637 Clinical Trials for Various Conditions
This Phase 3 trial (Study SRK-015-003) was conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA) and were receiving an approved survival motor neuron (SMN) upregulator therapy (i.e., either nusinersen or risdiplam), to confirm the efficacy and safety of apitegromab as an adjunctive therapy to nusinersen and evaluate the efficacy and safety of apitegromab as an adjunctive therapy to risdiplam.
The TOPAZ study will assess the safety and efficacy of SRK-015 in later-onset Spinal Muscular Atrophy (SMA Type 2 and Type 3) in pediatric and adult patients.
This study will quantify and examine the extent to which the quadriceps femoris muscle group atrophies following tibial plateau fractures and the length of time that atrophy affects function. The study will also look at the effects of tibial plateau fractures on the quadriceps muscle and the effect this atrophy has on functional outcome. Quadriceps atrophy will be measured both clinically and by using MRI scans taken pre-operatively, 3 months post operatively, and 1 year post-operatively. The injured leg and the non-injured leg will be scanned in order for the non-injured leg to serve as the control. In this way muscle volume can be estimated from the muscle thickness at specific locations in the thigh. The muscle strength of the quadriceps will also be assessed at the 3 month and 1 year visit by measuring isokinetic knee-extension torque, and functional assessment questionnaires will be completed at each study visit.
To compare safety and efficacy of Perrigo's drug product compared to an FDA approved drug product in the treatment of vulvar and vaginal atrophy.
The purpose of this study is to assess the use of the Alma CO2 Pixel FemiLift™ in the treatment of VulvoVaginal Atrophy (VVA) in postmenopausal female subjects. The primary endpoint is to assess the change in the vaginal dryness by means of a Visual Analogic Scale (10 cm VAS) and/or Bachmann Index in VVA. The clinical improvement will be assessed by patient filled Vulvovaginal Atrophy.
Plantar heel pain is the most common problem of the foot. Plantar fasciitis is the leading cause of this pain, accounting for 11-15% of all foot symptoms seeking professional care, and occurs in 10% of the US population. The cause of heel pain can be from bone, soft tissue, nerve, or systemic disease. The second most common cause of heel pain is fat pad atrophy, followed by a combination of both fat pad atrophy and plantar fasciitis. \[1-4\] Fat pad displacement may be seen in the heel as well. Current treatments for plantar fasciitis include stretching and external support with orthotics with the goal of reducing local pressure as well as tissue breakdown.\[5\] However, patient compliance with extrinsic devices is challenging, and they may experience increased friction, irritation and breakdown at a different location on the foot due to thickness of the device in the shoe. Also, the patient must replace the device as soon as it breaks down but the breakdown often goes unnoticed. Failure of conservative management may lead to treatment with steroids or surgical intervention by endoscopic plantar fasciotomy, open fasciotomy or excision of bone spurs. Injection of steroids can instigate or exacerbate pre-existing heel fat pad atrophy. Autologous fat grafting to the heel may reduce plantar pressures, and thus serve as a treatment for heel pain. Autologous fat grafting is currently under investigation for a myriad of clinical scenarios.\[6-19\] Even though autologous fat grafting is not yet an established therapeutic approach for the cited indications, some of the reported results have been intriguing.\[19\] The variable resorption of fat, however, is an important confounding factor in all these studies. Currently the literature reports fat retention rates ranging from 25% to 80%.\[20\] The specific aim of this study is to assess the adipose stem cell characteristics in patients undergoing autologous fat grafting for heel fat pad atrophy and correlate these characteristics with fat retention. Data from this study will help determine the characteristics of the adipose stem cells in this population and open the door to additional studies. This pilot study will also help build new collaborative efforts between Foot and Ankle Specialists, Plastic Surgery, and Adipose Stem Cell Biologists, combining biomechanical expertise with fat grafting and basic science expertise.
