Clinical trials: Multiple System Atrophy
Spinal Muscular Atrophy
Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2
To evaluate the safety and efficacy of intravenous onasemnogene abeparvovec-xioi in pre-symptomatic patients with SMA and 2 or 3 copies SMN2
- Trial name
- SPR1NT (NCT03505099)
- Testing
- onasemnogene abeparvovec-xioi
- Where
- 16 sites in United States, Australia and 4 more
- Volunteers
- 30 people
- Sponsor
- Novartis Gene Therapies
Neurogenic Orthostatic Hypotension, Multiple System Atrophy (MSA) With Orthostatic Hypotension
TD-9855 Phase 2 in Neurogenic Orthostatic Hypotension (nOH)
This multiple-center, 3-part, single-blind dose escalation (Part A), randomized, double-blind (Part B), and open-label multiple dose extension (Part C) study will be conducted in male and female subjects with neurogenic…
- Conditions
- Neurogenic Orthostatic Hypotension, Multiple System Atrophy (MSA) With Orthostatic Hypotension, Pure Autonomic Failure, Parkinson Disease
- Trial name
- NCT02705755
- Testing
- TD-9855
- Where
- 6 sites in United States
- Volunteers
- 34 people
- Sponsor
- Theravance Biopharma
Autoimmune Diseases of the Nervous System, Multiple Sclerosis
Study of Tcelna (Imilecleucel-T) in Secondary Progressive Multiple Sclerosis
The purpose of this study is to determine whether Tcelna (imilecleucel-T, autologous T-Cell Immunotherapy) is effective in the treatment of secondary progressive multiple sclerosis (SPMS).
- Conditions
- Autoimmune Diseases of the Nervous System, Multiple Sclerosis, Secondary Progressive Multiple Sclerosis, Disease Progression
- Trial name
- Abili-T (NCT01684761)
- Testing
- Tcelna
- Where
- 36 sites in United States, Canada
- Volunteers
- 183 people
- Sponsor
- Opexa Therapeutics, Inc.
SMA II
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 2 (SMA 2) patients.
- Trial name
- NCT05901987
- Testing
- GC101
- Where
- 5 sites in China
- Volunteers
- 33 people
- Sponsor
- GeneCradle Inc
Oculopharyngeal Muscular Dystrophy
Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients
The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.
- Trial name
- HOPEMD (NCT02015481)
- Testing
- Cabaletta
- Where
- 4 sites in United States, Canada and 1 more
- Volunteers
- 25 people
- Sponsor
- Bioblast Pharma Ltd.
Muscular Dystrophies
A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route of Administration
The purpose of the extension phase of this study is to determine whether Drisapersen is effective in the treatment of boys with Duchenne muscular dystrophy resulting from a mutation thought to be corrected by exon 51 skipping.
- Trial name
- NCT01910649
- Testing
- Drisapersen
- Volunteers
- 12 people
- Sponsor
- BioMarin Pharmaceutical
Neurogenic Orthostatic Hypotension, Non-Diabetic Autonomic Neuropathy
Open-Label Clinical Study of Droxidopa in Patients With Neurogenic Orthostatic Hypotension (NOH)
The purpose of this study is to assess the durability of effect of Droxidopa in treating symptoms of neurogenic orthostatic hypotension in patients with Primary Autonomic Failure (Pure Autonomic Failure, Multiple System Atrophy…
- Conditions
- Neurogenic Orthostatic Hypotension, Non-Diabetic Autonomic Neuropathy, Multiple System Atrophy, Dopamine Beta Hydroxylase Deficiency
- Trial name
- NOH303 (NCT00738062)
- Testing
- Droxidopa
- Where
- 54 sites in United States, Australia and 2 more
- Volunteers
- 103 people
- Sponsor
- Chelsea Therapeutics
Parkinson's Disease, Multiple System Atrophy, Parkinson Variant
AB-1005 Long-Term Follow-up Study
This is an observational, long term, follow-up study for participants with Parkinson's disease (PD) or Multiple System Atrophy-Parkinsonian subtype (MSA-P) who participated in prior interventional studies with AB-1005.
- Trial name
- NCT07081841
- Testing
- Observation
- Where
- 2 sites in United States
- Volunteers
- 132 people
- Sponsor
- AskBio Inc
Prostate Cancer
A Trial to Learn How Safe AZD9750 is and How Well it Works in People With Metastatic Prostate Cancer When Given With or Without Other Anticancer Drugs
ANDROMEDA is a first-in-human, Phase I/II, open-label, multicenter study of AZD9750 in participants with metastatic prostate cancer.
- Trial name
- ANDROMEDA (NCT07336446)
- Testing
- AZD9750, AZD5305
- Where
- 5 sites in United States, Australia and 2 more
- Volunteers
- 6 people
- Sponsor
- AstraZeneca
Multiple System Atrophy
A Trial of Amlenetug (Lu AF82422) in Participants With Multiple System Atrophy (MSA)
The main goal of this trial is to evaluate the efficacy and safety of amlenetug for the treatment of participants with Multiple System Atrophy (MSA).
