Clinical trials: Atrophy
Spinal Muscular Atrophy (SMA)
Virtual Reality for the Mitigation of Anxiety During Intrathecal Administration in Participants With Spinal Muscular Atrophy
The primary objective of the study is to evaluate anxiety level during intrathecal administration (IT) under standard of care (SOC) and virtual reality (VR) conditions using a reliable self-rating scale.
- Trial name
- REALITY (NCT05354414)
- Testing
- Virtual reality, Standard of Care
- Where
- 12 sites in France
- Volunteers
- 63 people
- Sponsor
- Biogen
Congenital Myotonic Dystrophy, CDM
DMCRN-02-001: Assessing Pediatric Endpoints in DM1
The overall goal of the study is to establish valid clinical endpoint assessments for children with congenital myotonic dystrophy type 1 and develop biomarkers for the condition.
- Trial name
- ASPIRE-DM1 (NCT05224778)
- Where
- 5 sites in United States, Italy
- Volunteers
- 50 people
- Sponsor
- Virginia Commonwealth University
Dry Age-related Macular Degeneration, Geographic Atrophy
Study of the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ACU-4429 in Subjects With Geographic Atrophy
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of the investigational drug ACU-4429 in subjects with geographic atrophy.
- Trial name
- NCT01002950
- Testing
- ACU-4429
- Where
- 12 sites in United States
- Volunteers
- 72 people
- Sponsor
- Kubota Vision Inc.
Duchenne Muscular Dystrophy (DMD)
The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy
The aim of the BABY DUCHENNE study is to evaluate the natural history and characterize the early clinical outcomes in very young children (0-3 years) with Duchenne muscular dystrophy (DMD) identified by newborn screening programs.
- Trial name
- BABY DUCHENNE (NCT07092540)
- Where
- 4 sites in United States
- Volunteers
- 105 people
- Sponsor
- University of Rochester
Vulvar Atrophy, Breast Cancer
Vaginal Prasterone In Vaginal Atrophy In Breast Cancer Survivors
VAGINAL PRASTERONE in the treatment of VAGINAL ATROPHY IN PATIENTS WITH BREAST CANCER TREATMENT WITH AROMATASE INHIBITORS (VIBRA STUDY)
- Conditions
- Vulvar Atrophy, Breast Cancer, Genitourinary Syndrome of Menopause
- Trial name
- VIBRA (NCT04705883)
- Testing
- Prasterone (DHEA), Micronized
- Where
- Spain
- Volunteers
- 10 people
- Sponsor
- Hospital Clinic of Barcelona
Spinal Muscular Atrophy, Neuromuscular Disease
Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA)
This study is being conducted to test whether exercise can be effectively used as an intervention to treat Spinal Muscular Atrophy (SMA).
- Trial name
- NCT01166022
- Testing
- Exercise
- Where
- United States
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Columbia University
Muscular Dystrophy, Duchenne
Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy
The MoveDMD study is a 3-part, Phase 1/2, multi-site study to evaluate the safety, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of edasalonexent (also known as CAT-1004) in pediatric patients with a genetically…
- Trial name
- MoveDMD® (NCT02439216)
- Testing
- Edasalonexent
- Where
- 5 sites in United States
- Volunteers
- 31 people
- Sponsor
- Catabasis Pharmaceuticals
SMA
Study to Evaluate the Efficacy of Riluzole in Children and Young Adults With Spinal Muscular Atrophy (SMA)
This is a multicentric, randomized, double-blind study versus placebo, with two parallel groups treated to evaluate the efficacy and the tolerance of Riluzole in children and young adults (6 to 20 years of age) with SMA.
- Trial name
- ASIRI (NCT00774423)
- Testing
- Riluzole
- Where
- France
- Volunteers
- 141 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Alveolar Bone Loss
Comparison of Coronally Advanced Lingual Flap to Modified MPI for Flap Advancement in Partially Edentulous Patients
Comparison of cronally advanced lingual flap to modified periosteal releasing incision (MPRI)
- Trial name
- NCT03466840
- Testing
- The coronally advanced lingual flap, Modified periosteal releasing Incision
- Where
- Egypt
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- Cairo University
Spinal Muscular Atrophy
Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen
Risdiplam Exchange (RISE) is a study of spinal muscular atrophy (SMA) patients who crossover to 36 months of open-label risdiplam monotherapy following a comparable period of nusinersen treatment.
