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Found 6,914 clinical trials

Measuring Impact of Computer Gaming on Arm Use in Rett Syndrome

Using a tele-research approach, we will recruit, enroll, guide and support carers and participants to engage in computer based activities (modified virtual reality) with the primary outcome of reducing stereotypies and increasing independent arm and hand use and secondary outcome of improving quality of living. Because of our virtual approach, …

4-60 years All Phase N/A
J Jingqi Zhang, MSc

Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome

Rett syndrome (RTT) is a serious neurodevelopmental disorder that has a significant impact on patients and their families. Patients suffer from severe social dysfunction and poor quality of life, and there is currently no effective treatment available. The MECP2 functional loss mutation is the clear pathogenic factor. In recent years, …

4-10 years Female Phase 0
J Jennifer Thompson, MS

To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome

Primary Objective • To investigate the efficacy of treatment with oral Bionetide versus placebo in girls and women with Rett syndromeKey Secondary Objective• To investigate the efficacy of treatment with oral Bionetide versus placebo on ability to communicate in girls and women with Rett syndrome

5-20 years Female Phase 3
G Giuliana Guggino, MD

Wheat-free Diet in the Treatment of Sjogren's Syndrome

Recent data show that some foods can increase intestinal mucosa permeability and immune activation of subjects with gastrointestinal (GI) symptoms. Wheat seems the most frequent food which activates this inflammatory response and can cause both GI and extra-intestinal symptoms. Patients suffering from wheat-related troubles, in absence of celiac disease diagnosis, …

18-65 years All Phase N/A
P Pasquale Mansueto, MD

Tolerability of an Ancient Grain in Patients With Non-Celiac Wheat Sensitivity

Patients suffering from wheat-related troubles, in absence of celiac disease or wheat allergy diagnosis, can suffer from non-celiac wheat sensitivity (NCWS). This is characterized by both gastrointestinal (GI) and extra-intestinal symptoms, which improve with the elimination of wheat intake. To date no definitive explanation of pathogenetic mechanisms of NCWS has …

18-65 years All Phase N/A
S Sun Can

Clinical Study to Evaluate the Efficacy and Safety of FB1006 in the Treatment of ALS Patients

This is a randomized double-blind controlled exploratory clinical study to evaluate the efficacy and safety of FB1006 in the treatment of amyotrophic lateral sclerosis (ALS) patients.

18-80 years All Phase 4
C COMBRET Yann, PhD

The Importance of Positive Expiratory Pressure Associated With the In-exsufflator in ALS Patients

Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disorder that impairs motor neurons, with a life expectancy of 2 to 7 years after diagnosis. ALS manifests as 'spinal' when it primarily affects limbs, or 'bulbar' when it impairs speech and swallowing. The disease progressively weakens all skeletal muscles, causing respiratory …

18 years and older All Phase N/A
R Rachel Grace Kozik

RE104 Safety and Efficacy Study in Adjustment Disorder in Cancer and Other Medical Illnesses

The purpose of this study is to determine if treatment with a single dose of RE104 for Injection reduces depressive symptoms or depressive symptoms mixed with anxiety symptoms in participants with Adjustment Disorder due to cancer or other illnesses such as Amyotrophic Lateral Sclerosis (ALS), Multiple Sclerosis (MS), Parkinson's Disease …

18-80 years All Phase 2

Social Cognition in Patients With Amyotrophic Lateral Sclerosis

Amyotrophic Lateral Sclerosis, also known as Charcot disease, is a neurodegenerative disease evidenced by gradual paralysis of the muscles involved in voluntary motor function. The clinical hallmark of Amyotrophic Lateral Sclerosis is the combination of upper and lower motor neuron signs and symptoms. The most recent studies suggest that up …

18 years and older All Phase N/A
A Angel Qin

Gilteritinib for the Treatment of ALK NSCLC

This phase I trial is studying the safety, side effects, and best dose of gilteritinib in treating patients with stage IV ALK positive non-small cell lung cancer (NSCLC) who have progressed on other treatments. While there are many approved targeted drugs for ALK NSCLC, resistance to these drugs frequently occur. …

18 years and older All Phase 1

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