Clinical trials: Genetic Diseases
Mitochondrial Diseases, Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
The KHENEREXT Study
This is an open-label, multi-centre study in subjects with a genetically confirmed mitochondrial deoxyribonucleic acid (DNA) transfer ribonucleic acid (tRNA)Leu(UUR) m.3243A>G mutation who completed study KH176-202.
- Conditions
- Mitochondrial Diseases, Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation, Maternally Inherited Diabetes and Deafness (MIDD), Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke Like Episodes (MELAS)
- Trial name
- NCT04604548
- Testing
- Oral administration of 100 mg KH176 twice daily
- Where
- 4 sites in Denmark, Germany and 2 more
- Volunteers
- 11 people
- Sponsor
- Khondrion BV
Atopic Dermatitis
A First-In-Human Study to Evaluate the Safety, Tolerability, and Efficacy of Si-544 in Adults With Atopic Dermatitis
This is a multi-center, Phase 1b, double-blind, placebo-controlled, SAD and MAD, first-in-human study in subjects with mild to severe AD receiving si-544. The study consists of 2 parts, an SAD and an MAD part.
- Trial name
- NCT05383378
- Testing
- si-544
- Where
- 5 sites in Germany
- Volunteers
- 36 people
- Sponsor
- selectION Therapeutics GmbH
Gaucher Disease, Type 1
Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease
The purpose of this study is to evaluate the safety and efficacy of ISU302 in patients with Type 1 Gaucher disease.
- Trial name
- NCT02770625
- Testing
- ISU302
- Volunteers
- 8 people
- Sponsor
- ISU Abxis Co., Ltd.
Psoriasis, Atopic Dermatitis
Topical Application of BMX-010 in Subjects With Atopic Dermatitis and Plaque Psoriasis
This is a randomized, placebo-controlled Phase 2 trial consisting of up to 300 subjects with either psoriasis or atopic dermatitis. In this trial BMX-010 will be topically applied twice daily for up to 28 days.
- Trial name
- NCT03381625
- Testing
- BMX-010
- Where
- 10 sites in United States
- Volunteers
- 139 people
- Sponsor
- BioMimetix JV, LLC
Primary Immunodeficiency Disease
IVIG Boya: Safety, Efficacy, and Pharmacokinetics
The goal of this phase 3, open-label, single-group clinical trial is to assess the efficacy of Boya IVIG in maintaining the mean number of serious bacterial infections to less than one per year in participants with primary…
- Trial name
- BoyaIVIG (NCT06150833)
- Testing
- Boya IVIG
- Volunteers
- 50 people
- Sponsor
- Azidus Brasil
Hemophilia
LR769 in Congenital Hemophilia Patients With Inhibitors Undergoing Elective Surgery or Invasive Procedures
The purpose of this study, PerSept 3, is to evaluate LR769 for the prevention of excessive bleeding and achievement of hemostasis in congenital hemophilia A or B patients who have inhibitors to Factor VIII or Factor IX , are aged…
- Trial name
- PerSept3 (NCT02548143)
- Testing
- Coagulation Factor VIIa (Recombinant)
- Where
- 9 sites in United States, Mexico and 4 more
- Volunteers
- 12 people
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
Hypophosphatasia (HPP)
A Study to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALE1 in Healthy Adults and Adults With Hypophosphatasia in Order to Identify Suitable Doses of ALE1
This is a phase 1/2a randomised, placebo controlled, double-blind study investigating the safety, tolerability, pharmacokinetics, and pharmacodynamics of ALE1 on healthy adult subjects and adult patients with Hypophosphatasia…
- Trial name
- NCT07179640
- Testing
- ALE1
- Where
- 3 sites in Germany, New Zealand and 1 more
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Alesta Therapeutics
Cerebrovascular Accident, Stroke, Acute
Fabry and Cardiomyopathy (FaCard)
Primary objective and endpoint is the analysis of the long-term course of lyso-Gb3 and its clinical correlation to the progression of the cardiomyopathy in N215S-Fabry patients.
- Conditions
- Cerebrovascular Accident, Stroke, Acute, Cerebral Stroke
- Trial name
- FaCard (NCT01429597)
- Where
- Germany
- Sponsor
- CENTOGENE GmbH Rostock
Morquio Disease
Establishment of Human Cellular Disease Models for Morquio Disease
Establishment of human cellular disease models for Morquio disease for an individualized therapy development having the capacity to address both hepatic and neurologic forms of the disease
- Trial name
- IPSMORQUIO (NCT03872713)
- Where
- Pakistan
- Volunteers
- 40 people
- Sponsor
- CENTOGENE GmbH Rostock
Atopic Dermatitis
Pharmacodynamics of Omiganan BID in Patients With Atopic Dermatitis
This study has a randomized, double-blind, placebo-controlled design to assess the efficacy, pharmacodynamics and safety/tolerability of omiganan in patients with mild to moderate atopic dermatitis when applied BID to all atopic…
- Trial name
- NCT03091426
- Testing
- Omiganan
- Where
- Netherlands
- Volunteers
- 80 people
- Sponsor
- Maruho Co., Ltd.
