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Found 106 clinical trials
Oral Hydroxychloroquine (HCQ) for Retinitis Pigmentosa Caused by P23H- Rhodopsin (RHO)

is that treatment with HCQ is safe and tolerable in patients with autosomal dominant retinitis pigmentosa (adRP) caused by P23H-RHO, and may arrest progression of retinal degeneration by altering the

genetic analysis
pupil dilation
retinopathy
genetic testing
corrected visual acuity
  • 45 views
  • 03 Mar, 2021
  • 1 location
A Randomized Open-label LOSARTAN Therapy on the Progression of Aortic Root Dilation in Patients With Marfan Syndrome

To assess the efficacy of angiotensin II receptor blocker, Losartan, to prevent progressive dilation of aortic root in patients with Marfan syndrome.

  • 0 views
  • 07 Nov, 2020
Retraining Reaching in Cerebellar Ataxia

The purpose of this study is to test for benefits of reinforcement based training paradigm versus standard practice over weeks for improving reaching movements in people with ataxia.

stroke
ataxia
  • 1 views
  • 25 Jan, 2021
  • 1 location
Tolerability and Efficacy of L-Serine in Patients With GRIN-related Encephalopathy

GRIN-related disorders encompass a new group of Inborn Errors of Metabolism according to the recent nosology published by Ferreira et al (Genet Med, 2019). These rare conditions represent a subtype of paediatric encephalopathies leading to intellectual disability, hypotonia, communication deficits and motor impairment (Orphanet entries: 178469, 289266, 101685, for GRIN1, …

  • 0 views
  • 27 Jan, 2021
  • 1 location
Multi-modal Neuroimaging in Alzheimer's Disease

Alzheimer's disease (AD) is a major public health problem due to its socio-economic weight. An early diagnosis of AD is urgently needed as it would constitute a determinant breakthrough from a social, financial and research standpoints. Therefore, the investigators need predictive markers of AD, and neuroimaging is a particularly promising …

early diagnosis
mini-mental state examination
apolipoprotein e
  • 7 views
  • 08 Nov, 2020
  • 1 location
A Trial of Bardoxolone Methyl in Patients With ADPKD - FALCON

This international, multi-center, randomized, double-blind, placebo-controlled Phase 3 trial will study the safety, tolerability, and efficacy of bardoxolone methyl in qualified patients with ADPKD. Approximately 300 patients will be enrolled.

systolic blood pressure
cyst
diastolic blood pressure
  • 270 views
  • 03 Feb, 2021
  • 115 locations
A Study to Evaluate the Effects of GLPG2737 in Participants With Autosomal Dominant Polycystic Kidney Disease (ADPKD)

This exploratory, randomized, double-blind, placebo-controlled, multicenter, proof of concept, Phase 2a study will evaluate the efficacy, safety, tolerability, and pharmacokinetics (PK) of orally administered GLPG2737, for a treatment period of 52 weeks, in participants with rapidly progressing ADPKD.

  • 0 views
  • 19 Feb, 2021
  • 11 locations
An Extended Access Program for Bardoxolone Methyl in Patients With CKD (EAGLE)

This extended access study will assess the long-term safety and tolerability of bardoxolone methyl in qualified patients with chronic kidney disease (CKD) who previously participated in one of the qualifying clinical studies with bardoxolone methyl. Patients will remain in the study until bardoxolone methyl is available through commercial channels or …

nephropathy
chronic kidney disease
renal disease
renal failure
  • 133 views
  • 26 Jan, 2021
  • 37 locations
A Study of Guselkumab in Participants With Familial Adenomatous Polyposis

The purpose of this study is to determine the effect of treatment with guselkumab in participants with familial adenomatous polyposis (FAP) on rectal/pouch polyp burden.

colectomy
post colectomy
  • 184 views
  • 04 Mar, 2021
  • 37 locations
Ketogenic Dietary Interventions in Autosomal Dominant Polycystic Kidney Disease (ADPKD)

A mild reduction in food intake significantly inhibits renal cyst growth in mouse models of ADPKD. The underlying mechanism was unknown at the time. Recently published data show that the beneficial effect is not due to caloric restriction per se but due to the induction of the state of ketosis. …

  • 0 views
  • 02 Feb, 2021
  • 2 locations