Clinical trials: Newborn Diseases
To Determine Bioequivalence Under Fed Conditions
Bioavailability Study of Leflunomide Tablets Under Fed Conditions
To compare the single-dose Bioavailability of Kali and Aventis
- Trial name
- NCT00653003
- Testing
- Leflunomide, ARAVA
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- Par Pharmaceutical, Inc.
Congenital Bleeding Disorder, Haemophilia B
Safety, Efficacy and Pharmacokinetics of NNC-0156-0000-0009 in Previously Treated Children With Haemophilia B.
This trial is conducted in Asia, Europe and North America. The aim of the trial is to evaluate safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC-0156-0000-0009 (nonacog beta pegol, N9-GP)…
- Trial name
- paradigm™5 (NCT01467427)
- Testing
- nonacog beta pegol
- Where
- 51 sites in United States, Brazil and 10 more
- Volunteers
- 25 people
- Sponsor
- Novo Nordisk A/S
Dermatitis, Atopic
Study Of Atopic Dermatitis In Pediatrics
To verify of cetirizine dry syrup to ketotifen dry syrup in the change in the severity of pruritus of the treatment period.
- Trial name
- NCT00257569
- Testing
- Cetirizine Dry Syrup
- Volunteers
- 278 people
- Sponsor
- GlaxoSmithKline
Microcystic Lymphatic Malformation
SELVA: A Phase 3 Study Evaluating QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations
SELVA: A Multicenter, Phase 3 Baseline-Controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations The main purpose of this study is to assess…
- Trial name
- SELVA (NCT06239480)
- Testing
- QTORIN 3.9% Rapamycin Anhydrous Gel
- Where
- 15 sites in United States
- Volunteers
- 51 people
- Sponsor
- Palvella Therapeutics, Inc.
Hereditary Angioedema
A Study of the Relative Bioavailability of a New Formulation of BCX4161 and the Effect of Food on BCX4161
The purpose of the study is to compare how the body takes up and then eliminates the study drug BCX4161 when it is taken orally as a hard gelatin capsule and as a soft gelatin capsule.
- Trial name
- NCT02125162
- Testing
- BCX4161
- Where
- United Kingdom
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- BioCryst Pharmaceuticals
Primary Immune Deficiency
Study of Subcutaneous Immunoglobulin in Patients With PID Requiring IgG Replacement Therapy
The objective of this study is to assess the efficacy, tolerability, safety and pharmacokinetics of IgPro20 in patients with primary humoral immunodeficiency (PID).
- Trial name
- NCT00419341
- Testing
- Human Normal Immunoglobulin for Subcutaneous Administration
- Where
- 13 sites in United States
- Volunteers
- 49 people
- Sponsor
- CSL Behring
Atopic Dermatitis
A Clinical Study of AK120 in Adolescents With Moderate-to-severe Atopic Dermatitis
This is a randomized double-blind, placebo-controlled, multicenter phase III clinical study evaluating the efficacy and safety of AK120 in the treatment of moderate-to-severe AD in adolescents.
- Trial name
- NCT06767540
- Where
- 28 sites in China
- Volunteers
- 198 people
- Sponsor
- Akeso
Congenital Bleeding Disorder, Glanzmann's Disease
Observational Registry of the Treatment of Glanzmann's Thrombasthenia
This observational registry is conducted in Europe, Asia, Africa and the United States of America (USA). The purpose of the registry is to evaluate the efficacy and safety of activated recombinant human factor VII (rFVIIa) during…
- Trial name
- NCT01476423
- Testing
- activated recombinant human factor VII
- Where
- 15 sites in United States, Algeria and 13 more
- Volunteers
- 218 people
- Sponsor
- Novo Nordisk A/S
Hemophilia B
BAX 326 Surgery Study in Hemophilia B Patients
The purpose of the study is to assess the hemostatic efficacy and safety of BAX 326 in subjects with severe (FIX level < 1%) or moderately severe (FIX level 1-2%) hemophilia B undergoing major or minor elective or emergency…
- Trial name
- NCT01507896
- Testing
- Recombinant factor IX
- Where
- 14 sites in Argentina, Bulgaria and 8 more
- Volunteers
- 30 people
- Sponsor
- Baxalta now part of Shire
Cystic Fibrosis
Study of VX-661 Alone and in Combination With Ivacaftor in Subjects Homozygous or Heterozygous to the F508del-Cystic Fibrosis Transmembrane Conductance Regulator(CFTR) Mutation
The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) effects of VX-661 alone and when coadministered with ivacaftor in participants with cystic fibrosis (CF) who are…
- Trial name
- NCT01531673
- Testing
- VX-661, Ivacaftor
- Where
- 36 sites in United States, Canada and 2 more
- Volunteers
- 194 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Hereditary Transthyretin-mediated (hATTR) Amyloidosis, Polyneuropathy
Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program
The purpose of this study is to collect and evaluate pregnancy outcomes, pregnancy complications, and fetal/neonatal/infant outcomes in women exposed to patisiran-LNP.
