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Found 91 clinical trials

• A Global Prospective Observational Registry of Patients with Pompe Disease

  This is an international Registry that would like to collect and evaluate outcomes on patients with Pompe. The Sponsor of the study will look at long-term…

  Ages

  0 Years - 100 Years

  Sexes

  All

• A Multi-Center Phase 2/3 Randomized, Double-Blind, Placebo-Controlled, Parallel- Group Safety and Efficacy Study of Dapansutrile Tablets in Subjects with an Acute Gout Flare

  The main purpose of this study is to see how well dapansutrile reduces joint pain and other joint symptoms (such as tenderness and swelling) because of an…

  Ages

  18 Years - 100 Years

  Sexes

  All

• A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 2 Study to Investigate the Efficacy and Safety of Asapiprant in Hospitalized Adults With COVID-19

  Individuals diagnosed with COVID-19 are being asked to take part in a research study of an investigational drug called Asapiprant (BGE 175). This drug is being…

  Ages

  N/A

  Sexes

  All

• A Natural History and Outcome Measure Discovery Study of PRPF31 Mutation–Associated Retinal Dystrophy (20226317)

  A Natural History and Outcome Measure Discovery Study of PRPF31 Mutation–Associated Retinitis Pigmentosa Patients with Retinitis Pigmentosa with PRPF31 Gene…

  Ages

  N/A

  Sexes

  All

• A Phase 1 Study of EG-70 as an Intravesical Administration to Patients with BCG -Unresponsive NMIBC

  A Phase 1/2 Study of EG-70 as an Intravesical Administration to Patients with BCG -Unresponsive NMIBC and High-Risk NMIBC Patients who are BCG Naïve or…

  Ages

  18 Years - 101 Years

  Sexes

  All

• A Phase 1/2 Study of EG-70 as an Intravesical Administration to Patients with BCG-Unresponsive NMIBC and High-Risk NMIBC who are BCG Naïve/Received Incomplete BCG Treatment

  This study will evaluate the safety and tolerability of EG-70, a gene therapy, which is given inside the bladder, and its effectiveness on eliminating bladder…

  Ages

  18 Years - 100 Years

  Sexes

  All

• A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Investigate the Safety and Efficacy of Oral Brepocitinib in Adults with Cutaneous Sarcoidosis

  The purpose of this study is to measure clinical safety and efficacy of oral brepocitinib in adult participants with cutaneous sarcoidosis. ### Study details…

  Ages

  18 Years - 75 Years

  Sexes

  All

• A Phase 2/3, Randomized, Controlled, Masked, Multi-center Study to Evaluate the Efficacy, Safety and Tolerability of Two Doses of AGTC-501, a Recombinant Adeno-associated Virus Vector Expressing RPGR (rAAV2tYF-GRK1-RPGR), Compared to an Untreated Control

  A Phase 2/3, Randomized, Controlled, Masked, Multi-center Study to Evaluate the Efficacy, Safety and Tolerability of Two Doses of AGTC-501, a Recombinant…

  Ages

  12 Years - 50 Years

  Sexes

  Male

• A Phase 2a Multiple Ascending Dose Study to Examine the Safety, Tolerability and Efficacy of AV-001 Injection in Patients with Pneumonia Due To COVID-19 or Respiratory Infections.

  The purpose of this research study is to assess the safety, tolerability (primary objective), efficacy, and target engagement (secondary objective) of multiple…

  Ages

  18 Years - 100 Years

  Sexes

  All

• A Phase 3, Double-blind, Randomized, Placebo-controlled Study to Assess the Efficacy, Safety, and Tolerability of EN3835 vs Placebo in the Treatment of Plantar Fibromatosis (PFI) (Also Known as Ledderhose Disease)

  This study will assess the efficacy, safety, and tolerability of EN3835, an investigational treatment, compared to placebo in the treatment of PFI (also known…

  Ages

  18 Years - N/A

  Sexes

  All