Clinical trials: Congenital Anomaly
Hereditary Angioedema, HAE
A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
This is a Phase 1/2, single-arm, open-label, dose-escalation and dose-expansion study of BMN 331 for the treatment of hereditary angioedema (HAE) due to C1 Esterase Inhibitor (C1-INH) protein deficiency.
- Trial name
- HAErmony-1 (NCT05121376)
- Testing
- Dose 1 of BMN 331, Dose 2 of BMN 331, Dose 3 of BMN 331
- Where
- 16 sites in United States, Australia and 1 more
- Volunteers
- 44 people
- Sponsor
- BioMarin Pharmaceutical
Healthy Volunteer, Becker Muscular Dystrophy
A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults
EDG-5506 is an investigational product intended to protect and improve function of dystrophic muscle fibers. This Phase 1 study of EDG-5506 will assess the safety, tolerability, and pharmacokinetics (PK) and of EDG-5506 in adult…
- Trial name
- NCT04585464
- Testing
- EDG-5506
- Where
- United States
- Volunteers
- 127 people, healthy volunteers welcome
- Sponsor
- Edgewise Therapeutics, Inc.
MRSA, Cystic Fibrosis
AeroVanc in the Treatment of Methicillin-resistant Staphylococcus Aureus Infection in Patients With Cystic Fibrosis
This is a multi-center, randomized phase III study to evaluate the clinical effectiveness of AeroVanc in persistent methicillin-resistant Staphylococcus aureus (MRSA) infection in patients with cystic fibrosis (CF).
- Trial name
- NCT03181932
- Testing
- Vancomycin inhalation powder
- Where
- 71 sites in United States, Canada
- Volunteers
- 188 people
- Sponsor
- Savara Inc.
Hypophosphatasia, Bone Disease
Non-interventional Substudy of ALX-HPP-502 to Assess Natural History of Patients With Juvenile-onset HPP Who Served as Historical Controls in ENB-006-09
The purpose of this study is to characterize the natural history of HPP in patients with Juvenile-onset HPP who served as historical controls in ENB-006-09.
- Trial name
- NCT02235493
- Testing
- Retrospective Case Only
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Exocrine Pancreatic Insufficiency
Phase 1 Study to Assess Safety and Efficacy of ANG003
Randomized, parallel, active-treatment Phase 1 study of a single dose of orally administered ANG003 with a test meal in adult subjects with cystic fibrosis-related exocrine pancreatic insufficiency.
- Trial name
- NCT06052293
- Testing
- ANG003
- Where
- 20 sites in United States
- Volunteers
- 57 people
- Sponsor
- Anagram Therapeutics, Inc.
Teeth Loss, Bone Loss
Maxillary Sinus Elevation With Bio-Oss or Straumann BoneCeramic
The purpose of the study was to test a clinically relevant non-inferiority of Straumann BoneCeramic treatment compared to Bio-Oss treatment 180 to 240 days after sinus grafting procedure.
- Trial name
- NCT00901121
- Testing
- Straumann BoneCeramic, Bio-Oss
- Where
- 8 sites in Italy
- Volunteers
- 48 people
- Sponsor
- Institut Straumann AG
Duchenne Muscular Dystrophy
Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old
This is a Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in younger DMD Patients.
- Trial name
- NCT06769633
- Testing
- Givinostat Hydrochloride, Givinostat Hydrochloride - Cohort 2
- Where
- 9 sites in Belgium, Italy and 2 more
- Volunteers
- 18 people
- Sponsor
- Italfarmaco
Spinocerebellar Ataxia Type 3
A Pharmacokinetics and Safety Study of BIIB132 in Adults With Spinocerebellar Ataxia 3
The primary objective of this study is to evaluate the safety and tolerability of multiple ascending doses of BIIB132 administered via intrathecal (IT) injection to participants with spinocerebellar ataxia type 3 (SCA3).
- Trial name
- NCT05160558
- Testing
- BIIB132
- Where
- 20 sites in United States, Germany and 4 more
- Volunteers
- 8 people
- Sponsor
- Biogen
Atopic Dermatitis
Safety Study Evaluating the Adrenal Suppression Potential of Product 0405 in Pediatric Subjects With Atopic Dermatitis
The aim of the trial is to assess Adrenal Suppression potential when pediatric subjects are administered Product 0405 for the treatment of Atopic Dermatitis
- Trial name
- 0405-01-04 (NCT01232543)
- Testing
- Product 0405
- Where
- United States
- Volunteers
- 114 people
- Sponsor
- Fougera Pharmaceuticals Inc.
Heart Failure, Dilated Cardiomyopathy
A Pivotal Trial to Establish the Efficacy and Safety of Algisyl in Patients With Moderate to Severe Heart Failure
AUGMENT-HF II is a study to evaluate the efficacy and safety of the Algisyl device. The purpose of this study is to investigate Algisyl employed as a method of left ventricular augmentation and restoration in patients with…
- Conditions
- Heart Failure, Dilated Cardiomyopathy, Heart Failure With Reduced Ejection Fraction
- Trial name
- AUGMENT-HFII (NCT03082508)
- Testing
- Algisyl, Standard Medical Therapy
- Volunteers
- 240 people
- Sponsor
- LoneStar Heart, Inc.