- Trial name
- NCT05040373
- Where
- 7 sites in United States, France and 5 more
- Volunteers
- 10 people
- Sponsor
- Alnylam Pharmaceuticals
Prader-Willi Syndrome
Phase 2 Trial to Evaluate Safety and Efficacy of Setmelanotide (RM-493) in Obese Participants With Prader-Willi Syndrome
The purpose of this study was to evaluate the effects of a once daily subcutaneous injectable formulation of setmelanotide in obese participants with Prader-Willi syndrome on tolerability, weight loss, and hyperphagia-related…
- Trial name
- NCT02311673
- Testing
- Setmelanotide
- Where
- 5 sites in United States
- Volunteers
- 40 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Wilson Disease
Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840
The main objective of the study is to evaluate the change in liver copper (Cu) concentration following 48 weeks of treatment with ALXN1840 in adult participants with Wilson Disease (WD) who have been previously treated for at…
- Trial name
- NCT04422431
- Testing
- Bis-Choline Tetrathiomolybdate
- Where
- 12 sites in United States, Canada and 6 more
- Volunteers
- 31 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Fuchs' Endothelial Dystrophy, Fuchs Dystrophy
TTHX1114(NM141) in Combination With DWEK/DSO
Open label, single-treatment, with a concurrent non-treatment control
- Conditions
- Fuchs' Endothelial Dystrophy, Fuchs Dystrophy, Fuchs
- Trial name
- NCT04676737
- Testing
- TTHX1114(NM141)
- Where
- 11 sites in United States
- Volunteers
- 49 people
- Sponsor
- Trefoil Therapeutics, Inc.
Cystic Fibrosis
Safety of Tobramycin Inhalation Powder (TIP) vs Tobramycin Solution for Inhalation in Patients With Cystic Fibrosis
This study compares the safety of the tobramycin solution for inhalation with the tobramycin dry powder formulation, used with a simple inhaler
- Trial name
- EAGER (NCT00388505)
- Testing
- Tobramycin Inhalation Powder, Tobramycin Solution for Inhalation
- Where
- 72 sites in United States, Australia and 12 more
- Volunteers
- 517 people
- Sponsor
- Novartis Pharmaceuticals
Acquired Angioedema Due to C1-Inhibitor Deficiency (AAE-C1-INH)
Study of Oral Deucrictibant XR Tablet for Prophylaxis and Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adults With Acquired Angioedema Due to C1 Inhibitor Deficiency
This is a Phase 3, multicenter, 3-part study, with 2 randomized, double-blind, placebo-controlled parts and an open-label extension part, to evaluate the efficacy and safety of orally administered deucrictibant XR tablet for…
- Trial name
- CREAATE (NCT07266805)
- Testing
- Deucrictibant
- Where
- 32 sites in United States, Australia and 14 more
- Volunteers
- 48 people
- Sponsor
- Pharvaris Netherlands B.V.
Neurofibromatosis Type 2
Efficacy and Safety of REC-2282 in Patients With Progressive Neurofibromatosis Type 2 (NF2) Mutated Meningiomas
This is a two-staged, Phase 2/3, randomized, multi-center study to investigate the efficacy and safety of REC-2282 in participants with progressive NF2 mutated meningiomas.
- Trial name
- POPLAR-NF2 (NCT05130866)
- Testing
- REC-2282
- Where
- 15 sites in United States, United Kingdom
- Volunteers
- 25 people
- Sponsor
- Recursion Pharmaceuticals Inc.
Hemophilia A
Pharmacokinetics and Safety of a Single Intravenous Infusion of BAY 79-4980
The primary objective of this study is to determine the pharmacokinetic profile after single administration of two doses of BAY 79-4980 (high and low: 35 IU FVIII/Kg reconstituted in 22 mg and 13 mg of liposomes/Kg, respectively)…
- Trial name
- NCT00629837
- Testing
- Recomb. Factor VIII (Kogenate FS Liposome, BAY79-4980), Recombinant Factor VIII (Kogenate FS, BAY14-2222)
- Where
- 2 sites in United States
- Volunteers
- 27 people
- Sponsor
- Bayer
Congenital Bleeding Disorder, Haemophilia B
Safety of 40K Pegylated Recombinant Factor IX in Non-Bleeding Patients With Haemophilia B
This trial is conducted in Europe, Japan and the United States of America (USA). The aim of this clinical trial is to investigate the safety and pharmacokinetics (the determination of the concentration of the administered…
- Trial name
- NCT00956345
- Testing
- nonacog beta pegol
- Where
- 19 sites in United States, Denmark and 6 more
- Volunteers
- 18 people
- Sponsor
- Novo Nordisk A/S
Inborn Errors of Metabolism
Induced Pluripotent Stem Cells for the Development of Novel Drug Therapies for Inborn Errors of Metabolism (iPSC-IEM)
Human induced pluripotent stem cells (iPSCs), are reprogrammed from somatic cells that can self-renew indefinitely and produce different types of cells. They provide human model cell lines for orphan drug development.
- Trial name
- iPSC-IEM (NCT04097275)
- Testing
- Skin biopsy, Withdraw of blood
- Where
- Pakistan
- Volunteers
- 1,000 people
- Sponsor
- CENTOGENE GmbH Rostock
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.