D supplements to see how well they raise vitamin D levels and affect bone mineral density. The purpose is to find the best dosing method for improving vitamin D intake. Participants are assigned to one of three treatment groups: Group A receives 0.266 mg of calcifediol once a month, Group B takes 25,000 IU of cholecalciferol every 15 days, and Group C uses 4 drops of calcifediol daily. Vitamin D blood levels are measured after one month of treatment to guide dosing. The study also monitors bone mineral density over an 18-month period. During the trial, participants have their vitamin D levels checked at one and six months after starting treatment. Bone density is assessed using DEXA scans between recruitment and 18 months. The study involves randomization and single masking to monitor and compare the effects of these vitamin D supplementation methods. Participation lasts up to 18 months, including follow-up assessments.