Primary Ciliary Dyskinesia
A Study to Assess the Safety, Tolerability, Ciliary Rescue, and Pharmacodynamics of RCT1100 in Adults With PCD
This is the second in-human study with RCT1100 and is designed to provide safety, tolerability and preliminary efficacy data for future clinical studies.
- Trial name
- NCT06600425
- Testing
- RCT1100
- Where
- 2 sites in United Kingdom
- Volunteers
- 7 people
- Sponsor
- ReCode Therapeutics
Cystic Fibrosis
Safety, Tolerability and Pharmacokinetics of BIIL 284 BS in Adult and Pediatric Cystic Fibrosis (CF) Patients
Safety, tolerability and pharmacokinetics following repeated doses (15-day dosing)
- Trial name
- NCT02269189
- Testing
- BIIL 284 BS, high dose, BIIL 284 BS, low dose
- Volunteers
- 36 people
- Sponsor
- Boehringer Ingelheim
Cystic Fibrosis
Safety, Tolerability, Pharmacokinetics, and Preliminary Pharmacodynamics of QBW251 in Healthy Subjects and Cystic Fibrosis Patients
This study is designed to assess the safety, tolerability, pharmacokinetics and preliminary pharmacodynamics (proof of concept) of QBW251 in healthy subjects and cystic fibrosis patients following single and multiple doses.
- Trial name
- NCT02190604
- Testing
- QBW251
- Where
- 23 sites in United States, Belgium and 5 more
- Volunteers
- 153 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
Neurologic Disorders, Nervous System Diseases
Neurologic Stem Cell Treatment Study
This is a human clinical study involving the isolation of autologous bone marrow derived stem cells (BMSC) and transfer to the vascular system and inferior 1/3 of the nasal passages in order to determine if such a treatment will…
- Conditions
- Neurologic Disorders, Nervous System Diseases, Neurodegenerative Diseases, Neurological Disorders
- Trial name
- NEST (NCT02795052)
- Testing
- Intravenous and Intranasal BMSC
- Where
- 3 sites in United States, United Arab Emirates
- Volunteers
- 500 people
- Sponsor
- MD Stem Cells
Hereditary Angioedema
A Study of Icatibant in Patients With Acute Attacks of Hereditary Angioedema (FAST-3)
This study is being conducted to evaluate the efficacy and safety of icatibant compared to placebo in patients experiencing acute attacks of hereditary angioedema (HAE).
- Trial name
- NCT00912093
- Testing
- Icatibant
- Where
- 64 sites in United States, Australia and 7 more
- Volunteers
- 98 people
- Sponsor
- Shire
Niemann-Pick Disease Type C, GM2 Gangliosidosis
Long-Term Safety and Tolerability of Nizubaglustat in Participants With Rare Lysosomal Disorders
This is an open-label extension trial to evaluate the long-term safety and tolerability of nizubaglustat (AZ-3102) in participants with rare lysosomal disorders who have participated in a previous nizubaglustat trial and wish to…
- Conditions
- Niemann-Pick Disease Type C, GM2 Gangliosidosis, GM1 Gangliosidosis, Gaucher Disease Type 3
- Trial name
- HARBOR (NCT07885917)
- Testing
- AZ-3102
- Volunteers
- 218 people
- Sponsor
- Azafaros B.V.
Atopic Dermatitis
Correlation Study Between PEST and SCORAD in Management of Atopic Dermatitis With Ceradan® Regimen
This is a prospective, open label, single arm, and observational and multicenter study to assess the correlation between PEST and SCORAD scores in the management of AD with the Ceradan® regimen.
- Trial name
- COPES (NCT02073591)
- Testing
- Ceradan Cream, Ceradan Wash
- Where
- 2 sites in Singapore
- Volunteers
- 58 people
- Sponsor
- Hyphens Pharma Pte Ltd
Myotonic Dystrophy 1
Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1
The primary purpose of the study is to evaluate the safety and tolerability of single intravenous (IV) doses of PGN-EDODM1 administered to participants with Myotonic Dystrophy Type 1 (DM1).
- Trial name
- FREEDOM-DM1 (NCT06204809)
- Testing
- PGN-EDODM1 for infusion
- Where
- 12 sites in United States, Canada and 1 more
- Volunteers
- 24 people
- Sponsor
- PepGen Inc
Cystic Fibrosis
A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis
The purpose of this study is to evaluate the safety, tolerability and efficacy of VX-121 combination therapy in subjects with cystic fibrosis (CF).
- Trial name
- NCT03912233
- Testing
- VX-121, TEZ, VX-561
- Where
- 26 sites in United States, Germany and 3 more
- Volunteers
- 87 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Asthma, COPD
A Clinical Study in Healthy Subjects to Evaluate 9MW1911 Injection
This is a Phase 1, randomized, placebo-controlled study, enrolling approximately 38 healthy adult subjects (18-65 yrs). The purpose of this study is to evaluate the safety, tolerability and PK of single ascending dose of 9MW1911…
- Conditions
- Asthma, COPD, Atopic Dermatitis
- Trial name
- NCT05170243
- Testing
- 9MW1911 Injection
- Where
- China
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- Mabwell (Shanghai) Bioscience Co., Ltd.
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.