- Trial name
- MASCOT (NCT06706622)
- Testing
- Amlenetug
- Where
- 74 sites in United States, Australia and 8 more
- Volunteers
- 401 people
- Sponsor
- H. Lundbeck A/S
the Treatment of Multiple System Atrophy Parkinsonian Variant (MSA-P)
The Safety, Tolerability, and Efficacy of Dopaminergic Neural Progenitor Cells (XS411CN Cell Injection) for the Treatment of Multiple System Atrophy Parkinsonian Variant (MSA-P).
A Phase I Study to Assess the Safety, Tolerability and Efficacy of dopaminergic neural progenitor cells (XS411CN cell injection) for the treatment of multiple system atrophy Parkinsonian variant (MSA-P).
- Trial name
- NCT07617012
- Testing
- XS411CN Cell Injection
- Volunteers
- 3 people, healthy volunteers welcome
- Sponsor
- XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd.
Duchenne Muscular Dystrophy
A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)
This Phase II study is an open-label, multiple dose study to evaluate the safety, tolerability, PK, PD, clinical efficacy, behavior and neuropsychology, and physical functioning vamorolone over a treatment period of 12 weeks in…
- Trial name
- NCT05185622
- Testing
- Vamorolone
- Where
- 5 sites in Canada
- Volunteers
- 54 people
- Sponsor
- Santhera Pharmaceuticals
Duchenne Muscular Dystrophy (DMD)
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy…
- Trial name
- DELIVER (NCT05524883)
- Testing
- DYNE-251
- Where
- 30 sites in United States, Australia and 7 more
- Volunteers
- 86 people
- Sponsor
- Dyne Therapeutics
Spinal Muscular Atrophy
A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)
The primary objective is to examine the clinical efficacy of multiple doses of nusinersen (ISIS 396443) administered intrathecally to participants with Infantile-Onset Spinal Muscular Atrophy (SMA).
- Trial name
- NCT01839656
- Testing
- nusinersen
- Where
- 4 sites in United States, Canada
- Volunteers
- 21 people
- Sponsor
- Biogen
Multiple System Atrophy (MSA)
Expanded Access Protocol of Verdiperstat in Patients With Multiple System Atrophy (MSA)
The purpose of this expanded access program is to provide access to the investigational drug verdiperstat in patients with Multiple System Atrophy (MSA).
- Trial name
- NCT05086094
- Testing
- BHV3241, verdiperstat
- Where
- United States
- Sponsor
- Biohaven Pharmaceuticals, Inc.
Duchenne Muscular Dystrophy
Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy
The main purpose of this study is to test the safety and tolerability of different, increasing doses of an experimental medication called HT-100 in boys and young men with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT01847573
- Testing
- HT-100
- Where
- 5 sites in United States
- Volunteers
- 17 people
- Sponsor
- Processa Pharmaceuticals
Symptomatic Neurogenic Orthostatic Hypotension
Sustained Effect of Droxidopa in Symptomatic Neurogenic Orthostatic Hypotension
To evaluate the time to treatment intervention in patients with Parkinson's Disease (PD), Multiple System Atrophy (MSA), Pure Autonomic Failure (PAF), Non-Diabetic Autonomic Neuropathy (NDAN) or Dopamine Beta Hydroxylase (DBH)…
- Trial name
- RESTORE (NCT02586623)
- Testing
- Droxidopa capsules
- Where
- 168 sites in United States
- Volunteers
- 453 people
- Sponsor
- H. Lundbeck A/S
Myotonic Dystrophy Type 1 (DM1)
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1
The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1).
- Trial name
- ACHIEVE (NCT05481879)
- Testing
- DYNE-101
- Where
- 20 sites in United States, Australia and 6 more
- Volunteers
- 127 people
- Sponsor
- Dyne Therapeutics
Sarcopenia in Elderly
A Study of PLH-2301 in Subjects With Sarcopenia
This is a Phase 2, randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of PLH-2301 in subjects with sarcopenia.
- Trial name
- PLH-2301_P2 (NCT07390552)
- Testing
- PLH-2301
- Where
- 5 sites in South Korea
- Volunteers
- 168 people
- Sponsor
- Pluto Inc.
Symptomatic Neurogenic Orthostatic Hypotension
Phase 3 Open-Label Extension Study of TD-9855 for Treating Symptomatic nOH in Subjects With Primary Autonomic Failure
A Phase 3, multi-center, open-label study to evaluate the safety and tolerability of ampreloxetine in subjects with primary autonomic failures (MSA, PD, and PAF) and symptomatic nOH over 182 weeks.
- Trial name
- OAK (NCT04095793)
- Testing
- ampreloxetine
- Where
- 55 sites in United States, Australia and 16 more
- Volunteers
- 110 people
- Sponsor
- Theravance Biopharma