Clinical trials: Congenital Anomaly
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Evaluating the Safety and effectivenesS in Adult KorEaN Patients Treated With Tolvaptan for Management of Autosomal domInAnt poLycystic Kidney Disease
To evaluate the safety and therapeutic effectiveness of tolvaptan when administered to slow the progression of cyst development and renal function insufficiency in adult Korean patients diagnosed with rapidly progressive ADPKD…
- Trial name
- ESSENTIAL (NCT03949894)
- Testing
- Tolvaptan
- Where
- 18 sites in South Korea
- Volunteers
- 118 people
- Sponsor
- Korea Otsuka Pharmaceutical Co., Ltd.
Intracardiac and Septal Defects, Valve and Anulus Repair
Prospective and Non-randomized Registry of CardioCel 3D
Post-market, prospective, multi-centre, open-label, registry designed to collect prospective safety and performance data on the use of CardioCel in patients with cardiovascular disorders and in accordance with local standard of…
- Conditions
- Intracardiac and Septal Defects, Valve and Anulus Repair, Great Vessel Reconstruction, Peripheral Vessel Reconstruction
- Trial name
- NCT04175327
- Testing
- CardioCel implantation
- Where
- 6 sites in Germany, Italy and 2 more
- Volunteers
- 200 people
- Sponsor
- LeMaitre Vascular
Congenital Adrenal Hyperplasia, CAH - Congenital Adrenal Hyperplasia
Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia
This is a Phase 2 study of SPR001 for the treatment of classic CAH that will provide 12 weeks of open-label treatment to eligible subjects.
- Conditions
- Congenital Adrenal Hyperplasia, CAH - Congenital Adrenal Hyperplasia, CAH - 21-Hydroxylase Deficiency
- Trial name
- NCT03687242
- Testing
- SPR001
- Where
- 8 sites in United States
- Volunteers
- 11 people
- Sponsor
- Spruce Biosciences
Thyroid-Associated Ophthalmopathy
Lanreotide (Somatuline Autogel) in Thyroid-associated Ophthalmopathy Treatment
The main purpose of this study is to evaluate the efficacy and safety of 3 doses of Somatuline Autogel 60mg to control the muscle infiltration and edema, eyelid retraction and extraocular muscular contraction in patients with…
- Trial name
- NCT00288522
- Testing
- Lanreotide (Autogel formulation). Duration of treatment - 3 injections, 1 every 28 days
- Where
- Spain
- Volunteers
- 5 people
- Sponsor
- Ipsen
Mucopolysaccharidosis IVA (Morquio A Syndrome)
BMN 110 Phase 3B in Australian Patients
There is currently no treatment for MPS IVA other than supportive care for the clinical manifestations of the disease. Enzyme replacement therapy (ERT) with BMN 110 to replace the deficient GALNS is a potential new treatment…
- Trial name
- MOR-AUS (NCT01966029)
- Testing
- BMN 110
- Where
- 5 sites in Australia
- Volunteers
- 13 people
- Sponsor
- BioMarin Pharmaceutical
Lennox-Gastaut Syndrome
A Placebo-Controlled, Double-Blind Comparative Study of E2080 in Lennox-Gastaut Syndrome Patients (Study E2080-J081-304)
To confirm that the combination therapy of rufinamide has superior efficacy compared to placebo in patients with Lennox-Gastaut syndrome.
- Trial name
- NCT01146951
- Testing
- Rufinamide (E2080)
- Where
- 23 sites in Japan
- Volunteers
- 66 people
- Sponsor
- Eisai Limited
Sickle Cell Disease
Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies
The goal of this research study is to establish chimerism and avoid graft-versus-host disease in patients with Hemoglobinopathies to halt disease progression.
- Trial name
- NCT00777231
- Testing
- Enriched Hematopoetic Stem Cell Infusion
- Where
- 3 sites in United States
- Volunteers
- 9 people
- Sponsor
- Talaris Therapeutics Inc.
Congenital Adrenal Hyperplasia
Comparison of Chronocort Versus Standard Hydrocortisone Replacement Therapy in Participants Aged 16 Years and Over With Congenital Adrenal Hyperplasia
This study is a randomized, double-blind, active-controlled, phase III study of Chronocort® compared with immediate-release hydrocortisone replacement therapy in participants aged 16 years and over with Congenital Adrenal…
- Trial name
- CONnECT (NCT05063994)
- Testing
- Chronocort, Cortef
- Where
- 21 sites in United States, France and 1 more
- Volunteers
- 55 people
- Sponsor
- Neurocrine UK Limited
Gaucher Disease
A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease
The main aim of this study is to learn if velaglucerase alfa (VPRIV) improves growth and symptoms in participants up to 5 years of age with Gaucher disease. Symptoms will be checked with blood tests.
