Clinical trials: Atrophy
Varicocele
Assessing the Rates of Major Complications in Combined Mini-incision Microscopic Varicocelectomy
The purpose of this study is to determine the rates of major post-varicocelectomy complications (Recurrence, Hydrocele and Testicular Atrophy) in patients operated with a new method named Combined Mini-incision Microscopic…
- Trial name
- CMMV (NCT02092311)
- Testing
- Combined Microscopic Varicocelectomy, Inguinal and Subinguinal varicocelectomy
- Where
- Iran
- Volunteers
- 570 people
- Sponsor
- Omid Fertility Center
Atypical Parkinsonism, Dementia With Lewy Bodies
Focused Ultrasound Thalamotomy for Tremor Relief in Atypical Parkinsonism
The goal of this retrospective observational study is to describe the efficacy of focused ultrasound ventral-intermediate nucleus thalamotomy in patients with atypical parkinsonism.
- Conditions
- Atypical Parkinsonism, Dementia With Lewy Bodies, Multiple System Atrophy, MSA
- Trial name
- FUSAP (NCT06204575)
- Testing
- Data collection
- Where
- Israel
- Volunteers
- 8 people
- Sponsor
- Rambam Health Care Campus
Muscular Dystrophy, Duchenne
A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)
Part 1 (dose escalation) will evaluate the safety and tolerability of 2 doses (100 milligrams/kilogram [mg/kg] and 200 mg/kg) of eteplirsen in approximately 10 participants with DMD; Part 2 (dose finding and dose comparison) will…
- Trial name
- MIS51ON (NCT03992430)
- Testing
- Eteplirsen
- Where
- 59 sites in United States, Colombia and 24 more
- Volunteers
- 160 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2
The proposed clinical trial study of rAAVrh74.MCK.GALGT2 for duchenne muscular dystrophy (DMD) patients that will involve direct intramuscular injection to the extensor digitorum brevis muscle (EDB).
- Trial name
- NCT02704325
- Testing
- rAAVrh74.MCK.GALGT2
- Where
- United States
- Sponsor
- Kevin Flanigan
Duchenne Muscular Dystrophy
Intermittent vs Alternate Daily Regimen of Prednisolone in Ambulatory Boys With Duchenne Muscular Dystrophy
The alternate-day regimen is hypothesized to reduce hypothalamic-pituitary-adrenal (HPA) axis suppression and decrease steroid-related toxicity.
- Trial name
- NCT07787689
- Testing
- Alternate-day prednisolone, Intermittent prednisolone
- Where
- Pakistan
- Volunteers
- 40 people
- Sponsor
- Muhammad Aamir Latif
SMA II, Cerebral Palsy
Paracetamol Study in Patients With Low Muscle Mass
To investigate the safety and toxicity related to paracetamol treatment in children and adults with respectively SMA and CP.
- Trial name
- NCT03648658
- Testing
- Paracetamol 120Mg/5mL Oral Suspension
- Where
- Denmark
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Mette Cathrine Oerngreen
Spinal Muscular Atrophy
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)
The primary objective of this study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally to participants with later-onset Spinal Muscular Atrophy (SMA).
- Trial name
- CHERISH (NCT02292537)
- Testing
- Nusinersen
- Where
- 24 sites in United States, Canada and 8 more
- Volunteers
- 126 people
- Sponsor
- Biogen
Muscular Dystrophies
Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
The purpose of this study is to explore long-term safety, tolerability and efficacy of GSK2402968 in DMD subjects who previously participated in either DMD114117 or DMD114044.
- Trial name
- NCT01480245
- Testing
- GSK2402968
- Where
- 59 sites in Argentina, Australia and 22 more
- Volunteers
- 233 people
- Sponsor
- GlaxoSmithKline
Vaginitis, Atrophic Vaginitis
a Flexible Wound Dressing for the Management of Genital Skin Conditions
The objective of the study is to determine the efficacy of 7-0940 in the management of genital skin conditions in female patients
- Conditions
- Vaginitis, Atrophic Vaginitis, Lichen Planus, Lichen Sclerosus
- Trial name
- NCT05396261
- Testing
- Film forming silicone gel (7-0940)
- Where
- Australia
- Volunteers
- 54 people
- Sponsor
- Stratpharma AG
Atrophy of Edentulous Maxillary Alveolar Ridge
Ascending Ramus Versus Chin Cortical Plates for Reconstruction of Atrophic Maxilla
Assessment of ascending ramus cortical plate for reconstruction of atrophic maxillary ridges versus chin cortical plates (Randomized clinical trial)
- Trial name
- NCT03629860
- Testing
- Atrophic maxillary alveolar ridge augmentation
- Where
- Egypt
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Cairo University
Fructose Metabolism, Inborn Errors, Glycogen Storage Disease
Biomarker for Glycogen Storage Diseases (BioGlycogen)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Glycogen Storage Diseases from plasma. Testing for clinical robustness, specificity and long-term stability of the biomarker.
