Clinical Research Directory

A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping

A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping

Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping

The purpose of this Managed Access Program is to allow access to delpacibart zotadirsen (AOC 1044) for eligible patients diagnosed with DMD mutations amenable to exon 44 skipping. The patient's Administering Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.

Hyperthermia in Patients With Chronic Primary Pain - Effects on Thermoregulation, Somatosensory System and Movement Evoked Pain

This study, in a quasi-experimental matched two-group pre-post design, investigates the effect of serial water-filtered whole-body hyperthermia on circadian core body temperature, the somatosensory system (nociception) and the movement evoked pain in healthy and patients with chronic primary pain (e.g., fibromyalgia). The intervention lasts 3 weeks with two treatment sessions per week.

Expert@Work - Effectiveness of a Digital Expert Team on Return to Work

Long-term sick leave is a challenge in Norway, and the healthcare and labor- and welfare services are often not coordinated in helping the sick-listed employees return to work. In this study, the investigators want to evaluate the effectiveness of an intervention where an expert team provides advice for an individualized patient pathway based on information from a digital questionnaire and a video meeting with the sick-listed employee and the participant's general practitioner. The patient pathway should include both healthcare and labor- and welfare services measures. Sick-listed patients with musculoskeletal- and/or common mental disorders are invited to join the study. They will receive a digital questionnaire and be randomized to either treatment as usual or personalized advice from an expert team. The team will be put together based on the response from the individual's questionnaire. The investigators will evaluate the effect on return to work and the use of health care services 12 months after the intervention.

Translation, Cultural Adaptation, and Standardization of E-NMQ

The primary objective of this study is to translate the Nordic Musculoskeletal (E-NMQ) Questionnaire Modified Version into Urdu following international guidelines. Presently, there exists no validated tool to comprehensively measure symptoms such as pain, stiffness and weakness in Urdu-speaking patients in Pakistan. Specifically, the widely used E-NMQ, having these three vital categories, has yet to be translated and validated for Urdu-speaking individuals with musculoskeletal disease (MSD).

Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients

AOC 1001-CS2 (MARINA-OLE) (NCT05479981) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) (NCT05027269) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients

Longevity Metrics AI/ML Development Study

This study builds AI models that score diagnostic screening tests, and that predict screening results, clinical judgment, and life expectancy. Longevity Metrics collects a battery of clinical tests on each participant, in whole or in part, and follows every participant for life. The sit-to-rise test and the timed walk are scored by hand today, from a person's count. A model scores the same test from video instead. It also measures what no one can count by eye - speed, asymmetry, steadiness - so one capture yields both the original score and additional measurements, intended to enrich the model and strengthen what it predicts. Every test in a participant's record measures the same body, so the tests are correlated: a test that was performed carries information about one that was not. A model trained across the library learns those relationships and estimates a missing result from the results that are present. Each estimate is checked against records where that part was actually measured, and over decades against death and disease through linkage to the 100-Year Human Aging Study (NCT07563777). The hypothesis is that the full battery can eventually be predicted across modalities with high accuracy using a few short video clips, replacing most in-person screening. That would let preventive screening reach people and places a physical laboratory cannot. How far the input can be reduced is the question this study exists to answer. Every model is a physician-reviewed clinical decision aid until it is cleared by the FDA.

100-Year Human Aging Study

The 100-Year Human Aging Study is a prospective, pragmatic, observational trial enrolling participants across fixed clinical sites, mobile clinical sites, and an online participation pathway (current and future) to undergo comprehensive multi-system health screening and longitudinal follow-up until death. Participants are followed to determine whether measurements taken at enrollment and repeated across the lifespan - individually and in combination - predict all-cause mortality, cause-specific mortality, incident serious disease, and functional disability. The study is designed to generate the surrogate endpoint validation data that longevity medicine currently lacks.

Sarcopenia and Related Factors in Lipedema

Lipedema is a common disease of subcutaneous adipose tissue. The most common complaint of patients with swelling in the affected extremity is pain. In addition, patients with lipedema may experience conditions that can greatly affect the health and quality of life of the individual, such as loss of muscle strength and exercise capacity, and deterioration in activity levels of daily living. It is still unknown whether the decrease in muscle strength in patients with lipedema is part of this condition or whether decreased activity levels lead to decreased muscle strength. Sarcopenia is an important health problem characterized by age-related loss of muscle mass and muscle function. The relationship between muscle weakness and sarcopenia in patients with lipedema has not been investigated before. Early recognition of possible sarcopenia and functional limitations in these patients may be important to increase the ability of patients to participate in physical activity as part of their conservative management. There is no study in the literature investigating sarcopenia in patients with a diagnosis of lipedema. The aim of this study is to evaluate patients with a diagnosis of lipedema in terms of sarcopenia. In addition, the relationship between sarcopenia and age, body mass index, exercise frequency, lipedema type, and stage will be investigated in patients diagnosed with lipedema.

