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NCT ID: NCT05769465 Recruiting - Clinical trials for Spinal Muscular Atrophy

MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular Atrophy

MAP_THE_SMA-01
Start date: April 1, 2023
Phase:
Study type: Observational

Spinal Muscular Atrophy (SMA) is caused by the homozygous loss of the Survival Motor Neuron (SMN) 1 gene, which leads to degeneration of spinal alpha-motor neurons and muscle atrophy. Three treatments have been approved for SMA but the available data show interpatient variability in therapy response and, to date, individual factors such as age or SMN2 copies,cannot fully explain this variance. The aim of this project is: - collect clinical data and patient-reported outcome measures (PROM) from patients treated with nusinersen, risdiplam, onasemnogene abeparvovec, - identify novel biomarkers and RNA molecular signature profiling, - develop a predictive algorithm using artificial intelligence (AI) methodologies based on machine learning (ML), able to integrate clinical outcomes, patients' characteristics, and specific biomarkers. This effort will help to better stratify the SMA patients and to predict their therapeutic outcome, thus to address patients towards personalized therapies.

NCT ID: NCT05761262 Recruiting - Clinical trials for Spinal Muscular Atrophy

SMN Circular RNAs as Potential Biomarkers for the Therapeutic Response to Nusinersen in Spinal Muscular Atrophy Patients

Start date: December 13, 2019
Phase:
Study type: Observational

The first cure for Spinal Muscular Atrophy (SMA; Nusinersen) has been approved by FDA in 2017. Although it improves the clinical picture of most SMA patients, not all exhibit the same response to treatment. In this project the aim will be: i. identifying cell-free SMN circular RNAs (circRNAs) in body fluids of SMA patients as potential biomarkers before and after Nusinersen; ii. evaluating their prognostic power as predictors of the clinical response of SMA patients to Nusinersen; iii. identifying human intronic polymorphisms that affect SMN circRNAs biogenesis and impact on the efficacy of Nusinersen. The results obtainable with this project will evaluate if different concentration of cell free SMN circRNAs in SMA patients could underlie the genotype-phenotype mismatch, usually observed, and the reduced response of a subset of SMA patients to therapy. Our research could highlight the need for these of combinatorial 'SMN-plus' and "personalized" therapies that account for individual differences.

NCT ID: NCT05760209 Recruiting - Clinical trials for Spinal Muscular Atrophy

SMN Circular RNAs as Potential New Targets and Biomarkers for SMA

CircSMA
Start date: July 22, 2021
Phase:
Study type: Observational

Spinal Muscular Atrophy (SMA) is a life-threatening disease in infancy that is caused by inactivating mutations in the Survival Motor Neuron 1 (SMN1) gene1,2. SMN1 mutations lead to deficiency in SMN protein, which results in degeneration of motor neurons in the spinal cord, progressive muscle weakness and atrophy. The almost identical SMN2 gene does not suffice SMN function, because skipping of exon 7 in its mRNA yields an unstable protein. Nevertheless, SMN2 represents a disease modifier gene and increasing its expression or rescuing its splicing defect have long been considered elective strategies for SMA1,2. After substantial translational research efforts, the first therapies eliciting clinical benefits for SMA patients have recently become available3. Nusinersen, an antisense oligonucleotide (ASO), and Risdiplasm, a small molecule, bind the SMN2 RNA and promote splicing of exon 7. On the other hand, Zolgesma, an adeno-associated virus delivering the SMN1 gene (scAAV9-SMN), bypasses the need to correct the splicing defect. Nevertheless, none of these therapies currently represents a complete cure for patients, because not all of them respond equally and in a significant portion of patients the symptoms are attenuated but not corrected3. It is believed that early treatment, possibly at a pre-symptomatic stage, would positively affect the clinical response and may significantly improve patient's management. However, another critical point is the current lack of information on the long-term efficacy and safety of the current treatments4. In this scenario, it is likely that further elucidation of the biological functions of the SMN genes and the identification of robust biomarkers for stratification of patients will set the ground for more "personalized" therapies, which may account for the clinical variability observed in patients and help improving the therapies in use.

NCT ID: NCT05760066 Not yet recruiting - Atrophy, Muscular Clinical Trials

Effects of Resistance Training Preconditioning on Skeletal Muscle Recovery From a Period of Disuse in Young Adults

Start date: August 1, 2023
Phase: N/A
Study type: Interventional

The goal of this clinical trial is to compare the effects of resistance training (RT) preconditioning vs no training on disuse-induced atrophy and post-disuse resistance training in young healthy individuals. The main questions it aims to answer are: - To determine if performing RT prior to a period of disuse enhances the regain of strength, skeletal muscle size, and skeletal muscle quality while performing RT after a period of disuse. - To determine if performing RT prior to a period of disuse dampens the maladaptive effects of disuse on muscle size, muscle quality, and strength. - To determine the anabolic and proteolytic mechanisms underpinning the observed outcomes. Participants will: 1. Perform either 6 weeks of resistance training or maintain an untrained lifestyle 2. Perform 2 weeks of limb immobilization induced disuse of a randomized leg 3. Perform 6 weeks of resistance training Researchers will compare the resistance training preconditioning condition vs the non-trained condition to see if resistance training prior to a period of disuse is beneficial during the disuse period and in the return to training period on skeletal muscle size, strength, and underpinning molecular markers.

