Clinical trials: Phase 1b
Relapsed Ewing Sarcoma
A Study of INCB059872 in Relapsed or Refractory Ewing Sarcoma
The purpose of this study is to evaluate the safety and preliminary antitumor activity of INCB059872 in participants with Ewing sarcoma who are refractory or relapsed from prior standard therapy and not eligible for further…
- Trial name
- NCT03514407
- Testing
- INCB059872
- Where
- 11 sites in United States, Italy and 2 more
- Volunteers
- 25 people
- Sponsor
- Incyte Corporation
Cystic Fibrosis
Study to Assess Amphotericin B Cystetic for Inhalation (ABCI) Doses in Healthy Volunteers & People with Cystic Fibrosis
This is a 3-part, single-ascending dose Phase 1a randomized, double-blind, placebo-controlled study in healthy volunteers (Part A) and multiple-ascending dose Phase 1a randomized, double-blind, placebo-controlled study in healthy…
- Trial name
- ABCI (NCT05802264)
- Testing
- ABCI
- Where
- 5 sites in Australia, New Zealand
- Volunteers
- 108 people, healthy volunteers welcome
- Sponsor
- Cystetic Medicines, Inc.
Central Nervous System Neoplasms, Brain Tumor
B7-H3.CD28Z.CART in CNS Neoplasms
The purpose of this research study is to test the safety and effectiveness of a cell therapy at different doses for children and young adults with recurrent or progressive brain tumors.
- Conditions
- Central Nervous System Neoplasms, Brain Tumor, Brain Tumor, Recurrent, Brain Tumor, Pediatric
- Trial name
- NCT07390539
- Testing
- B7-H3.CD28Z.CART, Fludarabine, Cyclophosphamide
- Where
- 2 sites in United States
- Volunteers
- 70 people
- Sponsor
- Robbie Majzner
Vanishing White Matter Disease
An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease
Fosigotifator is an investigational drug being researched for the treatment of Vanishing White Matter disease in adult, pediatric and infant participants.
- Trial name
- NCT05757141
- Testing
- Fosigotifator
- Where
- 5 sites in United States, Canada and 1 more
- Volunteers
- 50 people
- Sponsor
- Calico Life Sciences LLC
Classical Hodgkin Lymphoma
CD30 CAR T-cells Post AutoHSCT for Poor-risk Hodgkin Lymphoma
Patients with poor risk classical Hodgkin Lymphoma (cHL) will undergo myeloablative chemotherapy (MAC) with autologous stem cell transplantation (AutoHSCT) and subsequently receive autologous CD30+ CAR T-cells.
- Trial name
- NCT06617286
- Testing
- CD30 CAR T-cell
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- New York Medical College
FSHD, FSHD1
Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Participants with…
- Conditions
- FSHD, FSHD1, FSHD2, FMD
- Trial name
- FORTITUDE (NCT05747924)
- Testing
- AOC 1020
- Where
- 17 sites in United States, Canada and 1 more
- Volunteers
- 90 people
- Sponsor
- Avidity Biosciences, Inc.
Neoplasms
Clinical Trial of Safety, Tolerability and Antitumor Activity of Genetically Engineered T Cells in Combination With Anti-Cancer Agents in Relapsed and Refractory Synovial Sarcoma Expressing New York Esophageal Antigen-1 (NY-ESO-1) and/or LAGE-1a
Adoptive T-cell therapy is a therapeutic approach that aims to generate an anti-tumor T-cell immune response by infusing a cancer subjects own T-cells obtained by leukapheresis, engineered and expanded in-vitro to express a tumor…
- Trial name
- NCT03697824
- Testing
- GSK3377794, Pembrolizumab, Fludarabine
- Sponsor
- GlaxoSmithKline
Pediatric Growth Hormone Deficiency
Versartis Trial in Children to Assess Long-Acting Growth Hormone
This is a Phase 1b/2a study of VRS-317 (long-acting growth hormone) in pediatric patients with growth hormone deficiency. During Phase 1b, pediatric patients each will receive a single subcutaneous injection of VRS-317.
- Trial name
- VERTICAL (NCT01718041)
- Testing
- Experimental: VRS-317
- Where
- 23 sites in United States
- Volunteers
- 64 people
- Sponsor
- Versartis Inc.
Metastatic Solid Tumors
Study to Investigate the Safety and Activity of Aldoxorubicin Plus Gemcitabine in Subjects With Metastatic Solid Tumors
This is a Phase 1 study to investigate the safety and activity of aldoxorubicin plus gemcitabine in Subjects with solid tumors.
- Trial name
- NCT02235688
- Testing
- aldoxorubicin, gemcitabine
- Where
- 2 sites in United States
- Volunteers
- 30 people
- Sponsor
- ImmunityBio, Inc.
Cystic Fibrosis
Safety/Tolerability Study of Arikayce™ in Cystic Fibrosis Patients With Chronic Infection Due to Pseudomonas Aeruginosa
This is a study to determine the safety and tolerability of 28 days of daily dosing of 560 mg of Arikayce™ versus placebo and daily dosing of 70 mg and 140 mg of Arikayce™ versus placebo in patients who have Cystic fibrosis (CF)…
- Trial name
- NCT00558844
- Testing
- Arikayce™ 560 mg, Arikayce™ 70 mg, Arikayce™ 140 mg
- Where
- 19 sites in United States
- Volunteers
- 41 people
- Sponsor
- Insmed Incorporated
Severe Hemophilia A
A Study to Learn About How Changing Therapy From Emicizumab to Marstacimab Affects People With the Severe Hemophilia A.
The purpose of the study is to learn about safety, how the body processes marstacimab and how it works in patients with severe hemophilia A without inhibitors.
- Trial name
- NCT06703606
- Testing
- MARSTACIMAB
- Where
- 10 sites in United States, India and 1 more
- Volunteers
- 15 people
- Sponsor
- Pfizer
Duchenne Muscular Dystrophy
A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to determine whether increasing doses of SMT C1100 are safe, well tolerated and achieve appropriate blood levels in patients with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT02056808
- Testing
- SMT C1100
- Where
- 4 sites in United Kingdom
- Volunteers
- 12 people
- Sponsor
- Summit Therapeutics
Non Hodgkin Lymphoma, Richter Transformation
Study of Oral Administration of LP-118 in Patients With Relapsed or Refractory CLL, SLL, MDS, MDS/MPN, AML, CMML-2, MPN-BP, ALL, MF, NHL, RT, MM or T-PLL.
This is a Phase 1, multi-center, open-label study with a dose-escalation phase (Phase 1a) and a cohort expansion phase (Phase 1b), to evaluate the safety, tolerability, and PK profile of LP-118 under a once daily oral dosing…
- Conditions
- Non Hodgkin Lymphoma, Richter Transformation, Multiple Myeloma, T-cell-prolymphocytic Leukemia
- Trial name
- NCT04771572
- Testing
- LP-118
- Where
- 8 sites in United States
- Volunteers
- 100 people
- Sponsor
- Newave Pharmaceutical Inc