Video-based First-contact Physiotherapy in Primary Care

This study aims to evaluate the effects and implications of using video-based first-contact consultation in primary care physiotherapy, compared with traditional in-person consultation for musculoskeletal symptoms. The participants with musculoskeletal symptoms will receive either a video-based first contact physiotherapy consultation or an in-person first-contact physiotherapy consultation and will be followed for up to two years. The project addresses clinical effectiveness, healthcare utilisation, cost-effectiveness, and carbon footprint, according to the following specific aims. Objectives * To evaluate whether a video-based first-contact consultation with a physiotherapist is not clinically inferior to an in-person first-contact consultation, using the Patient-Specific Functional Scale (PSFS) as the primary outcome measure and multiple secondary outcomes assessed over time. * To analyse healthcare utilisation in primary and specialist care at 12 and 24 months following a video-based first-contact consultation, compared with an in-person first contact consultation. * To evaluate whether a video-based first-contact consultation with a physiotherapist is cost-effective from a healthcare perspective and in terms of environmental impact compared with an in-person first contact consultation.

Musculoskeletal Ultrasound in Patients With Hematologic Malignancies

This study evaluates the role and diagnostic value of musculoskeletal ultrasonography (MSUS) in adult patients with hematologic malignancies who present with musculoskeletal symptoms. Hematologic malignancies (such as leukemia, lymphoma, and multiple myeloma) frequently involve the musculoskeletal system through bone marrow infiltration, bone destruction, or soft-tissue lesions. Musculoskeletal ultrasound is a non-invasive, radiation-free, and high-resolution imaging technique that allows real-time evaluation of joints, muscles, tendons, bursae, and superficial bone changes. In this study, adult participants with confirmed blood cancers presenting with symptoms such as localized bone pain, joint swelling, muscle pain, or soft-tissue swelling will undergo a dedicated ultrasound examination of symptomatic areas. The study aims to characterize the patterns of musculoskeletal involvement (including cortical breaks, periosteal changes, soft-tissue masses, synovitis, and vascularity on Doppler imaging) and determine how these findings correlate with disease type, clinical presentation, and laboratory parameters.

Fecal Microbiota Transplantation in Axial Spondyloarthritis

Although biologic therapy have revolutionized the treatment of Spondyloarthrtitis (SpA), many patients do not experience complete relief of SpA related complaints. It has been established that patients with SpA have an altered composition of microorganisms (microbiota) in the gut compared to healthy controls, and that this correlates to disease activity and respons to therapy. The goal of this randomized double-blind study is to evaluate the efficacy of fecal microbiota transplantation (FMT) in patients with axial SpA with a suboptimal effect of biologic therapy. The main questions it aims to answer are: * Can FMT reduce disease activity in axial SpA? * Can FMT alleviate pain and reduce fatigue in axial SpA? * Is the composition of microorganisms restored to normal in patients with SpA after a treatment with FMT? Participants will receive a single treatment in the form of an enema with either donor FMT or placebo at baseline. The primary endpoint will be evaluated after 90 days, but efficacy and safety will be monitored from baseline until 365 days.

Impact of Exoskeleton Use on Perceived Effort and Pain During Resident Handling in Nursing Home Care Staff

The EXO 2 study assessed whether daily use of an exoskeleton - a wearable mechanical support device - could reduce physical effort and pain in nursing home care staff who regularly perform resident handling tasks such as bed baths and diaper changes. Care staff in nursing homes (EHPADs) are among the most affected workers by musculoskeletal disorders (MSDs), which are the leading cause of occupational illness and sick leave in the French healthcare sector. Handling of residents in lying positions (decubitus), such as turning, washing, or changing residents in bed, is one of the most physically demanding tasks and a major source of lower and upper back strain. The HAPO Version 4 exoskeleton is a wearable device worn around the torso that provides mechanical support to the upper body and back during forward-bending tasks. This study is part of a research program that progressively evaluated and improved the HAPO exoskeleton through four earlier phases with LNA Santé care staff. The main questions this study aimed to answer were: Does wearing the HAPO exoskeleton daily reduce the perceived effort (physical fatigue) felt in the upper body during resident handling? Does the exoskeleton reduce upper body pain (neck, shoulders, arms, back, hips) over time? How well do care staff integrate the exoskeleton into their daily work routine? Does exoskeleton use reduce workplace accidents and sick leave related to handling? What participants did: First 2 weeks (baseline): worked normally without the exoskeleton, completing a daily logbook to record pain and workload Following 11 weeks (intervention): wore the exoskeleton daily during all bed baths and resident changes in decubitus, continuing the daily logbook Completed weekly assessments of perceived effort and satisfaction with the device Were assessed at the end of the study (Day 91) with a final evaluation The study took place across 5 LNA Santé nursing homes in France and included 35 volunteer care staff members.

