Clinical trials: Congenital Disorder
Pruritus, Atopic Dermatitis
Study to Evaluate the Efficacy and Safety of Oral Difelikefalin as Adjunct Therapy to a Topical Corticosteroid for Moderate to Severe Pruritus in Subjects With Atopic Dermatitis
This is a two-part, multicenter, randomized, double-blind study to evaluate the efficacy and safety of oral difelikefalin as adjunct therapy to a topical corticosteroid (TCS) for moderate-to-severe pruritus in adult subjects with…
- Trial name
- KIND-1 (NCT05387707)
- Testing
- difelikefalin 0.25 mg, difelikefalin 0.5 mg, TCS Cream
- Where
- 61 sites in United States
- Volunteers
- 287 people
- Sponsor
- Cara Therapeutics, Inc.
Familial Adenomatous Polyposis (FAP)
A Safety Study of ST316 in Participants With Familial Adenomatous Polyposis (FAP).
This is a Phase 1b, open- label, dose-optimization study evaluating ST316 in adult participants with familial adenomatous polyposis (FAP) who have undergone colectomy and have recurrent polyps.
- Trial name
- FAP (NCT07729371)
- Testing
- ST316 IV
- Volunteers
- 40 people
- Sponsor
- Sapience Therapeutics
Infantile Colic
Lactobacillus Reuteri Supplementation in the Treatment of Infantile Colic
The purpose of this study is to evaluate the efficacy of the oral administration of Lactobacillus reuteri in treating infantile colic as defined by the Rome III criteria.
- Trial name
- PROBIOC (NCT01887444)
- Testing
- Lactobacillus reuteri DSM17938 probiotic
- Where
- France
- Sponsor
- BioGaia AB
Congenital Bleeding Disorder, Haemophilia A
Investigating Safety and Pharmacokinetics of NNC 0128-0000-2011 Compared to NNC 0128-0000-2021 in Healthy Male Subjects
This trial is conducted in Europe. The aim of this trial is to investigate the safety and pharmacokinetics (the rate at which the body eliminates the trial drug) of NNC 0128-0000-2011 compared to NNC 0128-0000-2021 when given for…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
- Trial name
- NCT01272206
- Testing
- NNC 0128-0000-2011, NNC 0128-0000-2021
- Where
- United Kingdom
- Volunteers
- 12 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
Pharmacokinetic Comparison of Advate rAHF-PFM With Recombinate rAHF in Patients With Severe Hemophilia A
The purpose of this study is to compare the pharmacokinetic parameters and safety of Advate rAHF-PFM versus Recombinate rAHF in well described previously treated patients with severe hemophilia A (factor VIII level < 1%).
- Trial name
- NCT00666406
- Testing
- Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method (rAHF-PFM), Recombinant Factor VIII (rAHF)
- Where
- Germany
- Volunteers
- 9 people
- Sponsor
- Baxalta now part of Shire
Prader-Willi Syndrome
A Study of Oral ARD-101 in Patients With Prader-Willi Syndrome
A Phase 2, Single-Arm, Open-Label Study to Evaluate the Safety and Efficacy of ARD-101 in Patients with Prader-Willi Syndrome
- Trial name
- NCT05153434
- Testing
- ARD-101
- Where
- 2 sites in United States
- Volunteers
- 19 people
- Sponsor
- Aardvark Therapeutics, Inc.
Pompe Disease, Glycogen Storage Disease Type II (GSD II)
Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.
Primary Objective: To evaluate the safety and tolerability of neoGAA in treatment naïve and alglucosidase alfa treated late-onset Pompe disease patients.
- Conditions
- Pompe Disease, Glycogen Storage Disease Type II (GSD II), Acid Maltase Deficiency
- Trial name
- NCT01898364
- Testing
- GZ402666
- Where
- 17 sites in United States, Belgium and 5 more
- Volunteers
- 24 people
- Sponsor
- Genzyme, a Sanofi Company
Solid Tumor, Renal Cell Carcinoma (Kidney Cancer)
A Study of NDI 1150-101 in Patients With Solid Tumors
This study is to determine the maximum tolerated dose (MTD) and the recommended Phase 2 dose (RP2D) and to investigate the safety, pharmacokinetics (PK), pharmacodynamics, and preliminary antitumor activity of NDI-101150 given as…
- Conditions
- Solid Tumor, Renal Cell Carcinoma (Kidney Cancer), Gastric and Gastroesophageal Junction (GEJ) Adenocarcinoma, Non-Small Cell Lung Cancer
- Trial name
- NCT05128487
- Testing
- NDI-101150, Pembrolizumab
- Where
- 19 sites in United States
- Volunteers
- 106 people
- Sponsor
- Nimbus Saturn, Inc.
Colorectal Adenocarcinoma
A Study to Evaluate the Efficacy of VVD-133214 in Combination With Pembrolizumab Compared With Pembrolizumab Alone in Participants With Advanced Cancer of the Colon or Rectum
Researchers are looking for a better way to treat people with advanced colorectal cancer. Colorectal cancer (cancer of the colon or rectum) is one of the most common cancers worldwide.
- Trial name
- NCT07811193
- Testing
- VVD-133214, Pembrolizumab
- Where
- 122 sites in United States, Argentina and 20 more
- Volunteers
- 140 people
- Sponsor
- Vividion Therapeutics, Inc.
Fabry Disease
A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme.
