Clinical trials: Congenital Disorder
Congenital Bleeding Disorder, Haemophilia A With Inhibitors
A Trial Evaluating the Efficacy and Safety of Prophylactic Administration of Concizumab in Haemophilia A and B Patients With Inhibitors
This trial is conducted in Africa, Asia, Europe and North America. The aim of the trial is to assess the efficacy of concizumab administered s.c.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Trial name
- explorer™4 (NCT03196284)
- Testing
- Concizumab, Eptacog alfa
- Where
- 22 sites in United States, Austria and 12 more
- Volunteers
- 26 people
- Sponsor
- Novo Nordisk A/S
Cystic Fibrosis
A Study Comparing the Efficacy and Tolerability of Tobrineb®/Actitob®/Bramitob® Versus TOBI®
The objectives of the study are to demonstrate that Tobrineb®/Actitob®/Bramitob® is non-inferior to TOBI® in the primary efficacy variable, forced expiratory volume in one second (FEV1) percent of predicted normal, and to compare…
- Trial name
- CT03 Core (NCT00885365)
- Testing
- tobramycin / Bramitob, tobramycin / TOBI
- Where
- 44 sites in Czechia, France and 6 more
- Volunteers
- 324 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Sanfilippo Syndrome Type A (MPS IIIA)
A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients
MPS IIIA, also known as Sanfilippo A, is an inherited lysosomal storage disease (LSD). MPS IIIA is caused by a deficiency in sulfamidase, one of the enzymes involved in the lysosomal degradation of the glycosaminoglycan (GAG)…
- Trial name
- NCT03811028
- Testing
- SOBI003
- Where
- 3 sites in United States, Turkey (Türkiye)
- Volunteers
- 6 people
- Sponsor
- Swedish Orphan Biovitrum
Muscular Dystrophy, Duchenne
A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy
This is a multicenter, double blind, placebo controlled, multiple dose study to examine the safety and efficacy of MNK-1411 in male subjects 4 to 8 years of age (inclusive) with Duchenne Muscular Dystrophy (DMD).
- Trial name
- BRAVE (NCT03400852)
- Testing
- MNK-1411
- Where
- 16 sites in United States, Bulgaria and 6 more
- Volunteers
- 44 people
- Sponsor
- Mallinckrodt ARD LLC
Seizures, Epilepsy
A Study of the Safety of Topiramate Given in Combination With Other Medications in Adults and Children With Seizures
The purpose of this observational study is to evaluate the safety of topiramate when used in combination with other medications for seizures in adults and children.
- Trial name
- NCT00297349
- Testing
- Topiramate
- Volunteers
- 153 people
- Sponsor
- Janssen Cilag Pharmaceutica S.A.C.I., Greece
Dystrophic Epidermolysis Bullosa
Study to Evaluate Safety and Efficacy of ALLO-ASC-SHEET in Subjects With Dystrophic Epidermolysis Bullosa
After confirming eligibility, a single subject with four selected target lesions will receive both ALLO-ASC-SHEET and Vehicle control, three target lesions for ALLO-ASC-SHEET and the other target for Vehicle control, and which…
- Trial name
- NCT05157958
- Testing
- ALLO-ASC-SHEET, Vehicle Control
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Anterogen Co., Ltd.
Cardiology
Bivalirudin as a Procedural Anticoagulant in Pediatrics
Pharmacokinetics and safety of weight based bivalirudin in children
- Trial name
- NCT00503126
- Testing
- bivalirudin
- Volunteers
- 110 people
- Sponsor
- The Medicines Company
Atopic Dermatitis
Dose Ranging Study to Assess Efficacy, Safety, Tolerability and Pharmacokinetics of PF-06700841 Topical Cream in Participants With Mild or Moderate Atopic Dermatitis
This study is being conducted to provide data on efficacy, safety, tolerability and PK of multiple topical formulation concentrations of PF-06700841 topical cream in the treatment of mild to moderate atopic dermatitis (AD).
- Trial name
- NCT03903822
- Testing
- PF-06700841
- Where
- 76 sites in United States, Australia and 8 more
- Volunteers
- 292 people
- Sponsor
- Pfizer
Barth Syndrome
Clinical Trial in Patients With Barth Syndrome- 4TAZPower
Phase 3b/4, randomized, double-blind, parallel-group, placebo-controlled clinical trial to evaluate the efficacy, safety, and pharmacokinetics of a once daily SC injection of elamipretide in subjects with genetically confirmed…
- Trial name
- 4TAZPower (NCT07531251)
- Testing
- Elamipretide
- Where
- 3 sites in United States, Canada and 1 more
- Volunteers
- 48 people
- Sponsor
- Stealth BioTherapeutics Inc.
Sickle Cell Disease, Umbilical Cord Blood
US Phase I Study of ECT-001-CB in Patients With Sickle-Cell Disease
The application of experimental hematopoietic cell transplantation (HCT) therapy in sickle-cell disease (SCD) must strike a balance between the underlying disease severity and the possibility of a direct benefit of the treatment…
- Conditions
- Sickle Cell Disease, Umbilical Cord Blood, Hematopoietic Cell Proliferation
- Trial name
- NCT04594031
- Testing
- ECT-001-CB (UM171-Expanded Cord Blood Transplant)
- Where
- 2 sites in United States
- Sponsor
- ExCellThera inc.
