Clinical trials: Congenital Disorders
Intrauterine Growth Restriction
Intraumbilical Amino Acids and Glucose Supplementation Via Port by Severe IUGR in Human Fetuses
Placental insufficiency is responsible for fetal loss in about 40% of all stillbirths and long term neurological deficits. The mean interval from diagnosis of brain sparing of severe IUGR fetuses to delivery has been recently…
- Trial name
- port-IUGR (NCT02596594)
- Testing
- fetal nutrition port system
- Volunteers
- 14 people
- Sponsor
- Martin-Luther-Universität Halle-Wittenberg
Macular Corneal Dystrophy
Fresh Corneal Lenticule Implantation in Macular Corneal Distrophy With Relex Smile Surgery
The aim of the study is to examine the effect of fresh corneal lenticule implantation as allogenic graft taken from myopic patients to implanted in patients with macular corneal dystrophy using Visumax Femtosecond Laser Smile…
- Trial name
- NCT04642729
- Testing
- Fresh Corneal Lenticule Implantation using Relex-Smile Surgery
- Where
- Kosovo
- Volunteers
- 15 people
- Sponsor
- Eye Hospital Pristina Kosovo
Atopic Dermatitis
Responses to Immunization With Keyhole Limpet Hemocyanin Administered by Scarification and the Intradermal Route
Atopic dermatitis (AD) is a skin disorder in which people often have swelling and skin infections. People with this disease cannot receive the smallpox vaccine because it could cause them to have a fatal reaction known as eczema…
- Trial name
- NCT00614731
- Testing
- KLH carrier-protein
- Where
- United States
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Spinocerebellar Degeneration
Confirmatory Study 3 of KPS-0373 in Patients With Spinocerebellar Degeneration
This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, phase III study to evaluate the efficacy and safety of KPS-0373 given once daily after breakfast in 142 patients with spinocerebellar…
- Trial name
- NCT07040137
- Testing
- KPS-0373
- Where
- Japan
- Volunteers
- 142 people
- Sponsor
- Kissei Pharmaceutical Co., Ltd.
Unilateral Cleft Lip
Philtrum Reconstruction Using Autogenous Fat Injection Versus a Surgical Repair in Secondary Unilateral Cleft Lip Revision
Two groups of patients with repaired unilateral cleft lip deformity having mild to moderate grooving and/or scarring of the philtral column and requiring a secondary cleft lip repair.
- Trial name
- NCT04735237
- Testing
- Autogenous fat injection, Surgical revision with orbicularis oris muscle reconstruction.
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Cairo University
Familial Mediterranean Fever (FMF )
Comparing Structured Neuromuscular Exercise and Exergaming Program in Adolescents With Familial Mediterranean Fever
Familial Mediterranean Fever (FMF) is an autosomal recessive, autoinflammatory disease characterized by recurrent fever attacks and inflammation of the serous membranes.
- Trial name
- NCT07013045
- Testing
- Structured Neuromuscular Exercise Group, Neuromuscular Exergaming Program Group
- Where
- Turkey (Türkiye)
- Volunteers
- 60 people
- Sponsor
- Istanbul University - Cerrahpasa
Esophageal Varices
Wired Magnetically Assisted Capsule Endoscopy and Esophageal Varices
The goal of this clinical trial is to compare the safeness and effectiveness of traditional esophagogastroduodenoscope (EGD) and wired magnetically assisted capsule endoscopy (MACE) in the diagnosis of esophageal varices in…
- Trial name
- NCT06017102
- Testing
- wired magnetic assisted capsule endoscopy, esophagogastroduodenoscopy
- Where
- Taiwan
- Volunteers
- 50 people
- Sponsor
- National Taiwan University Hospital
Wilson's Disease
Gandouling in the Treatment of Wilson's Disease
To evaluate the efficacy and safety of gandouling tablet in the treatment of clinical symptoms of Wilson's diasease.
- Trial name
- NCT05305872
- Testing
- Gandouling
- Where
- China
- Volunteers
- 240 people
- Sponsor
- Jun Li
Atopic Dermatitis (AD)
Study of ENV-294 in Adults With Moderate-to-Severe Atopic Dermatitis
The goal of this clinical trial is to learn about the safety and effectiveness of ENV-294 in adults with moderate to severe atopic dermatitis.
