Clinical trials: Genetic Diseases
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
Hemophilia A
Impact on French Physician's Haemophilia Treatment Management Decision Based on Systematic Joint Examination
This low interventional study aims to describe if and how the haemophilia treatment management decisions are impacted by a systematic joint examination (ultrasound, functional, physical) in patients with haemophilia A in France.
- Trial name
- A-MOVE (NCT04133883)
- Testing
- Ultrasound, Haemophilia Joint Health Score
- Where
- 20 sites in France
- Volunteers
- 88 people
- Sponsor
- Swedish Orphan Biovitrum
Atopic Dermatitis
A Multicenter, Randomized, Double-blind, Vehicle Controlled Phase II Clinical Study to Evaluate the Efficacy and Safety of VC005 Gel in Adult Subjects With Mild to Moderate Atopic Dermatitis
This is a multicenter, randomized, double-blind, vehicle controlled phase II clinical study to evaluate the efficacy and safety of VC005 gel in adult subjects with mild to moderate atopic dermatitis
- Trial name
- NCT07598383
- Testing
- VC005 group
- Where
- China
- Volunteers
- 120 people
- Sponsor
- Jiangsu vcare pharmaceutical technology co., LTD
Atopic Dermatitis, Atopic
A Study of the Effects of Camoteskimab in Adults With Moderate to Severe Atopic Dermatitis
This is a Phase 2a, multicenter, randomized, double-blind, placebo-controlled study with an open-label extension to evaluate the efficacy and safety of camoteskimab in adults with moderate to severe AD.
- Conditions
- Atopic Dermatitis, Atopic, Dermatitis, Dermatologic Disease
- Trial name
- NCT06436183
- Testing
- Camoteskimab
- Where
- 35 sites in United States, Canada
- Volunteers
- 62 people
- Sponsor
- Apollo Therapeutics Ltd
Hemochromatosis
Study With Deferiprone and/or Desferrioxamine in Iron Overloaded Patients
Comparison of efficacy and toxicity of the combination treatment of deferiprone and desferrioxamine with the single agent treatment of either drug
- Trial name
- NCT00350662
- Testing
- Deferiprone (L1), Desferrioxamine
- Where
- 2 sites in Egypt, Turkey (Türkiye)
- Volunteers
- 95 people
- Sponsor
- Lipomed
Genetic Disorders
Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
The purpose of this multi-center study is to explore the efficacy, safety, tolerability and pharmacokinetics/pharmacodynamics of recombinant human C1 inhibitor in the treatment of acute attacks in patients with hereditary…
- Trial name
- NCT00262288
- Testing
- i.v. recombinant human C1 inhibitor
- Where
- Netherlands
- Volunteers
- 14 people
- Sponsor
- Pharming Technologies B.V.
Pulmonary Hypertension
Efficacy and Safety of Bosentan in Sickle Cell Disease (SCD) Patients Diagnosed With Pulmonary Hypertension (PH)
The study will assess the effect of bosentan on pulmonary vascular resistance and exercise capacity in Sickle Cell Disease (SCD) patients diagnosed with Pulmonary Hypertension.
- Trial name
- ASSET-2 (NCT00313196)
- Testing
- bosentan
- Volunteers
- 12 people
- Sponsor
- Actelion
X-Linked Hypohidrotic Ectodermal Dysplasia
Phase 2 Study to Evaluate Safety, Pharmacokinetics, Immunogenicity and Pharmacodynamics/Efficacy of EDI200 in Male Infants With X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)
This Phase 2 first-in-neonate EDI200 study will enroll treatment-naïve, XLHED-affected male newborns in the first two weeks of life.
- Trial name
- ECP-002 (NCT01775462)
- Testing
- EDI200
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 6 people
- Sponsor
- Edimer Pharmaceuticals
Prostate Cancer
A Trial to Learn How Safe AZD9750 is and How Well it Works in People With Metastatic Prostate Cancer When Given With or Without Other Anticancer Drugs
ANDROMEDA is a first-in-human, Phase I/II, open-label, multicenter study of AZD9750 in participants with metastatic prostate cancer.
