Clinical trials: Progressive Muscular Atrophy
Spinal and Bulbar Muscular Atrophy, Kennedy's Disease
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients
This is a phase 1/2a randomized, double-blind study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of study drug AJ201 in subjects with Spinal and Bulbar Muscular Atrophy (SBMA).
- Trial name
- NCT05517603
- Testing
- AJ201
- Where
- 6 sites in United States
- Volunteers
- 25 people
- Sponsor
- AnnJi Pharmaceutical Co., Ltd.
Metastatic Breast Cancer
Efficacy Evaluation of Enobosarm Monotherapy in Treatment of AR+/ER+/HER2- Metastatic Breast Cancer
To demonstrate the efficacy of enobosarmin the treatment of androgen receptor positive (AR+) and estrogen receptor positive (ER+) metastatic breast cancer (MBC) as measured by radiographic progression free survival (rPFS).
- Trial name
- ARTEST (NCT04869943)
- Testing
- Enobosarm, Exemestane
- Where
- 54 sites in United States, Poland and 2 more
- Volunteers
- 52 people
- Sponsor
- Veru Inc.
Breast Cancer, Vulvovaginal Signs and Symptoms
WISH, Feasibility of a Factorial Design
The primary aim of this study is to evaluate the feasibility of testing a multi-component intervention for sexual function using a factorial design.
- Conditions
- Breast Cancer, Vulvovaginal Signs and Symptoms, Sexual Desire Disorder, Body Image
- Trial name
- NCT07023822
- Testing
- Hyaluronic acid (HLA) Vaginal Moisturizer, Polycarbophil Vaginal Moisturizer, Hypnotic Relaxation
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- The University of Tennessee, Knoxville
Healthy Volunteer, Muscular Atrophy, Spinal
Ability of Muscle Imaging and Motor Function Measure (MFM) to Detect Changes in Disease Progression in Ambulant Spinal Muscular Atrophy Patients Compared to Healthy Volunteers.
This non-drug, single center, 24-week, longitudinal study in ambulant spinal muscular atrophy (SMA) patients and in age- and gender-matched healthy volunteers will assess the detection of disease progression by magnetic resonance…
- Trial name
- NCT02044029
- Where
- Switzerland
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Spinal Muscular Atrophy
A Trial of Hydroxyurea in Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is an autosomal recessive disorder in humans which results in the loss of motor neurons. It is caused by reduced levels of the survival motor neuron (SMN) protein as a result of loss or mutation of…
- Trial name
- NCT00485511
- Testing
- Hydroxyurea
- Where
- Taiwan
- Sponsor
- Kaohsiung Medical University Chung-Ho Memorial Hospital
Spinal Muscular Atrophy
Measuring Levels of SMN in Blood Samples of SMA Patients
Spinal muscular atrophy (SMA) is a disorder that affects the motor neurons. SMA is caused by a mutation in a part of the DNA called the survival motor neuron (SMN1) gene, which normally produces a protein called SMN.
- Trial name
- NCT00061607
- Where
- United States
- Volunteers
- 73 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Muscular Atrophy, Spinal
A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular Atrophy
The aim of this study was to evaluate treatment patterns, SMA-related complications and services, and all-cause medical encounters of patients with SMA receiving multiple DMTs in the real world.
- Trial name
- NCT07403214
- Where
- United States
- Volunteers
- 4,805 people
- Sponsor
- Novartis Pharmaceuticals
Muscular Diseases, Spinal Muscular Atrophy
New Imaging Biomarkers for Muscular Diseases - Multispectral Optoacoustic Imaging in Spinal Muscular Atrophy
This study aims to refine the capability of MSOT to characterise muscle tissue and to determine non-invasive, quantitative biomarkers for the disease assessment in patients with spinal muscular atrophy (SMA) using Multispectral…
- Trial name
- MSOT_SMA (NCT04115475)
- Testing
- Multispectral Optoacoustic Tomography (MSOT)
- Where
- Germany
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- University of Erlangen-Nürnberg Medical School
Spinal Muscular Atrophy
Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen
The Motor Function Measure (MFM), a reliable tool assessing motor function and its progression in most neuromuscular diseases, is widely used in France in many teams.
