Find a Clinical Trial - United States

Follicular Lymphoma

Refractory Follicular Lymphoma

Tazverik

United States of America (the)

Not Yet Recruiting

A study to assess efficacy and safety of oral tazemetostat in adult participants with relapsed/refractory follicular lymphoma that does not have an “EZH2 gain-of-function” genetic mutation

The purpose of this research study is to learn about the effectiveness and safety of the study drug, tazemetostat, in adults with relapsed/refractory follicular lymphoma whose tumours do not have an “EZH2” genetic mutation. Follicular lymphoma is a blood cancer. It affects white blood cells called lymphocytes. White blood cells normally help to fight infections, but when you have follicular lymphoma, the blood cells can form tumours in your body. ‘Relapsed/refractory’ follicular lymphoma means the disease has either not improved or is getting worse (progressing) during or after previous treatment. Tazemetostat already has approval in the United States for the treatment of adult patients with relapsed/refractory follicular lymphoma with or without the “EZH2” mutation who have no satisfactory alternative treatment options. This study is being conducted to better understand the effectiveness in patients whose tumours do not have an “EZH2” genetic mutation and who previously received therapies commonly used in the U.S. in your body. ‘Relapsed/refractory’ follicular lymphoma means the disease has either not improved or is getting worse (progressing) during or after previous – 5 – SM D form protocol master data -INT / Version 1 treatment. Tazemetostat already has approval in the United States for the treatment of adult patients with relapsed/refractory follicular lymphoma with or without the “EZH2” mutation who have no satisfactory alternative treatment options. This study is being conducted to better understand the effectiveness in patients whose tumours do not have an “EZH2” genetic mutation and who previously received therapies commonly used in the U.S. In this study, all participants will receive the study drug. It will be taken by mouth (orally), as a tablet, twice daily. The sizes and number of tumours according to scan results will be collected as well as results of safety tests (such as physical examinations and laboratory tests). The study consists of 3 or 4 periods: ? Screening period may take up to 4 weeks and require at least 1 visit. ? Treatment period will require 2 visits for each of the first 2 months, followed by 1 visit every month for the remainder of the first 6 months, followed by 1 visit every 3 months (except for women of childbearing potential [WOCBP], who will continue to have a visit every month) until unacceptable toxicity, disease progression, or the start of new systemic anticancer therapy, whichever is first. ? Safety follow -up period will last for 1 month after the last dose of tazemetostat, and it will end with 1 visit or telephone call. ? Long -term follow -up period is only for participants who stop taking tazemetostat while their disease continues to respond; this period will last until disease progression, start of new cancer treatment, or death from any cause, whichever is first. Tazemetostat will be provided to participants who tolerate it for as long as their disease does not progress. Participants may be transferred to another study or program after about 2 years for continued treatment with tazemetostat or for long-term follow-up. Patients may withdraw consent to participate at any time.

Osteosarcoma|Osteosarcoma In Children

Cabometyx|Cometriq

France

Netherlands (Kingdom of the)

Spain

Not Yet Recruiting

A study to assess whether adding maintenance cabozantinib (XL184) to best supportive care helps children, adolescents and young adults with osteosarcoma.

The participants of this study will be children, adolescents, and young adults with residual osteosarcoma, which cannot be removed completely through surgery. Participants will have achieved a partial response or stable disease at the end of conventional chemotherapy. Osteosarcoma is cancer of the bone. The cancer cells make immature bone cells, known as osteoid. Osteosarcoma is very rare, but it is the most common type of bone cancer in children and teens. It is most common in teens and young adults. In this study, participants will receive either cabozantinib and best supportive care or the best supportive care alone. Best supportive care will be provided at the investigator’s discretion and according to institutional guidelines. It includes antibiotics, nutritional support, correction of metabolic disorders, optimal symptom control and pain management (including radiotherapy), etc. but does not include tumor specific therapy. Cabozantinib will be taken by mouth (orally), as a tablet, once a day. Cabozantinib will be provided to participants who tolerate it for as long as their disease does not progress. Participants in the study receiving best supportive care alone may switch to treatment with cabozantinib and best supportive care if their disease progresses and if other eligibility criteria are met. Participants may withdraw consent to participate at any time. The estimated duration of the study for participants is 24 months, however a participant could remain in the study longer if demonstrating treatment benefit.

