Clinical trials: Multiple Congenital Anomaly
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
Netherton Syndrome
Evaluation of Topical ATR12-351 in Adults With Netherton Syndrome
The objectives of this clinical trial are to evaluate the safety and tolerability of topically applied ATR12-351, to understand what the body does to ATR12-351, and to observe treatment benefits of the drug in approximately 12…
- Trial name
- NCT06137157
- Testing
- ATR12-351
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Azitra Inc.
Cystic Fibrosis
A First in Human Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Single and Multiple Ascending Doses of Inhaled ETD001 in Healthy Subjects
This is a first in human study of ETD001 a new drug being developed for the treatment of cystic fibrosis. The study is a randomised, placebo-controlled, double-blind interventional study to assess the safety, tolerability and…
- Trial name
- NCT04926701
- Testing
- ETD001 single dose, ETD001 multiple twice daily doses, ETD001 multiple once daily doses
- Where
- United Kingdom
- Volunteers
- 98 people, healthy volunteers welcome
- Sponsor
- Enterprise Therapeutics Ltd
Gout
A Clinical Trial of HEC93077 in Chinese Healthy Subjects and HUA Patients With Single or Multiple Dosing
A phase 1 clinical trial of HEC93077,to evaluate the safety ,PK,PD and food effect in Chinese healthy subjects and HUA patients after single or multiple dosing.
- Trial name
- NCT05127772
- Testing
- HEC93077
- Where
- China
- Volunteers
- 126 people, healthy volunteers welcome
- Sponsor
- Sunshine Lake Pharma Co., Ltd.
Down Syndrome
Correlation Between Optic Nerve Vessel Anomalies, Serum Angiogenic Factors and Renal Anomalies in Down Syndrome Children
In approximately half of individuals with Down syndrome, an higher than normal number of vessels cross the optic disc margin. Investigator hypothesize that early retinal vessel branching occurs due to inhibition of angiogenesis…
- Trial name
- DOPANUR (NCT03206957)
- Testing
- Funduscopic examination and retinal photography, Serum levels of endostatin and angiogenesis factors, Renal and low urinary tract Doppler ultrasound
- Where
- Belgium
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Queen Fabiola Children's University Hospital
Genetic Disease, Nonsense Mutation
Phase 1 Study of ELX-02 in Healthy Adult Subjects
Phase 1 Multiple Ascending Dose Study in Normal Healthy Volunteers
- Trial name
- NCT03309605
- Testing
- ELX-02
- Where
- Belgium
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- Eloxx Pharmaceuticals, Inc.
Acute Intermittent Porphyria
Porphozym in the Treatment of Acute Attacks in AIP
A multi-centre, double-blind, randomized, placebo controlled, parallel group trial, investigating the efficacy and safety of Porphozym (recombinant human porphobilinogen deaminase)in the treatment of acute attacks in AIP.
- Trial name
- NCT00418795
- Testing
- recombinant human porphobilinogen deaminase (Porphozym)
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Zymenex A/S
Arteriovenous Fistula
An Efficacy and Safety Study of SRM003 in the Treatment of Subjects Undergoing Creation of an Arteriovenous Fistula to Facilitate Hemodialysis Access
A study to evaluate the efficacy of SRM003 treatment versus participating sites' standard practice treatment in improving the rate of AVF maturation and use in subjects with end-stage renal disease undergoing surgery for creation…
- Trial name
- NCT01806545
- Testing
- SRM003, Participating Site's standard practice
- Where
- 47 sites in United States
- Volunteers
- 64 people
- Sponsor
- Shire
Carcinoma, Basal Cell, Recurrent Skin Cancer
Pilot Study of Sonidegib and Buparlisib in Treating Patients With Advanced or Metastatic Basal Cell Carcinoma
This pilot trial studies how well sonidegib and buparlisib work in treating patients with basal cell carcinoma that has spread to other places in the body.
- Conditions
- Carcinoma, Basal Cell, Recurrent Skin Cancer, Skin Neoplasms, Basal Cell Nevus Syndrome
- Trial name
- NCT02303041
- Testing
- Buparlisib, Sonidegib
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Anne Chang
Homozygous Familial Hypercholesterolemia
Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients With Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy
This is a Phase 3 single-arm, open-label, international, multi-center clinical trial to evaluate the efficacy and safety of lomitapide in pediatric patients with HoFH who are receiving stable lipid-lowering therapy, including LDL…
- Trial name
- NCT02765841
- Testing
- Lomitapide
- Sponsor
- Aegerion Pharmaceuticals, Inc.
Multiple Sclerosis, Exercise Intervention
Regular Exercise Improves Physical Capacity and Promotes Neurotrophins in Patients With Multiple Sclerosis
ABSTRACT Objective: The investigators aimed to determine the effect of regular exercise on aerobic capacity, strength values, and plasma levels of nerve growth factor (NGF) and Neurotrophin-3 (NT-3) in patients with multiple…
- Conditions
- Multiple Sclerosis, Exercise Intervention, Neuro-Degenerative Disease
- Trial name
- NCT04944251
- Testing
- Regular Exercise
- Where
- Turkey (Türkiye)
- Volunteers
- 43 people
- Sponsor
- Ege University
Autosomal Dominant Polycystic Kidney, ADPKD
Study of the Efficacy and Safety of Tesevatinib in Subjects With ADPKD
The goal of the study was to compare and evaluate safety and efficacy of tesevatinib 50 milligrams (mg) versus placebo in participants with autosomal dominant polycystic kidney disease (ADPKD).
