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NCT ID: NCT05415618 Enrolling by invitation - Clinical trials for Ultrasound Evaluation of the Effect of Manual Lymph Drainage

Ultrasonographic Evaluation of the Effectiveness of Different Techniques

Start date: March 12, 2024
Phase: N/A
Study type: Interventional

Carpal tunnel syndrome (CTS) occurs as a result of compression of the median nerve under the transverse ligament along the carpal tunnel. The main cause of median nerve compression and carpal tunnel syndrome is increased volume in the carpal tunnel. The pressure that edema puts on the nerve must be controlled in the early period. Edema that persists beyond the inflammatory process can contribute to the fibrotic stage, delay healing, and even cause complications such as pain and stiffness. Manual lymphatic drainage (MLD) is a specialized technique that involves gentle massage techniques and follows lymphatic pathways from proximal to distal and then from distal to proximal. On the basis of this concept; Increasing circulation by stimulating the lymph system, removing biochemical residues, reducing edema and pain, and regulating sympathetic and parasympathetic system responses. It is known that MLD rapidly regulates lymphatic circulation by creating a change in interstitial fluid pressure, thus preventing even arthrofibrotic tissue that may form after a traumatic situation, reducing edema that predisposes to pain, and increasing mobility. Nerve gliding exercises are another method used in treatment. By providing a sliding movement of the tendons and median nerve in the distal-proximal direction, mobilization of the surrounding soft tissues is achieved and dynamic ischemia is terminated. The aim of this study is to determine the effectiveness of manual lymphatic drainage and nerve mobilization on clinical and ultrasonographic findings in carpal tunnel syndrome.

NCT ID: NCT05400278 Enrolling by invitation - Clinical trials for Bardet-Biedl Syndrome

Characterizing the Genotype and Phenotype in Adults With Bardet-Biedl Syndrome

Start date: February 1, 2022
Phase:
Study type: Observational

Bardet-Biedl syndrome (BBS; OMIN #209900) is a rare genetic disorder characterized by six core features: rod-cone dystrophy (retinitis pigmentosa), polydactyly, obesity, genital anomalies, renal anomalies, and learning difficulties. This study aims to contribute to genetic and medical knowledge of BBS, and to provide information on quality of life in adults with BBS and their close relatives. Participants will undergo medical assessments (ocular, oral, and physical examinations) and self-reporting of quality of life, diet, cognitive and emotional symptoms. There are some known genotype-phenotype associations in BBS and participants will be offered genetic testing. It is important to map both genotype and associated phenotype in order to provide optimal treatment and follow-up. Individuals with BBS, age 16 years or older, will be invited to participate. The investigators expect to enroll at least 25 male and female adults with BBS and 15 of their parents to participate in qualitative interviews. These interviews will investigate parents' experiences having a child with BBS, satisfaction with health care services, experience with social and family life, and psychological health.

NCT ID: NCT05367960 Enrolling by invitation - Fragile X Syndrome Clinical Trials

An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome

Start date: November 1, 2022
Phase: Phase 3
Study type: Interventional

This is a 2-year, open-label extension (OLE) study for subjects completing one of two double-blind clinical trials with BPN14770, Study BPN14770-CNS-301(in adult males) and Study BPN14770-CNS-204 (in adolescent males).

NCT ID: NCT05366114 Enrolling by invitation - Clinical trials for Ehlers-Danlos Syndrome

