Clinical trials: Genetic Diseases
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
Major Depressive Disorder (MDD, Anxiety Disorder, Generalized
Serotonin Variants & Gut Microbiota in Mental Health
This observational pilot study investigates whether a broad panel of genetic variants relevant to the serotonin pathway (including serotonergic system, methylation cycle, intestinal barrier integrity, and inflammatory response…
- Conditions
- Major Depressive Disorder (MDD, Anxiety Disorder, Generalized, Stress Disorders, Post-Traumatic, Panic Disorder
- Trial name
- NCT07634458
- Testing
- SNP Genotyping and Gut Metagenomics
- Volunteers
- 30 people
- Sponsor
- Neurobiologix
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
Peripheral T-cell Lymphoma
A Study of Brentuximab Vedotin and CHP in Frontline Treatment of PTCL With Less Than 10% CD30 Expression
This clinical trial will study brentuximab vedotin with CHP to find out if the drugs work for people who have certain types of peripheral T-cell lymphoma (PTCL).
- Trial name
- NCT04569032
- Testing
- brentuximab vedotin, cyclophosphamide, doxorubicin
- Where
- 63 sites in United States, France and 3 more
- Volunteers
- 82 people
- Sponsor
- Seagen, a wholly owned subsidiary of Pfizer
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
Hemophilia A or B
PF-06741086 Long-term Treatment in Severe Hemophilia
This study is designed to evaluate the safety, tolerability and efficacy of long-term treatment with PF-06741086 in subjects with severe hemophilia who participated in the 3-month Phase 1b/2 B7841002 study.
- Trial name
- NCT03363321
- Testing
- PF-06741086
- Where
- 8 sites in United States, Brazil and 5 more
- Volunteers
- 20 people
- Sponsor
- Pfizer
Thyroid Eye Disease (TED)
MHB018A Treatment in Patients With Chronic Thyroid Eye Disease
The primary objective of this study is to investigate the efficacy, safety, and tolerability of MHB018A, a humanized anti-IGF1R antibody, administered Q4W for 6 months, in comparison to placebo, in the treatment of participants…
- Trial name
- Interventional (NCT07257185)
- Testing
- MHB018A injection
- Where
- China
- Volunteers
- 150 people
- Sponsor
- Minghui Pharmaceutical (Hangzhou) Ltd
Congenital Bleeding Disorder, Haemophilia A
Study for Turoctocog Alfa Treatment Regimen in Iraqi Haemophilia A Patients
The study has descriptive purposes, with aim of assessing how turoctocog alfa is used in the everyday practice and to provide a baseline for the management of haemophilia A and does not involve any change in the clinical…
- Trial name
- NCT06574984
- Testing
- Turoctocog alfa
- Where
- 7 sites in Iraq
- Volunteers
- 329 people
- Sponsor
- Novo Nordisk A/S
HIV Infections
Case-Control Viramune (Nevirapine) Toxicogenomics Study
Attempt to identify genetic polymorphisms in interrogated pathways which may be associated with symptomatic hepatotoxicity or severe cutaneous toxicity observed in case patients within the first 8 weeks of nevirapine therapy.
- Trial name
- NCT00310843
- Testing
- Nevirapine
- Where
- 102 sites in United States, Argentina and 9 more
- Volunteers
- 889 people
- Sponsor
- Boehringer Ingelheim
Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis
Efficacy and Safety of Vamifeport in Adult Participants With Homeostatic Iron Regulator Gene (HFE)-Related Hereditary Hemochromatosis
This is a phase 2, multicenter, randomized, placebo-controlled, double-blind, parallel-group, proof-of-concept study to assess vamifeport in adult participants with homeostatic iron regulator gene-related hereditary…
- Trial name
- NCT07332091
- Testing
- Vamifeport
- Where
- 100 sites in United States, Australia and 16 more
- Volunteers
- 84 people
- Sponsor
- CSL Behring
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