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Results for “Inherited Neuromuscular Conditions”

374 trials

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Showing 20 of 374 results

Large-scale testing (Phase 3)Study completedNCT05156320
What this trial is testing

Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

Who this might be right for
Spinal Muscular AtrophySpinal Muscular Atrophy Type 3Spinal Muscular Atrophy Type 2+7 more
Scholar Rock, Inc. 188
Early research (Phase 1)Ended earlyNCT02292225
What this trial is testing

Duvelisib With Obinutuzumab in Patients With CLL/SLL Previously Treated With a BTKi (SYNCHRONY)

Who this might be right for
Lymphocytic Leukemia, ChronicLymphoma, Small Lymphocytic
SecuraBio 3
Not applicableLooking for participantsNCT06539169
What this trial is testing

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

Who this might be right for
Alpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more
xCures 1,000
Not applicableNo Longer AvailableNCT03317366
What this trial is testing

Expanded Access to Provide ARQ 092 for the Treatment of Overgrowth Diseases and/or Vascular Anomalies

Who this might be right for
Proteus SyndromePIK3CA-Related Overgrowth Spectrum (PROS)Growth Disorders
ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)
Not applicableStudy completedNCT00622453
What this trial is testing

Arrhythmias in Myotonic Muscular Dystrophy

Who this might be right for
Muscular DystrophyArrhythmiaSudden Cardiac Death
Indiana University 448
Not applicableStudy completedNCT04862273
What this trial is testing

CMR T1 Mapping for Diagnosis of Cardiac Amyloidosis

Who this might be right for
Heart Failure NYHA Class IIHeart Failure NYHA Class IIIHeart Failure NYHA Class IV+4 more
University of Leipzig 112
Not applicableUnknownNCT05940922
What this trial is testing

RWE-based Treatment Patterns and Outcomes in CIDP

Who this might be right for
Chronic Inflammatory Demyelinating PolyneuropathyGuillain-Barre SyndromeHereditary Amyloidosis
RwHealth 170
Not applicableStudy completedNCT05131542
What this trial is testing

Assessment of Hypotonia in Children With Down Syndrome

Who this might be right for
Muscle HypotoniaDown SyndromeMuscle, Floppy+2 more
Universidad de Murcia 50
Not applicableActive Not RecruitingNCT05227287
What this trial is testing

ADH1 and ADH2 Disease Monitoring Study (DMS)

Who this might be right for
Autosomal Dominant Hypocalcemia
Calcilytix Therapeutics, Inc., a BridgeBio company 95
Not applicableLooking for participantsNCT05361811
What this trial is testing

Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial

Who this might be right for
Neurofibromatosis 1Noonan SyndromeLegius Syndrome+2 more
National Cancer Institute (NCI) 70
Large-scale testing (Phase 3)Study completedNCT03746522
What this trial is testing

Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Participants With Moderate to Severe Obesity

Who this might be right for
Bardet Biedl Syndrome (BBS)Alström Syndrome (AS)
Rhythm Pharmaceuticals, Inc. 52
Testing effectiveness (Phase 2)Study completedNCT01252628
What this trial is testing

Phase 1 and 2 Study of PX-866 and Cetuximab

Who this might be right for
Incurable Metastatic Colorectal CarcinomaIncurable Progressive, Recurrent or Metastatic Squamous Cell Carcinoma of the Head and Neck
Cascadian Therapeutics Inc. 178
Not applicableStudy completedNCT06240000
What this trial is testing

Radiofrequency Ablation of the Superior Cluneal Nerve

Who this might be right for
Nerve Entrapment SyndromeRadiofrequency AblationLow Back Pain
Ankara City Hospital Bilkent 25
Not applicableStudy completedNCT05436210
What this trial is testing

Postural and Anthropometric Properties of Foot and Ankle of Patients With DMD

Who this might be right for
Duchenne Muscular DystrophyAmbulation DifficultyPosture Disorders in Children+1 more
Hacettepe University 48
Testing effectiveness (Phase 2)Study completedNCT00030264
What this trial is testing

Combination Chemotherapy in Treating Patients With Neurofibromatosis and Progressive Plexiform Neurofibromas

Who this might be right for
Neurofibromatosis Type 1Precancerous Condition
Children's Hospital of Philadelphia 23
Not applicableUnknownNCT05215964
What this trial is testing

The Association Between Skeletal Muscle Mass and Severity of Polycystic Liver Disease and Polycystic Kidney Disease

Who this might be right for
Polycystic Kidney DiseasesPolycystic Liver DiseaseMuscle Loss
National Taiwan University Hospital 600
Not applicableStudy completedNCT00521794
What this trial is testing

Characteristics of Andersen-Tawil Syndrome

Who this might be right for
Andersen-Tawil SyndromeAndersen Syndrome
University of Rochester 28
Not applicableStudy completedNCT05545774
What this trial is testing

Neuromuscular Characterisation in Late Adolescent and Adult Cystinosis Patients

Who this might be right for
Cystinosis
Assistance Publique - Hôpitaux de Paris 20
Testing effectiveness (Phase 2)Study completedNCT01279590
What this trial is testing

Study of the Safety and Tolerability Associated With PPD10558 Versus Atorvastatin in Patients Previously Intolerant to Statins Due to Statin-associated Myalgia (SAM)

Who this might be right for
MyalgiaHypercholesterolemiaHyperlipidemia
Furiex Pharmaceuticals, Inc 282
Testing effectiveness (Phase 2)Study completedNCT01329770
What this trial is testing

Safety and Efficacy Study of Antioxidants for the Treatment of the Fragile X Syndrome

Who this might be right for
Fragile X Syndrome
Yolanda de Diego Otero 30
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