- Trial name
- PEDS (NCT04721366)
- Testing
- Standard of Care
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Takeda
Atopic Dermatitis
Maximal Use Study of Tapinarof Cream, 1% in Pediatric Subjects With Extensive Atopic Dermatitis
This is an open-label, multicenter study to evaluate the systemic exposure and safety of topical tapinarof cream, 1% under conditions of maximal use in pediatric subjects with atopic dermatitis
- Trial name
- NCT05186805
- Testing
- Tapinarof cream, 1%
- Where
- 10 sites in United States, Canada
- Volunteers
- 36 people
- Sponsor
- Organon and Co
ARB, BVMD
Safety and Tolerability of Subretinally Injected OPGx-BEST1 in Patients With Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB)
The goal of this clinical trial is to learn if drug OPGx-BEST1 works to treat BVMD and ARB Bestrophinopathy. It will also learn about the safety of drug OPGx-BEST1.
- Conditions
- ARB, BVMD, Autosomal-Dominant Bestrophinopathy, Best Vitelliform Macular Dystrophy
- Trial name
- BIRD-1 (NCT07185256)
- Testing
- OPGx-BEST1
- Where
- 5 sites in United States
- Volunteers
- 10 people
- Sponsor
- Opus Genetics, Inc
Atopic Dermatitis
A Study to Determine the Safety and Tolerability of Dupilumab (REGN668/SAR231893) in Patients Aged ≥6 to <18 Years With Atopic Dermatitis (Eczema)
The primary objective of the study is to characterize the safety and pharmacokinetics (PK) of dupilumab in pediatric patients with moderate-to-severe atopic dermatitis (AD) (for adolescents ≥12 to <18 years of age) or severe AD…
- Trial name
- NCT02407756
- Testing
- Dupilumab
- Where
- 26 sites in Canada, Czechia and 4 more
- Volunteers
- 78 people
- Sponsor
- Regeneron Pharmaceuticals
Atopic Dermatitis
A Food Effect Study of VC005 Tablets in Healthy Adult Subjects.
This study will adopt a randomized, open-label, two-period, 2-way crossover design to evaluate Food effect of VC005 Tablets in healthy subjects
- Trial name
- NCT07533851
- Testing
- VC005 Tablets
- Where
- China
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Jiangsu vcare pharmaceutical technology co., LTD
Alpha 1-Antitrypsin Deficiency
Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-814 in PiZZ subjects.
- Trial name
- NCT04167345
- Testing
- VX-814
- Where
- 28 sites in United States, Canada and 2 more
- Volunteers
- 48 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Hemophilia A; Hemophilia B
A Study to Assess Pharmacokinetics and Pharmacodynamics Following Administration of BAY1093884 in Patients With Severe Hemophilia
The primary objective of this study is to assess the pharmacokinetics in patients with severe hemophilia. The secondary objective is to assess the pharmacodynamics of BAY1093884 based on tissue factor pathway inhibitor activity
- Trial name
- NCT03481946
- Testing
- BAY1093884
- Where
- Israel
- Volunteers
- 6 people
- Sponsor
- Bayer
Septal Defect, Atrial
Safety and Efficacy of the GORE® Septal Occluder to Treat Ostium Secundum Atrial Septal Defects
The primary objective of the GORE® Septal Occluder Study is to evaluate the safety and efficacy of the occluder device in the treatment of transcatheter closure of ostium secundum atrial septal defects (ASDs).
- Trial name
- NCT01711983
- Testing
- GORE® CARDIOFORM Septal Occluder
- Where
- United States
- Volunteers
- 400 people
- Sponsor
- W.L.Gore & Associates
Persistent Pulmonary Hypertension of the Newborn
Remodulin as Add-on Therapy for the Treatment of Persistent Pulmonary Hypertension of the Newborn
This study assessed the safety and treatment effect of intravenous (IV) Remodulin as an add-on therapy in neonates with persistent pulmonary hypertension of the newborn (PPHN).
- Trial name
- NCT02261883
- Testing
- IV Remodulin
- Where
- 14 sites in United States
- Volunteers
- 42 people
- Sponsor
- United Therapeutics
Achondroplasia
A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia
This is a Phase 3, multicenter, double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of infigratinib in children and adolescents with achondroplasia (ACH) who have completed at least 26 weeks of…
- Trial name
- PROPEL3 (NCT06164951)
- Testing
- Infigratinib 0.25 mg/kg/day
- Where
- 27 sites in United States, Argentina and 8 more
- Volunteers
- 114 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Atopic Dermatitis
Safety and Efficacy of FURESTEM-AD Inj. in Patients With Moderate to Severe Chronic Atopic Dermatitis(AD)
This is multi-center, randomized, double-blind, parallel, placebo-controlled phase III clinical trial to evaluate the efficacy and safety of FURESTEM-AD Inj. for moderate to severe chronic atopic dermatitis
- Trial name
- NCT03269773
- Testing
- FURESTEM-AD Inj.
- Where
- 11 sites in South Korea
- Volunteers
- 197 people
- Sponsor
- Kang Stem Biotech Co., Ltd.
Primary Immunodeficiency
Pharmacokinetics and Safety of IVIG Nanogam 100 mg/ml
Intravenous immunoglobulin (IVIG) is used for treatment of a heterogeneous group of immune related disorders both as immune-replacement and immune-modulating therapy.
- Trial name
- Nanogam (NCT01985373)
- Testing
- Intravenous immunoglobulin infusion
- Where
- 4 sites in Netherlands
- Volunteers
- 23 people
- Sponsor
- Prothya Biosolutions
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