- Trial name
- NCT03203642
- Testing
- Tesevatinib
- Where
- 19 sites in United States
- Volunteers
- 80 people
- Sponsor
- Kadmon, a Sanofi Company
Healthy Volunteers, Cryopyrin Associated Periodic Syndrome
Safety and Tolerability, Pharmacokinetic and Pharmacodynamic Study With IZD334
This is a first in human (FIH), single-centre, double -blind, randomised, cross-over, SAD followed by a MAD study of IZD334 conducted in healthy adult participants as well as an open-label cohort in adult patients with CAPS.
- Trial name
- NCT04086602
- Testing
- IZD334
- Where
- Australia
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- Inflazome UK Ltd
Cryopyrin-Associated Periodic Syndromes, Familial Cold Autoinflammatory Syndrome
Efficacy and Safety of ACZ885 in Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease
This will provided long-term safety and efficacy data for ACZ885 (a fully human anti-interleukin-1β [anti-IL-1β] monoclonal antibody) given as an injection subcutaneously in patients who participated in the CACZ885A2102…
- Conditions
- Cryopyrin-Associated Periodic Syndromes, Familial Cold Autoinflammatory Syndrome, Muckle Wells Syndrome, Neonatal Onset Multisystem Inflammatory Disease
- Trial name
- NCT00685373
- Testing
- Canakinumab (ACZ885)
- Where
- 28 sites in United States, Belgium and 7 more
- Volunteers
- 166 people
- Sponsor
- Novartis
Duchenne Muscular Dystrophy (DMD)
A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
The study consists of 3 periods: A Screening Period (up to 45 days), a double-blind, placebo-controlled Multiple Ascending Dose (MAD) Period (28 weeks), and a Long-Term Extension (LTE) Period (108 weeks).
- Trial name
- CONNECT2-EDO51 (NCT06833931)
- Testing
- IV infusion
- Sponsor
- PepGen Inc
Graft Versus Host Disease, Leukemia
Laboratory-Treated Donor Bone Marrow in Treating Patients Who Are Undergoing a Donor Bone Marrow Transplant for Hematologic Cancer
RATIONALE: Giving chemotherapy and total-body irradiation before a donor bone marrow transplant or peripheral blood stem cell transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune…
- Conditions
- Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00265837
- Testing
- graft versus host disease prophylaxis/therapy, allogeneic bone marrow transplantation, in vitro-treated bone marrow transplantation
- Where
- United States
- Volunteers
- 72 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Pneumonia, Bronchoalveolar Lavage
HCAPPED I: HCAP-Pcr in Emergency Department
Prospective Observational Study (on Diagnostic Procedure) - Single-Center, Non-Profit. A pilot study will be conducted on a prospective cohort of patients with HCAP (Healthcare-Associated Pneumonia).
- Conditions
- Pneumonia, Bronchoalveolar Lavage, Antimicrobial Treatment, Sputum
- Trial name
- HCAPPED-I (NCT06506617)
- Where
- Italy
- Volunteers
- 93 people
- Sponsor
- Azienda Ospedaliero-Universitaria Careggi
Clear Cell Renal Cell Carcinoma, VHL Syndrome
Overtaking Intra and Inter Tumoral Heterogeneity In Von Hippel-Lindau Related Renal Cancer
The goal of this study is to comprehensively map intra and inter tumor heterogeneity of ccRCC in VHL patients through the use of imaging, molecular biology and genomics techniques.
- Trial name
- ITHORinVHL (NCT06195150)
- Testing
- Blood collection
- Where
- Italy
- Volunteers
- 3 people
- Sponsor
- IRCCS San Raffaele
Homozygous Familial Hypercholesterolemia (HoFH)
A Study to Evaluate the Safety of Rosuvastatin in Children and Adolescents With Homozygous Familial Hypercholesterolemia
The purpose of the study is to evaluate the safety of Rosuvastatin in Children and Adolescents with Homozygous Familial Hypercholesterolemia.
- Trial name
- NCT02434497
- Testing
- Rosuvastatin 20mg
- Where
- 6 sites in Belgium, Canada and 4 more
- Volunteers
- 9 people
- Sponsor
- AstraZeneca
Chronic Myeloproliferative Disorders, Leukemia
Donor Stem Cell Transplant in Treating Older or Frail Patients With Hematologic Cancer
RATIONALE: Giving low doses of chemotherapy, such as fludarabine and busulfan, before a donor bone marrow or peripheral blood stem cell transplant helps stop the growth of cancer cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00296023
- Testing
- anti-thymocyte globulin, filgrastim, therapeutic allogeneic lymphocytes
- Where
- 2 sites in United States
- Volunteers
- 25 people
- Sponsor
- University of California, San Francisco