Found 34 clinical trials
Capivasertib+Abiraterone as Treatment for Patients With Metastatic Hormone-sensitive Prostate Cancer and PTEN Deficiency
This study will assess the efficacy and safety of capivasertib plus abiraterone (+prednisone/prednisolone) plus androgen deprivation therapy (ADT) versus placebo plus abiraterone (+prednisone/prednisolone) plus ADT in participants with mHSPC whose tumours are characterised by PTEN deficiency. The intention of the study is to demonstrate that in participants with mHSPC, the …
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- 19 Feb, 2024
A Phase 1/2 Study of SHP648 an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects
The purpose of this study is to evaluate the safety and dose escalation of SHP648 an adeno-associated viral vector for gene transfer in hemophilia B participants.
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- 19 Feb, 2024
Safety Tolerability and Pharmacokinetics of ARCT-810 in Healthy Adult Subjects
Determine the safety, tolerability and pharmacokinetics of single doses of ARCT-810 in healthy adult subjects.
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- 19 Feb, 2024
Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-864 in PiZZ subjects.
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- 19 Feb, 2024
AAV Gene Therapy Study for Subjects With PKU
This is a Phase 1/2, open-label, dose escalation study to evaluate the safety, efficacy and tolerability of BMN 307 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of BMN 307 and will be followed for safety and efficacy.
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- 19 Feb, 2024
Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia
This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 24 weeks in approximately 165 adult subjects with classic CAH due to 21-hydroxylase deficiency. The study consists of a 6 month randomized, double blind, placebo-controlled period, followed by 1 year of …
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- 19 Feb, 2024
Gene Therapy for X Linked Severe Combined Immunodeficiency
A safety and efficacy clinical study of a lentiviral vector to transfer IL2RG complementary DNA to bone marrow stem cells in ten children with genetic diagnosed X-SCIDsevere combined immune deficiency .The ten children will be followed for 3-5 years and be evaluated by clinical characteristics, vector marking (vector copy number …
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- 19 Feb, 2024
A Multinational Randomized Double-blind Placebo-controlled Study to Assess the Efficacy Pharmacodynamics Pharmacokinetics and Safety of Venglustat in Late-onset GM2
Primary Objectives: Primary population (adult participants with late-onset GM2 gangliosidosis): To assess the efficacy and pharmacodynamics (PD) of daily oral dosing of venglustat when administered over a 104-week period Secondary population (participants with juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1 or juvenile/adult galactosialidosis): To assess …
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- 19 Feb, 2024
- 2 locations
Determinants of Neonatal Anemia in Women Carrying Multiples
Recent data indicate that multiple births now comprise 3-4.5% of all births in the United States. Pregnant women are at risk for iron (Fe) deficiency anemia yet there are virtually no data on Fe status in women carrying multiples and current recommendations do not necessitate Fe screening among this high …
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- 22 Feb, 2024
- 2 locations
Tissue-engineered Skin Graft Repair of Autologous Scar Dermal Scaffolds
It affects the appearance and some scar contracture often leads to joint dysfunction.Patients have low quality of life, long treatment cycle, heavy social burden and high medical costs.Skin grafting is currently the gold standard for scar repair.However, there are often insufficient skin sources, easy to scar recurrence, lack of skin …
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- 19 Feb, 2024