Clinical trials: Infant, Newborn Diseases
Congenital Bleeding Disorder, Haemophilia A
Investigation of the Pharmacokinetics of NNC172-2021, at Two Different Dose Levels, in Healthy Japanese Subjects
This trial is conducted in Europe. The aim of this trial is to investigate the pharmacokinetics (how the trial drug is distributed in the body) of NNC172-2021 administered subcutaneously, at two different dose levels, in healthy…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B, Healthy
- Trial name
- NCT01555749
- Testing
- NNC172-2021
- Where
- United Kingdom
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Hereditary Angioedema, HAE
A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
This is a Phase 1/2, single-arm, open-label, dose-escalation and dose-expansion study of BMN 331 for the treatment of hereditary angioedema (HAE) due to C1 Esterase Inhibitor (C1-INH) protein deficiency.
- Trial name
- HAErmony-1 (NCT05121376)
- Testing
- Dose 1 of BMN 331, Dose 2 of BMN 331, Dose 3 of BMN 331
- Where
- 16 sites in United States, Australia and 1 more
- Volunteers
- 44 people
- Sponsor
- BioMarin Pharmaceutical
Deformity, Defect, Congenital
Observational Study on JuniOrtho Plating System for Deformities and Fractures Treatment in Lower Limb
Orthofix Srl put the JPS on the European market (2019) by the mean of a pre-market clinical evaluation made under the Medical Device Directive (MDD) requirements that were based on the analysis of the scientific literature of…
- Conditions
- Deformity, Defect, Congenital, Trauma
- Trial name
- JPS (NCT05245617)
- Testing
- JuniOrtho™ Plating System™
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- Orthofix s.r.l.
Sjogren-Larsson Syndrome
RESET Trial - Part 1 - A Phase 3 Trial in Subjects With Sjögren-Larsson Syndrome (SLS)
A Phase 3 Randomized, Double-Blind, Vehicle-Controlled, Parallel Group Trial to Evaluate the Safety and Efficacy of ADX-102 1% Topical Dermal Cream in Subjects with Sjögren-Larsson Syndrome (SLS).
- Trial name
- NCT03445650
- Testing
- ADX-102 1% Topical Dermal Cream (reproxalap), Vehicle of ADX-102 Topical Dermal Cream
- Where
- 2 sites in United States
- Volunteers
- 11 people
- Sponsor
- Aldeyra Therapeutics, Inc.
Port-wine Stain
A Pilot Study of Hemoporfin PDT in Children(2-7 Years Old) With Port-wine Stain
This pilot study aims to evaluate the safety and efficacy of hemoporfin photodynamic therapy (PDT) with different light doses for port-wine stain (PWS）in 2-7 years old children.
- Trial name
- NCT04106258
- Testing
- Hemoporfin PDT
- Where
- China
- Volunteers
- 40 people
- Sponsor
- Shanghai Fudan-Zhangjiang Bio-Pharmaceutical Co., Ltd.
Primary Immune Deficiency Disorder
Study to Evaluate the Efficacy, Tolerability and Safety of Octanorm in Patients With Primary Immunodeficiency Diseases
Clinical phase 3 study to evaluate the efficacy, tolerability and safety of subcutaneous human immunoglobulin (octanorm) in patients with primary immunodeficiency diseases.
- Trial name
- NCT03988426
- Testing
- Octanorm
- Where
- 5 sites in Russia
- Volunteers
- 25 people
- Sponsor
- Octapharma
Pectus Excavatum
Pectus Excavatum Camouflage
The clinical study is testing a new treatment for pectus excavatum called "custom-made 3D printed scaffold-based soft tissue reconstruction".
- Trial name
- NCT05451108
- Testing
- PCL Pectus Scaffold implantation and autologous fat grafting
- Where
- Australia
- Volunteers
- 7 people
- Sponsor
- BellaSeno Pty Ltd
Achondroplasia
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
This is a multicenter, multinational, randomized, active-controlled, operationally seamless Phase 2/3 study of BMN 333 in treatment-naïve pediatric participants with achondroplasia (ACH).
- Trial name
- ASPEN (NCT07441876)
- Testing
- BMN 333, Vosoritide Injection [Voxzogo]
- Where
- 28 sites in United States, Australia and 7 more
- Volunteers
- 160 people
- Sponsor
- BioMarin Pharmaceutical
Acquired Factor X Deficiency
A Study Investigating Coagadex in the Treatment AFXD Associated With AL Amyloidosis
The purpose of this study is to evaluate the efficacy and safety of Coagadex in treating active bleeds and to manage peri-operative bleeding in participants with acquired factor X deficiency associated with AL amyloidosis.
- Trial name
- NCT06963216
- Testing
- Coagadex(R)
- Sponsor
- Kedrion S.p.A.
Cystic Fibrosis
Phase 1b Safety, Tolerability, and PK Study to Assess GS-5737 in Subjects With CF
This study will determine the safety, tolerability, and pharmacokinetics of a single dose of GS-5737 administered with a 2.8% saline solution vehicle in adult subjects with CF.
- Trial name
- NCT01840735
- Testing
- GS-5737
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Gilead Sciences
Hemophilia B, Hemophilia A
A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)
To establish baseline prospective efficacy data of current FIX prophylaxis replacement therapy in the usual care setting of hemophilia B subjects, who are negative for nAb to AAV-Spark100, prior to the Phase 3 gene therapy study.
