Clinical trials: Progressive Muscular Atrophy
Prostate Cancer
Genomic Guided Therapy With Dasatinib or Nilutamide in Metastatic Castration-Resistant Prostate Cancer
This is a phase II multi-center study to determine the clinical impact of using a patient-specific genomic expression signature of androgen receptor (AR) activity to determine therapy for patients with castration-resistant…
- Trial name
- ARS (NCT00918385)
- Testing
- Nilutamide, Dasatinib
- Where
- 3 sites in United States
- Volunteers
- 57 people
- Sponsor
- Duke University
Spinal Muscular Atrophy
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy
The primary objective of the study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally (IT) to participants with infantile-onset with infantile-onset spinal muscular atrophy (SMA).
- Trial name
- ENDEAR (NCT02193074)
- Testing
- nusinersen
- Where
- 31 sites in United States, Australia and 11 more
- Volunteers
- 122 people
- Sponsor
- Biogen
Muscular Atrophy, Spinal
Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy
The primary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA), as measured by change in Children's Hospital…
- Trial name
- DEVOTE (NCT04089566)
- Testing
- Nusinersen
- Where
- 45 sites in United States, Brazil and 16 more
- Volunteers
- 145 people
- Sponsor
- Biogen
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
Metastatic Castration-Resistant Prostate Cancer
Study to Evaluate Exicorilant (CORT125281) in Combination With Enzalutamide in Patients With Metastatic Castration-Resistant Prostate Cancer
This study consists of a dose escalation study with an expansion phase to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD), and preliminary efficacy of exicorilant (CORT125281) in combination…
- Trial name
- NCT03437941
- Testing
- Exicorilant, Enzalutamide
- Where
- 9 sites in United States, United Kingdom
- Volunteers
- 39 people
- Sponsor
- Corcept Therapeutics
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 Atrophy, Spinal
A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA)
The purpose of this open-label, single arm study is to further evaluate long-term tolerability, safety and efficacy outcomes of olesoxime in participants with Spinal Muscular Atrophy (SMA) who previously participated in one of…
- Trial name
- NCT02628743
- Testing
- Olesoxime
- Where
- 24 sites in Belgium, France and 5 more
- Volunteers
- 131 people
- Sponsor
- Hoffmann-La Roche
Spinal Muscular Atrophy
An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)
An open-label, multi-part, first-in-human study of oral branaplam in infants with Type 1 spinal muscular atrophy. The purpose of this study was to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD)…
- Trial name
- NCT02268552
- Testing
- branaplam
- Where
- 14 sites in Belgium, Bulgaria and 5 more
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
Type 2 Spinal Muscular Atrophy
Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)
This was a Phase III multi-center, single dose (1.2 x 10^14 vector genomes), randomized, sham controlled, double-blind study that investigates the efficacy, safety and tolerability of OAV101B in treatment naive, sitting and never…
- Trial name
- STEER (NCT05089656)
- Testing
- OAV101
- Where
- 42 sites in United States, Brazil and 11 more
- Volunteers
- 126 people
- Sponsor
- Novartis Pharmaceuticals
Muscular Atrophy, Spinal
A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
A global study of oral risdiplam in pre-symptomatic participants with spinal muscular atrophy (SMA).
- Trial name
- Rainbowfish (NCT03779334)
- Testing
- Risdiplam
- Where
- 7 sites in United States, Australia and 5 more
- Volunteers
- 26 people
- Sponsor
- Hoffmann-La Roche
Breast Cancer
Phase II Trial of SAHA & Tamoxifen for Patients With Breast Cancer
Phase II trial to explore the efficacy of vorinostat and tamoxifen combined.
- Trial name
- NCT00365599
- Testing
- suberoylanilide hydroxamic acid (SAHA, Vorinostat), tamoxifen citrate (Tamoxifen)
- Where
- 8 sites in United States
- Volunteers
- 43 people
- Sponsor
- H. Lee Moffitt Cancer Center and Research Institute
Spinal Muscular Atrophy
Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy
In this single-center trial, we will evaluate the effects of NaPB on presymptomatic Spinal Muscular Atrophy (SMA) type I (cohort 1)and presymptomatic SMA type II (cohort 2) infants.
- Trial name
- STOPSMA (NCT00528268)
- Testing
- Sodium phenylbutyrate
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- University of Utah
Muscular Atrophy, Spinal
A Study to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of a Single Oral Dose of Risdiplam Compared to Matched Healthy Participants With Normal Hepatic Function
This is a multi-center, open-label, non-randomized, parallel-group, 2-part study to evaluate the effect of hepatic impairment on the PK and safety and tolerability of a single oral dose of risdiplam compared to matched healthy…
- Trial name
- NCT03920865
- Testing
- Risdiplam
- Where
- 3 sites in United States
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III
Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III
The purpose of this study is to identify the maximum tolerated dosage of sodium phenylbutyrate in children with spinal muscular atrophy types II or III; and to determine if the drug has an effect on SMN mRNA and protein levels.
- Trial name
- NPTUNE01 (NCT00439569)
- Testing
- sodium phenylbutyrate
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Westat
Spinal Muscular Atrophy
A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy
This is a multi-center, exploratory, non-comparative, and open-label study to investigate the safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants with Spinal Muscular Atrophy (SMA)…
- Trial name
- Jewelfish (NCT03032172)
- Testing
- Risdiplam
- Where
- 24 sites in United States, Belgium and 7 more
- Volunteers
- 174 people
- Sponsor
- Hoffmann-La Roche
Small Cell Neuroendocrine Carcinoma, Prostate Cancer
Apalutamide Plus Cetrelimab in Patients With Treatment-Emergent Small Cell Neuroendocrine Prostate Cancer
Despite the low androgen receptor (AR) transcriptional activity of treatment-emergent small cell neuroendocrine prostate cancer, there is persistent AR expression observed in the majority of treatment-emergent small-cell…
- Conditions
- Small Cell Neuroendocrine Carcinoma, Prostate Cancer, Small Cell Carcinoma
- Trial name
- NCT04926181
- Testing
- Apalutamide, Cetrelimab
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Rahul Aggarwal
Spinal Muscular Atrophy
An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy
This study will test the safety, tolerability, and pharmacokinetics of escalating doses of nusinersen (ISIS 396443) administered into the spinal fluid either two or three times over the duration of the trial, in participants with…
- Trial name
- NCT01703988
- Testing
- Nusinersen
- Where
- 4 sites in United States
- Volunteers
- 34 people
- Sponsor
- Biogen
Spinal Muscular Atrophy
Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam
This was a Phase IIIb open-label, single arm, multi-center study to evaluate the safety, tolerability and efficacy of OAV101B in participants with SMA aged 2 to <18 years after the discontinuation of treatment with nusinersen or…
- Trial name
- STRENGTH (NCT05386680)
- Testing
- OAV101
- Where
- 13 sites in United States, Australia and 7 more
- Volunteers
- 27 people
- Sponsor
- Novartis Pharmaceuticals