Vision-based Assessment of Joint Extensibility in Ehlers Danlos Syndrome

Start date: April 26, 2022
Phase:
Study type: Observational

Ehlers Danlos Syndrome (EDS) is a heterogenous group of genetic disorders with 13 identified subtypes. Hypermobile EDS (hEDS), although the most common subtype of EDS, does not yet have an identified genetic mutation for diagnostic confirmation. Generalized joint hypermobility (GJH) is one of the hallmark features of hEDS. The scoring system used in measurement of GJH was described by Beighton. The Beighton score is calculated using a dichotomous scoring system to assess the extensibility of nine joints. Each joint is scored as either hypermobile (score = 1) or not hypermobile (score = 0). The total score (Beighton score) can vary between a minimum of 0 and a maximum of 9, with higher scores indicating greater joint laxity. While there is moderate validity and inter-rater variability in using the Beighton score, there continue to be several challenges with its widespread and consistent application by clinicians. Some of the barriers reported in the literature include: i) In open, non-standardized systems there can be significant variation in the method to perform these joint extensibility tests including assessing baseline measurements, ii) Determining consistent and standard measurement tools/methodology e.g. goniometer use can vary widely iii) Assessing the reliability of the cut off values and, iv) Performing full assessment prior to informing patients of possible classification of GJH positivity (low specificity and low positive predictive). Inappropriate implementation of tests to assess GJH results in inaccurate identification of GJH and potentially unintended negative consequences of making the wrong diagnosis of EDS. The objective of this study is to create a more robust and valid method of joint mobility measurement and reduce error in the screening of EDS through use of a smartphone-based machine learning application systems for measurement of joint extensibility. The project will: i) Create a smart-phone enabled visual imaging app to assess the measurement of joint extensibility, ii) Assess the feasibility of using the smart-phone app in a clinical setting to screen potential EDS patients, iii) Determine the validity of the application in comparison to in person clinical assessment in a tertiary care academic EDS program. If successful, the smart-phone application could help standardize the care of potential EDS patients in an efficient and cost-effective manner.

NCT ID: NCT05340816 Enrolling by invitation - Clinical trials for Premenstrual Syndrome

The Effects of Manual Lymphatic Drainage and Transcutaneous Electrical Nerve Stimulation in Premenstrual Syndrome

Start date: April 4, 2022
Phase: N/A
Study type: Interventional

The aim of this study is to (a) investigate the effects of manual lymphatic drainage and transcutaneous electrical nerve stimulation on the severity of syndrome symptoms, pain, heart rate variability, and quality of life in women with premenstrual syndrome, and (b) compare the effectiveness of these two methods. Female participants who met the inclusion criteria and volunteered to participate in the study will be called approximately one week before menstruation. After the participants are randomly divided into 3 groups, their baseline assessments will be made. Then, the participants will be treated according to the group they are assigned to. Treatments will be applied for 5 days and once a day. No treatment will be applied to the control group. The assessments will be repeated after the treatment, on 1st day of menstruation, two days before the next menstruation, and on the first day of the next menstruation. The hypothesis of this study is that manual lymphatic drainage and transcutaneous electrical nerve stimulation have positive effects on the severity of premenstrual syndrome symptoms, pain, heart rate variability, and quality of life.

NCT ID: NCT05311293 Enrolling by invitation - Clinical trials for Irritable Bowel Syndrome With Diarrhea

Study on the Molecular Mechanism of Diarrhea-predominant Irritable Bowel Syndrome With Anxiety and Depression Based on Multi-omics Correlation Analysis

Start date: November 1, 2021
Phase:
Study type: Observational

The research group intends to carry out a case-control study to recruit IBS-D patients with anxiety and depression symptoms, by collecting intestinal mucosa for single-cell transcriptome sequencing, collecting peripheral blood for proteomic analysis, the two groups and patient symptoms are associated, and then discover the characteristics of molecular level changes associated with brain-gut axis dysfunction, explore the pathophysiological mechanism of comorbid anxiety and depression and IBS, and discover potential targets for effective treatment. This project can help to construct the colonic single-cell map of IBS-D patients and explore the differentially expressed genes in the colon of IBS patients and their signaling pathways related to neuroregulation, providing an effective therapeutic target for the treatment of comorbid anxiety and depression and IBS.

NCT ID: NCT05308927 Enrolling by invitation - Noonan Syndrome Clinical Trials

French Registry of Children Treated With Norditropin® for Short Stature Associated With Noonan Syndrome

Start date: March 16, 2022
Phase:
Study type: Observational

This is a non-interventional registry of children treated with Norditropin® for short stature due to Noonan Syndrome (NS). This study aims to provide data on long-term growth evolution and safety of Norditropin® as well as Health Related Quality of Life (HRQoL) data. This registry will include the entirety of children treated with Norditropin® for short stature due to NS over the inclusion period. The decision to initiate treatment with commercially available Norditropin® is made by the patient/parents/Legally Acceptable Representative (LAR) and the treating physician before and independently from the decision to include the patient in this study.

