Clinical trials: Congenital Diseases
Emphysema
International Study Evaluating the Safety and Efficacy of Inhaled, Human, Alpha-1 Antitrypsin (AAT) in Alpha-1 Antitrypsin Deficient Patients With Emphysema
This is a randomised , placebo controlled, double blind , multicentre, Phase II/III study evaluating the safety and efficacy of Kamada AAT for inhalation in patients with Emphysema caused by Alpha-1 Antitrypsin (AAT) deficiency.
- Trial name
- NCT01217671
- Testing
- Kamada AAT for inhalation
- Where
- 12 sites in Canada, Denmark and 5 more
- Volunteers
- 168 people
- Sponsor
- Kamada, Ltd.
Congenital Heart Disease, Telerehabilitation
The Effect of Telerehabilitation Exercises in Children With Congenital Heart Disease
This study aims to investigate the effects of functional inspiratory muscle training on physical performance and quality of life in pediatric patients with congenital heart disease.
- Conditions
- Congenital Heart Disease, Telerehabilitation, Functional Inspiratory Muscle Training
- Trial name
- CHD (NCT07854171)
- Testing
- Inspiratory Muscle Training, Functional Inspiratory Muscle Training
- Where
- Turkey (Türkiye)
- Volunteers
- 32 people
- Sponsor
- Izmir Democracy University
Hemophilia A, Hemophilia B
Vitamin K Antagonist Versus Direct Oral Anticoagulant Treatments in Hemophilia
Hemophilia is a rare X-linked bleeding disorder responsible for deficiency of coagulation factor VIII (FVIII) or IX (FIX). The main clinical manifestation is increased bleeding throughout the life which is directly correlated to…
- Conditions
- Hemophilia A, Hemophilia B, Anticoagulant-induced Bleeding
- Trial name
- KADOAH (NCT05804734)
- Testing
- Vitamin K Antagonist - Drug
- Where
- 6 sites in France
- Volunteers
- 54 people
- Sponsor
- Groupe Maladies hémorragiques de Bretagne
Pain, Sickle Cell Disorder
Ketamine Infusion for Acute Sickle Cell Crisis in the Emergency Department
Pain associated with sickle cell disease is a common emergency department visit. It is also frequently associated with a high emergency department recidivism rate for pain control and admissions to the hospital.
- Trial name
- KISS (NCT02417298)
- Testing
- Normal saline, Ketamine
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Billy Sin
Behcet Disease, Behcet Disease and Vascular Involvement
Immunosuppressive Therapy Alone Versus Plus Oral Anticoagulation in the Treatment of VT Associated With Behcet's Disease
This study is a Phase III, multicenter, prospective, randomized, double-blind, placebo-controlled clinical trial investigating the treatment of lower extremity venous thrombosis associated with Behçet's Disease.
- Conditions
- Behcet Disease, Behcet Disease and Vascular Involvement, Deep Venous Thromboses, Post Thrombotic Syndrome
- Trial name
- BETTER (NCT06925698)
- Testing
- Rivaroxaban plus immunosupression
- Where
- 2 sites in Turkey (Türkiye)
- Volunteers
- 110 people
- Sponsor
- Marmara University
Hemophilia B
Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)
The primary purpose of this study is to assess the long-term safety and efficacy in male adults with hemophilia B who were treated with CSL222 (CSL222) in parent studies CSL222_2001 (NCT03489291) or CSL222_3001 (NCT03569891).
- Trial name
- IX-TEND 3003 (NCT05962398)
- Testing
- AAV5-hFIXco-Padua
- Where
- 28 sites in United States, Belgium and 6 more
- Volunteers
- 56 people
- Sponsor
- CSL Behring
Pompe Disease, Glycogen Storage Disease Type II
A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease
The purpose of this study is to see how molecules called pharmacological chaperones affect the cells of patients with Pompe disease.
- Trial name
- NCT00515398
- Testing
- Observation
- Where
- 4 sites in United States
- Volunteers
- 30 people
- Sponsor
- Amicus Therapeutics
Muscular Dystrophies, Scapular Dyskinesis
Evaluation of a Textile Scapula Orthosis
Shoulder instability due to muscle weakness is a common problem in disorders of the upper extremities. During arm motion, the scapula acts as a dynamic base for the humeral head.
- Conditions
- Muscular Dystrophies, Scapular Dyskinesis, Muscular Dystrophy, Facioscapulohumeral, Muscle Weakness
- Trial name
- ScapOrthosis (NCT04154098)
- Testing
- Scapula orthosis assistance, Manual scapula assistance, No assistance
- Where
- 2 sites in Germany, Switzerland
- Volunteers
- 8 people
- Sponsor
- Swiss Federal Institute of Technology
Infant, Premature, Diseases, Nutrition, Enteral
Evaluation of Gastric Residuals and Feedings Progression
This study evaluates the utility of measuring gastric residuals in premature infants born between 26+0 and 33+6 weeks and to determine if its routine use is related with a delay in feedings advancement in this babies compared…
- Trial name
- REGAP (NCT04064398)
- Testing
- Aspiration of gastric residuals
- Where
- Chile
- Volunteers
- 203 people
- Sponsor
- Pontificia Universidad Catolica de Chile
Hypospadias
Is Dorsal Inlay Graft (DIG) With TIP Repair Superior to TIP Alone for Primary Hypospadias?