It is believed that the average person with an eighty year old life span will walk the distance of the world twice in their lifetime. The foot is comprised of a specialized fat pad to provide shock absorption and protection against breakdown. With all the walking humans do however, foot fat pad breakdown is inevitable. Trauma to the foot is compounded in the diabetic patients due to many reasons: loss of protective sensation secondary to neuropathy, reduced skin hydration, decreased soft tissue elasticity, elevated blood sugars and increased body weight to name a few. Increased foot pressure and decreased fat pad protection are the main contributing factors to callus formation, foot pain and ulcer formation. Fat grafting is a cosmetic and reconstructive procedure that is used sometimes to help improve one's soft tissue thickness, shape and integrity. Autologous fat transplantation is a procedure using a patient's own fat that is taken by a small liposuction tube, from areas with a substantial amount of fat ( i.e. abdomen or thighs) and then transferred into the fat atrophied (decreased or worn out) area (in this case, the foot). The investigators are performing this research in an effort to decrease foot pressure during activity and to increase the soft tissue thickness of the sole of the foot during one's lifetime. Ultimately this could help reduce foot pain, callus formation and even ulcer formation. The goal of this research is to see whether fat grafting will help decrease the prevalence of the aforementioned foot complications. The investigators hope that by using one's own fat tissue, he or she may have a longer duration of relief than by using other methods. This is a randomized, cross-over designed study. Randomized means that patients will be enrolled by chance (like the flip of a coin) to one of two groups for the first part of the study. Cross-over means that, after the first part of the study or at one year, patients will move into the opposite group. One group will receive the fat grafting procedure with one year follow up (year A pathway) and the other will receive standard of care treatment from the patient's primary podiatrist during the first year (year B pathway). After the first year, patients will switch to the opposite year pathway for the next 12 months. Participation in this study will last approximately 24-26 months.
This study is being done to help determine whether patients with severe sepsis (overwhelming inflammation in the body as a result of an infection) lose muscle and become weak more rapidly than patients with other severe illnesses. Weakness and muscle loss that develops after a severe illness is a serious problem. Patients who develop weakness and have a decrease in muscle size often have to stay in the hospital longer and have a higher chance of dying. At the current time, it is not clear whether certain severe illnesses are more likely to cause weakness and muscle loss. This study will be done to measure the changes in muscle size and strength as a result of each patient's illness
Severe sepsis will provoke signals leading to muscle atrophy and weakness. Electrical stimulation will reduce the impact of sepsis.
This protocol is designed to study the effect and safety of 2 low-dose regimens of conjugated estrogen cream (PVC) given vaginally in postmenopausal women with atrophic vaginitis.
The main purpose of this study is to assess the ocular and systemic safety and tolerability of RO7669330 in GA secondary to AMD after multiple unilateral intravitreal (IVT) doses.
A Phase 2a Study of Foralumab Nasal in Patients with Multiple System Atrophy (MSA)
This is a Phase 2, double-blind, placebo-controlled, multi-center, Phase II, dose escalation study to evaluate the safety, tolerability, pharmacokinetics and efficacy of Ya-101 in subjects with multiple system atrophy.
This study is for people who have geographic atrophy due to age-related macular degeneration (AMD). AMD happens when the macula, the light-sensitive layer at the back of the eye called the retina, becomes damaged and causes a person's central vision to worsen. Geographic atrophy is an advanced form of AMD where cells in the retina waste away and die. Over time this can lead to permanent loss of vision. Avacincaptad pegol can help slow down the worsening or progression of geographic atrophy. Avacincaptad pegol is a treatment approved in the US to treat geographic atrophy. This study is about collecting information on how people with geographic atrophy are treated in routine clinical practice. This includes recording any medical problems from avacincaptad pegol. This is known as an observational study. Information will be collected from the peoples' medical records during and after treatment. The people in this study will have geographic atrophy in 1 or both eyes and they and their doctor has decided they will be treated with avacincaptad pegol. The individual's doctor decides on treatment, not the study sponsor (Astellas). People that want to take part in the study will have eye examinations that they would usually have as part of their routine care. People will also be asked to complete surveys about their eye health. These surveys will occur when treatment starts and then every 6 months for the first 2 years. After 2 years the surveys will happen once a year. The people on the study can take part if their doctor provides treatment with avacincaptad pegol and they want to continue with the study. The people on the study can take part for up to 3 years or up to 5 years, depending on when they start the study.
This study is open to adults aged 55 years and older with geographic atrophy that was diagnosed by imaging of the retina. The purpose of this study is to find out whether a medicine called BI 1584862 improves the eyes in people with geographic atrophy and to find the most suitable dose. This study has 4 treatment groups. Participants are put into groups randomly, which means by chance. Initially participants can join a BI 1584862 and a placebo group; then more participants are put randomly in 2 additional BI 1584862 groups and a placebo group. Participants are in the study for 1 year. During this time, they visit the study site 13 times. At the visits, doctors check the severity of participants' eye disease and collect information on any health problems. They take detailed pictures of the back of the eye, the retina, with imaging methods called fundus autofluorescence (FAF) and optical coherence tomography (OCT). They measure areas of the retina that appear healthy (bright) or start to waste away (dark, atrophy). The changes over time are then compared between the treatment groups.