- Trial name
- NCT03587116
- Testing
- Standard of Care FIX Replacement therapy, Standard of Care FVIII Replacement therapy
- Where
- 75 sites in United States, Australia and 16 more
- Volunteers
- 212 people
- Sponsor
- Pfizer
Von Willebrand Disease (VWD)
A Study of Recombinant Von Willebrand Factor (rVWF) in Chinese Participants With Von Willebrand Disease (vWD)
The main aim of this study is to find out if VONVENDI is safe for adult Chinese participants with VWD. The study will also check how well VONVENDI helps control bleeding with or without product ADVATE in the participants who may…
- Trial name
- NCT07129343
- Testing
- VONVENDI, ADVATE
- Where
- 7 sites in China
- Volunteers
- 20 people
- Sponsor
- Takeda
Cystic Fibrosis
Pharmacokinetic Study of Bramitob® Administered for Inhalation by PARI eFlow® vs PARI LC® PLUS Nebulizer
The purpose of this study is to assess pharmacokinetic and safety comparability of Bramitob® when administered for inhalation by using PARI eFlow® rapid electronic nebulizer vs PARI LC® PLUS nebulizer in Cystic Fibrosis Patients…
- Trial name
- NCT01116089
- Testing
- Bramitob® administered by PARI LC® PLUS nebulizer, Bramitob® administered by PARI eFlow® rapid electronic nebulizer
- Where
- 5 sites in Czechia, Moldova and 1 more
- Volunteers
- 25 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Atopic Dermatitis
Study to Assess the Safety and Efficacy of Lebrikizumab (LY3650150) in Adolescent Participants With Moderate-to-Severe Atopic Dermatitis
This is an open-label, single arm study of 52 weeks duration. The study will assess the safety and efficacy of lebrikizumab in adolescent participants (≥12 to <18 years weighing ≥40 kilograms) with moderate-to-severe atopic…
- Trial name
- ADore (NCT04250350)
- Testing
- Lebrikizumab
- Where
- 70 sites in United States, Australia and 2 more
- Volunteers
- 206 people
- Sponsor
- Eli Lilly and Company
Hemophilia A
Safety,Efficacy and Pharmacokinetics Evaluation of SCT800 in Previously Treated Paediatric Patients With Severe Haemophilia A.
This study is a multicenter phase III uncontrolled open-label trial to evaluate the efficacy,safety and pharmacokinetics of SCT800 in regular prophylaxis and perioperative treatment in patients (<12 years old) with severe…
- Trial name
- NCT03947320
- Testing
- Recombinant Human Coagulation FVIII
- Volunteers
- 70 people
- Sponsor
- Sinocelltech Ltd.
Fabry Disease
Extension Study of PRX-102 for up to 60 Months
To evaluate the ongoing safety, tolerability, and efficacy parameters of PRX-102 in adult Fabry patients who have successfully completed treatment with PRX-102 in studies PB-102-F01 and PB-102-F02.
- Trial name
- NCT01981720
- Testing
- PRX-102 (pegunigalsidase alfa)
- Where
- 8 sites in United States, Paraguay and 2 more
- Volunteers
- 15 people
- Sponsor
- Protalix
Infantile Colic
Evaluation of Safety and Efficacy of New Infant Formula in Infantile Colic
The study is conducted to examine the safety and efficacy of a new infant formula and its effects on the gastrointestinal tolerance in infants suffering from colic.
- Trial name
- Coco (NCT01721850)
- Testing
- control formula, intervention formula 1, intervention formula 2
- Where
- 18 sites in Germany, Italy
- Volunteers
- 169 people, healthy volunteers welcome
- Sponsor
- HiPP GmbH & Co. Vertrieb KG
Duchenne Muscular Dystrophy
Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy
The FOX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics, and biomarkers in children and adolescents with Duchenne muscular dystrophy previously treated with gene therapy including a randomized…
- Trial name
- FOX (NCT06100887)
- Testing
- Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3
- Where
- 7 sites in United States
- Volunteers
- 43 people
- Sponsor
- Edgewise Therapeutics, Inc.
Fistulas Arteriovenous
A Safety and Efficacy Study of Hemodialysis Arteriovenous Fistulae Stenosis Treated With DissolveAV
Drug-coated balloons delivering paclitaxel at the angioplasty site have proved their superiority in the treatment of coronary and peripheral arterial stenoses.
- Trial name
- DissolveAVF (NCT04226599)
- Testing
- Dissolve AVF, Armada 35
- Where
- 10 sites in China
- Volunteers
- 220 people
- Sponsor
- DK Medical Technology (Suzhou) Co., Ltd.
Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1
Study of CAD-1883 for Spinocerebellar Ataxia
This is a randomized, double-blind, placebo-controlled Phase 2 study evaluating oral administration of CAD-1883 in the treatment of adults with a genotypic diagnosis of Spinocerebellar Ataxia (SCA).
- Conditions
- Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3
- Trial name
- Synchrony-1 (NCT04301284)
- Testing
- CAD-1883
- Where
- 6 sites in United States
- Sponsor
- Cadent Therapeutics
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