This prospective, randomized study included all patients who presented with primary hypospadias without chordee, Patients were randomized into two groups as group 1 or group 2,Group 1: Repaired with standard TIP repair as…
- Trial name
- NCT07086963
- Testing
- Repaired with standard TIP repair as described by Snodgrass, Repaired with TIP with GIP using preputial graft
- Where
- Egypt
- Volunteers
- 584 people
- Sponsor
- Mohammad Daboos
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
Donor T Cells After Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies
This pilot phase II trial studies how well giving donor T cells after donor stem cell transplant works in treating patients with hematologic malignancies.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
- Trial name
- NCT01839916
- Testing
- therapeutic allogeneic lymphocytes, laboratory biomarker analysis
- Where
- United States
- Volunteers
- 77 people
- Sponsor
- University of Chicago
Pectus Excavatum
Lung Ultrasound for the Postoperative Diagnosis of Pneumothorax in Children
This study will be conducted to determine the advantages and limitations of sonography compared with chest radiography, in the detection of post procedure iatrogenic pneumothorax in patients underwent to Pectus Excavatum (PE)…
- Trial name
- ECO_NUSS (NCT03073616)
- Testing
- Lung ultrasound
- Where
- Italy
- Volunteers
- 66 people
- Sponsor
- Nicola Disma, MD
Hereditary Hemochromatosis
A Study of LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis
This study is a Phase 2 multicenter, randomized, placebo controlled, single-blind study. The primary objective of the study is to compare the effect of weekly dosing of LJPC-401 (synthetic human hepcidin) versus placebo on…
- Trial name
- NCT03395704
- Testing
- LJPC-401
- Where
- 31 sites in United States, Australia and 2 more
- Volunteers
- 70 people
- Sponsor
- La Jolla Pharmaceutical Company
Neonatal Hyperbilirubinemia
Comparison of Double Pad Fiber Optic Device Versus Conventional Phototherapy
A randomized study of phototherapy for neonatal hyperbilirubinemia. The intervention group is treated with a double pad fiber optic device and the control group with conventional blue ligt phototherapy from above.
- Trial name
- NCT04585828
- Testing
- Phototherapy with double sided fiber optic pads with blue light at irradiance 30 uW/cm2/nm using Bili Cocoon, Phototherapy from above using NeoBlue at irradiance of 30 uW/cm2/nm
- Volunteers
- 80 people
- Sponsor
- University of Aarhus
Atopic Dermatitis
A Phase 2 Study of QLM3003 Ointment in Participants With Mild or Moderate Atopic Dermatitis
The purpose of this study is to assess efficacy, safety, and pharmacokinetics (PK) of QLM3003 Ointment in participants with mild or moderate atopic dermatitis.
- Trial name
- NCT06058000
- Testing
- 1.5% QLM3003, 2% QLM3003
- Where
- 2 sites in China
- Volunteers
- 120 people
- Sponsor
- Qilu Pharmaceutical Co., Ltd.
Infant Conditions, Infant, Premature, Diseases
The Alberta BLOOM Premature Child Study
This is a prospective, observational clinical cohort study involving 405 children born premature (at less than 37 weeks gestation) and their mother/parent/guardian.
- Conditions
- Infant Conditions, Infant, Premature, Diseases, Asthma, Asthma in Children
- Trial name
- BLOOM-PCS (NCT05011071)
- Where
- Canada
- Volunteers
- 535 people
- Sponsor
- University of Calgary
Cystic Fibrosis, Pseudomonas Aeruginosa
Safety of AZLI in Children With Cystic Fibrosis (CF) and Chronic Pseudomonas Aeruginosa in the Lower Airways
This was an open-label, multicenter study in children ≤ 12 years of age with cystic fibrosis (CF) and chronic Pseudomonas aeruginosa (PA) infection in the lower airways using three 28-day courses of Aztreonam for Inhalation…
- Trial name
- PALS (NCT01404234)
- Testing
- AZLI
- Where
- 29 sites in United States, France and 4 more
- Volunteers
- 61 people
- Sponsor
- Gilead Sciences
Cystic Fibrosis-related Diabetes, Cystic Fibrosis
Continuous Glucose Monitoring and OGTT Screen for Cystic Fibrosis Related Diabetes in Cystic Fibrosis
Cystic Fibrosis (CF) related diabetes (CFRD) is a unique form of diabetes mellitus, different from type 1 diabetes and type 2 diabetes.
- Trial name
- NCT06560463
- Testing
- Access to Dexcom G7 CGM Data
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Medical College of Wisconsin
Osteoporosis
Skeletal Health of Klinefelter Patients
This study compared the bone health of KS patients who were actively monitored in our clinic by dual-energy X-ray absorptiometry (DXA) with that of a control group of healthy volunteers.
- Trial name
- NCT03704987
- Testing
- dual-energy X-ray absorptiometry
- Where
- Turkey (Türkiye)
- Volunteers
- 247 people, healthy volunteers welcome
- Sponsor
- TC Erciyes University
Alopecia Areata, Depressive Episode
The Epidemiology, Management, and the Associated Burden of Related Conditions in Alopecia Areata
This study series consists of four related studies and aims to explore and describe many important elements of alopecia areata over three key areas: (1) the current epidemiology of alopecia areata, (2) the prevalence and…
- Conditions
- Alopecia Areata, Depressive Episode, Recurrent Depressive Disorder, Anxiety Disorders
- Trial name
- NCT04239521
- Testing
- Exposure of interest (studies 2 & 3).
- Where
- United Kingdom
- Volunteers
- 51,955 people
- Sponsor
- Momentum Data
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