Clinical trials: Congenital Malformation
Atopic Eczema
Study to Investigate Skin Conditions and Patient Assessment of LAS 41002 in the Treatment of Atopic Eczema
The aim of the study is an intra-individual comparison of skin conditions in addition to a patient based assessment of product appearance in the topical treatment of atopic eczema.
- Trial name
- NCT01119313
- Testing
- LAS 41002, Active
- Where
- Germany
- Volunteers
- 20 people
- Sponsor
- Almirall, S.A.
Gout
The Percentage of Cases of Acute Treatment With Allopurinol in Gout Patients
The aim of the study is to determine the percentage of cases of acute treatment with Allopurinol in gout patients who have been previously treated.
- Trial name
- NCT03601260
- Testing
- Allopurinol
- Where
- Israel
- Volunteers
- 700 people
- Sponsor
- Hillel Yaffe Medical Center
Bronchopulmonary Dysplasia, Child Development
Maternal Omega-3 Supplementation to Reduce Bronchopulmonary Dysplasia
The aim of this randomized controlled trial is to determine whether docosahexaenoic acid (or DHA, an omega-3 lipid) supplementation in lactating mothers providing breast-milk to their infant born below 29 0/7 weeks of gestational…
- Conditions
- Bronchopulmonary Dysplasia, Child Development, Neonatal and Perinatal Conditions
- Trial name
- MOBYDIck (NCT02371460)
- Testing
- DHA-rich algal oil
- Where
- 16 sites in Canada
- Volunteers
- 461 people
- Sponsor
- CHU de Quebec-Universite Laval
Cystic Fibrosis
Evaluating High Flow Humidification Therapy in Patients With Cystic Fibrosis
This study is a single center, randomized pilot study to evaluate the clinical effectiveness of nasal high flow 20LPM humidification therapy in subjects with Cystic Fibrosis.
- Trial name
- NCT02129803
- Testing
- High-Flow, 20 LPM (via Optiflow cannula), Low FLow, 5 LPM (via Optiflow cannula)
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Virginia Commonwealth University
Infant, Premature, Diseases, Motor Development
Standardized Infant NeuroDevelopmental Assessment (SINDA) in 0-24 Months Age in Turkey
This study aims to assess Turkish validity and reliability of Standardized Infant Neurodevelopmental Assessment and to spread its use for evaluation in early rehabilitation in our country.
- Trial name
- NCT04889027
- Testing
- Standardized Infant NeuroDevelopmental Assessment
- Where
- Turkey (Türkiye)
- Volunteers
- 50 people
- Sponsor
- Ondokuz Mayıs University
Retinitis Pigmentosa, Retinal Degeneration
Safety and Efficacy of Subretinal Implants for Partial Restoration of Vision in Blind Patients
Patients suffering from hereditary retinal degeneration receive a retinal implant to restore sight. Subretinal implant "ON" results in significant visual acuity improvement, when compared to "OFF" condition.
- Trial name
- NCT01024803
- Testing
- Device name: "Retina Implant model Alpha". Surgical implantation of medical device into eye
- Where
- 6 sites in Germany, Hungary and 1 more
- Volunteers
- 39 people
- Sponsor
- Retina Implant AG
Lysosomal Storage Diseases
Screening of Lysosomal Storage Disorders Diseases in Minority Groups
Aim is to undertake a screening study that identifies undiagnosed patients with LSDs and determine the prevalence of these diseases with special focus on underrepresented minority groups.
- Trial name
- NCT03812042
- Testing
- Enzyme assay and molecular sequencing
- Where
- United States
- Volunteers
- 100,000 people, healthy volunteers welcome
- Sponsor
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Multiple Osteochondroma, Osteogenesis Imperfecta
Resilience and Coping in a Rare Skeletal Disease Population to Face Coronavirus (COVID-19) Outbreak Distress: a Longitudinal Study
In the COVID-19 outbreak context, people living with rare diseases have been highly troubled with anxiety, loneliness, and depression.
- Conditions
- Multiple Osteochondroma, Osteogenesis Imperfecta, Ollier Disease, Maffucci Syndrome
- Trial name
- RELOAD (NCT04844697)
- Where
- Italy
- Volunteers
- 15 people
- Sponsor
- Istituto Ortopedico Rizzoli
Benign Breast Disease
Nuclear Morphology of Breast Cells in Ductal Lavage
Breast nipple aspirate fluids (NAF) are useful for non-invasive monitoring of the breast. NAF has been shown to exhibit large inter-individual differences in lipid peroxidation.
- Trial name
- NCT00259597
- Testing
- breast ductal lavage
- Where
- United States
- Volunteers
- 57 people, healthy volunteers welcome
- Sponsor
- University of Michigan
Muscular Dystrophies, Muscular Dystrophy
Muscle Tissue Bank for Muscular Dystrophy
This protocol proposes to establish gene expression profiles of muscular dystrophies for correct diagnosis and for development of experimental therapies for these diseases.