This is a randomized, double-blind, placebo-controlled trial in de novo kidney transplant patients to determine if the addition of belumosudil (brand name- REZUROCK®) on the background of standard immunosuppression will prevent fibrosis in the kidney transplant. Interstitial expansion is the precursor of interstitial fibrosis and graft loss. The hypothesis underlying the study is that abgrogating the fibrogenic effects of the RhoA pathway with belumosudil will reduce structural damage in transplanted kidneys and possible subsequent transplant failure.
This study is open to people 50 years or older with an eye condition called geographic atrophy. The purpose of this study is to compare a medicine called BI 771716 with a medicine called pegcetacoplan. BI 771716 is being developed to treat people with geographic atrophy. Pegcetacoplan is a medicine already used to treat people with geographic atrophy. In this study, participants receive either BI 771716 or pegcetacoplan as injections in the eye. Participants are in the study for a little longer than a year and visit the study site every 4 weeks. At the visits, the study doctor checks the eyes of the participants. The results are compared between the groups of participants to see whether the treatment works. The study doctor also regularly checks participants' health and takes note of any unwanted effects.
The main goal of this trial is to evaluate the efficacy and safety of Lu AF82422 for the treatment of participants with Multiple System Atrophy (MSA).
Age-related macular degeneration (AMD) affects millions of elderly patients. When advanced, there is Geographic Atrophy (GA) in the retina. This means that there is an area with a loss of light-sensitive cells, called photoreceptors. That part of the retina can no longer see. Atrophy begins as a small spot in the retina distant from the fovea which is the part of the retina responsible for sharp central vision. The GA grows, and when it reaches the fovea, vision is severely diminished, and details cannot be seen anymore. The purpose of the eDREAM study is to understand if GAL-101 can slow the growth of GA and prevent it from reaching the fovea. GAL-101 is given as eyedrops. eDREAM patients will administer study eyedrops every day. Patients will be assigned by chance (randomly) to receive either eye-drops that contain the new medication, GAL-101, or eyedrops without the active drug (Placebo). Neither patients nor doctors will know which treatment was assigned to each patient until the end of the study.
The purpose of this study is to assess the long-term safety and tolerability after an intravitreal injection (a shot of medicine into the eye) of JNJ-81201887 administered in parent clinical studies.
An observational, retrospective, cross-sectional, multicenter study. Real-world data were obtained from medical records of Spanish public hospitals (9 hospitals).
The aging Veteran population has more frequent inpatient hospitalization than non-Veterans. In addition, older Veterans have an inability to fully recover muscle mass and function after hospital-acquired weakness. The problem of hospital-acquired weakness is greatly increased in at-risk Veteran populations such as those with chronic kidney disease (CKD). The purpose of this collaborative study involving Denver and Baltimore VAMC sites is to improve health through the detection and rehabilitation management of hospital-acquired weakness in those with CKD. The study goals include identifying and monitoring the optimal muscle assessment sites in clinical settings, examining how muscle health impacts length of hospital stay and physical resilience, and determining how home-based and supervised exercise comparatively addresses post-hospitalization physical resilience using performance tests and Veteran feedback.
The primary objective of the study is to evaluate the efficacy of TEV-56286 administered orally for the treatment of adult participants with Multiple System Atrophy (MSA). A secondary objective of the study is to evaluate specific efficacy parameters of TEV-56286. Another secondary objective is to evaluate the safety and tolerability of TEV-56286. The planned study period per participant is 56 weeks including a screening period (up to 4 weeks), a 48-week double-blind treatment period, and a follow-up visit (approximately 4 weeks after the end of the double-blind treatment period). The study duration will be approximately 27 months.
In this PIERRE-PK study, researchers will learn how the body processes nusinersen when it is given through the ThecaFlex DRx™ System, compared to when nusinersen is given by lumbar puncture (LP). The ThecaFlex DRx system is an investigational implantable medical device developed by Alcyone Therapeutics, Inc. It consists of a catheter, which is a flexible tube, connected to a port which is placed under the skin. Alcyone Therapeutics, Inc. has an ongoing study called PIERRE to test the ThecaFlex DRx system. Participants with spinal muscular atrophy (SMA) in the PIERRE study may be enrolled in the PIERRE-PK study. The main objective of the PIERRE-PK study is to learn how the body processes nusinersen when given by the ThecaFlex DRx system compared to a lumbar puncture. The main questions researchers want to answer are: * What is the highest amount of nusinersen found in the blood after dosing? * How much nusinersen is found in the blood over the first 24 hours after dosing? The PIERRE-PK study will be done as follows: * Participants will be screened to check if they can join the study. The screening period will be up to 30 days for this study and may overlap with the PIERRE study. * Participants will receive a dose of nusinersen by lumbar puncture. * The ThecaFlex DRx system will be implanted after the lumbar puncture, as part of the PIERRE study. * Participants will receive a dose of nusinersen by the ThecaFlex DRx system, as part of the PIERRE study. * Researchers will take blood samples before and after each dose. The last blood sample will be taken 24 hours after the dose. * The total study duration for each participant in the PIERRE-PK study will be approximately 5 months. This period will overlap with the participant's first 5 months in the PIERRE study.
