Clinical trials: Congenital Diseases
Atopic Dermatitis
Secukinumab for Treatment of Atopic Dermatitis
Atopic Dermatitis, also known as atopic eczema, or eczema, is a common skin disease that can affect males and females of all ages, but often starts in childhood.
- Trial name
- NCT02594098
- Testing
- Secukinumab
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
Leukemia, Myelodysplastic Syndromes
Combination Chemotherapy With or Without Filgrastim and/or Tretinoin in Treating Patients With Acute Myeloid Leukemia
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Colony-stimulating factors such as filgrastim may increase the number of immune cells found in bone marrow…
- Conditions
- Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
- Trial name
- NCT00005863
- Testing
- filgrastim, cytarabine, daunorubicin hydrochloride
- Where
- United Kingdom
- Sponsor
- Medical Research Council
Hemophilia A
Effects of Ultrasound and Infrared on Joint Pain and Movement in Hemophilia A Patients.
The study aims to compare the effectiveness of therapeutic ultrasound and infrared in managing hemarthrosis in patients with Hemophilia A, focusing on specific outcomes like joint pain, swelling, muscle atrophy, and range of…
- Trial name
- Hemophilia A (NCT07116993)
- Testing
- theraputic ultrasound, infrared radiation
- Where
- Pakistan
- Volunteers
- 30 people
- Sponsor
- Danyal Ahmad
Hemophilia A
A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants With Inhibitors
This multicenter, open-label study will evaluate the safety, efficacy and pharmacokinetics of prophylactic emicizumab treatment in participants previously treated with episodic or prophylactic bypassing agents.
- Trial name
- HAVEN 1 (NCT02622321)
- Testing
- Emicizumab, rFVIIa, aPCC
- Where
- 44 sites in United States, Australia and 12 more
- Volunteers
- 113 people
- Sponsor
- Hoffmann-La Roche
Hyperlipemia, Carbohydrate Inducible
Milk as a Recovery Beverage After Exercise for Improving Metabolic Health
Fasting blood fat levels (triglycerides) are often used to assess risk of heart disease but the level of fats in the blood after a meal is actually a stronger risk factor.
- Trial name
- NCT03154216
- Testing
- Exercise, Drink
- Where
- Canada
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- University of Saskatchewan
Mitochondrial Diseases, Mitochondrial Respiratory Chain Deficiencies
A Phase Ia/Ib, SAD and MAD Study of of KL1333 in Healthy Subjects and Patients With Primary Mitochondrial Disease
This will be a double blind, randomised, placebo controlled, single and multiple oral dose study conducted in 3 parts: Part A, Part B and Part C.
- Conditions
- Mitochondrial Diseases, Mitochondrial Respiratory Chain Deficiencies, MELAS Syndrome, Mitochondrial Myopathies
- Trial name
- NCT03888716
- Testing
- KL1333
- Where
- 2 sites in United Kingdom
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- Abliva AB
Turner Syndrome, Infertility, Female
Turner Syndrome Minipuberty Study
Rationale: Due to accelerated germ cell loss, infertility is a major problem in girls with Turner syndrome (TS). Therefore, cryopreservation of ovarian tissue or oocytes before exhaustion of the ovarian reserve may preserve…
- Conditions
- Turner Syndrome, Infertility, Female, Premature Ovarian Failure, Gonadal Dysgenesis
- Trial name
- Minipuberty (NCT04189406)
- Testing
- Venapunction
- Where
- 13 sites in Denmark, Germany and 3 more
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Radboud University Medical Center
Chronic Myeloproliferative Disorders, Leukemia
Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Other Diseases
RATIONALE: Giving chemotherapy, such as fludarabine, busulfan, and melphalan, before a donor peripheral stem cell transplant or bone marrow transplant helps stop the growth of cancer or abnormal cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00453206
- Testing
- allogeneic bone marrow transplantation, allogeneic hematopoietic stem cell transplantation, nonmyeloablative allogeneic hematopoietic stem cell transplantation
- Where
- United States
- Volunteers
- 66 people
- Sponsor
- Wake Forest University Health Sciences
Sphingomyelin Lipidosis
A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
The primary objective of this study was to obtain data regarding the safety of olipudase alfa in participants with acid sphingomyelinase deficiency (ASMD) who were exposed to long term treatment with olipudase alfa.
- Trial name
- NCT02004704
- Testing
- GZ402665
- Where
- 9 sites in United States, Belgium and 5 more
- Volunteers
- 25 people
- Sponsor
- Genzyme, a Sanofi Company
Adrenal Hyperplasia, Congenital
LC-MS / MS Adrenal Steroids Assayed on Dried Blot Spot for the Congenital Adrenal Hyperplasia Neonatal Screening (SPECTROSPOT)
This research uses the Liquid Chromatography coupled to tandem Mass Spectrometry (LC-MS / MS) technique on dried blot spot samples for the neonatal screening of congenital adrenal hyperplasia.
