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NCT ID: NCT05794139 Recruiting - Clinical trials for Spinal Muscular Atrophy

Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy

SYNAPSE-SMA
Start date: September 21, 2023
Phase: Phase 2
Study type: Interventional

The purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of NMD670 in the treatment of ambulatory adults with spinal muscular atrophy type 3

NCT ID: NCT05791968 Recruiting - Insulin Sensitivity Clinical Trials

Blood Donation Could Improve Insulin Sensitivity

GLUCFER
Start date: September 2023
Phase: N/A
Study type: Interventional

Studies in the literature suggest that people with obesity have an excess of stored iron. There is possibly an inverse relationship between ferritin levels and the actions of insulin on glycemic control. The reduction of stored iron by simply donating blood could result in improvements in glycemic control in people with obesity and prediabetes. We propose, to reduce ferritin levels through a standard donation of a unit of whole blood, and to measure if it positively affects glycemic control.

NCT ID: NCT05791708 Recruiting - Clinical trials for Cold Agglutinin Disease (CAD)

Cold Agglutinin Disease Real World Evidence Registry

CADENCE
Start date: December 12, 2019
Phase:
Study type: Observational [Patient Registry]

This is a multinational, multi-center, observational, prospective, longitudinal disease registry designed to collect data on participants with cold agglutinin disease (CAD) or cold agglutinin syndrome (CAS). Among them, a minimum of 30 patients with CAD treated with sutimlimab are expected to take part in the sutimlimab cohort study. Patients with CAD who have been enrolled in previous sutimlimab clinical trials (e.g., BIVV009-01/LTS16214 [NCT02502903,CAD patients], BIVV009-03/EFC16215 [NCT03347396], and BIVV009-04/EFC16216 [NCT03347422]) and who either completed or discontinued the corresponding clinical trial are eligible to participate in the registry.

NCT ID: NCT05789537 Recruiting - Hemophilia B Clinical Trials

A Study of SerpinPC in Participants With Hemophilia B (HemB) With Inhibitors

PRESent-3
Start date: July 28, 2023
Phase: Phase 2
Study type: Interventional

The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetic (PK) profile of prophylactic SerpinPC in participants with Hemophilia B with inhibitors, as part of the SerpinPC registrational program.

NCT ID: NCT05789524 Recruiting - Hemophilia A Clinical Trials

The Efficacy and Safety of SerpinPC in Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B

PRESent-2
Start date: July 6, 2023
Phase: Phase 2
Study type: Interventional

The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of prophylactic SerpinPC administered subcutaneously (SC) to participants with severe hemophilia A (HemA) (with or without inhibitors) or moderately severe to severe hemophilia B (HemB) (without inhibitors) as part of the SerpinPC registrational program. This study consists of 3 parts: Part 1: dose-justification phase, Part 2: dose-confirmatory phase, Part 3: extension phase for participants who complete either Part 1 or Part 2. This adaptive design study has a randomized dose-justification component to investigate the efficacy and safety of SerpinPC as a therapeutic option, principally for participants with HemB without inhibitors. SerpinPC has a novel mechanism of action compared with marketed treatments and those that are in development.

NCT ID: NCT05789082 Recruiting - Clinical trials for Non-Small Cell Lung Cancer

A Study Evaluating the Safety, Activity, and Pharmacokinetics of Divarasib in Combination With Other Anti-Cancer Therapies in Participants With Previously Untreated Advanced or Metastatic Non-Small Cell Lung Cancer With a KRAS G12C Mutation

Krascendo 170
Start date: June 20, 2023
Phase: Phase 1/Phase 2
Study type: Interventional

The purpose of this study is to evaluate the safety, pharmacokinetics (PK), and activity of divarasib combined with other anti-cancer therapies in participants with previously untreated, advanced or metastatic non-small cell lung cancer (NSCLC).

NCT ID: NCT05789069 Recruiting - Colorectal Cancer Clinical Trials

A Study of HFB200603 as a Single Agent and in Combination With Tislelizumab in Adult Patients With Advanced Solid Tumors

Start date: May 9, 2023
Phase: Phase 1
Study type: Interventional

The purpose of this study is to test the safety and tolerability of HFB200603 as a single agent and in combination with tislelizumab in patients with advanced cancers. There are two parts in this study. During the escalation part, groups of participants will receive increasing doses of HFB200603 as a monotherapy or in combination with tislelizumab until a safe and tolerable dose of HFB200603 as a single agent or combination therapy is determined. During the expansion part, participants will take the doses of HFB200603 as a monotherapy (optional arm) or in combination with tislelizumab that were determined from the escalation part of the study and will be assigned to a group based on the type of cancer the participants have.

NCT ID: NCT05788614 Recruiting - Clinical trials for Shoulder Osteoarthritis

Retroversion in Reverse Shoulder Arthroplasty

RV-RSA
Start date: June 30, 2022
Phase: N/A
Study type: Interventional

The goal of this clinical trial is to compare function in reverse shoulder arthroplasty (RSA) using two different degrees of humeral retroversion (RV). The main question it aims to answer is: • Does external rotation improves when using a higher retroversion? Participants will randomly receive a 0 degree or 30 degree RV for their RSA. They will be followed for two years and clinical outcomes will be recorded and analyzed.

NCT ID: NCT05788536 Recruiting - Clinical trials for Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)

A Study of DB-OTO, an Adeno-associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations

CHORD
Start date: May 12, 2023
Phase: Phase 1/Phase 2
Study type: Interventional

Regeneron is conducting a study of an investigational new drug called DB-OTO. DB-OTO is a gene therapy that is being developed to treat children who have hearing loss due to changes in the otoferlin gene. The purpose of this study is to: - Learn about the safety of DB-OTO - Determine how well DB-OTO is tolerated (does not cause ongoing discomfort) - Evaluate the efficacy of DB-OTO (how well DB-OTO works)

NCT ID: NCT05788432 Recruiting - Clinical trials for Coronary Artery Disease

Sequent Extended Study

rEpic06
Start date: March 28, 2023
Phase:
Study type: Observational

Multicenter, prospective, non-randomized, post-market clinical follow-up (PMCF) study to confirm and support the clinical safety and performance of Sequent Please Neo to meet EU Medical Device regulation (MDR) requirements in all the consecutive patients treated with Sequent Please Neo.