Clinical trials: Newborn Diseases
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
Alport Syndrome, Focal Segmental Glomerulosclerosis
Study to Evaluate R3R01 in Patients With Alport Syndrome and Patients With Focal Segmental Glomerulosclerosis
This is a Phase 2, Multi-center, Open-Label Study to Assess Safety, Tolerability, Efficacy and Pharmacokinetics of R3R01 in Alport Syndrome Patients with Uncontrolled Proteinuria on ACE/ARB Inhibition and in Patients with Primary…
- Trial name
- NCT05267262
- Testing
- R3R01
- Where
- 22 sites in United States, Belgium and 4 more
- Volunteers
- 43 people
- Sponsor
- River 3 Renal Corp.
Graves Ophthalmopathy
A Safety and Tolerability Study of Otelixizumab in Thyroid Eye Disease
The purpose of this study is to investigate the safety and tolerability of otelixizumab in patients with Graves' ophthalmopathy (thyroid eye disease).
- Trial name
- NCT01114503
- Testing
- Otelixizumab - low dose, Otelixizumab - medium low dose, Otelixizumab - medium high dose
- Where
- United Kingdom
- Volunteers
- 2 people
- Sponsor
- GlaxoSmithKline
Atrial Fibrillation, Stroke
Philips Intracardiac Echocardiography (ICE) Clinical Registry
The purpose of this observational registry is to report real-world safety and performance of VeriSight for ultrasound guided ICE imaging in percutaneous cardiac intervention procedures when used in standard clinical practice.
- Conditions
- Atrial Fibrillation, Stroke, Valve Disease, Heart, Atrial Septal Defect
- Trial name
- NCT04950192
- Testing
- ICE Image guidance
- Where
- 5 sites in United States
- Volunteers
- 155 people
- Sponsor
- Philips Clinical & Medical Affairs Global
Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS
ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
A first-in-human study using ISP-001 in patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
- Trial name
- NCT05682144
- Testing
- Autologous Plasmablasts (B cells)
- Where
- 2 sites in United States
- Volunteers
- 11 people
- Sponsor
- Immusoft of CA, Inc.
FAP, Familial Amyloid Polyneuropathy
Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy
The purpose of this study is to evaluate the efficacy and safety of inotersen given for 65 weeks in participants with Familial Amyloid Polyneuropathy (FAP).
- Conditions
- FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin
- Trial name
- NCT01737398
- Testing
- Inotersen
- Where
- 24 sites in United States, Argentina and 8 more
- Volunteers
- 173 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
May-Thurner Syndrome, Peripheral Vascular Disease
BARD® The VENOVO™ Venous Stent Study for Treatment of Iliofemoral Occlusive Disease
The BARD® Venovo™ Venous Stent Study is a non-randomized clinical study intended to collect confirmatory evidence of the safety and effectiveness of the Venous Stent for the treatment of iliofemoral occlusive disease.
- Conditions
- May-Thurner Syndrome, Peripheral Vascular Disease, Venous Disease, Iliofemoral Occlusive Disease
- Trial name
- VERNACULAR (NCT02655887)
- Testing
- VENOVO™ Venous Stent
- Where
- 21 sites in United States, Australia and 5 more
- Volunteers
- 170 people
- Sponsor
- C. R. Bard
Primary Immunodeficiencies, Inborn Errors of Metabolism
Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases
The aim of the trial is to describe the safety and efficacy of intravenous (i.v.) Treosulfan compared to the conventional (myeloablative) dose of i.v.
- Conditions
- Primary Immunodeficiencies, Inborn Errors of Metabolism, Haemoglobinopathies, Bone Marrow Failure Syndromes
- Trial name
- NCT02349906
- Testing
- Treosulfan, Busilvex
- Where
- 21 sites in Czechia, Germany and 2 more
- Volunteers
- 106 people
- Sponsor
- medac GmbH
Atopic Dermatitis
Study of Dupilumab in Adult Patients With Extrinsic Moderate-to-Severe Atopic Dermatitis
The primary objective was to assess the clinical efficacy of repeated subcutaneous (SC) doses of Dupilumab in adult participants with moderate-to-severe atopic dermatitis (AD).
- Trial name
- NCT01548404
- Testing
- Dupilumab
- Where
- 21 sites in Czechia, France and 3 more
- Volunteers
- 109 people
- Sponsor
- Regeneron Pharmaceuticals
Lax Skin
Evaluation of Facial and Neck Skin Laxity With SofWave Treatment in Adults Initiating Tirzepatide for Weight Loss
Open-label, randomized double-arm prospective, single-center, self-controlled clinical study.