Primary Sclerosing Cholangitis

Iqirvo

Germany

Canada

Italy

United States of America (the)

United Kingdom of Great Britain and Northern Ireland (the)

Spain

Portugal

Recruiting

A Study to Assess Safety and Effectiveness of Elafibranor in Adult Participants with Primary Sclerosing Cholangitis.

This study will evaluate the effects of elafibranor (the study drug) in participants with Primary Sclerosing Cholangitis (PSC). PSC is a rare disease of the liver that leads to injury and destruction of bile ducts. Damage to bile ducts leads to buildup of bile in the liver, which then causes further damage, and leads to disease progression. This study will compare elafibranor to a placebo, a dummy treatment. The main objective of the trial will be to study the safety and side effects of the study drug. The trial will also study the study drug’s effects on blood tests and other tests related to PSC disease activity.

Primary Biliary Cholangitis (PBC)

Iqirvo

New Zealand

Romania

Spain

Poland

United States of America (the)

Italy

Korea (the Republic of)

Czechia

France

Recruiting

A Long-Term Study of Elafibranor in Adult Participants with Primary Biliary Cholangitis

The participants of this study will have confirmed Primary Biliary Cholangitis (PBC) and cirrhosis (scarring). PBC is a disease that progresses slowly. It causes damage to the bile ducts in the liver, leading to a build-up of bile acids which causes further damage. The liver damage in PBC may lead to cirrhosis. PBC may also be associated with multiple symptoms. Many people with PBC may require liver transplant or may die if the disease progresses and a liver transplant is not done. This study will compare a daily dose of elafibranor (the study drug) to a daily dose of placebo (a dummy treatment). Each participant will be in the study for about 3.5 years. The main aim of this study is to determine if elafibranor is better than placebo in preventing adverse (bad) clinical outcomes including progression of disease leading to liver transplant or death. This study will also examine the safety of long-term treatment with elafibranor, as well as the impact on symptoms like itching and tiredness.

Fibrodysplasia Ossificans Progressiva

fidrisertib

United States of America (the)

United Kingdom of Great Britain and Northern Ireland (the)

Sweden

Belgium

Spain

Portugal

France

Canada

China

Korea (the Republic of)

Japan

Argentina

Netherlands (Kingdom of the)

Mexico

Italy

Australia

Recruiting

A study to assess the effectiveness and safety of 2 dosage regimens of oral fidrisertib (IPN60130) for the treatment of Fibrodysplasia Ossificans Progressiva (FOP)

Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by the presence of bone in soft tissue where bone normally does not exist, known as Heterotopic Ossification (HO). It is often associated with painful, recurrent episodes of soft tissue swelling (flare-ups) that lead to abnormal stiffening and immobility (ankyloses) of major joints with cumulative and irreversible loss of movement and disability. This study will evaluate the efficacy of 2 dosing regimens of IPN60130 in inhibiting new HO volume compared with placebo (a dummy treatment) in adult and paediatric participants with FOP. It will be assessed by a scan (provides internal images of the body) called low dose Whole Body Computed Tomography (WBCT), excluding head. Adults and participants 15 years of age or older are also eligible for a sub study to evaluate HO lesions assessed by another type of scan, Fluorine-18-labelled natrium fluoride Positron Emission Tomography-Computed Tomography ([18F]NaF PETCT ). This will be a 3-part study: Part A will be a 12-month, 3-arm, parallel-group, randomised, double-blind, placebo-controlled period during which participants will be randomised to low or high weight-based IPN60130 dosage arms or placebo, Part B will be a 12-month, 2-arm, randomised, double-blind period during which participants will receive high or low weight-based daily dosages of IPN60130. Participants completing Part A will enter Part B: placebo recipients from Part A will be randomised to one of the IPN60130 dosage arms with a 1:1 ratio, whereas participants receiving IPN60130 will remain in their respective dosage arms. Part C will be a 36-month, extension period. All participants completing Part B will continue in the same IPN60130 treatment arm for additional 36-month treatment in Part C.