Clinical trials: Congenital Deformity
Cholangiocarcinoma
A Study of E7090 in Participants With Unresectable Advanced or Metastatic Cholangiocarcinoma With Fibroblast Growth Factor Receptor (FGFR) 2 Gene Fusion
The primary purpose of the study is to assess the objective response rate (ORR) of E7090 by Response Evaluation Criteria In Solid Tumors (RECIST) 1.1 based on independent imaging review (IIR) in participants with unresectable…
- Trial name
- NCT04238715
- Testing
- E7090
- Where
- 56 sites in China, Japan
- Volunteers
- 63 people
- Sponsor
- Eisai Co., Ltd.
Atopic Dermatitis
Oral DS107 in Moderate to Severe Atopic Dermatitis
This study will compare the safety and efficacy of Oral DS107 (2 g) to placebo in patients with moderate to severe atopic dermatitis. Oral DS107 (2 g) will be orally administered for 8 weeks, and will be compared against placebo.
- Trial name
- NCT02211417
- Testing
- Oral DS107
- Where
- 14 sites in United States, Canada
- Volunteers
- 102 people
- Sponsor
- Dignity Sciences Ltd.
Hereditary Periodic Fever Syndromes, Cryopyrin-associated Periodic Syndromes (CAPS)
Ilaris NIS in Korea
This is a study to evaluate safety and effectiveness of Ilaris in adult and pediatric patients receiving the drug in a clinical setting for any of the following indications, Hereditary Periodic Fever Syndromes…
- Conditions
- Hereditary Periodic Fever Syndromes, Cryopyrin-associated Periodic Syndromes (CAPS), Colchicine Resistance Familial Mediterranean Fever (crFMF), TNF Receptor Associated Periodic Syndrome (TRAPS)
- Trial name
- REASSURE (NCT06838143)
- Testing
- Ilaris
- Where
- South Korea
- Volunteers
- 25 people
- Sponsor
- Novartis Pharmaceuticals
Hyalinosis, Hyaline Membrane Disease
Biomarker for Hyaline Fibromatosis Syndrome (BioHFS)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Hyaline fibromatosis syndrome from the blood
- Conditions
- Hyalinosis, Hyaline Membrane Disease, Juvenile Hyaline Fibromatosis, Fibromatosis Hyalinica Multiplex Juvenilis
- Trial name
- BioHFS (NCT03196115)
- Where
- 3 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
Genetic Disorder, Turner Syndrome
Growth Response in Girls With Turner Syndrome
This trial is conducted in Europe. The aim of the trial is to evaluate growth response of two somatropin dose regimens in girls with Turner Syndrome.
- Trial name
- NCT01734486
- Testing
- somatropin
- Where
- 15 sites in France
- Volunteers
- 31 people
- Sponsor
- Novo Nordisk A/S
Cockayne Syndrome
Pharmacokinetics and Safety Study of Single and Multiple Oral Doses Prodarsan™ in Patients With Cockayne Syndrome
This study is to compare the exposure of orally administered Prodarsan to the intravenous dosed Osmitrol (10% solution) in Cockayne Syndrome (CS) patients.
- Trial name
- NCT01142154
- Testing
- Prodarsan
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- DNage B.V.
Metastatic Head and Neck Squamous Cell Carcinoma, Recurrent Head and Neck Squamous Cell Carcinoma
FORTIFI-HN01: A Study of Ficerafusp Alfa (BCA101) or Placebo in Combination With Pembrolizumab in First-Line PD-L1-pos, R or M HNSCC
Ficerafusp alfa is directed against two targets, Epidermal Growth Factor Receptor (EGFR) and Transforming Growth Factor beta (TGF-β).
- Trial name
- FORTIFI-HN01 (NCT06788990)
- Testing
- Ficerafusp alfa, Pembrolizumab (KEYTRUDA®)
- Where
- 231 sites in United States, Argentina and 26 more
- Volunteers
- 650 people
- Sponsor
- Bicara Therapeutics
Limb Girdle Muscular Dystrophy
A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)
The primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4).
- Trial name
- NCT05876780
- Testing
- SRP-9003
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Sarepta Therapeutics, Inc.
Cystic Fibrosis
Evaluation of an Airway Clearance System
The aim of this study is to assess the effectiveness of an Airway Clearance System.
- Trial name
- NCT03091062
- Testing
- Vest® Airway Clearance System, Monarch™ System
- Where
- Belgium
- Volunteers
- 9 people
- Sponsor
- Hill-Rom
Non-small Cell Lung Cancer, Colorectal Cancer
A Study of JAB-3312 in Adult Patients With Advanced Solid Tumors in China
This is a Phase 1, open-label dose-escalation study to determine the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D) and assess the DLT of JAB-3312.
- Conditions
- Non-small Cell Lung Cancer, Colorectal Cancer, Pancreatic Ductal Carcinoma, Esophageal Squamous Cell Carcinoma
- Trial name
- NCT04121286
- Testing
- JAB-3312
- Where
- 5 sites in China
- Volunteers
- 17 people
- Sponsor
- Allist Pharmaceuticals, Inc.