This study is researching experimental (study) drugs called pozelimab and cemdisiran. The study is focused on participants who have geographic atrophy (GA) caused by age-related macular degeneration (AMD). Geographic atrophy is a medical term that refers to later-stage cases of AMD which is an eye condition affecting central vision (what one sees straight ahead). The purpose of this study is to evaluate the progression rate of Geographic Atrophy in eyes of patients treated with cemdisiran alone or in combination with pozelimab compared to those treated with placebo. The study is looking at several other research questions, including: * What side effects may happen from taking the study drug(s) * How much study drug(s) are in the blood at different times * Whether the body makes antibodies against the study drug(s) (which could make the study drug(s) less effective or could lead to side effects)
In this observational study, researchers are looking at the effects of spinal muscular atrophy (SMA) drugs on the muscles and nerve cells in patients with SMA. Primary Objectives * To evaluate the feasibility and reliability of performing MR functional imaging in exercising muscle in patients with SMA. * To evaluate patients with SMA types 2 and 3 at baseline and longitudinally at 6 and 12 months Secondary Objectives * To describe the MR functional bioenergetics response in the leg muscles in four potential groups of patients with spinal muscular atrophy: untreated, actively treated with nusinersen (Spinraza®) or onasemnogene abeparvovec (Zolgensma®), actively treated with risdiplam (Evrysdi®), and switching from Spinraza or Zolgensma to Evrysdi. * To identify changes in motor function in patients with SMA types 2 and 3 who initiate treatment with risdiplam. * To obtain biomarkers in blood, urine, and muscle tissue to provide evidence for risdiplam effect on skeletal muscle. * To obtain quality of life and disability data from participants in this study.
The primary purpose of the study is to determine if IVT injections of ANX007 every month reduce vision loss in participants with GA secondary to age-related macular degeneration (AMD).
Spinal cord stimulation (SCS) has shown remarkable efficacy in restoring motor function in people with spinal cord injury by recruiting afferent input to enhance the responsiveness of spared neural circuits to residual cortical inputs. This pilot will test if SCS can show evidence to improve motor deficits in people with Type 2, 3, or 4 spinal muscular atrophy (SMA). The investigators will enroll up to six subjects with Type 2, 3, or 4 SMA aged 16 or older that show quantifiable motor deficits of the upper body. The investigators will then implant the subjects with percutaneous, linear spinal leads near the cervical spinal cord for a period of up to 29 days. Although these leads are not optimized for motor function but rather for their clinically approved indication of treating pain, the investigators believe they provide a safe technology enabling our team to perform scientific measurement necessary to evaluate potential for effects of SCS in motor paralysis with SMA. After the end of the study, the leads will be explanted.
DRPLA Natural History and Biomarkers Study (DRPLA NHBS) is a prospective observational study that will lay the foundation for clinical trials in DRPLA. The aims of this project are: * To characterize the natural history of DRPLA in both juvenile- and adult-onset patients and study different modalities of biomarkers in this condition. * To identify genetic factors and biomarkers that could predict disease progression. * To provide a platform to support the design and conduct of clinical trials. This study has three arms: 1. Adult Participants: this arm of the study will require participants to be 16 years old or over to participate. 2. Pediatric Participants: this arm of the study will require participants to be under 16 years old to participate. 3. Remote Participants: patients that cannot or do not wish to travel to one of the study sites can participate in this arm of the study, irrespective of their age. Participants will have an annual visit for three years (baseline visit and two follow-up visits, three visits in total). Subjects who complete the whole protocol will be assessed on two consecutive days to reduce patient burden. This project will allow for a better understanding of DRPLA and its course, and therefore allow for future clinical trials on this condition to be more precisely and effectively conducted.
This study will aim to assess the fertility status of men with Spinal Muscular Atrophy (SMA) not on disease-modifying therapies. Participants will: 1. Complete online questionnaires that will assess SMA diagnosis and disease burden, medical and surgical history, medication usage, and fertility status and perspectives. 2. Over the 3-month initial study baseline period participants will provide two separate ejaculates for semen analysis and a single determination of sperm quality using DNA fragmentation testing using home collection and subsequent shipment to a central laboratory. 3. Over the initial study baseline period of 3 months study participants will obtain a blood test to determine male reproductive hormone levels. During the 24-month study duration, participants will be requested to undergo a yearly semen analysis and complete online relevant questionnaires.