Telerehabilitation Intervention for Individuals With Shoulder Pain

Introduction: Shoulder pain is a condition of high prevalence in the general population. Studies indicate that physiotherapeutic treatment with exercise is effective in reducing pain and restoring function in patients with shoulder pain. Patients may have difficulty accessing the physiotherapy service due to the cost of treatment, transportation to the service, and long waiting lines. A possible solution is the use of a telerehabiliation intervention to increase access to physiotherapy for shoulder pain patients. Objective: To assess the effectiveness of telerehabilitation when compared to in-person physiotherapy on pain and disability in individuals with persistent rotator cuff related shoulder pain. Methods: This study is a controlled, randomized, blinded clinical trial. A total of 184 individuals with shoulder pain were randomly assigned to two groups. One group received a telerehabiliation intervention and the other group received the in-person intervention. The intervention consisted of strengthening exercises with emphasis on the lateral rotator and scapulothoracic muscles. The primary outcome was pain and disability (SPADI, Shoulder Pain and Disability Index), and the secondary outcomes were pain intensity (NPRS, Numerical Pain Rating Scale), function (DASH questionnaire, Disabilities of the Arm, Shoulder and Hand), self-efficacy (CPSS, Chronic Pain Self-Efficacy Scale), kinesiophobia (Cover Scale), patient expectation of treatment (7-point Likert Scale), and patient self-perceived improvement (Global Change Assessment Scale). All outcomes were measured before and after 12 weeks of treatment (2x/week). Normality of data was verified by Kolmogorov Smirnov's test. Differences between groups were assessed using the mixed linear models including the group × time interaction. The effect size was calculated for the variables between the groups. The significance level was set at 5%.

Shoulder Health After Rehabilitation and Performance Training

The goal of this study is to investigate the impact of a home-based shoulder strength training on the overall shoulder health in manual wheelchair users, and if the timepoint of such a training makes a difference. The study is focusing on persons with a spinal cord injury in the thoracic or lumbar region of the spine, that have only recently been injured and will soon be discharged from primary rehabilitation. The shoulder training will take place either 3 or 12 months after discharge from primary rehabilitation and will be carried out twice a week for 12 weeks. There are six measurements occurring every three months, which leads to an overall duration of 15 months. The measurements consist of: * Questionnaires about independence in daily life, participation, quality of life and physical activity * Assessment of shoulder strength, range of motion and function * Measurement of the daily wheelchair use during one week via sensors that are fixed to the wheelchair and wrist * one further questionnaire at the end of the measurement week about the occurrence of shoulder pain Additionally, on four of the six measurement timepoints, a magnet resonance image (MRI) of the shoulder will be taken to assess the shoulder status (pathology, muscle volume and quality). Though all these measurements the researchers can additionally assess the load of daily life on the shoulders, and how well this matches the preparation during the primary rehabilitation.

Turkish Adaptation Study of the Generic Functional Index

The Generic Functional Index is a scale developed to assess the general functional status of individuals. This study aims to evaluate the validity and reliability of the Turkish version of the Generic Functional Index. The adaptation process was designed to follow a standardized translation protocol, including forward translation, expert committee review, back-translation, pilot testing, and psychometric analyses. Data will be collected from individuals with musculoskeletal problems. Psychometric testing will include test-retest reliability, internal consistency, and construct validity analyses. The main hypotheses of this study are as follows: 1. The Turkish version of the Generic Functional Index will demonstrate high test-retest reliability when administered to individuals with musculoskeletal disorders. 2. The scores of the Turkish version of the Generic Functional Index will show a significant positive correlation with the scores of the Functional Independence Measure in individuals with musculoskeletal disorders.

Randomized Comparison of PARtial Wrist Fusion With or Without Triquetral Excision (PARTE)

This randomized clinical trial (RCT) aims to compare clinical and radiographic outcomes of different partial wrist fusion techniques in participants with post-traumatic wrist arthritis. Participants with stage II or III scapholunate advanced collapse (SLAC) or scaphoid nonunion advanced collapse (SNAC) who meet the eligibility criteria will be randomly assigned to one of two parallel groups: Group A (partial wrist arthrodesis without triquetral excision i.e. four-corner arthrodesis), or Group B (partial wrist arthrodesis with triquetral excision i.e. three-corner or capitolunate arthrodesis with triquetral excision). The results of this study will provide evidence to guide surgeons in determining the ideal wrist fusion technique in the management of patients with post-traumatic wrist arthritis requiring surgery.

