Clinical trials: Newborn Diseases
Gastric Adenocarcinoma
Derazantinib Alone or in Combination With Paclitaxel, Ramucirumab or Atezolizumab in Gastric Adenocarcinoma
The purpose of this study was to evaluate the efficacy of derazantinib monotherapy or derazantinib in combination with paclitaxel and ramucirumab in patients with gastric adenocarcinoma (GAC) i.e.
- Trial name
- FIDES-03 (NCT04604132)
- Testing
- Derazantinib, Derazantinib-paclitaxel-ramucirumab combination
- Where
- 81 sites in United States, Argentina and 13 more
- Volunteers
- 47 people
- Sponsor
- Basilea Pharmaceutica
Infertility, Hereditary Diseases
Embryo Health Study
This study intends to determine the patients' perception and motivation to obtain additional information on their preimplantation embryos' risks of polygenic disorders.
- Trial name
- EHS (NCT04528498)
- Testing
- Preimplantation Genetic Testing
- Where
- United States
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- Genomic Prediction Inc.
22Q11.2 Deletion Syndrome
Open-Label Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With 22q11.2 Deletion Syndrome (INSPIRE)
To evaluate the safety and tolerability of ZYN002 administered as a transdermal gel formulation, for up to 38 weeks, in participants ages 4 to <18 years, in the treatment of 22q.11.2 Deletion syndrome (22qDS).
- Trial name
- INSPIRE (NCT05149898)
- Testing
- ZYN002
- Where
- 3 sites in United States, Australia
- Volunteers
- 20 people
- Sponsor
- Harmony Biosciences Management, Inc.
Thrombocytopenia, Neonatal Alloimmune
A Study of Nipocalimab or Intravenous Immunoglobulin (IVIG) in Pregnancies At Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)
The purpose of this study is to assess the efficacy and safety of nipocalimab in reducing the risk of severe fetal and neonatal alloimmune thrombocytopenia (FNAIT).
- Trial name
- FREESIA-3 (NCT06533098)
- Testing
- Nipocalimab, Intravenous immunoglobulins (IVIG), Prednisone
- Where
- 38 sites in United States, Austria and 9 more
- Volunteers
- 50 people
- Sponsor
- Janssen Research & Development, LLC
Hemophilia A
A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors
This is a phase IIIb, single arm, open-label, multi-center study to evaluate the safety and tolerability of emicizumab in participants with congenital hemophilia A who have documented inhibitors against Factor VIII (FVIII) at…
- Trial name
- STASEY (NCT03191799)
- Testing
- Emicizumab
- Where
- 72 sites in Australia, Belgium and 22 more
- Volunteers
- 195 people
- Sponsor
- Hoffmann-La Roche
Rhizomelic Chondrodysplasia Punctata
RCDP Natural History Study
A prospective, longitudinal observational trial in patients with RCDP. Study participants will be evaluated at baseline and approximately every 6 months by the study team.
- Trial name
- NCT04031287
- Where
- United States
- Volunteers
- 75 people
- Sponsor
- MED-LIFE DISCOVERIES LP
Atopic Dermatitis (AD), Psoriasis (PsO)
Safety and Pharmacokinetics of LPX-TI641 in Atopic Dermatitis and Psoriasis
The goal of this clinical trial is to study the drug LPX-TI641 in patients with atopic dermatitis and psoriasis. We will compare the safety and tolerability of LPX-TI641 to placebo ( a look-alike solution) that contains no drug.
- Trial name
- NCT06982352
- Testing
- LPX-TI641
- Where
- 3 sites in United States, Jordan
- Volunteers
- 48 people
- Sponsor
- LAPIX Therapeutics Inc.
Alpha 1-Antitrypsin Deficiency
A Study of Belcesiran in Patients With AATLD
This is a multiple dose, randomized, placebo-controlled, double-blind study of belcesiran to evaluate the safety, tolerability, PK, and PD in adult patients with PiZZ AATD-associated liver disease (AATLD).
- Trial name
- ESTRELLA (NCT04764448)
- Testing
- Belcesiran
- Where
- 23 sites in United States, Australia and 12 more
- Volunteers
- 16 people
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Lung Disease, Pulmonary Disease
NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease
This study will be conducted to evaluate the safety, tolerability, clinical activity, pharmacokinetics, and pharmacodynamics of NTLA-3001 in adults with alpha-1 antitrypsin deficiency (AATD) -associated lung disease
- Conditions
- Lung Disease, Pulmonary Disease, AATD, Alpha-1 Antitrypsin Deficiency
- Trial name
- AATD (NCT06622668)
- Testing
- Biological NTLA-3001
- Where
- New Zealand
- Sponsor
- Intellia Therapeutics
Gene Mutations, Pseudoxanthoma Elasticum
ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency
The purpose of this study (Study INZ701-304 [ADAPT]) is to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to…
- Conditions
- Gene Mutations, Pseudoxanthoma Elasticum, Arterial Calcification, Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency
- Trial name
- NCT06462547
- Testing
- INZ-701
- Where
- 5 sites in United States, France and 2 more
- Volunteers
- 22 people
- Sponsor
- Inozyme Pharma
Skin Laxity
Rollover Study to Evaluate Histological Results of Radiofrequency Device Treatments on the Flanks
The purpose of this study is to evaluate histological results of the treatments with the TempSure Firm handpiece on the flanks performed in the CYN20-FIRM-LIPO study.
