Clinical trials: Genetic Diseases
Metastatic Castration Resistant Prostate Cancer
A Study of AC176 for the Treatment of Metastatic Castration Resistant Prostate Cancer
This clinical trial is evaluating a drug called AC176 in participants with metastatic castration resistant prostate cancer (mCRPC) who have progressed on at least two prior systemic therapies.
- Trial name
- NCT05241613
- Testing
- AC176
- Where
- 5 sites in United States
- Volunteers
- 28 people
- Sponsor
- Accutar Biotechnology Inc
Beta-Thalassemia
To Document the Burden of Illness on the Quality of Life and the Impact on Healthcare Utilization in (Beta) β-thalassemia Subjects Who Are Transfusion Dependent (TD) and Non-transfusion Dependent (NTD) Receiving Standard of Care
This is a multi-site, prospective, observational study implemented in β-thalassemia treatment centers from 5 countries (Italy, Turkey, Greece, Lebanon, and Thailand).
- Trial name
- NCT02626689
- Testing
- Quality of Life (QOL) questionnaires, Healthcare Resource Utilization
- Where
- 5 sites in Greece, Italy and 3 more
- Volunteers
- 100 people
- Sponsor
- Celgene
Behçet's Syndrome
A Phase 3 Randomized, Double-blind Study to Evaluate the Efficacy and Safety of Apremilast (CC-10004) in Subjects With Active Behçet's Disease
The main objective of this study is to evaluate the efficacy and safety of apremilast in the treatment of oral ulcers in adults with active Behçet's disease (BD).
- Trial name
- NCT02307513
- Testing
- Apremilast
- Where
- 63 sites in United States, France and 8 more
- Volunteers
- 207 people
- Sponsor
- Amgen
Retinitis Pigmentosa
Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene
The goal of this phase 1 clinical trial is to learn about the safety and efficacy of a gene therapy, VG901, in patients with a rare disorder of the eye called Retinitis Pigmentosa.
- Trial name
- NCT06291935
- Testing
- VG901
- Where
- Germany
- Volunteers
- 6 people
- Sponsor
- VeonGen Therapeutics GmbH
Elevated LDL-Cholesterol (LDL-C)
Trial to Evaluate Safety and Tolerability of ALN-PCS02 in Subjects With Elevated LDL-Cholesterol (LDL-C)
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-PCS02 in subjects with Elevated LDL-Cholesterol (LDL-C).
- Trial name
- NCT01437059
- Testing
- ALN-PCS02, Sterile Normal Saline (0.9% NaCl)
- Where
- 2 sites in United Kingdom
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Facioscapulohumeral Muscular Dystrophy
Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)
Study A083-02 is a multi-center, Phase 2 study to evaluate the safety, tolerability, pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of locally-acting ACE-083 in patients with Facioscapulohumeral muscular dystrophy…
- Trial name
- NCT02927080
- Testing
- ACE-083
- Where
- 24 sites in United States, Canada and 1 more
- Volunteers
- 95 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Blood and Lymphatic Diseases
Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.
Primary Objectives: Long-term safety of BIVV003 in participants with severe sickle cell disease (SCD) and ST- 400 in participants with transfusion-dependent beta-thalassemia (TDT) Secondary Objectives: Long-term efficacy of the…
- Trial name
- NCT05145062
- Testing
- BIVV003, ST-400
- Where
- 7 sites in United States
- Volunteers
- 8 people
- Sponsor
- Sangamo Therapeutics
Skin Laxity
Hybrid Fractional Laser for Treatment of Off Face Body Skin Quality
To demonstrate effectiveness of hybrid fractional laser for the treatment of poor skin quality on the decolletage, back of the hands and lower legs
- Trial name
- NCT03969485
- Testing
- Hybrid Fractional Laser
- Where
- United States
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- Sciton
Acute Gout Flare
Study Utilizing Rilonacept in Gout Exacerbations
This purpose of this clinical research was to determine the efficacy and safety of an experimental drug called Rilonacept in participants with an acute gout attack. Participants participated in this study for 30 days.
- Trial name
- SURGE (NCT00855920)
- Testing
- Rilonacept, Indomethacin
- Where
- 44 sites in United States
- Volunteers
- 225 people
- Sponsor
- Regeneron Pharmaceuticals
Hemophilia B
HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile. The study drug is identified as AAV5-hFIXco-Padua (AMT- 061).
- Trial name
- NCT03569891
- Testing
- AAV5-hFIXco-Padua, Factor IX (FIX)
- Where
- 33 sites in United States, Belgium and 6 more
- Volunteers
- 67 people
- Sponsor
- CSL Behring
Huntington's Disease
A Study in Subjects With Late Prodromal & Early Manifest HD to Assess the Safety, Tolerability, pk, and Efficacy of Pepi
The purpose of this study is to evaluate the safety, tolerability, PK, and efficacy of Pepinemab in subjects with late prodromal and early manifest Huntington's disease.