Dermatitis Atopic
A Study to Evaluate the Efficacy and Safety of Subcutaneous Amlitelimab on Background Topical Corticosteroids Therapy in Participants Aged 12 Years and Older With Moderate-to-severe AD Who Have Had an Inadequate Response to Prior Biologic Therapy or an Oral JAK Inhibitor
This is a parallel group, Phase 3, multinational, multicenter, randomized, double-blind, placebo-controlled, 3-arm study for treatment of participants diagnosed with moderate-to-severe AD on background TCS who have had inadequate…
- Trial name
- AQUA (NCT06241118)
- Testing
- Amlitelimab, Topical corticosteroids, Topical tacrolimus or pimecrolimus
- Where
- 154 sites in United States, Argentina and 20 more
- Volunteers
- 464 people
- Sponsor
- Sanofi
Cardiac Amyloidosis, AL Amyloidosis
Efficacy of [18F]Florbetaben PET for Diagnosis of Cardiac AL Amyloidosis
This is an open-label, multi-center pivotal Phase 3 study to visually and quantitatively assess PET images obtained after single application of 300 MBq [18F]florbetaben and PET scanning of patients with suspected cardiac…
- Conditions
- Cardiac Amyloidosis, AL Amyloidosis, ATTR Amyloidosis
- Trial name
- CArdiag (NCT05184088)
- Testing
- [18F]florbetaben
- Where
- 14 sites in United States, Germany and 2 more
- Volunteers
- 244 people
- Sponsor
- Lantheus Germany GmbH
Atopic Dermatitis
Evaluate Efficacy, PK, and Safety of FB825 in Adults With Atopic Dermatitis
The study aims to evaluate the efficacy, improvement from baseline in Eczema Area and Severity Index (EASI) score, of multiple intravenous (IV) doses of FB825 in subjects with atopic dermatitis
- Trial name
- NCT04413942
- Testing
- FB825
- Where
- 18 sites in United States
- Volunteers
- 99 people
- Sponsor
- Oneness Biotech Co., Ltd.
Urea Cycle Disorders
Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)
This non-randomized, open-label study was approximately one year in duration and consisted of a short term NaPBA to HPN-100 switchover part involving two overnight stays followed by a 12-month long term treatment period involving…
- Trial name
- NCT01347073
- Testing
- HPN-100
- Where
- 8 sites in United States
- Volunteers
- 23 people
- Sponsor
- Amgen
Atopic Dermatitis
A First-in-Human Study of CEE321 in Adult Subjects
Open label, non-randomized, uncontrolled, First-in-Human Phase 1 Study in Healthy Subjects and Subjects with Atopic Dermatitis
- Trial name
- NCT04612062
- Testing
- CEE321
- Where
- 3 sites in United States, Japan
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
Osteoarthritis Shoulder, Avascular Necrosis of the Head of Humerus
Catalyst CSR Shoulder System for Semi or Total Shoulder Arthroplasty
Evaluation of the performance and safety of the Catalyst CSR Shoulder System with clinical and radiographic results at multiple time points through 24 months postoperatively This study will be a prospective multi-center study…
- Conditions
- Osteoarthritis Shoulder, Avascular Necrosis of the Head of Humerus, Rheumatoid Arthritis Shoulder
- Trial name
- NCT04968405
- Testing
- Catalyst CSR Total Shoulder System
- Where
- 3 sites in United States
- Volunteers
- 87 people, healthy volunteers welcome
- Sponsor
- Catalyst OrthoScience
Atopic Dermatitis
An Observational Study to Assess Change in Disease Activity and Use of Upadacitinib Tablets in Adolescent and Adult Participants With Moderate to Severe Atopic Dermatitis in Real World
Atopic dermatitis (AD) is a skin condition that may cause a rash and itching due to inflammation of the skin. This study will assess how effective upadacitinib is in treating AD. Upadacitinib is an approved drug for treating AD.
- Trial name
- UPDATE (NCT05669794)
- Where
- 59 sites in France
- Volunteers
- 300 people
- Sponsor
- AbbVie
Atopic Dermatitis
IB/IIa Study of MG-K10 in Adolescent Moderately Severe Patients
This study was designed to reflect the safety pharmacokinetics and preliminary efficacy of MG-K10 Humanized Monoclonal Antibody Injection in adolescent patients 12-18 weeks of age with moderate to severe atopic dermatitis…
- Trial name
- NCT06765005
- Testing
- MG-K10
- Where
- China
- Volunteers
- 24 people
- Sponsor
- Shanghai Mabgeek Biotech.Co.Ltd
Duchenne Muscular Dystrophy
Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy
The purpose of the study is to see whether PRO045 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 45 in the DNA for the dystrophin protein.
- Trial name
- NCT01826474
- Testing
- PRO045, 0.15 mg/kg/week, PRO045, 1.0 mg/kg/week, PRO045, 3.0 mg/kg/week
- Where
- 6 sites in Belgium, France and 3 more
- Volunteers
- 15 people
- Sponsor
- BioMarin Pharmaceutical
Phenylketonuria
A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks
The primary objective of the study is to evaluate the effect of dosing regimens of multiple subcutaneous (SC) doses of rAvPAL-PEG to induce an early and sustained Phe reduction while decreasing the frequency and severity of…
- Trial name
- NCT01560286
- Testing
- BMN 165 (rAvPAL-PEG)
- Where
- 6 sites in United States
- Volunteers
- 24 people
- Sponsor
- BioMarin Pharmaceutical
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