- Trial name
- NCT04881149
- Testing
- TempSure treatment
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Cynosure, Inc.
Hypothalamic Obesity
A Long-Term Study of Bivamelagon in Participants With Hypothalamic Obesity (HO)
This is a long-term, open-label extension study of bivamelagon in participants with Hypothalamic Obesity (HO) who have transitioned from the index study, LG-MCCL005. The study will last up to 2 years.
- Trial name
- NCT07156578
- Testing
- Bivamelagon
- Where
- 9 sites in United States, United Kingdom
- Volunteers
- 25 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Thyroid Eye Disease
A Phase III Study of KHN939 in Chinese Patients With Moderate-to-Severe Active Thyroid Eye Disease
This is a multicenter, randomized, double-masked, placebo-controlled Phase 3 study to evaluate the efficacy, safety, immunogenicity, and population pharmacokinetic (PopPK) characteristics of KHN939 in Chinese patients with…
- Trial name
- Halo-1 (NCT07720635)
- Testing
- KHN939
- Where
- China
- Volunteers
- 60 people
- Sponsor
- Beijing Kanghong Biological Medicine Co., Ltd.
Alopecia Areata, Depressive Episode
The Epidemiology, Management, and the Associated Burden of Related Conditions in Alopecia Areata
This study series consists of four related studies and aims to explore and describe many important elements of alopecia areata over three key areas: (1) the current epidemiology of alopecia areata, (2) the prevalence and…
- Conditions
- Alopecia Areata, Depressive Episode, Recurrent Depressive Disorder, Anxiety Disorders
- Trial name
- NCT04239521
- Testing
- Exposure of interest (studies 2 & 3).
- Where
- United Kingdom
- Volunteers
- 51,955 people
- Sponsor
- Momentum Data
Fabry Disease
A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the alpha-galactosidase A enzyme.
- Trial name
- NCT00196716
- Testing
- Fabrazyme (agalsidase beta)
- Where
- 4 sites in Czechia, Estonia and 2 more
- Volunteers
- 21 people
- Sponsor
- Genzyme, a Sanofi Company
Alström Syndrome
Open-Label Rollover Study of PBI 4050 in Subjects With Alström Syndrome
Phase 2, open-label, single-arm, multi-centre study evaluating the long term safety and tolerability of PBI-4050 in subjects with Alström Syndrome who have completed a preceding ProMetic-sponsored Alström Syndrome study with…
- Trial name
- NCT03184584
- Testing
- PBI-4050
- Where
- United Kingdom
- Volunteers
- 10 people
- Sponsor
- Liminal BioSciences Ltd.
Atopic Dermatitis
Probiotic on Atopic Dermatitis in Infant
The clinical trial will be carried out in China Medical University Hospital. The overall plan is a double-blind trial. All recruited 3 months-3 years old atopic dermatitis (AD) patients are divided into two groups, 50 in each…
- Trial name
- NCT05286047
- Testing
- Bifidobacterium longum CCFM1029
- Where
- Taiwan
- Volunteers
- 100 people
- Sponsor
- Glac Biotech Co., Ltd
Epidermolysis Bullosa Dystrophica, Epidermolysis Bullosa, Junctional
CACIPLIQ20 in Wound Healing in Subjects With Epidermolysis Bullosa
Epidermolysis bullosa (EB) is a group of inherited disorders characterized by fragility of the skin and mucous membranes within the basement membrane zone.
- Trial name
- MATHBULL (NCT06007235)
- Testing
- CACIPLIQ20
- Where
- France
- Volunteers
- 16 people
- Sponsor
- Organ, Tissue, Regeneration, Repair and Replacement
Charcot-Marie-Tooth Disease, Hereditary Motor and Sensory Neuropathy IV
A Natural History Study of Charcot-Marie-Tooth 4J (CMT4J)
Charcot-Marie-Tooth 4J (CMT4J) is a rare inherited peripheral neuropathy often characterized by rapidly progressive, asymmetrical upper and lower extremity weakness, muscle atrophy leading to loss of ambulation, respiratory…
- Trial name
- CMT4J (NCT03810508)
- Where
- 2 sites in United States
- Volunteers
- 21 people
- Sponsor
- Neurogene Inc.
Congenital Bleeding Disorder, Haemophilia A
Safety and Efficacy of Turoctocog Alfa During Long-Term Treatment of Severe and Moderately Severe Haemophilia A
This study is conducted in Europe, and North and South America. The aim of this study is to provide additional documentation of the immunogenicity, and obtain additional clinical data, of turoctocog alfa in the setting of normal…
- Trial name
- guardian™ 5 (NCT02035384)
- Testing
- turoctocog alfa
- Where
- 41 sites in United States, Austria and 13 more
- Volunteers
- 69 people
- Sponsor
- Novo Nordisk A/S
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