- Trial name
- RISE (NCT05522361)
- Testing
- Risdiplam
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Clinic for Special Children
Myocardial Fibrosis, Muscular Dystrophies
Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy - ACE Inhibitor Therapy Trial
This trial intends to evaluate myocardial Fibrosis progression in Duchenne and Becker Muscular Dystrophy, as well the influence of ACE inhibitors in fibrosis progression.
- Trial name
- NCT02432885
- Testing
- Enalapril
- Volunteers
- 76 people
- Sponsor
- InCor Heart Institute
Duchenne Muscular Dystrophy
Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
The study will evaluate the safety and efficacy of gene therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study with two thirds of participants assigned to gene therapy.
- Trial name
- NCT04281485
- Testing
- PF-06939926
- Where
- 53 sites in United States, Australia and 13 more
- Volunteers
- 114 people
- Sponsor
- Pfizer
Duchenne Muscular Dystrophy
Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons
The purpose of this study is to determine the safety of giving intravenous (IV) gentamicin to boys with Duchenne muscular dystrophy who have stop codon mutations.
- Trial name
- NCT00451074
- Testing
- Gentamicin infusions twice a week for six months
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- Nationwide Children's Hospital
Muscular Atrophy, Spinal
A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients
The objectives of this trial are: to establish a safety profile for use of Hydroxyurea in children with Type I Spinal Muscular Atrophy; to identify reliable outcome measures for HU treatment in Type I SMA; and to detect the…
- Trial name
- NCT00568698
- Testing
- Hydroxyurea
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Stanford University
Congenital Myotonic Dystrophy, Childhood Myotonic Dystrophy
Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension
This is a natural history study to improve the types of assessments and biological samples that will be used in clinical drug trials in both congenital myotonic dystrophy and childhood myotonic dystrophy.
- Conditions
- Congenital Myotonic Dystrophy, Childhood Myotonic Dystrophy, Myotonic Dystrophy
- Trial name
- TREAT-EXT (NCT06747884)
- Where
- 4 sites in United States, Brazil
- Volunteers
- 200 people
- Sponsor
- Virginia Commonwealth University
Muscular Dystrophy, Duchenne
A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to evaluate the safety and efficacy of exogenous gene transfer in DMD participants by measuring biological and clinical endpoints in three parts: two 48-week randomized, double-blinded…
- Trial name
- NCT03769116
- Testing
- delandistrogene moxeparvovec
- Where
- 2 sites in United States
- Volunteers
- 41 people
- Sponsor
- Sarepta Therapeutics, Inc.
Increase Muscle Strength in Patients With Muscular Dystrophy
Efficacy Safety of Granulocyte Colony-stimulating Factor Treatment Children and Adolescents With Muscular Dystrophy
Importance: Currently the gold standard treatment for ambulant patients is corticosteroids. Granulocyte colony-stimulating factor (G-CSF) has been reported to exert the proliferation of satellite cells, the regulation of myoblast…
- Trial name
- NCT02814110
- Testing
- Granulocyte colony-stimulating factor (Filgrastim)
- Where
- Poland
- Volunteers
- 27 people
- Sponsor
- Medical University of Bialystok
Spinal Muscular Atrophy
An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701)
The primary objective of this study is to examine the safety and tolerability of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA) who previously participated in ISIS…
- Trial name
- NCT01780246
- Testing
- nusinersen
- Where
- 4 sites in United States
- Volunteers
- 18 people
- Sponsor
- Biogen
Vaginal Atrophy
A Clinical Trial to Assess the Safety of 0.005 % Estriol Vaginal Gel in Hormone Receptor-Positive Postmenopausal Women With Early Stage Breast Cancer in Treatment With Aromatase Inhibitor in the Adjuvant Setting
This is a phase II, prospective, randomized, double-blind, placebo-controlled, international (Spain and Sweden) and multicentre study to explore the safety of 0.005% estriol vaginal gel in women with early stage breast cancer in…
- Trial name
- BLISSAFE (NCT02413008)
- Testing
- estriol
- Where
- 6 sites in Spain, Sweden
- Volunteers
- 61 people
- Sponsor
- ITF Research Pharma, S.L.U.
Multiple System Atrophy
Open-Label Access to ATH434 for Patients Who Completed Study ATH434-201 in France
This multicenter open-label extension study conducted in France is designed to provide ATH434 orally BID to eligible patients who completed the Phase 2 study ATH434-201, and who may benefit from this treatment according to the…
- Trial name
- NCT07729852
- Testing
- ATH434
- Where
- 4 sites in France
- Volunteers
- 7 people
- Sponsor
- Alterity Therapeutics
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