Clinical trials: Birth Defects
Corneal Endothelial Decompensation, Fuchs' Endothelial Dystrophy
Impact of Donor Diabetes on DMEK Success and Endothelial Cell Loss
This double-masked multi-center trial will evaluate the association of diabetes in the cornea donor with transplant success and loss of endothelial cells through 1 year following Descemet membrane endothelial keratoplasty (DMEK).
- Trial name
- DEKS (NCT05134480)
- Testing
- Descemet membrane endothelial keratoplasty
- Where
- 2 sites in United States
- Volunteers
- 1,097 people
- Sponsor
- Case Western Reserve University
Retinoblastoma
Topotecan Episcleral Plaque for Treatment of Retinoblastoma
This single site, single-arm, non-randomized, dose escalation phase I toxicity clinical trial will assess primarily the safety and secondarily the efficacy of episcleral topotecan in patients with active de novo or residual or…
- Trial name
- STEP-RB (NCT04428879)
- Testing
- Topotecan Episcleral Plaque
- Where
- Canada
- Volunteers
- 26 people
- Sponsor
- The Hospital for Sick Children
Sickle Cell Anemia
Risk-based Therapy for Sickle Cell Anemia: A Feasibility Study
Sickle cell anemia (SCA) patients experience organ damage that begins at an early age and results in significant morbidity and early mortality.
- Trial name
- NCT02090296
- Testing
- Hydroxyurea
- Where
- United States
- Sponsor
- Children's National Research Institute
Prader-Willi Syndrome
Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome
RGH-706 is a novel, potent, and orally active MCHR1 antagonist drug candidate discovered and being developed by Gedeon Richter Plc. for weight management.
- Trial name
- NCT05322096
- Testing
- RGH-706
- Where
- 23 sites in United States, Czechia and 3 more
- Volunteers
- 39 people
- Sponsor
- Gedeon Richter Plc.
Severe Combined Immunodeficiency, X Linked, Gene Therapy
Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning
This is a phase I/II open label multi-center study in which patients will receive low dose targeted busulfan followed by infusion of autologous CD34+ selected bone marrow or mobilized peripheral blood cells transduced with the…
- Trial name
- NCT03311503
- Testing
- autologous CD34+ cell transduced with G2SCID vector
- Where
- 4 sites in United States
- Volunteers
- 12 people
- Sponsor
- David Williams
Alpha1-antitrypsin Deficiency
GLASSIA Safety, Immunogenicity, and Bronchoalveolar Lavage Study
The purpose of the study is 2-fold: (1) to evaluate the safety and potential immunogenicity of GLASSIA following intravenous (IV) administration via in-line filtration; and (2) to assess the effects of GLASSIA augmentation…
- Trial name
- NCT02525861
- Testing
- GLASSIA
- Where
- 18 sites in United States, Canada
- Volunteers
- 34 people
- Sponsor
- Baxalta now part of Shire
Fed
Bioequivalence Study of Indomethacin Extended-Release Capsules 75 mg Under Fed Condition
This is a randomized, balanced, open Label, two-treatment, two-period, two-sequence, single dose, crossover pivotal study. The purpose of this study is to assess the bioequivalence between Test Product and the corresponding…
- Trial name
- NCT01722110
- Testing
- Indomethacin Extended-Release Capsules USP 75 mg, Indomethacin Extended Release Capsules USP 75 mg
- Where
- India
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- IPCA Laboratories Ltd.
Sickle Cell Anemia, Sickle Cell Thalassemia
Clinical and Laboratory Characteristics of Sickle Cell Anemia Patients Admitted With Fever
This study will summarized the clinical and laboratory data and the outcome of all the patients suffering from Sickle Cell Anemia (Including Sickle cell thalassemia) admitted to the pediatric ward.
- Trial name
- NCT00736060
- Testing
- Observational
- Where
- Israel
- Volunteers
- 60 people
- Sponsor
- HaEmek Medical Center, Israel
Atopic Dermatitis, Eczema
The Utility of Functionally Relevant Signature Genes in Assessing the Clinical Outcomes of Dupilumab Treatment
The utility of functionally relevant signature genes in assessing the clinical outcomes of dupilumab treatment in the adult onset atopic dermatitis
- Trial name
- NCT04358224
- Testing
- Dupilumab Prefilled Syringe
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Jonathan A. Bernstein, MD
Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Monoblastic Leukemia (M5a)
Lithium Carbonate and Tretinoin in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia
This phase I trial studies the side effects and best dose of tretinoin when given together with lithium carbonate in treating patients with relapsed or refractory acute myeloid leukemia.
- Conditions
- Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b), Adult Acute Myeloblastic Leukemia With Maturation (M2)
- Trial name
- NCT01820624
- Testing
- tretinoin, lithium carbonate, laboratory biomarker analysis
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Paolo Caimi, MD
Friedreich's Ataxia, Motor Disorders
Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.
Prospective, exploratory, multicenter pilot study investigating the structural and functional connectome in patients with Friedreich's Ataxia (FRDA) using high-density electroencephalogram (HD-EEG).
