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Progressive Muscular Atrophy clinical trials

View clinical trials related to Progressive Muscular Atrophy.

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NCT ID: NCT06315673 Not yet recruiting - Clinical trials for Amyotrophic Lateral Sclerosis

Digital Assessment of Speech and Fine Motor Control in ALS

Start date: June 1, 2024
Phase:
Study type: Observational

This is a single-session, case-control study that incorporates digital tools for assessing speech and motor function in motor neuron disease. Patients with motor neuron disease (including amyotrophic lateral sclerosis (ALS), primary lateral sclerosis (PLS), and progressive muscular atrophy (PMA)) and age-matched healthy controls will be enrolled. Subjects will complete a speech and handwriting assessment during the study visit on a tablet computer (BioSensics LLC, Newton, MA). We will explore whether these digital biomarkers are sensitive to functional disease severity as reported by the ALS Functional Rating Scale - Revised (ALFRS-R) [1]. We will also compare assessment data between the patient and control groups.

NCT ID: NCT05204017 Recruiting - Clinical trials for Amyotrophic Lateral Sclerosis

Comprehensive Analysis Platform To Understand, Remedy and Eliminate ALS

CAPTURE ALS
Start date: September 12, 2021
Phase:
Study type: Observational

CAPTURE ALS is a long-term data and biorepository platform that will facilitate future ALS research. CAPTURE ALS will provide the standardized systems and tools necessary to collect, store, and analyze vast amounts of multimodal information about ALS. These multimodal datasets and biosamples will be made available for use by researchers or industry across Canada and around the world in accordance with the CAPTURE ALS Data Sharing Policy to advance research on ALS.

NCT ID: NCT04875416 Recruiting - Clinical trials for Amyotrophic Lateral Sclerosis

Phenotype, Genotype and Biomarkers 2

PGB2
Start date: January 8, 2021
Phase:
Study type: Observational

The purpose of this study is to learn more about amyotrophic lateral sclerosis (ALS) and other related neurodegenerative diseases, including frontotemporal dementia (FTD), primary lateral sclerosis (PLS), hereditary spastic paraplegia (HSP), progressive muscular atrophy (PMA) and multisystem proteinopathy (MSP). More precisely, the investigator wants to identify the links that exist between the disease phenotype (phenotype refers to observable signs and symptoms) and the disease genotype (genotype refers to your genetic information). The investigator also wants to identify biomarkers of ALS and related diseases.

NCT ID: NCT03912987 Active, not recruiting - Clinical trials for Amyotrophic Lateral Sclerosis

TRIAL READY (Clinical Trial Readiness)

Start date: January 22, 2019
Phase:
Study type: Observational

This study, being conducted under the auspice of the CReATe Consortium, will enroll patients with ALS and related disorders as well as healthy controls, with the goal of facilitating clinical validation of leading biological-fluid based biomarker candidates that may aid therapy development for patients with ALS and related disorders.

NCT ID: NCT03706391 Completed - Clinical trials for Amyotrophic Lateral Sclerosis

Study of ALS Reversals 4: LifeTime Exposures

StARLiTE
Start date: September 26, 2018
Phase:
Study type: Observational [Patient Registry]

Hypothesis: There exists patients who have met ALS or PMA diagnostic criteria and subsequently experienced robust and sustained improvement, i.e. a "reversal." Thirty-eight of these patients were identified in the prior Duke University study, Documentation of Known ALS Reversals (St.A.R. Protocol 1, Duke IRB Pro00076395). The investigators hypothesize these patients have had different environmental exposures than patients with typically progressive ALS. Identification of specific environmental influences may point to exposures which are protective or exposure that lead to the development of a rare and novel reversible ALS-like disease. Objective: This study seeks to identify environmental exposures associated with ALS reversals.

NCT ID: NCT03489278 Recruiting - Clinical trials for Amyotrophic Lateral Sclerosis

Clinical Procedures to Support Research in ALS

CAPTURE-ALS
Start date: February 15, 2018
Phase:
Study type: Observational

The purpose of the Clinical Procedures To Support Research (CAPTURE) study is to utilize information collected in the medical record to learn more about a disease called amyotrophic lateral sclerosis (ALS) and related disorders.

NCT ID: NCT03464903 Completed - Clinical trials for Amyotrophic Lateral Sclerosis

Study of ALS Reversals 2: Genetic Analyses

StAR2
Start date: June 22, 2018
Phase:
Study type: Observational [Patient Registry]

The purpose of this study is to try to understand why reversals of amyotrophic lateral sclerosis (ALS) and primary muscular atrophy (PMA) take place. The study will enroll patients with ALS or PMA reversals to give saliva samples in order to determine if the ALS or PMA reversal is because of certain changes in the genetic code.

NCT ID: NCT03067857 Active, not recruiting - Clinical trials for Amyotrophic Lateral Sclerosis

Autologous Bone Marrow-Derived Stem Cell Therapy for Motor Neuron Disease

Start date: September 2016
Phase: Phase 1/Phase 2
Study type: Interventional

Herein, the investigators study the safety and efficacy of transplanting purified autologous bone marrow-derived stem cells transplanted via the intrathecal route by interventional radiology and the intravenous route.

NCT ID: NCT02852278 Completed - Clinical trials for Amyotrophic Lateral Sclerosis

A Patient Centric Motor Neuron Disease Activities of Daily Living Scale

Start date: December 2016
Phase:
Study type: Observational

The purpose of this study is to learn about rates of patient-reported disease progression in patients with motor neuron diseases (amyotrophic lateral sclerosis, progressive muscular atrophy, primary lateral sclerosis, hereditary spastic paraplegia) outside the clinical setting, and the patient-reported clinical characteristics that influence this rate of progression. All patients enrolled in CReATe Connect, a Rare Diseases Clinical Research Network (RDCRN) Contact Registry, will be invited via email to participate in this study.

NCT ID: NCT02574390 Completed - Clinical trials for Amyotrophic Lateral Sclerosis

Answer ALS: Individualized Initiative for ALS Discovery

AnswerALS
Start date: December 2015
Phase:
Study type: Observational [Patient Registry]

Creation of a large repository of induced pluripotent stem cells (iPSC), bio-fluid samples (blood and spinal fluid (optional)), and cell lines for ALS gene identification. This will be combined carefully with collected measures of the pattern of the symptoms people with ALS have and how these change over time. People with other motor neuron diseases and healthy controls will be included as comparisons