- Conditions
- Fructose Metabolism, Inborn Errors, Glycogen Storage Disease, Glycogen Storage Disease Type I, Glycogen Storage Disease Type II
- Trial name
- BioGlycogen (NCT02385162)
- Where
- 4 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
Duchenne Muscular Dystrophy, Muscular Dystrophies
Non-invasive Imaging of Muscle Structure in Duchenne Muscular Dystrophy Using Multispectral Optoacoustic Tomography
This pilot study aims to assess subcellular muscle structure in patients with Duchenne X-linked progressive Duchenne muscular dystrophy (DMD) in comparison to healthy volunteers using multispectral optoacoustic tomography (MSOT).
- Conditions
- Duchenne Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy, Duchenne
- Trial name
- MSOT_DMD (NCT03490214)
- Testing
- Multispectral Optoacoustic Tomography
- Where
- Germany
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- University of Erlangen-Nürnberg Medical School
Spinal Muscular Atrophy
Development of a Multidisciplinary Network for Clinical and Laboratory Research for SMA
The goal of this observational study is to to establish profiles of clinical progression in patients affected by the different types of SMA (type I, II and III) treated with the currently approved drugs using a structured battery…
- Trial name
- NCT05866939
- Volunteers
- 400 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Congenital Muscular Dystrophy Due to Lamin A/C Mutation
HBCMD01- Expanded Access for the Treatment of Congenital Muscular Dystrophy.
This individual patient expanded access IND is requested for a patient diagnosed with LMNA-related congenital muscular dystrophy (L-CMD).
- Trial name
- NCT05154851
- Testing
- HB-adMSCs
- Where
- United States
- Sponsor
- Hope Biosciences Research Foundation
Duchenne Muscular Dystrophy
A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
The primary objective of this study is to evaluate the safety of a 0.9 milligrams per kilogram (mg/kg) and 0.45 mg/kg daily dose of deflazacort with a comparable natural history control group after 52 weeks of treatment in males…
- Trial name
- PTCEMF (NCT03642145)
- Testing
- Deflazacort
- Sponsor
- PTC Therapeutics
Muscular Dystrophy, Duchenne, Muscular Dystrophies
An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
The primary objective of this study is to obtain long term safety data of ataluren in male participants with nonsense mutation dystrophinopathy (who participated and completed a previous Phase 3 study of ataluren…
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT02090959
- Testing
- Ataluren
- Where
- 58 sites in United States, Australia and 17 more
- Volunteers
- 219 people
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy (DMD)
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45
This is a study of the investigational medicine ENTR-601-45 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition.
- Trial name
- ELEVATE-45 (NCT07038824)
- Testing
- ENTR-601-45
- Where
- 15 sites in Belgium, Italy and 3 more
- Volunteers
- 24 people
- Sponsor
- Entrada Therapeutics, Inc.
Gingival Recession, Generalized
Comparison Between Connective Graft and Xenogeneic Matrix, in Patients With Multiple Gingival Recessions
Subepithelial connective tissue graft (SCTG) is considered the gold standard for root coverage treatment. However, it is associated with postoperative morbidity, limited amount of donor tissue, and longer surgical time.
- Trial name
- NCT03207984
- Testing
- Root coverage surgery
- Where
- Brazil
- Volunteers
- 37 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Spinal Muscular Atrophy (SMA)
A Study of the Safety and Preliminary Efficacy of NKG001 in Pediatric Patients With Type 1 or Type 2 Spinal Muscular Atrophy
This study is a single-center, open-label, single-arm, non-randomized, single-dose, dose-escalation investigator-initiated trial (IIT) designed to evaluate the safety, tolerability, and preliminary efficacy of NKG001 Injection…
- Trial name
- NCT07617779
- Testing
- NKG001
- Where
- China
- Volunteers
- 21 people
- Sponsor
- Nikegen Pharmaceutical (Hangzhou) Company Limited
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