- Trial name
- NCT00074984
- Testing
- Fabrazyme (agalsidase beta)
- Where
- 26 sites in United States, Canada and 4 more
- Volunteers
- 82 people
- Sponsor
- Genzyme, a Sanofi Company
Atopic Dermatitis
A First-In-Human Study to Evaluate the Safety, Tolerability, and Efficacy of Si-544 in Adults With Atopic Dermatitis
This is a multi-center, Phase 1b, double-blind, placebo-controlled, SAD and MAD, first-in-human study in subjects with mild to severe AD receiving si-544. The study consists of 2 parts, an SAD and an MAD part.
- Trial name
- NCT05383378
- Testing
- si-544
- Where
- 5 sites in Germany
- Volunteers
- 36 people
- Sponsor
- selectION Therapeutics GmbH
Osteoarthritis, Arthritis
Periprosthetic Bone Mineral Density After Total Hip Arthroplasty Performed Through a Minimally Invasive Anterior Approach (AMIS) With Either an AMIStem or a Quadra Femoral Component
This is a monocentric, prospective, randomized clinical survey to verify if the periprosthetic remodelling brought about either the AMIStem or the Quadra femoral component is equivalent.
- Conditions
- Osteoarthritis, Arthritis, Avascular Necrosis, Fracture of the Femoral Neck or Head
- Trial name
- NCT01285843
- Testing
- Anterior Minimally Invasive Approach (AMIS)
- Where
- Germany
- Volunteers
- 40 people
- Sponsor
- Medacta International SA
Primary Hyperoxaluria Type 1, PH1
Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1)
This study will be conducted to evaluate the efficacy and safety of YOLT-203 in children and adults with Primary Hyperoxaluria Type 1.
- Trial name
- NCT07587021
- Testing
- YOLT-203
- Where
- 4 sites in United States
- Volunteers
- 36 people
- Sponsor
- YolTech Therapeutics Co., Ltd
Breast Cancer
Validation of Molecular Diagnostic Assays to Detect Cancer Biomarkers in Blood & Primary Tumor in ER+/HER2- MBC
Conventional biopsy and surgical tumor resection are invasive procedures that capture only one instance of the progression of the tumor. However, the genome of tumor is not static, but it is constantly altered during treatment.
- Trial name
- NCT04480814
- Testing
- PIK3CA kit
- Where
- 4 sites in Greece
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Pharmassist Ltd
HFpEF - Heart Failure With Preserved Ejection Fraction, HFmrEF
US Benchmarking Clinical Study
The goal of this observational study is to measure shear wave velocity (SWV) in patients with non-reduced left ventricular ejection fraction (LVEF) heart failure and left ventricular hypertrophy, with or without transthyretin…
- Conditions
- HFpEF - Heart Failure With Preserved Ejection Fraction, HFmrEF, ATTR-CM (Transthyretin Amyloid Cardiomyopathy), Left Ventricular Hypertrophy
- Trial name
- EMS-CT-1 (NCT07215715)
- Where
- 3 sites in United States
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- eMyosound SAS
Atopic Dermatitis
A Study on the Bioavailability of SHR-1819 Injection Administered Subcutaneously Using Different Injection Devices in Healthy Subjects
This study is a randomized, parallel-group, open-label Phase I clinical trial designed to compare the bioavailability and safety of subcutaneous SHR-1819 injection administered via different injection devices in healthy subjects.
- Trial name
- NCT07264335
- Testing
- SHR-1819 Injection
- Where
- China
- Volunteers
- 160 people, healthy volunteers welcome
- Sponsor
- Guangdong Hengrui Pharmaceutical Co., Ltd
Dermatitis, Atopic
Efficacy and Safety of AN2898 and AN2728 Topical Ointments to Treat Mild-to-Moderate Atopic Dermatitis
The purpose of this study is to determine whether AN2898 and AN2728 ointments are safe and effective treatments for mild-to-moderate atopic dermatitis (AD).
- Trial name
- NCT01301508
- Testing
- AN2728 ointment, 2%, AN2898 ointment, 1%, AN2898 ointment vehicle
- Where
- 13 sites in Australia
- Volunteers
- 46 people
- Sponsor
- Pfizer
Atopic Dermatitis
Phase II Clinical Study to Evaluate the Efficacy and Safety of VC005 in Adult Subjects With Mild to Moderate Atopic Dermatitis
A multicenter, randomized, double-blind, vehicle controlled phase II clinical study
- Trial name
- NCT06891040
- Testing
- VC005 groups
- Where
- China
- Volunteers
- 118 people
- Sponsor
- Jiangsu vcare pharmaceutical technology co., LTD
Alpha-1 Antitrypsin Deficiency
Respreeza® Self-administration and Learning Program (AmAREtTI Study)
According to the Respreeza® Summary of Product Characteristics, the initial infusions must be administered under the supervision of a health professional experienced in the treatment of alpha-1 antitrypsin deficiency, although…
- Trial name
- AmAREtTI (NCT04262284)
- Where
- 11 sites in France
- Volunteers
- 15 people
- Sponsor
- CSL Behring
Niemann-Pick Disease, Type C
Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease
This is a Phase-2, multicenter, multiple dose, open-label, 2-part evaluation study which will primarily assess the safety and tolerability of VTS-270 (2-hydroxypropyl beta-cyclodextrin [HP-β-CD]) in pediatric participants with…
- Trial name
- NCT03687476
- Testing
- VTS-270
- Sponsor
- Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company
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