NCT ID: NCT05755451 Recruiting - Clinical trials for Muscular Atrophy, Spinal

Natural History of SMA

iSMAR
Start date: June 21, 2018
Phase:
Study type: Observational [Patient Registry]

This is an investigator initiated observational study with the aim to record several aspects of function, care and adverse events in a large cohort of SMA patients followed longitudinally by using a structured academic disease registry.

NCT ID: NCT05747924 Recruiting - Clinical trials for Muscular Dystrophies

Phase 1/2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD)

FORTITUDE
Start date: April 4, 2023
Phase: Phase 1/Phase 2
Study type: Interventional

A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

NCT ID: NCT05747898 Completed - Scar; Atrophy Clinical Trials

Cannula-based Versus Needle-based Subcision for Posttraumatic Atrophic Facial Scars

Start date: November 1, 2021
Phase: N/A
Study type: Interventional

This study aims to evaluate the effectiveness of cannula- based subcision versus common needle subcision in treatment of posttraumatic atrophic facial scars

NCT ID: NCT05747261 Recruiting - Clinical trials for Spinal Muscular Atrophy (SMA)

Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy (BLUEBELL)

BLUEBELL
Start date: February 2, 2023
Phase: Phase 1/Phase 2
Study type: Interventional

The goal of this multicenter, open-label, non-comparative, cohort study is to investigate the safety, immunogenicity, and efficacy of ANB-004 in children with spinal muscular atrophy. The study will have a standard 3+3 dose-escalation design.

NCT ID: NCT05746052 Completed - Acne Scars Clinical Trials

Punch Elevation and Microneedling in Treatment of Atrophic Acne Scars

Start date: April 1, 2021
Phase: N/A
Study type: Interventional

The ultimate goal of this study was to compare punch elevation and micro needling with PRP versus micro needling and PRP only in treatment of post acne scars, in an attempt to achieve better management of such condition. This is a prospective study that was carried out on 15 patients (their ages ranged from 19 to 32 years with a mean of 23 years. They are 6 males and 7 females, 7 patients were of skin photo type III, and 10 were rural residents), they presented with post acne facial scars, and attending the Dermatology and Andrology outpatient clinic of Al-Azhar University Hospital in (Assiut), between April 2021 and March 2022. Left side of face of the lesion of each patient will be treated by punch elevation two weeks before microneeedling with platlets rich plasma (PRP),the right side will be treated by microneedling with (PRP) only from the start, three sessions of microneedling will be done with 4 weeks interval. Each patient had punch elevation for scars in left side at first session then dressing removed after 7 days after three weeks all patents received treatment on both sides of the face by micro needling with PRP. During each session, topical anesthesia was applied over the area of interest on face and removed after 20 mints. Patients were placed in supine position with head stable, the skin was stretched and micro needling was carried out in vertical, horizontal and both diagonal directions for about 4-5 times. PRP (2 ml) were applied on the face. A total of three sessions of microneedling were performed at monthly intervals. Follow-up of the patients was done before and after treatment by clinical examination and photography by comparing the photographs before and after therapy; Evolution of clinical response included extent of improvement and possible adverse effects including bleeding, and erythema. And PIH Clinical photos of the lesions had been taken before the first session and one month after the last session and assessed clinically to grade the severity of scarring proposed by Goodman and Baron's quantitative scale for acne scars at the baseline and the end of study. Patients' satisfaction had been done by using a quartile grading system (1 poorly satisfied, 2 satisfied or 3 very satisfied). As regard efficacy of the procedures, we found significant improvement of atrophic acne scars, with significant reduction in number of acne scars as well as significant reduction in goodman score after treatment by punch elevation and micro needling with PRP, most of patients were satisfied after treatment, the side treated with punch elevation have statistically significant reduction in the number of the scar when compared to the right side.

NCT ID: NCT05734248 Completed - Aging Clinical Trials

Fractional Laser Drug Delivery of a Local Anesthetic

Start date: February 6, 2023
Phase: N/A
Study type: Interventional

The purpose of this study is to assess the clinical efficacy of fractional CO2 laser-assisted delivery of a topically applied anesthetic to the perceived pain during filler injection.