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NCT ID: NCT05168202 Active, not recruiting - Clinical trials for Myelodysplastic Syndromes

A Study to Assess the Effect of CC-95251 in Participants With Acute Myeloid Leukemia and Myelodysplastic Syndromes

Start date: January 19, 2022
Phase: Phase 1
Study type: Interventional

The purpose of this study is to evaluate the safety, tolerability, and preliminary clinical activity of CC-95251 alone and in combination with antineoplastic agents in participants with relapsed or refractory acute myeloid leukemia and relapsed or refractory and treatment-naive higher risk melodysplastic syndromes.

NCT ID: NCT05165628 Active, not recruiting - Diabetic Foot Ulcer Clinical Trials

Safety, Tolerability and Efficacy of CYP-006TK in Adults With Diabetic Foot Ulcers

Start date: January 4, 2022
Phase: Phase 1
Study type: Interventional

Design: A randomised, controlled, prospective trial. Participants will be patients with non-healing diabetic foot ulcers. The study will aim to recruit 15 participants per study group (30 participants in total). Participants will be randomly allocated to one of two treatment groups: - Group 1: CYP-006TK - Group 2: Standard care This will be an open label study with respect to treatment allocation. However, the person reviewing images of the study ulcers to assess healing will be blind to the participant's treatment allocation. Participants assigned to Group 1 will be treated with CYP-0006TK dressings on 8 occasions over 4 weeks. The dressings will be changed every 3 or 4 days. After the first 4 weeks, participants in Group 1 will revert to standard care for the rest of the study. Participants assigned to Group 2 will have their ulcer treated with standard care throughout the study. Participants will attend a total of 16 scheduled visits over 24 weeks. There will be a mixture of on-site (hospital/clinic) visits, and home visits. The study will end 24 weeks after the initiation of treatment, unless the study ulcer is completely headed before then.

NCT ID: NCT05163288 Active, not recruiting - Clinical trials for Niemann-Pick Disease, Type C

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

Start date: June 30, 2022
Phase: Phase 3
Study type: Interventional

A pivotal, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Niemann Pick disease type C (NPC). The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.

NCT ID: NCT05162768 Active, not recruiting - Clinical trials for Mitochondrial Diseases

Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)

NuPower
Start date: April 29, 2022
Phase: Phase 3
Study type: Interventional

SPIMD-301 is a 48-week, randomized, double-blind, parallel-group, placebo-controlled trial to assess efficacy and safety of single daily subcutaneous (SC) administration of elamipretide as a treatment for subjects with primary mitochondrial myopathy associated with nuclear DNA mutations (nPMD).

NCT ID: NCT05162586 Active, not recruiting - Clinical trials for Systemic Lupus Erythematosus

The WILLOW Study With M5049 in SLE and CLE (SCLE and/or DLE) (WILLOW)

Start date: March 31, 2022
Phase: Phase 2
Study type: Interventional

The purpose of this Proof of Concept (PoC) and Dose-finding (DF) basket study is to evaluate the efficacy and safety of orally administered Enpatoran over 24 weeks in systemic lupus erythematosus (SLE) and cutaneous lupus erythematosus (CLE; subacute cutaneous lupus erythematosus [SCLE] and/or discoid lupus erythematosus [DLE]) participants in a randomized, double-blind, placebo-controlled, parallel, adaptive and dose-ranging setting. Study Duration: 33 weeks Visit Frequency: every 2 or 4 weeks Enpatoran is not available through an expanded access program.

NCT ID: NCT05160805 Active, not recruiting - Open Angle Glaucoma Clinical Trials

A Phase 1b Study of ONL1204 Ophthalmic Solution in Patients With Progressing Open Angle Glaucoma

Start date: May 2, 2022
Phase: Phase 1
Study type: Interventional

The purpose of this study is to demonstrate the safety and tolerability of ONL1204 Ophthalmic Solution in patients with progressing open angle glaucoma. ONL1204 Ophthalmic Solution is a first-in-class inhibitor of fragment apoptosis stimulator (Fas) receptor-mediated cell death that has demonstrated protection of multiple retinal cell types in numerous preclinical models of retinal disease. Apoptosis of retinal ganglion cells is associated with progressive glaucoma. Nonclinical data on ONL1204 Ophthalmic Solution suggest that ONL1204 Ophthalmic Solution may inhibit the cell death pathways in these cells.

NCT ID: NCT05155254 Active, not recruiting - Metastatic Melanoma Clinical Trials

IO102-IO103 in Combination With Pembrolizumab Versus Pembrolizumab Alone in Advanced Melanoma (IOB-013 / KN-D18)

Start date: May 17, 2022
Phase: Phase 3
Study type: Interventional

Phase 3, multicenter, international, open-label, randomized, 2-arm trial investigating the safety and efficacy of IO102-IO103 in combination with pembrolizumab as first-line treatment for patients with previously untreated unresectable or metastatic (advanced) melanoma. Patients will be stratified on the basis of the following factors; Disease stage: Stage III (unresectable) and IV M1a-b versus stage IV M1c-d and BRAFV600 mutation status: mutated vs wild type. All patients will receive pembrolizumab 200 mg intravenously every 3 weeks for a maximum of 35 cycles (up to 2 years treatment). Patients randomized to IO102-IO103 dual-antigen, immunotherapeutic arm will also be given IO102-IO103 Q3W with an additional dose given during the induction period on Day 8 of cycles 1 and 2. IO102 IO103 will thereafter be administered subcutaneous every 3 weeks during the maintenance period. Each patient can be treated for a maximum of 37 administrations in total (up to 2 years of treatment). The primary objective is to investigate the efficacy of IO102-IO103 in combination with pembrolizumab (compared with pembrolizumab alone) in terms of progression free survival.

NCT ID: NCT05153317 Active, not recruiting - Cystic Fibrosis Clinical Trials

Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis (CF) Participants 2 Years and Older

Start date: January 17, 2022
Phase: Phase 3
Study type: Interventional

The purpose of this study is to evaluate the long term safety, tolerability, efficacy and pharmacodynamics of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) in CF participants 2 years of age and older.

NCT ID: NCT05151601 Active, not recruiting - Anxiety Clinical Trials

Combined Human Milk Oligosaccharides (HMOs) and Probiotics Intervention for Children With Autism

Start date: May 17, 2022
Phase: N/A
Study type: Interventional

This research project will investigate if a supplement containing a unique combination of prebiotics and probiotics can influence behaviour in children diagnosed with autism spectrum disorder. The study will use a combination of human milk oligosaccharides (HMOs, prebiotics) and probiotics as an oral powder.This clinical trial will have two consecutive phases. Phase 1A is an 8-week randomised, double-blinded, placebo-controlled trial. Participants will be recruited and randomised (1:1) to receive either the investigational product (treatment group, n=30) or the placebo (control group, n=30). Phase 1B is an 8-week open-label study. All participants that complete Phase 1A will move into Phase 1B (n=60). This allows all participants to receive the investigational product and will provide additional information on increased duration of treatment.

NCT ID: NCT05150236 Active, not recruiting - Prostate Cancer Clinical Trials

EVOLUTION: 177Lu-PSMA Therapy Versus 177Lu-PSMA in Combination With Ipilimumab and Nivolumab for Men With mCRPC

ANZUP2001
Start date: April 29, 2022
Phase: Phase 2
Study type: Interventional

This phase II study will investigate the activity and safety of radionuclide 177Lu-PSMA therapy versus 177Lu-PSMA in combination with Ipilimumab and Nivolumab in patients with metastatic castrate resistant prostate cancer (mCRPC).