- Trial name
- SPECTROSPOT (NCT03589144)
- Where
- France
- Volunteers
- 132 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Congenital Heart Disease, Inflammation
Ultrafiltration in Pediatric Cardiac Surgery: An Observational Cohort Study
Infants and children undergoing cardiac surgery with cardiopulmonary bypass (CPB) can experience systemic inflammation that prolongs post-operative recovery.
- Trial name
- NCT05154864
- Testing
- SBUF-SMUF
- Where
- Canada
- Volunteers
- 40 people
- Sponsor
- IWK Health Centre
Tourette Syndrome
Effect of RNS in Treatment-refractory Tourette's Syndrome
The study is to investigate the effect of personalized responsive neurostimulation (RNS) therapy guided by stereoelectroencephalography (SEEG) in patients with treatment-resistant Tourette's Syndrome (TR-TS).
- Trial name
- NCT06785532
- Testing
- Responsive Neurostimulation (RNS)
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Xuanwu Hospital, Beijing
Molar Incisor Hypomineralization, Anesthesia, Local
Effect of Silver Diamine Fluoride on Local Anesthesia in Hypomineralized Molars
The purpose of this randomized, controlled clinical study is to evaluate if application of 38% SDF increases the efficacy of local anesthesia during operative dentistry on permanent molars affected by molar-incisor hypoplasia…
- Trial name
- NCT05964387
- Testing
- SDF
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University of Michigan
Breast Cancer, Chronic Myeloproliferative Disorders
Campath-1H + FK506 and Methylprednisolone for GVHD
RATIONALE: Alemtuzumab, tacrolimus, and methylprednisolone may be an effective treatment for graft-versus-host disease caused by a donor stem cell transplant.
- Conditions
- Breast Cancer, Chronic Myeloproliferative Disorders, Gestational Trophoblastic Tumor, Graft Versus Host Disease
- Trial name
- NCT00109993
- Testing
- alemtuzumab, methylprednisolone, tacrolimus
- Where
- 6 sites in United States
- Volunteers
- 34 people
- Sponsor
- Case Comprehensive Cancer Center
MPS II, Hunter Syndrome (MPS II)
RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)
RGX-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system.
- Trial name
- NCT07236606
- Testing
- RGX-121-3102
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- REGENXBIO Inc.
Tetralogy of Fallot (TOF), Alpha-tocopherol
Effect of Parenteral Alpha-Tocopherol in the Definitive Surgery of Tetralogy of Fallot
Introduction: Tetralogy of Fallot (ToF) correction with cardiopulmonary bypass (CPB) poses a risk of ischemia-reperfusion injury, especially in cyanotic myocardium.
- Conditions
- Tetralogy of Fallot (TOF), Alpha-tocopherol, Cardiac Surgery
- Trial name
- NCT07194304
- Testing
- alpha-Tocopherol, Sterile Water for Injection
- Where
- Indonesia
- Volunteers
- 60 people
- Sponsor
- Dr Cipto Mangunkusumo General Hospital
Atopic Dermatitis
Evaluate Efficacy and Safety of Repeat Subcutaneous Doses of FB825 in Adults With Moderate-to-Severe Atopic Dermatitis
This is a randomized, placebo-controlled and double-blind study to evaluate the efficacy, pharmacokinetics, and Safety of repeat subcutaneous doses of FB825 in adults with moderate-to-severe atopic dermatitis.
- Trial name
- NCT06397911
- Testing
- FB825
- Where
- 16 sites in United States, Taiwan
- Volunteers
- 114 people
- Sponsor
- Oneness Biotech Co., Ltd.
Ehlers-Danlos Syndrome, Multiple Sclerosis
Vascular Fundus Changes in Patients With High Probability of Chronic Cerebrospinal Venous Insufficiency (CCSVI)
The investigators propose that evidence of chronic cerebrospinal venous insufficiency (CCSVI) may be evident in the vasculature of the fundus.
- Trial name
- NCT01356134
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Genetic Disease Investigators
MELAS Syndrome, Mitochondrial Respiratory Chain Deficiencies
Safety, Tolerability and Pharmacokinetic Study of KL1333 in Healthy Male Volunteers
The purpose of this First In Human study is to investigate the safety and tolerability of KL1333 after a single oral dose and to investigate the pharmacokinetic characteristics of KL1333 after a single oral dose.
- Trial name
- NCT03056209
- Testing
- KL1333 25 mg, KL1333 50 mg, KL1333 100 mg
- Where
- South Korea
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Yungjin Pharm. Co., Ltd.
Laminopathy, Lamin A/C Gene Mutation
Subanalysis in Patients With CARDIoLAMinopathy Enrolled to REPORT-CCM Registry
Observational, retrospective registry with acute and chronic endpoints
- Conditions
- Laminopathy, Lamin A/C Gene Mutation, Heart Failure
- Trial name
- CARDILAM-CCM (NCT04904393)
- Testing
- Implant of the device for Cardiac Contractility Modulation (CCM) Therapy
- Where
- Italy
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Monaldi Hospital
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