Mode of Exercise and Bone Biomarkers in Older Veterans

Adults are often encouraged to exercise to maintain or improve bone health. However, there is evidence that exercise does not always lead to increases in bone mass, and exercise could lead to bone loss under certain conditions. Endurance exercise can increase bone resorption following an exercise bout, which may explain why bone does not always favorably adapt to exercise, but it is unclear if this also happens with resistance exercise. Further, it is not known how exercise training influences blood markers of bone resorption for either endurance or resistance exercise. The purpose of this study is to determine 1) if resistance exercise causes a similar increase in bone resorption as endurance exercise; and 2) if exercise training influences the increase in bone resorption following exercise for both endurance and resistance exercise.

New Clinical Rehabilitation Approach for the Management of Falls Risks

Accidental falls in older adults are one of the world's major pubic health problem, because of their strong association with injuries and mortality rates. In Quebec, falls are responsible for a high rate of hospitalization (more than 1800 emergency department visits every day) and deaths (more than 10,000 in recent years). Preventing falls is therefore a key mission for health professionals. This research program aims to develop a new clinical approach to the rehabilitation management of the older with a neuro-musculoskeletal disorder and a risk of falling. This program is part of a new partnership project between UQAC and specialized geriatric services at the CIUSSS Saguenay-Lac-St-Jean (La Baie site). These geriatric services admit more than 400 new patients per year, representing a large pool of participants for the new program's development. Specifically, this program has 4 phases: 1) Create a clinical profile of patients in rehabilitation care from specialized geriatric services (ex: reasons for consultation, neuro-musculoskeletal disorders, rates and causes of falls, etc.); 2) to diagnose functional deficits of these patients on different dimensions of functional and physical evaluations, using standardized tests and high-tech instruments (ex: platform of force); 3) determine the effectiveness of a new exercise intervention program (OTAGO) for falls prevention; and 4) Measure client and professional team satisfaction as well as long-term impact of this new approach used to prevent falls. The most significant impact of this new program will be to reduce public health expenditure for care of older adults with balance disorder and risk for falls; and therefore, be implanted in other CIUSSS institutions from Quebec.

Strength Training Effects on Musculoskeletal Health

The objective here is to assess the efficacy of variable resistance muscle training on bone, sleep parameters and quality of life in healthy populations of men and women age 30 or older.

Development of Multidimensional Screening Tool for WMSDs

The purpose of this study is to develop a multidimensional screening tool for Work-Related Musculoskeletal Disorders (WMSDs) and to evaluate its psychometric properties. The study involves a cross-sectional survey of approximately 250 workers in the bio-pharmaceutical industry to assess the tool's structural validity, internal consistency, construct validity, and measurement invariance following COSMIN guidelines.

Association Between Office Ergonomics, Work Organization, and Musculoskeletal Complaints in Medical Secretaries

Work-related musculoskeletal complaints are among the most common occupational health issues affecting healthcare staff, especially medical secretaries who spend long hours in sedentary positions and repetitive office tasks. This observational, cross-sectional study aims to investigate the relationship between office ergonomics, work organization, and musculoskeletal system complaints among medical secretaries. Participants will complete a structured questionnaire that includes demographic and occupational data, ergonomic and work organization characteristics (such as desk and chair setup, posture, computer use, and daily working hours), and self-reported measures of pain and well-being. Postural alignment will be evaluated using the New York Posture Rating Scale (NYPRS), while pain intensity will be assessed with the Visual Analog Scale (VAS). In addition, participants' physical activity level, disability score and stress level will be recorded to examine their potential influence on musculoskeletal symptoms. The study aims to determine whether poor ergonomic conditions, inadequate posture, low physical activity, and high stress are associated with increased musculoskeletal discomfort, particularly in the neck, shoulders, and lower back regions. This study is observational and does not include any intervention or experimental treatment. All participation will be voluntary, and data will be collected anonymously. The findings are expected to contribute to preventive strategies and ergonomic improvements to enhance occupational health and comfort among medical secretaries working in healthcare institutions.

Musculoskeletal Disorders of Postgraduate Dentistry Students and Specialist Dentists

This study aimed to assess the prevalence of MSDs and the level of ergonomics knowledge among dentists who are specializing in different branches or have completed specialist training.

SakuraBead Used as Resorbable Embolic for Genicular Artery Embolization

An open label, prospective, two-arm, multicenter, randomized controlled trial comparing SakuraBead genicular artery embolization (GAE) with a control (corticosteroid injection).