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33 recruiting studies matching your filters

Multi-Modal Digital Monitoring of Disease Symptoms in Spinocerebellar Ataxia

This is an observational study. Subjects with a documented diagnosis of spinocerebellar ataxia (SCA) will be screened and recruited to participate at two clini…

Spinocerebellar Ataxia (SCA)

Estimated

~$10 – $1,125

BioSensicsNCT07851376

Phase 1

A Phase 1b First-in-Human, Open-Label, Dose-Finding Trial to Evaluate the Safety and Tolerability of SGT-212 Delivered Via Dual Intradentate Nucleus (IDN) and Intravenous (IV) Administration to Participants With Friedreich's Ataxia (FA)

This is a phase 1b, first in-human, open-label, dose-finding study investigating the safety and tolerability of SGT-212 in participants with Friedreich's ataxi…

Friedreich's Ataxia (FA)

Estimated

~$10 – $8,750

Solid Biosciences Inc.NCT07180355

Phase 1

Channeling Hope Trial 1: A Phase 1, Open-label, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK) of Chronic Aprepitant Use in Individuals With Congenital Contractures of the Limbs and Face, Hypotonia, and Development Delay (CLIFAHDD)

There are no drug treatments for individuals with Congenital Contractures of the Limb and Face, Hypotonia, and Developmental Delay (CLIFAHDD), an ultra-rare an…

CLIFAHDDCongenital Contractures of the Limbs and Face, Hypotonia, and Developmental DelayNALCN Channelopathy+2 more

Estimated

~$10 – $6,250

The University of Texas Health Science Center at San AntonioNCT07845825

Phase 1

Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia

The purpose of this study is to test the safety and preliminary efficacy of AAVrh.10hFXN to treat the cardiomyopathy associated with Friedreich's ataxia (FA). …

Friedreich AtaxiaCardiomyopathiesCardiac Hypertrophy+1 more

Estimated

~$10 – $6,250

Weill Medical College of Cornell UniversityNCT05302271

Observational

Cardiac Output and Fatigue in Friedreich's Ataxia

This is a clinical trial examining to impact of aerobic training plus omaveloxolone in FRDA. Thirty individuals with FRDA will be recruited; 20 individuals wil…

Friedreich's Ataxia

Estimated

~$10 – $7,500

Scott BarbutoNCT07444333

Observational

Study of Adapted Exercise and Mindfulness Interventions to Improve Motor Function and Sleep Quality in Individuals With Neurodegenerative Disease

The primary goal of this study is to address the need for targeted therapeutic interventions for impairments that impact walking in related neurodegenerative d…

Neurodegenerative DiseasesLeukodystrophyAtaxia+5 more

Estimated

~$10 – $8,750

Hugo W. Moser Research Institute at Kennedy Krieger, Inc.NCT05443906

Phase 3

Effects of N-Acetyl-L-Leucine on CACNA1A Disorders: A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study

A pivotal, randomized, double-blind, placebo-controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of CACNA1A. The…

CACNA1ASpinocerebellar Ataxia Type 6Episodic Ataxia Type 2+1 more

Estimated

~$10 – $8,750

IntraBio IncNCT07221292

Phase 2

An Open-Label Extension Study to Assess the Long-Term Safety, Efficacy, Pharmacodynamics, Pharmacokinetics, and Tolerability of Subcutaneous CTI-1601 in Subjects With Friedreich's Ataxia

An open label study designed to evaluate the safety, PK, PD, and clinical effects of long-term daily administration of CTI-1601 enrolling adolescent and adult …

Friedreich Ataxia

Estimated

~$10 – $8,750

Larimar Therapeutics, Inc.NCT06447025

Phase 2

A Phase 2, Multicenter, Open-label, Randomized, Controlled Study of LX2006 Gene Therapy in Participants With Friedreich Ataxia Cardiomyopathy (SUNRISE-FA 2)

The purpose of Study LX2006-03, a multicenter, Phase 2, open-label, randomized, controlled study, is to evaluate the efficacy and safety of LX2006 gene therapy…

Friedreich AtaxiaCardiomyopathy, Secondary

Estimated

~$10 – $8,750

Lexeo TherapeuticsNCT07721025

Characteristics And Clinical Course Of Disease In Participants With Cardiomyopathy Associated With Friedreich Ataxia (CLARITY-FA)

Characteristics and clinical course of disease In participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA)

Friedreich AtaxiaCardiomyopathy

Estimated

~$10 – $1,050

Lexeo TherapeuticsNCT06865482

Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study

This project is a global, multicenter, prospective, longitudinal, observational natural history study that can be used to understand the disease progression an…

Friedreich Ataxia

Estimated

~$10 – $1,125

Friedreich's Ataxia Research AllianceNCT06016946

Phase 3

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia

To evaluate the efficacy and safety of subcutaneous nomlabofusp in adult and pediatric subjects with Friedreich's ataxia

Friedreich AtaxiaFriedreich's Ataxia

Estimated

~$10 – $8,750

Larimar Therapeutics, Inc.NCT07778836