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Syndrome clinical trials

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NCT ID: NCT03882918 Terminated - Angelman Syndrome Clinical Trials

An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome

ELARA
Start date: January 31, 2019
Phase: Phase 3
Study type: Interventional

This open-label study (OV101-18-002) will evaluate the long-term (52 weeks) safety of OV101 in subjects with AS and provide additional OV101 treatment to those subjects who completed Study OV101-15-001 (NCT02996305). Subjects with AS who completed the pharmacokinetic Study OV101-16-001 (NCT03109756) will also be permitted to participate, provided they meet all entry criteria.

NCT ID: NCT03878914 Terminated - Clinical trials for Nephrotic Syndrome in Children

Steroid Sensitive Nephrotic Syndrome in Children

Start date: August 6, 2019
Phase: Phase 4
Study type: Interventional

Idiopathic nephrotic syndrome (INS) is one of the most common glomerular pathologies in children and corticosteroid therapy is its most effective treatment. The total duration of treatment ranges anywhere from two to six months, generally about 3 months. The main objective of our study is to test the feasibility of a shorter total duration (two months) of corticosteroid therapy in patients who show a quicker treatment response to the initial treatment.

NCT ID: NCT03878524 Terminated - Anemia Clinical Trials

Serial Measurements of Molecular and Architectural Responses to Therapy (SMMART) PRIME Trial

Start date: April 1, 2020
Phase: Phase 1
Study type: Interventional

This phase Ib trial determines if samples from a patient's cancer can be tested to find combinations of drugs that provide clinical benefit for the kind of cancer the patient has. This study is also being done to understand why cancer drugs can stop working and how different cancers in different people respond to different types of therapy.

NCT ID: NCT03852771 Terminated - Clinical trials for Restless Legs Syndrome

REST Study (CompRESsion Therapy for RLS)

REST
Start date: March 12, 2019
Phase: N/A
Study type: Interventional

This study in patients with restless leg syndrome (RLS) will evaluate the use of Cirvo™ therapy for the treatment of this disorder when applied to both legs.

NCT ID: NCT03848832 Terminated - Rett Syndrome Clinical Trials

Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome

ARCH
Start date: July 29, 2019
Phase: Phase 3
Study type: Interventional

To evaluate the efficacy of cannabidiol oral solution (GWP42003-P, CBD-OS) in reducing symptom severity when compared with placebo, in participants with Rett syndrome.

NCT ID: NCT03817554 Terminated - Clinical trials for Restless Legs Syndrome

Pramipexole for Restless Leg Syndrome in Peritoneal Dialysis Patients

Start date: July 1, 2019
Phase: Phase 4
Study type: Interventional

A randomized, double-blind controlled Study from a single center to evaluate the effect and safety of pramipexole on peritoneal dialysis patients with restless legs syndrome

NCT ID: NCT03803722 Terminated - Dry Eye Syndrome Clinical Trials

To Demonstrate the Non-inferiority of Eyestil Protection® Compared to Vismed® in Terms of Clinical Performance

Start date: July 8, 2019
Phase: N/A
Study type: Interventional

This is a national, prospective, multicenter, comparative, randomized, single-blinded non-inferiority study performed in two parallel groups. 3 months (plus a run in period of 15 days prior inclusion) Patients with moderate to severe dry eye syndrome.

NCT ID: NCT03794024 Terminated - Clinical trials for Complex Regional Pain Syndrome

Comparison of Dorsal Column Stimulation to Dorsal Root Ganglion Stimulation for Complex Regional Pain Syndrome

Start date: January 8, 2019
Phase:
Study type: Observational

Complex Regional Pain Syndrome (CRPS) is a constellation of pain symptoms which are associated with impairment in mood, social and physical function. Spinal Cord Stimulation (SCS), a technique of placing electrodes into the epidural space is a validated treatment for Complex Regional Pain Syndrome . Treatment of CRPS with SCS, in combination with physical therapy, reduced pain to a greater degree than physical therapy alone. 40%-50% of CRPS patients achieve >50% pain relief with SCS using dorsal column stimulation . Dorsal Root Ganglion (DRG) SCS has also recently demonstrated clinical efficacy in patients with CRPS and peripheral causalgia . The hypothesis is that DRG stimulation is non-inferior to dorsal column SCS in patients with CRPS who have failed to respond to a course of analgesics and physical therapy. The aim to assess functional, quality of life, patient satisfaction and medication requirements in subjects treated with neuromodulation for CRPS and contrast outcomes amongst subjects treated with DRG SCS and dorsal column SCS.

NCT ID: NCT03790865 Terminated - Clinical trials for Prader-Willi Syndrome

Effects of Livoletide (AZP-531) on Food-related Behaviors in Patients With Prader-Willi Syndrome

ZEPHYR
Start date: March 25, 2019
Phase: Phase 2/Phase 3
Study type: Interventional

This Phase 2b/3 double-blind, placebo-controlled study will evaluate the safety, tolerability, and effects of livoletide on food-related behaviors in patients with Prader-Willi Syndrome (PWS).

NCT ID: NCT03783975 Terminated - Long QT Syndrome Clinical Trials

KCNQ1 Cascade Screening

Start date: March 7, 2019
Phase: N/A
Study type: Interventional

The objectives of the protocol are to: (1) evaluate the uptake of cascade screening and preventative therapies after the implementation of a simplified screening process and (2) assess proband and family member perspectives about the return of research results and cascade screening for the KCNQ1 Thr224Met variant. The investigators will conduct a mixed methods study in the Old Order Amish community where the KCNQ1 variant is enriched over 100,000-fold compared to other populations. The intervention will offer free, mail-in, saliva-based genetic testing for family members of probands. The rate of uptake of testing and preventative therapy after the intervention is implemented (i.e. when 'simplified' free, mail-in, saliva-based testing was available) will be compared to data from before the intervention (i.e. when 'traditional' $50 blood-based testing was available to family members) when uptake was essentially zero. The primary outcome is the rate of uptake of cascade screening with the intervention ('simplified'). The secondary outcomes include: extent of disclosure of genotype results before and after the intervention, proportion of informed relatives who get screened before and after the intervention, and the uptake of appropriate preventative care (e.g. seeing a cardiologist and/or taking beta-blocker). The tertiary outcomes are demographic characteristics associated with uptake of cascade screening or uptake of preventative therapy. The investigators will also assess qualitative themes surrounding the return of results process and cascade screening using interviews.