Clinical trials: Genetic Diseases
Primary Immunodeficiency Diseases (PID)
A Study of Immune Globulin Subcutaneous (Human), 20% Solution (IGSC, 20%) in Japanese Participants With Primary Immunodeficiency Diseases (PID)
In this study, Japanese participants with primary immunodeficiency diseases were treated with Immune Globulin Subcutaneous (Human), 20% solution, (IGSC, 20%).
- Trial name
- NCT04346108
- Testing
- Immune Globulin Intravenous (IGIV), Immune Globulin Subcutaneous, 20% Solution (IGSC, 20%)
- Where
- 8 sites in Japan
- Volunteers
- 17 people
- Sponsor
- Baxalta now part of Shire
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes
- Trial name
- NCT03301168
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 10 sites in United States
- Volunteers
- 120 people
- Sponsor
- Bellicum Pharmaceuticals
Digestive Disease, Gut Health
Impact of B. Adolescentis iVS-1 Probiotic on Gastrointestinal Outcomes
The study team is conducting a consumer-driven, decentralized clinical research study to assess the impact of the consumer-grade probiotic supplement, iVS-1, on adults with moderate to severe gastrointestinal discomfort.
- Trial name
- NCT07408518
- Testing
- Probiotic
- Where
- United States
- Volunteers
- 92 people, healthy volunteers welcome
- Sponsor
- Synbiotic Health
Nevus, Dysplasia; Epithelial
Skin Fluorescent Imaging (SFI) System in Patients With Nevi
The Sponsor has developed the Skin Fluorescent Imaging (SFI) system, an in vivo imaging modality, for the purpose of providing physicians with insight into the biological changes occurring during tissue remodeling in a nevus.
- Trial name
- NCT04368247
- Testing
- Skin Fluorescent Imaging
- Where
- 4 sites in United States
- Volunteers
- 300 people
- Sponsor
- Orlucent, Inc
HEMOPHILIA B
Safety and Efficacy of Benefix in Patients With Hemophilia B in Usual Care Settings in China
The purpose of this post-approval study is to evaluate the safety and efficacy of Benefix in subjects with hemophilia B in usual care settings in China.
- Trial name
- NCT02336178
- Testing
- Benefix
- Where
- 16 sites in China
- Volunteers
- 70 people
- Sponsor
- Pfizer
Carcinoma, Non-Small-Cell Lung
A Study of SGN-B6A Versus Docetaxel in Previously Treated Non-small Cell Lung Cancer
This clinical trial is studying nonsquamous non-small cell lung cancer (NSCLC). Participants in this study must have cancer that has spread through their body or can't be removed with surgery.
- Trial name
- NCT06012435
- Testing
- sigvotatug vedotin, docetaxel
- Where
- 331 sites in United States, Argentina and 23 more
- Volunteers
- 762 people
- Sponsor
- Seagen, a wholly owned subsidiary of Pfizer
Atopic Dermatitis
Effect of OC000459 on Moderate to Severe Atopic Dermatitis
The purpose of this study is to determine whether OC000459 is in reducing disease severity and preventing flares in people with moderate to severe atopic dermatitis (AD).
- Trial name
- NCT02002208
- Testing
- OC000459
- Where
- United Kingdom
- Volunteers
- 142 people
- Sponsor
- Atopix Therapeutics, Ltd.
Gout
Allopurinol Outcome Study
This is a study of allopurinol in gout patients with hyperuricemia that will evaluate the safety and serum urate (sUA) lowering capability of allopurinol as a urate lowering therapy (ULT) for up to six months.
- Trial name
- LASSO (NCT01391325)
- Testing
- Allopurinol
- Where
- 173 sites in United States, Australia and 5 more
- Volunteers
- 1,735 people
- Sponsor
- Ardea Biosciences, Inc.
Atopic Dermatitis
In-home Use Test of the New Modified Diprobase Formulation to Assess the Safety and Tolerability in Infants and Children Under Physician's Control
To investigate the safety and tolerability of the modified Diprobase formulation over 14 days in infants and children with a history of Atopic Dermatitis (AD).
- Trial name
- NCT03441568
- Testing
- Modified Diprobase formulation (BAY987534)
- Where
- United Kingdom
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Bayer
Solid Tumor, Adult
Nous-209 Genetic Vaccine for the Treatment of Microsatellite Unstable Solid Tumors
Ref: Protocol Version 9.1 05 December 2024. NOUS-209-01 is a multicenter, open-label, multiple cohorts, clinical study, designed to evaluate safety, tolerability, and immunogenicity, and to detect any preliminary evidence of…
- Trial name
- NCT04041310
- Testing
- GAd-209-FSP low dose, MVA-209-FSP low dose, GAd-209-FSP high dose
- Where
- 46 sites in United States, Belgium and 4 more
- Volunteers
- 115 people
- Sponsor
- Nouscom SRL
Friedreich's Ataxia
GV101 for Friedreich's Ataxia (FA) Treatment
This is a dose-ranging, randomized, double-blind, placebo-controlled 12-week Phase 2a trial with an up to two week loading dose period.
