Clinical trials: Newborn Diseases
Sickle Cell Disease
ESCORT-HU Extension: European Sickle Cell Disease Cohort - Hydroxyurea - Extension Study
As safety information pertaining to the long-term use of HU remains incomplete in spite of the first safety study (ESCORT-HU), an extension of the latter is proposed.
- Trial name
- NCT04707235
- Testing
- Hydroxycarbamide
- Where
- 87 sites in France, French Guiana and 6 more
- Volunteers
- 2,093 people
- Sponsor
- Theravia
Wilson Disease
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
The primary objectives of this study are to evaluate the safety of single IV doses of UX701 in patients with Wilson disease, to select the UX701 dose with the best benefit/risk profile based on the totality of safety and efficacy…
- Trial name
- NCT04884815
- Testing
- UX701, Standard of Care (SOC)
- Where
- 16 sites in United States, Portugal and 2 more
- Volunteers
- 82 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Transfusion-dependent Iron Overload
A Study Assessing the Efficacy and Safety of Deferasirox in Patients With Transfusion-dependent Iron Overload
This study uses a single arm, multi-center, open-label trial design. The study will assess the efficacy and safety of 52 weeks of treatment with deferasirox (ICL670) in patients with evidence of transfusion induced iron overload.
- Trial name
- NCT00171821
- Testing
- Deferasirox
- Where
- 97 sites in Australia, Austria and 21 more
- Volunteers
- 1,784 people
- Sponsor
- Novartis Pharmaceuticals
Advanced Nonhaematologic Malignancies
Safety, Efficacy, and Tolerability of BOS172738 in Patients With Advanced Rearranged During Transfection (RET) Gene-Altered Tumors
This study will be conducted to assess the safety and tolerability of BOS172738 when administered to patients with advanced solid tumors with rearranged during transfection (RET) gene alterations and also to establish the maximum…
- Trial name
- NCT03780517
- Testing
- BOS172738
- Where
- 21 sites in United States, Belgium and 5 more
- Volunteers
- 117 people
- Sponsor
- Boston Pharmaceuticals
Hemophilia A, Congenital
Study Evaluating "Real World" Treatment Pattern in Previously Treated Hemophilia A Patients Receiving KOVALTRY (Octocog Alfa) for Routine Prophylaxis
The primary objective of this study is to investigate weekly prophylaxis dosing regimens used in standard clinical practice. In addition the study will capture reported bleed rate, pattern of change in KOVALTRY prophylaxis dose…
- Trial name
- TAURUS (NCT02830477)
- Testing
- Kovaltry (Antihemophilic Factor [Recombinant], BAY81-8973
- Where
- 25 sites in United States, Belgium and 11 more
- Volunteers
- 313 people
- Sponsor
- Bayer
Hemophilia
A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia
This is a multicenter, multinational, open-label, one-way cross-over, Phase 3, single-arm study for treatment of hemophilia. The purpose of this study is to measure the frequency of treated bleeding episodes with fitusiran in…
- Trial name
- ATLAS-NEO (NCT05662319)
- Testing
- Fitusiran, Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC)
- Where
- 50 sites in United States, Canada and 15 more
- Volunteers
- 91 people
- Sponsor
- Sanofi
Phenylketonuria
Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria
This is an open-label, non-comparative, Phase 3 study to evaluate the degree, frequency of response and safety of Kuvan® (sapropterin dihydrochloride) in subjects aged 4 to 18 years who have phenylketonuria and with elevated…
- Trial name
- NCT01732471
- Testing
- Kuvan®
- Where
- Germany
- Volunteers
- 90 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Haemophilia A
Efficacy and Safety Evaluation Study of SCT800 in Previously Untreated Hemophilia A Patients.
This is an Open Label, Uncontrolled Study to Evaluate the the Safety and Efficacy of SCT800 for Prophylaxis Treatment in Severe Previously Untreated Hemophilia Patients.
- Trial name
- NCT07437404
- Testing
- SCT800 prophylaxis and treatment for bleeding events
- Where
- China
- Volunteers
- 36 people
- Sponsor
- Sinocelltech Ltd.
Friedreich's Ataxia
Safety and Pharmacology Study of VP 20629 in Adults With Friedreich's Ataxia
The objectives of the study are: To evaluate the safety and tolerability of single and multiple oral doses of VP 20629 in subjects with Friedreich's ataxia (FA).
- Trial name
- NCT01898884
- Testing
- VP 20629
- Where
- 5 sites in United States
- Volunteers
- 46 people
- Sponsor
- Shire
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.
Hepatitis B
LT F-up Study 16-20 Yrs After Vaccine Dose of Hepatitis B With/Without HBIg in Newborns to HBeAg+ Mothers
To evaluate the persistence of anti-HBs antibodies up to 16, 17, 18, 19 and 20 years after administration of the first dose of the study vaccine.
- Trial name
- NCT00240526
- Testing
- Engerix™ -B, Hepatitis B immunoglobulin (HBIg)
- Where
- Thailand
- Volunteers
- 79 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
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
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.