- Trial name
- NusiMFM (NCT04602195)
- Where
- 10 sites in France
- Volunteers
- 33 people
- Sponsor
- Hospices Civils de Lyon
Spinal Muscular Atrophy
Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy
The purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of NMD670 in the treatment of ambulatory adults with spinal muscular atrophy type 3
- Trial name
- SYNAPSE-SMA (NCT05794139)
- Testing
- NMD670
- Where
- 25 sites in United States, Belgium and 6 more
- Volunteers
- 52 people
- Sponsor
- NMD Pharma A/S
Cancer Cachexia, Omega 3
Effects of Dietary Supplementation With omega3-fatty Acids (Fish Oil) and Progressive Resistance Training on Skeletal Muscle Status in Gynaecological, Gastrointestinal and Urological Cancer Patients
Cancer cachexia is a common and prognostically relevant complication of advanced malignancies, characterized by systemic inflammation, increased catabolism, and reduced nutritional intake, leading to a progressive loss of…
- Conditions
- Cancer Cachexia, Omega 3, Resistance Training, Cancer
- Trial name
- SUPPORT (NCT07643558)
- Testing
- Omega 3 FA (Fish Oil), Training
- Where
- Germany
- Volunteers
- 288 people
- Sponsor
- University of Erlangen-Nürnberg Medical School
Spinal Muscular Atrophy
Mechanisms and Treatment of Exercise Intolerance and Persistent Fatigue in Spinal Muscular Atrophy
This study will focus on the pathophysiological underpinnings of reduced exercise capacity and fatigue in ambulatory patients with spinal muscular atrophy (SMA).
- Trial name
- NCT05518773
- Testing
- Observational
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- Columbia University
Type I Spinal Muscular Atrophy
Clinical Trial to Evaluate the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type I
The purpose of this study was to evaluate the safety and preliminary efficacy of a single intravenous injection of exg001-307 in patients with type I spinal muscular atrophy.
- Trial name
- NCT06576388
- Testing
- EXG001-307 injection
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
Spinal Muscular Atrophy, SMA
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects <2 years old with 5q autosomal recessive SMA who have delayed motor milestones…
- Conditions
- Spinal Muscular Atrophy, SMA, Spinal Muscular Atrophy Type 2, Spinal Muscular Atrophy Type 3
- Trial name
- OPAL (NCT07047144)
- Testing
- Apitegromab, Nusinersen, Risdiplam
- Where
- 23 sites in United States, Belgium and 5 more
- Volunteers
- 52 people
- Sponsor
- Scholar Rock, Inc.
Muscular Atrophy, Spinal
A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients
The objectives of this trial are: to establish a safety profile for use of Hydroxyurea in children with Types II and III Spinal Muscular Atrophy; to identify reliable outcome measures for HU treatment in Types II and III SMA; and…
- Trial name
- NCT00568802
- Testing
- Hydroxyurea
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Stanford University
Spinal Muscular Atrophy
Personalized Medicine for SMA: a Translational Project
Major breakthroughs in the treatment for Spinal muscular atrophy (SMA) have been recently achieved with various therapeutic approaches that increase full-length SMN protein levels.
- Trial name
- NCT05779956
- Where
- 2 sites in Italy
- Volunteers
- 90 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
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
SMA
EAP of Apitegromab for Patients With Spinal Muscular Atrophy
The purpose of this expanded access program (EAP) is to provide access to apitegromab for eligible patients with spinal muscular atrophy (SMA) prior to approval by the local regulatory agency .
- Trial name
- NCT06877689
- Testing
- Apitegromab
- Sponsor
- Scholar Rock, Inc.
Prostatic Neoplasm
A Phase 1a/1b Study to Evaluate the Safety of EZN-4176, in Adult Patients With Castration-Resistant Prostate Cancer
This study will evaluate an experimental drug called EZN-4176 to determine the anticancer effects when it is given to patients with an advanced form of prostate cancer called castration-resistant prostate cancer (CRPC).
- Trial name
- NCT01337518
- Testing
- EZN-4176
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 150 people
- Sponsor
- Enzon Pharmaceuticals, Inc.
Spinal Muscular Atrophy
A Review of the Management and Outcomes of Children With SMA in the West Midlands During 2017-2022
Spinal Muscular Atrophy (SMA) is a rare neuromuscular condition, characterised by loss of motor neurons as a result of a mutation in the survival motor neuron gene.
- Trial name
- NCT05994950
- Where
- 2 sites in United Kingdom
- Volunteers
- 30 people
- Sponsor
- Robert Jones and Agnes Hunt Orthopaedic and District NHS Trust