NCT ID: NCT05301361 Enrolling by invitation - Clinical trials for Attention Deficit Hyperactivity Disorder

Sensitivity of the NIH Toolbox to Stimulant Treatment in Intellectual Disabilities

Start date: February 1, 2023
Phase: Phase 1
Study type: Interventional

This study is a randomized, double-blind, placebo-controlled, crossover trial of extended-release liquid methylphenidate (XRMPH) to evaluate the sensitivity of the NIH Toolbox Cognition Battery (NIHTB-CB) to changes in cognition in children and adolescents ages 6 to 17 with intellectual disability (D) and comorbid Attention Deficit Hyperactivity Disorder (ADHD). The sample will include 68 males or females (expected male: female ratio of 1.8:1 with ID and ADHD as determined by structured diagnostic interview and Conners 3 scores. Additional inclusion criteria will include Full Scale IQ above 50 and mental age greater than or equal to 3 years. In addition, participants must be able to complete NIHTB-CB testing and provide valid scores at baseline. After baseline testing, participants will then be randomized to drug or placebo in a 1:1 ratio (N=34 per group) at the end of the baseline visit. XRMPH in oral suspension supplied as Quillivant XR in 5 mg/ml (Tris Pharma, Monmouth Junction, NJ) will be the active treatment. The XRMPH or matching placebo will be started at a dose of 0.3 mg/kg/day and individually titrated over two weeks. Phone calls at the end of weeks 1, 2, and 3 will be used to collect adverse event and response data. If there is no evidence of side effects and ongoing symptoms of ADHD, the dose will be increased to 0.5 mg/kg/day at one week and 0.7 mg/kg/day at 2 weeks (maximum dose of 60 mg per day consistent with FDA labeled use in youth). The Clinical Global Impression (CGI) will be used as a guide to define optimal dose. If side effects occur the dose will be reduced to the dose level at which there were no side effects. Final optimal dose will be established by the end of week 3 and this will be maintained for 2 weeks until 5 weeks post randomization, at which time the follow-up parent and teacher Conners scales, NIHTB-CB, Go/No-Go, and PedsQL will be completed. Participants will have a washout period of 1 week, will then complete re-assessment at the second baseline, and then will cross over to the other treatment (Quillivant to placebo; placebo to Quillivant), also in a double-blind fashion. In the second treatment arm, patients will have the same titration, monitoring and treatment periods as in the first arm, again followed by repeated assessments at the conclusion of 5 weeks. The accrual of participants and number of visits is shown in the Timeline per 6-month period.

NCT ID: NCT05226780 Enrolling by invitation - Clinical trials for SCN8A Developmental and Epileptic Encephalopathy Syndrome

Extension Study to Evaluate How Safe and Tolerable NBI-921352 is as an Adjunctive Therapy for Participants With SCN8A-DEE

Start date: July 12, 2022
Phase: Phase 2
Study type: Interventional

Extension study to evaluate how safe and tolerable the drug NBI-921352 is when used as adjunctive therapy in participants with SCN8A developmental and epileptic encephalopathy syndrome (SCN8A-DEE).

NCT ID: NCT05223621 Enrolling by invitation - Turner Syndrome Clinical Trials

Sexuality and Sexual Quality of Life in Adult Patients With Turner Syndrome (TS) Compared With Patients With Primary Ovarian Insufficiency Different From TS

SEXATURN
Start date: February 2022
Phase:
Study type: Observational

Turner syndrome (TS) is a rare disease affecting 1/2500 female. It is defined by a complete or partial loss of an X chromosome associated with clinical signs. The most frequent signs are a small height and primary ovarian insufficiency (POI). POI occurs in 95% of patients with TS. Clinically, patients have amenorrhea with elevated FSH levels (> 25 IU/L), before the age of 40. In most cases, patients receive hormonal replacement therapy. Among patients with POI, TS is present in less than 10% of cases. Therefore POI may occur in patients with normal karyotype, therefore without TS. Preliminary data suggest altered sexual function in patients with TS. The first goal of our study is to evaluate sexual function and sexual quality in patients with TS using a questionnaire, the Female Sexual Function Index (FSFI). The second goal is to compare sexual quality in patients in patients with TS compared to female patients with POI not related to TS. Our study should identify predictive markers of altered sexual function. The final endpoint is to optimize the quality of life of patients with TS and to enhance, if necessary psychological support in such patients.