- Trial name
- ANDROMEDA (NCT07336446)
- Testing
- AZD9750, AZD5305
- Where
- 5 sites in United States, Australia and 2 more
- Volunteers
- 6 people
- Sponsor
- AstraZeneca
Cystic Fibrosis
A Study Evaluating the Long-Term Safety of Elexacaftor Combination Therapy
This study evaluate the long-term safety and tolerability of elexacaftor (ELX)/tezacaftor (TEZ)/ ivacaftor (IVA) triple combination (TC) in participants with cystic fibrosis (CF) who are homozygous for F508del.
- Trial name
- NCT04362761
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 29 sites in Australia, Belgium and 2 more
- Volunteers
- 172 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Hemophilia A
Patient Functioning and Well-being, Economic, and Clinical Impact of Hemophilia A and Its Treatment
The purpose of the study is to improve the understanding of key patient reported outcomes such as quality of life as well as clinical outcomes in hemophilia A, in a global real world setting.
- Trial name
- ECHO (NCT02396862)
- Testing
- Available Recombinant, and Human Factor VIII products incl. Kogenate FS (Recombinant Factor VIII, BAY14-2222)
- Where
- 10 sites in United States, Japan and 2 more
- Volunteers
- 272 people
- Sponsor
- Bayer
Pediatric Solid Tumor, Pediatric Lymphoma
Dose Escalation Study of CLR 131 in Pediatric Relapsed/Refractory Malignant Tumors Including Neuroblastoma and Sarcomas
The study evaluates CLR 131 in children, adolescents, and young adults with relapsed or refractory malignant solid tumors and lymphoma and recurrent or refractory malignant brain tumors for which there are no standard treatment…
- Conditions
- Pediatric Solid Tumor, Pediatric Lymphoma, Pediatric Brain Tumor, DIPG
- Trial name
- CLOVER-2 (NCT03478462)
- Testing
- CLR 131
- Where
- 8 sites in United States, Australia and 1 more
- Volunteers
- 30 people
- Sponsor
- Cellectar Biosciences, Inc.
Alpha-Mannosidosis
Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3
The goal of this observational study is to learn the effects of the drug velmanase alfa (Lamzede®) in the bodies of children under the age of 3 with Alpha-Mannosidosis.
- Trial name
- LAMPO(0-3) (NCT06184503)
- Testing
- Velmanase Alfa
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Urea Cycle Disorder
A Safety and Efficacy Study of Infusions of HepaStem in Urea Cycle Disorders Pediatric Patients
This is a phase2, prospective, open label study designed to investigate the safety and efficacy of several infusions of HepaStem. This study will include 5 pediatric Urea Cycle Disorder (UCD) patients under 12 years old.
- Trial name
- NCT03884959
- Testing
- HepaStem Infusion
- Where
- South Korea
- Sponsor
- HLB Cell Co., Ltd.
Cystic Fibrosis, Pseudomonas Aeruginosa Airway Infection
Aztreonam Lysine for Inhalation in Patients With Cystic Fibrosis and Pseudomonas Aeruginosa Airway Infection
The primary objective of this program is to provide expanded access to aztreonam lysine for inhalation (AZLI) 75 mg prior to its commercial availability to patients with cystic fibrosis (CF) and chronic P.
- Trial name
- NCT00499720
- Testing
- Aztreonam Lysine for Inhalation
- Where
- 65 sites in United States, Puerto Rico
- Sponsor
- Gilead Sciences
Thyroid Eye Disease
An Open-label Study (OLE) for Non-responders of VRDN-001-101 and VRDN-001-301
The investigational drug, VRDN-001, is a monoclonal antibody that inhibits the activity of a cell surface receptor called insulin-like growth factor-1 receptor (IGF-1R).
- Trial name
- OLE (NCT06179875)
- Testing
- Veligrotug
- Where
- 45 sites in United States, France and 6 more
- Volunteers
- 139 people
- Sponsor
- Viridian Therapeutics, Inc.
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