- Trial name
- CONNETTOMA (NCT07095062)
- Testing
- HD-EEG recordings, combined with cognitive and motor assessment
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- IRCCS Eugenio Medea
Colon Cancer
Screening for Gynecologic Cancers in Hereditary Nonpolyposis Colorectal Cancer (HNPCC) Patients
Among women with HNPCC, this study will assess: 1. Knowledge of screening recommendations for endometrial and ovarian cancers. 2. Perceived risk and cancer worries regarding endometrial, ovarian and colorectal cancers. 3.
- Trial name
- NCT00508846
- Testing
- Questionnaire
- Where
- United States
- Volunteers
- 93 people
- Sponsor
- M.D. Anderson Cancer Center
Idiopathic Pulmonary Fibrosis
Assess the Safety, Tolerability and Pharmacokinetics of AZD5055 Following Single and Multiple Ascending Doses in Healthy Participants
This is a phase I, First-in-Human study in healthy participants, performed at a single study center, consisting of 2 parts: Part 1 is a single ascending dose (SAD) study and Part 2 is a multiple ascending dose (MAD) study.
- Trial name
- NCT05134727
- Testing
- AZD5055
- Where
- United States
- Volunteers
- 63 people, healthy volunteers welcome
- Sponsor
- AstraZeneca
Retinitis Pigmentosa
Natural History Study of Patients With X-linked Retinal Dystrophy Associated With Mutations in Retinitis Pigmentosa GTPase Regulator (RPGR)
The rod-cone dystrophies (often referred to as retinitis pigmentosa (RP)) are a clinically and genetically heterogeneous group of disorders in which there is progressive loss of rod and later cone photoreceptor function leading…
- Trial name
- NCT03349242
- Where
- 8 sites in United States, Canada and 1 more
- Volunteers
- 140 people
- Sponsor
- MeiraGTx UK II Ltd
Primary Ciliary Dyskinesia
Glycine and Magnesium+Thiamine for the Treatment of Primary Ciliary Dyskinesia
Patients with primary ciliary dyskinesia (PCD) have trouble with clearing their bronchi from mucus, which in the long-term may produce severe damage to the lungs.
- Trial name
- NCT06959251
- Testing
- Glycine, Magnesium+thiamine
- Where
- Mexico
- Volunteers
- 60 people
- Sponsor
- Instituto Nacional de Enfermedades Respiratorias
Tourette Syndrome
A Study to Evaluate Long-term Safety of Ecopipam Tablets in Children, Adolescents and Adults With Tourette's Disorder
The primary objective of this study is to evaluate the long-term safety and tolerability of ecopipam tablets in children (greater than or equal to [>=] 6 and less than [<] 12 years of age), adolescents (>=12 and <18 years of…
- Trial name
- NCT06021522
- Testing
- Ecopipam
- Where
- 67 sites in United States, Bulgaria and 6 more
- Volunteers
- 150 people
- Sponsor
- Emalex Biosciences Inc.
Congenital Heart Disease, Sedated for Cardiac Catheterization
Does Ketamine Attenuate Depression of Respiratory and Cardiac Functions
Normal cardiac and respiratory functions should be maintained during pediatric cardiac catheterization. Propofol has become a popular choice for sedation in children, however, it depresses cardiac and respiratory functions.
- Trial name
- NCT01501786
- Testing
- control, Ket10, Ket20
- Where
- Japan
- Volunteers
- 150 people
- Sponsor
- Ibaraki Children's Hospital
Congenital Heart Disease
Vancomycin and Cefoxitin During Pediatric Cardiopulmonary Bypass
The aim of this study will be to evaluate vancomycin and cefoxitin blood levels during elective cardiopulmonary bypass (CPB) surgery in four pre-determined pediatric strata: neonates, infants, children weighting less than 40 kg…
- Trial name
- VANCOCEF (NCT02839486)
- Testing
- vancomycin pharmacokinetics, cefoxitin pharmacokinetics
- Where
- Italy
- Volunteers
- 40 people
- Sponsor
- Bambino Gesù Hospital and Research Institute
Hemorrhage, Obstetric, Uterine Abnormality
Evaluation of the Non-inferiority of Resorbable Gelatin Embolization Compared to Embolization Combined With Endometrial Aspiration for the Management of Hemorrhagic Uterine Vascular Abnormalities Following Premature Termination of Pregnancy
The goal of this clinical trial is to determine the best treatment for patients who experience vaginal bleeding following a premature termination of pregnancy.
- Conditions
- Hemorrhage, Obstetric, Uterine Abnormality, Uterine Artery Embolization, Vaginal Bleeding During Pregnancy
- Trial name
- NIEMBUT (NCT07206342)
- Testing
- embolization therapy, embolization followed by aspiration
- Where
- 4 sites in France
- Volunteers
- 110 people
- Sponsor
- University Hospital, Grenoble
Hereditary Cancer Syndrome
CfDNA in Hereditary And High-risk Malignancies 2
The goal of this study is to understand the performance of an experimental blood test that aims to detect early tumors in patients with hereditary cancer syndromes.
- Trial name
- CHARM2 (NCT06726642)
- Testing
- Cell-free DNA analysis
- Where
- 8 sites in Canada
- Volunteers
- 1,000 people
- Sponsor
- University Health Network, Toronto