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Found 6,914 clinical trials

A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies

In this study, researchers will know more about the effects of nusinersen, also known as Spinraza®, in pregnant participants with spinal muscular atrophy, also known as SMA. This is a drug available for doctors to prescribe for people with SMA. Due to the current treatment options that exist, people with …

18-54 years Female Phase N/A
C Claire Boutoleau-Bretonniere, MD

Premorbid Personality Profile of Patients With Cognitive and Behavioral Disorders

Damages in frontal area present in neurodegenerative disease (frontotemporal degeneration, frontal variant of Alzheimer disease) and in psychiatric disease (bipolar disorder) can affect behavior and cognition including social cognition. Symptoms vary both quantitatively and qualitatively from disease to another and from person to person. It cannot be completely excluded that …

18 years and older All Phase N/A
F For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)

Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C

Background:Niemann-Pick type C (NPC) disease is a rare, progressive neurodegenerative disease that affects mainly the brain, liver, and spleen but also other parts of the body. There is no cure for NPC, and symptoms only get worse over time. Symptoms can include seizures, difficulty moving or talking, or dementia. But …

3 years and older All Phase N/A
J Jacqueline Montes, PT EdD

Gait and Bone Health in SMA

The objective of this study is to understand how spatiotemporal and kinetic gait parameters are associated with bone health in pwSMA who are receiving DMT. We hypothesize that gait parameters are associated with BMD and can determine fracture risk in pwSMA. This is an observational study that involves one in-clinic …

8-50 years All Phase N/A

A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies

In this study, researchers will know more about the effects of nusinersen, also known as Spinraza®, in pregnant participants with spinal muscular atrophy, also known as SMA. This is a drug available for doctors to prescribe for people with SMA. Due to the current treatment options that exist, people with …

18-54 years Female Phase N/A
C Claire Boutoleau-Bretonniere, MD

Premorbid Personality Profile of Patients With Cognitive and Behavioral Disorders

Damages in frontal area present in neurodegenerative disease (frontotemporal degeneration, frontal variant of Alzheimer disease) and in psychiatric disease (bipolar disorder) can affect behavior and cognition including social cognition. Symptoms vary both quantitatively and qualitatively from disease to another and from person to person. It cannot be completely excluded that …

18 years and older All Phase N/A
F For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)

Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C

Background:Niemann-Pick type C (NPC) disease is a rare, progressive neurodegenerative disease that affects mainly the brain, liver, and spleen but also other parts of the body. There is no cure for NPC, and symptoms only get worse over time. Symptoms can include seizures, difficulty moving or talking, or dementia. But …

3 years and older All Phase N/A
J Jacqueline Montes, PT EdD

Gait and Bone Health in SMA

The objective of this study is to understand how spatiotemporal and kinetic gait parameters are associated with bone health in pwSMA who are receiving DMT. We hypothesize that gait parameters are associated with BMD and can determine fracture risk in pwSMA. This is an observational study that involves one in-clinic …

8-50 years All Phase N/A
K Kotsakis

Hellenic Evaluation of the Long-Term Safety and Efficacy of NalIriFOx as a 1st-line Therapy in Metastatic Pancreatic Ductal Adenocarcinoma (mPDAC)

This is multicenter, prospective, open label, non-interventional study that will collect real-world data (RWD) on the clinical efficacy and safety of NalIriFOx as a first-line therapy in metastatic pancreatic ductal adenocarcinoma (mPDAC).

18 years and older All Phase N/A
P Per Hedenström, MD

Safety and Efficacy of EUS-RFA in Pancreatic Insulinoma

Transcutaneous and EUS-guided radiofrequency ablation are both modern modalities to apply in the managemnet of various malignancies.In the current study the investigators aim to study the technical feasibility, patient safety and clinical efficacy of EUS-RFA for the treatment of pancreatic insulinoma.

15-80 years All Phase N/A

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