Clinical trials: Congenital Disorder
Hereditary Angioedema (HAE)
A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE) in the Kingdom of Saudi Arabia
Hereditary angioedema (HAE) is a rare condition. It causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals.
- Trial name
- NCT07263685
- Testing
- No intervention
- Where
- Saudi Arabia
- Volunteers
- 50 people
- Sponsor
- Takeda
Hemophilia A
Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Congenital Hemophilia A
This study is to test whether the study drug (OBI-1) is safe and effective for the treatment of serious bleeding episodes in people with congenital hemophilia A.
- Trial name
- NCT01434511
- Testing
- OBI-1
- Where
- 3 sites in United States, South Africa and 1 more
- Volunteers
- 1 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A
A Long-term Study of ADYNOVI/ADYNOVATE in Participants With Haemophilia A
The main aim of this study is to check for long-term side effects from ADYNOVI/ADYNOVATE prophylaxis in participants with haemophilia A when used under standard clinical practice in the real-world clinical setting.
- Trial name
- NCT04158934
- Testing
- ADYNOVI/ADYNOVATE
- Where
- 36 sites in United States, Bulgaria and 11 more
- Volunteers
- 207 people
- Sponsor
- Baxalta now part of Shire
Retinitis Pigmentosa
Object Finder for a Retinal Prosthesis
The proposed project seeks to provide object recognition as a feature in a retinal implant system. Participants will be able to direct an object recognition application to find a desired object in the field of view of the…
- Trial name
- NCT04319809
- Testing
- Object recognition subsystem
- Where
- United States
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- Minnesota HealthSolutions
Hereditary Angioedema
Long-Term Follow-Up (LTFU) of Subjects Treated With NTLA 2002
This is a follow-up study of subjects who received NTLA-2002 in a previous clinical trial as an observational evaluation of the long-term effects of the investigational therapy.
- Trial name
- NCT06262399
- Where
- 7 sites in Australia, France and 4 more
- Volunteers
- 100 people
- Sponsor
- Intellia Therapeutics
Arrhythmogenic Right Ventricular Dysplasia, Arrhythmogenic Left Ventricular Cardiomyopathy
IC14 (Atibuclimab) in Arrhythmogenic Cardiomyopathy
The goal of this clinical trial is to test IC14 (atibuclimab) in patients with arrhythmogenic cardiomyopathy (ACM) and who have an implantable cardoverter/defibrillator in place.
- Conditions
- Arrhythmogenic Right Ventricular Dysplasia, Arrhythmogenic Left Ventricular Cardiomyopathy, Arrhythmogenic Right Ventricular Cardiomyopathy 1, Arrhythmogenic Cardiomyopathy
- Trial name
- NCT06275893
- Testing
- IC14
- Where
- 2 sites in United States
- Volunteers
- 1 people
- Sponsor
- Implicit Bioscience
Healthy Volunteers
A Study Evaluating Relative Bioavailability of an Oral Suspension of Abrocitinib and Effect of an Acid Reducing Agent on the Bioavailability of Abrocitinib and Assessing the Taste of Abrocitinib Oral Formulations.
This study consists of 2 parts: Part A is to estimate the relative bioavailability of a single 200 mg dose of abrocitinib oral suspension (Test formulation) compared to the commercial abrocitinib tablet (200 mg) (Reference…
- Trial name
- NCT04903093
- Testing
- Abrocitinib tablet, Abrocitinib Suspension F1, Abrocitinib Suspension F2
- Where
- United States
- Volunteers
- 19 people, healthy volunteers welcome
- Sponsor
- Pfizer
Hereditary Angioedema (HAE)
A Study About the Diagnosis and Management of Hereditary Angioedema (HAE) in Egypt
Hereditary angioedema (HAE) is a rare condition. It causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals. This swelling happens because of a temporary leak in blood vessels.
- Trial name
- EXPLORHAE (NCT07218393)
- Testing
- No intervention
- Where
- 7 sites in Egypt
- Volunteers
- 100 people
- Sponsor
- Takeda
FSHD - Facioscapulohumeral Muscular Dystrophy
Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD)
The goal of this clinical trial is to learn about how an umbilical cord lining-derived stem cell product (ULSC) performs when treating Facioscapulohumeral Muscular Dystrophy (FSHD) 1 or 2.
- Trial name
- NCT07086521
- Testing
- ULSC
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Restem, LLC.
Hemophilia A
A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants With Inhibitors
This multicenter, open-label study will evaluate the safety, efficacy and pharmacokinetics of prophylactic emicizumab treatment in participants previously treated with episodic or prophylactic bypassing agents.
- Trial name
- HAVEN 1 (NCT02622321)
- Testing
- Emicizumab, rFVIIa, aPCC
- Where
- 44 sites in United States, Australia and 12 more
- Volunteers
- 113 people
- Sponsor
- Hoffmann-La Roche
Neurofibromatosis Type 1
A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1
The trial will be an open label, single arm, phase 2 study to assess the tolerability and efficacy of HLX-1502 in participants with NF1 that are 16 years or older in age with progressive and/or symptomatic PN.