- Trial name
- SIGNAL-HD (NCT02481674)
- Testing
- VX15/2503
- Where
- 32 sites in United States, Canada
- Volunteers
- 301 people
- Sponsor
- Vaccinex Inc.
Transthyretin Amyloid Polyneuropathy (ATTR-PN)
The Effect Of Tafamidis Meglumine In Transthyretin Amyloid Polyneuropathy Patients
This is a single-arm, open-label, multicenter study designed to evaluate the efficacy, safety, tolerability as well as pharmacodynamics of tafamidis meglumine in ATTR-PN participants in China.
- Trial name
- NCT04828993
- Testing
- tafamidis meglumine
- Where
- 9 sites in China
- Volunteers
- 15 people
- Sponsor
- Pfizer
Primary Hyperoxaluria Type 1, PH1
Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1)
This study will be conducted to evaluate the efficacy and safety of YOLT-203 in children and adults with Primary Hyperoxaluria Type 1.
- Trial name
- NCT07587021
- Testing
- YOLT-203
- Where
- 4 sites in United States
- Volunteers
- 36 people
- Sponsor
- YolTech Therapeutics Co., Ltd
Beta-Thalassemia, Thalassemia
A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participans with transfusion-dependent β-thalassemia (TDT).
- Conditions
- Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases
- Trial name
- NCT03655678
- Testing
- CTX001
- Where
- 14 sites in United States, Canada and 3 more
- Volunteers
- 59 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Hereditary Periodic Fever Syndromes, Cryopyrin-associated Periodic Syndromes (CAPS)
Ilaris NIS in Korea
This is a study to evaluate safety and effectiveness of Ilaris in adult and pediatric patients receiving the drug in a clinical setting for any of the following indications, Hereditary Periodic Fever Syndromes…
- Conditions
- Hereditary Periodic Fever Syndromes, Cryopyrin-associated Periodic Syndromes (CAPS), Colchicine Resistance Familial Mediterranean Fever (crFMF), TNF Receptor Associated Periodic Syndrome (TRAPS)
- Trial name
- REASSURE (NCT06838143)
- Testing
- Ilaris
- Where
- South Korea
- Volunteers
- 25 people
- Sponsor
- Novartis Pharmaceuticals
Huntington's Disease
Efficacy and Safety of Tetrabenazine in Chorea
The primary objective of this study was to establish the absolute reduction of chorea in participants with Huntington's disease(HD) treated with tetrabenazine or placebo
- Trial name
- NCT00219804
- Sponsor
- Prestwick Pharmaceuticals
Atopic Dermatitis
A Clinical Trial to Compare Topical Agents in Adults With Mild to Moderate Atopic Dermatitis (AD)
An Explorative Clinical Trial to Evaluate an Intra Patient Comparison Design of Topical Agents in Adults with Mild to Moderate Atopic Dermatitis.
- Trial name
- NCT02376049
- Testing
- Pimecrolimus cream, Betamethasone dipropionate cream, Clobetasol propionate cream
- Where
- 2 sites in Canada, Israel
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- LEO Pharma
Sickle Cell Anemia, Sickle Cell Disease
Safety of Rivipansel (GMI-1070) in the Treatment of One or More Vaso-Occlusive Crises in Hospitalized Subjects With Sickle Cell Disease
This is an open label extension study in subjects with Sickle Cell Disease (SCD) who have completed the double blind Phase 3 study (B5201002).
- Conditions
- Sickle Cell Anemia, Sickle Cell Disease, Sickle Cell Disorders, Pain Crisis
- Trial name
- NCT02433158
- Testing
- Rivipansel
- Where
- 97 sites in United States, Canada
- Volunteers
- 154 people
- Sponsor
- GlycoMimetics Incorporated
Sickle Cell Disease
A 3-year, Prospective, Non-interventional, Multicenter Registry in Sickle Cell Disease (SCD) Patients
A long term observational study in sickle cell disease will enhance the understanding of the disease patterns, current transfusion practices, treatments and outcomes in sickle cell disease.
- Trial name
- FISCO (NCT01220115)
- Where
- 42 sites in United States
- Volunteers
- 498 people
- Sponsor
- Novartis
Age-related Macular Degeneration
Genetic Load and Phenotype in Aggressive AMD
Patients with AMD will provide cheek cell samples to determine if their is a correlation between genotype (DNA markers) and phenotype (the type of AMD the patient has).
- Trial name
- RPED Genetics (NCT01650948)
- Testing
- RetnaGene AMD LDT
- Where
- 3 sites in United States
- Volunteers
- 100 people
- Sponsor
- Sequenom, Inc.
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