- Trial name
- NCT07298395
- Testing
- ENV-294
- Where
- 32 sites in United States
- Volunteers
- 105 people
- Sponsor
- Enveda Therapeutics
Sickle-cell Disease (SCD)
Evaluation of Impact of Disease on Quality of Life, Education and Socio-professional Integration of Adults and Parents of Children Living with Sickle- Cell Disease in France
Sickle cell disease (SCD) is the most common genetic disease in France. Its consequences on patient's life-course and quality of life need to be precisely identified among French patients and their family to be able to improve…
- Trial name
- DREPAtient (NCT04413539)
- Where
- France
- Volunteers
- 1,088 people
- Sponsor
- Argo Sante
Congenital Heart Disease
Epidemiology of Congenital Heart Disease in France
Congenital heart disease (CHD) is the leading cause of birth defects, with an incidence of 0.8%. Since the 1980s, France has been a pioneer in neonatal CHD surgery (Pr. Fontan, Pr. Lecomte, Pr.
- Trial name
- EPIDEMIO-CHD (NCT04740177)
- Where
- France
- Volunteers
- 450,000 people
- Sponsor
- University Hospital, Montpellier
Angelman Syndrome
A Trial of Levodopa in Angelman Syndrome
This study is designed to determine whether levodopa will lead to an improvement in the development and tremor in children with Angelman syndrome (AS).
- Trial name
- NCT01281475
- Testing
- Levodopa
- Where
- 7 sites in United States
- Volunteers
- 67 people
- Sponsor
- Wen-Hann Tan
Hemophilia A
Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A
Primary Objective: To evaluate the long-term safety of BIVV001 in previously treated subjects with hemophilia A Secondary Objectives: To evaluate the efficacy of BIVV001 as a prophylaxis treatment.
- Trial name
- XTEND-ed (NCT04644575)
- Testing
- efanesoctocog alfa (BIVV001)
- Where
- 85 sites in United States, Argentina and 21 more
- Volunteers
- 261 people
- Sponsor
- Bioverativ, a Sanofi company
Cardiac Amyloidosis, Pacemaker
Increased Pacemaker Lower Rate in ATTR Cardiac Amyloidosis
In cardiac amyloidosis, the heart muscle becomes thick and stiff, making it difficult to pump enough blood with each beat. The heart also often cannot increase its stroke volume, making patients with cardiac amyloidosis more…
- Trial name
- PACE-ATTR (NCT07413081)
- Testing
- Increasing the pacemaker lower rate, Standard setting
- Where
- 9 sites in Denmark, Sweden
- Volunteers
- 34 people
- Sponsor
- Region Skane
Dermatitis, Atopic
Pimecrolimus Cream 1% Plus Topical Corticosteroid in Patients (2-17 Years of Age) With Severe Atopic Dermatitis
Atopic dermatitis, also called eczema, is characterized by redness, papulation (skin elevation) and pruritus (skin itching). The active ingredient of pimecrolimus inhibits the T-cell activation.
- Trial name
- NCT00121381
- Testing
- Pimecrolimus plus topical corticosteroid (TCS)
- Where
- 13 sites in United States
- Volunteers
- 400 people
- Sponsor
- Novartis
Cleft Palate, Cleft Lip and Palate
Book Sharing for Toddlers With Clefts
Children with clefts exhibit difficulty with language and literacy compared to children without clefts. However, little is known about interventions to address these difficulties in the cleft population.
- Trial name
- BOOST (NCT06338319)
- Testing
- Book-Sharing for Toddlers with Clefts (BOOST)
- Where
- United States
- Volunteers
- 320 people
- Sponsor
- Seattle Children's Hospital
Skin Carcinoma
Topical Tazarotene in Treating Patients With Basal Cell Skin Cancer and Basal Cell Nevus Syndrome on the Chest and Back
RATIONALE: Drugs used in chemotherapy, such as tazarotene, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing.
- Trial name
- NCT00783965
- Testing
- tazarotene
- Where
- 3 sites in United States
- Volunteers
- 34 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Adrenomyeloneuropathy, Adrenoleukodystrophy
MD1003-AMN MD1003 in Adrenomyeloneuropathy
The primary objective of the trial is to demonstrate the superiority of biotin at 300 mg/day over placebo in the clinical improvement (walking tests) of patients with adrenomyeloneuropathy
- Conditions
- Adrenomyeloneuropathy, Adrenoleukodystrophy, AMN
- Trial name
- NCT02961803
- Testing
- MD1003 100 mg capsule
- Where
- 4 sites in France, Germany and 1 more
- Volunteers
- 67 people
- Sponsor
- MedDay Pharmaceuticals SA
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
Down Syndrome
Effects of Stretch-shortening Cycle Exercises Postural Control in Down Syndrome
To investigate the effect of a 3-month, trampoline-based stretch-shortening cycle (SSC) exercises on muscle strength and postural control in children with Down syndrome.
- Trial name
- NCT04966403
- Testing
- Standard physical therapy, trampoline-based stretch-shortening cycle exercises
- Where
- Saudi Arabia
- Volunteers
- 32 people
- Sponsor
- Qassim University