Wet Age-related Macular Degeneration
A Study to Evaluate the Efficacy and Safety of Intravitreal KSI-301 Compared With Intravitreal Aflibercept in Participants With Neovascular (Wet) Age-related Macular Degeneration (wAMD)
This Phase 3 study will evaluate the efficacy and safety of KSI-301 compared to aflibercept, in participants with neovascular (wet) age-related macular degeneration (wAMD)
- Trial name
- DAYLIGHT (NCT04964089)
- Testing
- KSI-301, Aflibercept
- Where
- 66 sites in United States, Puerto Rico
- Volunteers
- 557 people
- Sponsor
- Kodiak Sciences Inc
Non-Small Cell Lung Cancer
Combination Study of RMC-4630 and Sotorasib for NSCLC Subjects With KRASG12C Mutation After Failure of Prior Standard Therapies
The purpose of this study is to evaluate the antitumor effects of sotorasib and RMC-4630 in subjects with KRASG12C mutant NSCLC
- Trial name
- NCT05054725
- Testing
- RMC-4630, Sotorasib
- Where
- 60 sites in United States, Australia and 8 more
- Volunteers
- 47 people
- Sponsor
- Revolution Medicines, Inc.
Facial Photoaging, Deepening of Facial Wrinkles
Regenmedic Cream 2.0 12-week Long-term Clinical Test
The goal of this study is to evaluate the anti-wrinkle, firming, moisturizing and repairing efficacy of a test facial cream after single use and 12 weeks of continuous use via clinical evaluation, instrumental measurement, image…
- Conditions
- Facial Photoaging, Deepening of Facial Wrinkles, Skin Laxity, Decreased Skin Elasticity
- Trial name
- NCT07874113
- Testing
- Test facial cream (topical skin care product)
- Where
- China
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- ChinaNorm
Growth Hormone Deficiency, Turner's Syndrome
Evaluation of the Adherence and the Patient Acceptability of Zomacton® Treatment With the Zomajet® Vision X Device
The study is performed to collect long-term data on the treatment adherence and patient's acceptability when Zomacton®10 mg is administered with the Zomajet® Vision X device in patients with a growth hormone deficiency or…
- Trial name
- ZOMAXEPT (NCT02018172)
- Testing
- somatropin
- Where
- France
- Volunteers
- 3 people
- Sponsor
- Ferring Pharmaceuticals
Advanced Solid Tumor, Advanced Solid Malignancies
A Phase I/II Study to Evaluate the Safety, Pharmacokinetics and Efficacy of PRJ1-3024 in Subjects with Advanced Solid Tumors
This is a multicenter, open-label study to assess the safety and preliminary efficacy and to determine the maximum tolerated dose (MTD) or maximum administration dose (MAD) and recommended Phase 2 doses (RP2D) of PRJ1-3024 in…
- Trial name
- NCT05315167
- Testing
- PRJ1-3024
- Where
- 5 sites in China
- Volunteers
- 267 people
- Sponsor
- Zhuhai Yufan Biotechnologies Co., Ltd
Huntington Disease
28-Day Study of SAGE-718 on Functioning Capacity in Participants With Huntington's Disease
The primary purpose of this study is to assess the magnitude of the baseline difference between participants with early Huntington's Disease (HD) and healthy participants (HP) with respect to measures of cognitive performance.
- Trial name
- NCT05358821
- Testing
- SAGE-718
- Where
- 14 sites in United States, Canada
- Volunteers
- 69 people, healthy volunteers welcome
- Sponsor
- Supernus Pharmaceuticals, Inc.
Healthy Subjects, Cystic Fibrosis
A Phase 1/2a Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of IONIS-ENaCRx in Healthy Volunteers and Patients With Cystic Fibrosis
This Phase 1/2a study is a double-blinded (subject and Investigator), randomized, placebo-controlled, dose-escalation study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple…
- Trial name
- NCT03647228
- Testing
- IONIS-ENaCRx
- Where
- 8 sites in Germany, United Kingdom
- Volunteers
- 98 people, healthy volunteers welcome
- Sponsor
- Ionis Pharmaceuticals, Inc.
Meningococcal Vaccine
Trial to Describe the Safety, Tolerability, and Immunogenicity of Trumenba When Administered to Immunocompromised Participants ≥10 Years of Age
The aim of this study is to evaluate the safety, tolerability, and immunogenicity of 2 doses of Trumenba® (on a 0- and 6-month schedule) in immunocompromised participants by functionally assessing antibody production in asplenic…
- Trial name
- NCT04893811
- Testing
- Trumenba
- Where
- 14 sites in Czechia, Poland and 1 more
- Volunteers
- 53 people, healthy volunteers welcome
- Sponsor
- Pfizer
Exocrine Pancreatic Insufficiency, Cystic Fibrosis
SOLUTION: Study of Oral Liprotamase Unit-Matched Therapy Of Non-Porcine Origin in Patients With Cystic Fibrosis
Liprotamase powder is a non-porcine, soluble and stable mixture of three digestive enzymes including lipase, protease, and amylase.
- Trial name
- NCT02279498
- Testing
- Liprotamase, porcine (pig) PERT
- Where
- 54 sites in United States, Canada and 5 more
- Volunteers
- 128 people
- Sponsor
- Anthera Pharmaceuticals
Fanconi Anemia
Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102
Long-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of RP-L102
- Trial name
- NCT07527975
- Testing
- RP-L102
- Where
- 3 sites in United States, Spain and 1 more
- Volunteers
- 14 people
- Sponsor
- Rocket Pharmaceuticals Inc.
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