- Trial name
- NCT07870590
- Testing
- GV101
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Graviton Bioscience Corporation
Cellulite
Treatment for Cellulite Appearance
Open-label, non-randomized, prospective, multi-center, self-controlled clinical study with masked evaluation.
- Trial name
- NCT05358847
- Testing
- Sofwave
- Where
- 4 sites in United States
- Volunteers
- 69 people, healthy volunteers welcome
- Sponsor
- Sofwave Medical LTD
Atopic Dermatitis
Study of MG-K10 Humanized Monoclonal Antibody Injection in Patients With Atopic Dermatitis
The study is to reflect the effectiveness and safety of MG-K10 humanized monoclonal antibody injection in patients with moderate to severe atopic dermatitis.administered every 4 weeks for 52 week
- Trial name
- NCT06026891
- Where
- China
- Volunteers
- 498 people
- Sponsor
- Shanghai Mabgeek Biotech.Co.Ltd
Atopic Dermatitis, Eczema
Effectiveness and Safety of Lebrikizumab Treatment in Adults and Adolescents With Moderate-to-Severe Atopic Dermatitis
The main purpose of this study is to evaluate the effectiveness of 24 weeks of lebrikizumab in improving disease severity, signs, and symptoms in adults and adolescents with moderate-to-severe atopic dermatitis (AD).
- Trial name
- ADhope (NCT05990725)
- Testing
- Lebrikizumab
- Where
- 33 sites in Germany, Netherlands and 1 more
- Volunteers
- 260 people
- Sponsor
- Almirall, S.A.
Joint Damage
Effect of Febuxostat on Joint Damage in Hyperuricemic Subjects With Early Gout
This purpose of this study is to assess the effect of febuxostat, once daily (QD), on joint damage in patients with elevated serum urate levels and gout.
- Trial name
- NCT01078389
- Testing
- Febuxostat
- Where
- 56 sites in United States
- Volunteers
- 314 people
- Sponsor
- Takeda
Cystic Fibrosis
A Safety Study of SPX-101 Inhalation Solution in Subjects With Cystic Fibrosis
Ascending dose, 7-day, open label safety trial of SPX-101 Inhalation Solution in adult subjects with cystic fibrosis.
- Trial name
- NCT03056989
- Testing
- SPX-101
- Where
- Canada
- Volunteers
- 5 people
- Sponsor
- Spyryx Biosciences, Inc.
Hypertriglyceridemia, Familial Hypercholesterolemia
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS ANGPTL3-LRx in Healthy Volunteers With Elevated Triglycerides and Participants With Familial Hypercholesterolemia
The purpose is to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of IONIS ANGPTL3-LRx (ISIS 703802) given to healthy volunteer subjects with elevated triglycerides and subjects with familial…
- Trial name
- NCT02709850
- Testing
- IONIS ANGPTL3-LRx
- Where
- Canada
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Ionis Pharmaceuticals, Inc.
Advanced Solid Tumor
A Phase I Study of RGT-264 in Subjects With Advanced Solid Tumors
This is a Phase 1 dose escalation and dose expansion study to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of RGT-264 as monotherapy in subjects with advanced solid tumors.
- Trial name
- NCT05764915
- Testing
- RGT-264 phosphate tablets
- Where
- 3 sites in China
- Volunteers
- 11 people
- Sponsor
- Regor Pharmaceuticals Inc.
Dermatitis, Atopic
Study of the Effectiveness and Safety of Desloratadine (Aerius) Syrup in Children With Allergic Skin Inflammation (P03475)
The purpose of this study was to test the effectiveness and safety of desloratadine (Aerius) syrup in children with allergic skin inflammation. Patients took desloratadine syrup once a day for 28 days.
- Trial name
- NCT00817076
- Testing
- desloratadine
- Volunteers
- 40 people
- Sponsor
- Organon and Co
Hereditary Hemochromatosis
Study to Evaluate the Efficacy and Safety of Deferasirox Film-coated Tablet Versus Phlebotomy in Patients With Hereditary Hemochromatosis (HH)
The purpose of this study was to evaluate the efficacy and safety of deferasirox film coated tablet (FCT) versus phlebotomy for the management of iron overload in adults with Hereditary Hemochromatosis (HH) at risk of…
- Trial name
- NCT03203850
- Testing
- Deferasirox FCT, Phlebotomy
- Where
- 11 sites in Belgium, France and 5 more
- Volunteers
- 45 people
- Sponsor
- Novartis Pharmaceuticals
The first 5,000 results can be paged through. Add a filter or change the sort order to reach the rest.