- Trial name
- NCT01950897
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- Wake Forest University Health Sciences
MELAS Syndrome, Ketogenic Dieting
Effectiveness of Ketogenic Diet in MELAS Syndrome
The goal of this clinical trial is to evaluate the effectiveness of ketogenic diet in patients with MELAS syndrome. The main questions it aims to answer are: Clarify the curative effects of ketogenic diet in the treatment of…
- Trial name
- NCT06013397
- Testing
- Ketogenic diet
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Second Affiliated Hospital, Zhejiang University, School of Medicine
Fabry Disease
Study to Evaluate the Safety and EffIcacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease
The study will be a randomized, double blind, placebo-controlled study of the safety and efficacy of PRX-102 in ERT naïve male patients randomized 1:1. Patient age will be 14 to 45 years.
- Trial name
- NCT02921620
- Testing
- PRX-102
- Sponsor
- Protalix
Childhood Glaucoma
Efficacy and Safety of Topically Applied Medical Therapy for the Treatment of Pediatric Glaucoma
Objectives: The study will assess the ocular hypotensive effect of latanoprost and dorzolamide in a selected sample of patients affected by primary Pediatric Glaucoma (PG), refractory to surgical procedures.
- Trial name
- NCT01527682
- Testing
- Latanoprost, Dorzolamide
- Where
- 2 sites in Italy
- Volunteers
- 37 people
- Sponsor
- Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia
Health, Subjective, Nutrition Disorders
Relative Energy Deficiency in Sport Multicenter Study
Relative Energy Deficiency in Sport (RED-S) characterizes a range of negative health and performance outcomes that result from chronically low energy availability.
- Conditions
- Health, Subjective, Nutrition Disorders, Athletic Injuries, Hormone Disturbance
- Trial name
- NCT04766203
- Testing
- Treatment of REDS using a holistic treatment arm
- Where
- Canada
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- Trent Stellingwerff
Mucopolysaccharidoses, Mucopolysaccharidosis I
Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics
This study is being done to learn how many children and young adults who come to pediatric rheumatology clinics may have mucopolysaccharidosis (MPS). The study tests for 4 of the types of MPS: I, II, IVA, and VI.
- Conditions
- Mucopolysaccharidoses, Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV
- Trial name
- NCT01675674
- Testing
- Dried blood spot test for MPS
- Where
- 2 sites in United States
- Volunteers
- 3,000 people
- Sponsor
- National MPS Society
Atopic Dermatitis
A Study to Assess the Efficacy and Safety of Ruxolitinib Cream in Children and Adolescents (6 to <18 Years Old) With Moderate Atopic Dermatitis
The purpose of the study is to assess the efficacy and safety of ruxolitinib cream in children and adolescents (6 to <18 Years Old) with moderate atopic dermatitis.
- Trial name
- TRuE-AD5 (NCT06832618)
- Testing
- Ruxolitinib, Vehicle Cream
- Where
- 93 sites in United States, Belgium and 8 more
- Volunteers
- 159 people
- Sponsor
- Incyte Corporation
Hemophilia B
Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug
This is a multi-center, Phase 1/2/3, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of BBM-H901 injection in Hemophilia B subjects with ≤2 International unit per…
- Trial name
- NCT05203679
- Testing
- Single dose intravenous injection of BBM-H901
- Where
- 9 sites in China
- Volunteers
- 32 people
- Sponsor
- Shanghai Xinzhi BioMed Co., Ltd.
Ehlers-Danlos Syndrome, Healthy Subjects
ABVN Phase 2 - EDS Wearable Device
The vagus nerve is a nerve that controls many bodily functions, including stomach function. Investigators hope that electrically stimulating the nerve around the external ear will also stimulate the internal vagus nerve.
- Trial name
- ABVN2 (NCT07856459)
- Testing
- ML1101 Stimulus Isolator, Cardiowatch Bracelet
- Where
- United States
- Volunteers
- 58 people, healthy volunteers welcome
- Sponsor
- Indiana University
Rett Syndrome
Telehealth Support to Increase Physical Activity in Rett Syndrome
This study will recruit families with a daughter with Rett syndrome living in either Australia, Denmark or Israel, and thereafter deliver individually designed participation programs using telehealth strategies.
- Trial name
- ActivRett (NCT04167059)
- Testing
- Telehealth-delivered participation strategies to increase physical activity
- Where
- 2 sites in Denmark, Israel
- Volunteers
- 60 people
- Sponsor
- Telethon Kids Institute
Myofacial Pain, Pain, Neck
Trigger Point Injections in Anterior Cervical Surgery
To achieve appropriate exposure for an anterior neck surgery (for example an Anterior Cervical Discectomy and Fusion or ACDF), patients are positioned supine with their neck extended.
- Conditions
- Myofacial Pain, Pain, Neck, Pain, Back, Cervical Fusion
- Trial name
- NCT04640896
- Testing
- Trigger point injection with bupivacaine, Trigger point injection with normal saline, Lidocaine skin wheal
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- George Washington University
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