Acute Intermittent Porphyria
Porphozym in the Treatment of Acute Attacks in AIP
A multi-centre, double-blind, randomized, placebo controlled, parallel group trial, investigating the efficacy and safety of Porphozym (recombinant human porphobilinogen deaminase)in the treatment of acute attacks in AIP.
- Trial name
- NCT00418795
- Testing
- recombinant human porphobilinogen deaminase (Porphozym)
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Zymenex A/S
Eczema
Effectiveness of Polymyxin B Sulphate + Prednisolone + Benzocaine + Clioquinol in Acute and Sub-acute Dermatitis Eczematous
Dermatitis eczematous is a recurrent pruritic skin disorder which has a significant morbidity and impaired quality of life due specially pruritus and physical visible skin lesions.
- Trial name
- NCT01429701
- Testing
- polymyxin B sulphate + prednisolone + benzocaine + clioquinol, betamethasone + gentamicin + tolnaftato + cleoquinol
- Volunteers
- 76 people
- Sponsor
- EMS
Cystic Fibrosis
BI 443651 Multiple Rising Dose in Healthy Volunteers Followed by a Cross-over in CF Subjects
The objective of this study is to investigate the safety, tolerability, and pharmacokinetics of BI 443651 in male and female healthy volunteers and subjects with Cystic Fibrosis (CF).
- Trial name
- NCT02976519
- Testing
- BI 443651
- Where
- 5 sites in Germany, United Kingdom
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- Boehringer Ingelheim
Haemophilia A, Haemophilia B
Post-marketing Surveillance (Special Use-results Surveillance) on Treatment With Alhemo
The purpose of the study is to investigate the safety and effectiveness of Alhemo in participants under real-world clinical practice in Japan. Total duration of this study is about 6 years.
- Trial name
- NCT06831734
- Testing
- Concizumab
- Where
- 23 sites in Japan
- Volunteers
- 30 people
- Sponsor
- Novo Nordisk A/S
Metastatic Castration Resistant Prostate Cancer
A Study of AC176 for the Treatment of Metastatic Castration Resistant Prostate Cancer
This clinical trial is evaluating a drug called AC176 in Chinese participants with metastatic castration resistant prostate cancer (mCRPC) who have progressed on at least one prior systemic therapy.
- Trial name
- NCT05673109
- Testing
- AC176
- Where
- 5 sites in China
- Volunteers
- 8 people
- Sponsor
- Accutar Biotechnology Inc
Normal Eyes, Eye Abnormalities
The Heidelberg Engineering BI US Evaluation Study
Evaluation of defined parameters measured in pathological and non-pathological eyes with the Heidelberg BI
- Trial name
- NCT03578302
- Testing
- BI Anterior Segment Mode, BI Anterior Segment and IOL Biometry Mode
- Where
- United States
- Volunteers
- 39 people, healthy volunteers welcome
- Sponsor
- Heidelberg Engineering GmbH
Cytomegalovirus Infections, HIV Infections
A Randomized, Double-Blind Study of the Efficacy and Safety of Oral Ganciclovir for the Prevention of CMV Disease in People Infected With the Human Immunodeficiency Virus
To evaluate the efficacy of oral ganciclovir in preventing progression to cytomegalovirus (CMV) disease (e.g., CMV retinitis, gastrointestinal CMV disease) in CMV-infected people with HIV infection and CD4 lymphocyte counts <=…
- Trial name
- NCT00002095
- Testing
- Ganciclovir
- Where
- 19 sites in United States
- Volunteers
- 700 people
- Sponsor
- Hoffmann-La Roche
Atopic Dermatitis
Drug-drug Interaction Trial With Tralokinumab in Moderate to Severe Atopic Dermatitis - ECZTRA 4
The purpose of this trial is to investigate if tralokinumab changes the metabolism of selected CYP substrates in adults with moderate-to-severe AD after: 14 weeks of treatment with tralokinumab a single dose of tralokinumab
- Trial name
- NCT03556592
- Testing
- Tralokinumab, Caffeine, Warfarin
- Where
- 12 sites in United States, France and 1 more
- Volunteers
- 40 people
- Sponsor
- LEO Pharma
Haemophilia A, Haemophilia B
A Study to Evaluate the Real-world Usage and Effectiveness of Elocta and Alprolix in Patients With Haemophilia A or B
Elocta (rFVIIIFc) and Alprolix (rFIXFc) are recombinant extended half-life coagulation factor products. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Elocta and Alprolix in…
- Trial name
- PREVENT (NCT03055611)
- Testing
- ELOCTA, ALPROLIX
- Where
- 22 sites in Germany
- Volunteers
- 201 people
- Sponsor
- Swedish Orphan Biovitrum
Osteogenesis Imperfecta
Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta
The primary objective of the study is to evaluate the effect of setrusumab on reduction in fracture rate, including morphometric vertebral fractures.
- Trial name
- NCT06636071
- Testing
- setrusumab
- Where
- 3 sites in Japan
- Volunteers
- 6 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
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