- Trial name
- INSPIRE-NF1 (NCT06541847)
- Testing
- HLX-1502
- Where
- 14 sites in United States
- Volunteers
- 25 people
- Sponsor
- Healx Limited
Lennox-Gastaut Syndrome
Post-marketing Surveillance of Long-term Administration of Inovelon Tablets in Patients With Lennox-Gastaut Syndrome
This surveillance's objectives are 1. Unknown adverse reactions 2. Incidences of adverse drug reaction 3. Efficacy during long-term administration 4. Factors considered to have effect to safety and effectiveness 5.
- Trial name
- NCT02175173
- Testing
- Rufinamide
- Where
- 2 sites in Japan
- Volunteers
- 707 people
- Sponsor
- Eisai Co., Ltd.
Cystic Fibrosis
Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor
Study 110 is a Phase 3, multicenter study in subjects aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation and who participated in Study 109…
- Trial name
- NCT02544451
- Testing
- LUM/IVA
- Where
- 60 sites in United States, Australia and 7 more
- Volunteers
- 246 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Fractures, Bone, Impending Fracture
Retrospective Study on Clinical Performance and Safety Profile of Agile Nail
This study aims to retrospectively collect data from routine clinical practice in order to evaluate the safety profile of the Agile intramedullary nail, used in pediatric patients according to the manufacturer Instructions For…
- Conditions
- Fractures, Bone, Impending Fracture, Deformity; Bone
- Trial name
- Agile (NCT06412939)
- Testing
- Agile Nail
- Where
- United Kingdom
- Volunteers
- 20 people
- Sponsor
- Orthofix s.r.l.
Prader-Willi Syndrome, Obesity
Double-Blind, Placebo Controlled, Phase 3 Trial of ZGN-440 (Beloranib) in Obese Subjects With Prader-Willi Syndrome
The purpose of this study is to evaluate efficacy and safety of ZGN-440 (beloranib) in obese adolescent and adult subjects with Prader-Willi Syndrome.
- Trial name
- bestPWS (NCT02179151)
- Testing
- ZGN-440 for Injectable Suspension
- Where
- 15 sites in United States
- Volunteers
- 108 people
- Sponsor
- Zafgen, Inc.
Atopic Dermatitis
Delgocitinib Cream for the Treatment of Moderate to Severe Atopic Dermatitis During 8 Weeks in Adults, Adolescents, and Children
This is a multi-center, open-label trial to evaluate safety and pharmacokinetics of topical delgocitinib cream applied to pediatric subjects (2-17 years) and adult subjects (18 years and above) with atopic dermatitis.
- Trial name
- NCT03826901
- Testing
- delgocitinib
- Where
- 16 sites in United States, Canada
- Volunteers
- 46 people
- Sponsor
- LEO Pharma
Hereditary Angioedema
C1 Esterase Inhibitor (C1INH-nf) for the Treatment of Acute Hereditary Angioedema (HAE) Attacks
The study objective was to determine the safety and efficacy of C1INH-nf for the treatment of acute HAE attacks.
- Trial name
- NCT00289211
- Testing
- C1 esterase inhibitor [human] (C1INH-nf)
- Where
- 37 sites in United States
- Volunteers
- 83 people
- Sponsor
- Shire
Cystic Fibrosis, Bronchiectasis
A Study to Test How Well BI 1291583 is Tolerated by People With Cystic Fibrosis Bronchiectasis (Clairafly™)
This study is open to adults aged 18 years and older with cystic fibrosis bronchiectasis. The purpose of this study is to find out whether a medicine called BI 1291583 is tolerated by people with cystic fibrosis bronchiectasis.
- Trial name
- NCT05865886
- Testing
- BI 1291583
- Where
- 24 sites in United States, Belgium and 5 more
- Volunteers
- 22 people
- Sponsor
- Boehringer Ingelheim
Prader-Willi Syndrome
Open-Label Extension Study of DCCR in PWS Followed by Double-Blind, Placebo-Controlled, Randomized Withdrawal Period
This is a multi-center, multi-period study with an open-label period followed by a double-blind, placebo-controlled, randomized withdrawal period evaluating the safety and efficacy of DCCR treatment.
- Trial name
- NCT03714373
- Testing
- DCCR
- Where
- 28 sites in United States, United Kingdom
- Volunteers
- 115 people
- Sponsor
- Soleno Therapeutics, Inc.
Alzheimer's Disease, Huntington's Disease
A Phase I Study To Estimate The Effect Of Ketoconazole And Omeprazole On The Pharmacokinetics Of Dimebon In Healthy Subjects Who Are Normal Or Poor CYP2D6 Metabolizers
This study will evaluate the potential for a drug-drug interaction of Dimebon with ketoconazole and omeprazole, potent inhibitors of the drug metabolizing enzymes CYP3A4 and CYP2C19, respectively.
- Trial name
- NCT00931073
- Testing
- Dimebon alone, Dimebon + Ketoconazole, Dimebon + Omeprazole
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Pfizer
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