- Trial name
- NCT07685834
- Testing
- SUPERB
- Where
- Canada
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Sofwave Medical LTD
Pediatric Homozygous Familial Hypercholesterolemia (HoFH), Severe Hypertriglyceridemia (HTG)
Compassionate Use of Evinacumab
Provide compassionate use of evinacumab
- Conditions
- Pediatric Homozygous Familial Hypercholesterolemia (HoFH), Severe Hypertriglyceridemia (HTG), Dysbetalipoproteinemia (DBL)
- Trial name
- NCT06500598
- Testing
- Evinacumab
- Sponsor
- Regeneron Pharmaceuticals
Catheters, Oxygen Saturation
Evaluation of Intravascular Monitoring of Partial PRessure of Oxygen for Neonatal intensiVE Care Patients
The study is a non-blinded trial with one single intervention in sick newborn infants. The Neosense umbilical catheter will be used instead of the routinely used umbilical catheter on infants where the treating physician has…
- Conditions
- Catheters, Oxygen Saturation, Newborn, Infant, Disease
- Trial name
- IMPROVE (NCT03967587)
- Testing
- Neosense Umbilical Catheter
- Where
- 2 sites in Sweden
- Volunteers
- 6 people
- Sponsor
- Neosense Technologies
Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome
RGX-111 Gene Therapy in Patients With MPS I
RGX-111 is a gene therapy which is intended to deliver a functional copy of the α-L-iduronidase (IDUA) gene to the central nervous system.
- Conditions
- Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome
- Trial name
- NCT03580083
- Testing
- RGX-111
- Where
- 2 sites in United States, Brazil
- Volunteers
- 21 people
- Sponsor
- REGENXBIO Inc.
Duchenne Muscular Dystrophy
Open Label Extension Study of HT-100 in Patients With DMD
This study is designed to provide 6-months continuous dosing with the study medication, called HT-100, on participants who successfully completed the predecessor study (HALO-DMD-01).
- Trial name
- NCT01978366
- Testing
- HT-100
- Where
- 5 sites in United States
- Volunteers
- 17 people
- Sponsor
- Processa Pharmaceuticals
Alpha 1-Antitrypsin Deficiency, Emphysema
Study of SAR447537 (INBRX-101) Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema
Phase 2 study to compare SAR447537 (INBRX-101) to plasma derived A1PI therapy in adults with AATD emphysema
- Trial name
- ELEVAATE (NCT05856331)
- Testing
- SAR447537, Zemaira
- Where
- 65 sites in United States, Australia and 6 more
- Volunteers
- 99 people
- Sponsor
- Sanofi
Healthy Volunteers
A Trial to Investigate Teprotumumab (High-concentration Formulation) Subcutaneous Administration in Healthy Adult Non-Japanese and Japanese Participants
The primary objective of this trial are to assess the pharmacokinetics (PK) of a single subcutaneous (SC) administration of teprotumumab high-concentration formula (HCF) in healthy adult non-Japanese and Japanese participants and…
- Trial name
- NCT06674941
- Testing
- Teprotumumab
- Where
- United States
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- Amgen
Atopic Dermatitis
Phase 2 Study of SAR444656 in Adult Participants With Moderate to Severe Atopic Dermatitis
This was a multinational, multicenter, double-blind, placebo-controlled, parallel-group Phase 2, 4-arm study in adult participants with moderate to severe AD who were inadequately controlled with topical therapies or for whom…
- Trial name
- ADVANTA (NCT06058156)
- Testing
- SAR444656 (KT-474)
- Where
- 22 sites in United States, Germany and 2 more
- Volunteers
- 70 people
- Sponsor
- Sanofi
Lysosomal Acid Lipase Deficiency
An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency
This is an open-label, multicenter expanded access protocol to allow patients with a confirmed diagnosis of Lysosomal Acid Lipase (LAL) Deficiency in the United States (US), access to sebelipase alfa (recombinant lysosomal acid…
- Trial name
- NCT02376751
- Testing
- sebelipase alfa
- Sponsor
- Alexion Pharmaceuticals, Inc.
Beta-Thalassemia, Thalassemia
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
This is a single-dose, open-label study in pediatric participants with TDT. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).
- Conditions
- Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases
- Trial name
- NCT05356195
- Testing
- CTX001
- Where
- 6 sites in United States, Canada and 3 more
- Volunteers
- 16 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Dermatitis
Clinical Investigation on the Performance and Safety of DermoRelizema Ecofoam
The scope of this open label clinical trial is to evaluate and confirm the performance of DermoRelizema ecofoam in the improvement of the dermatitis severity, by alleviating the symptomatology.
- Trial name
- NCT04826471
- Testing
- DermoRelizema ecofoam
- Where
- Italy
- Volunteers
- 40 people
- Sponsor
- Relife S.r.l.
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