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Chronic Migraine

Episodic Migraine

IPN10200

United States of America (the)

Recruiting

A study to evaluate IPN10200 safety and efficacy in the prevention of episodic or chronic migraine in adults

A migraine is a headache with severe throbbing pain or a pulsating sensation, usually on one side of the head. It is often accompanied by feeling or being sick and a sensitivity to bright lights and sound. Migraines are caused by a series of events when the brain gets stimulated or activated, which causes the release of chemicals that cause pain. IPN10200 is a medication that stops the release of these chemical messengers. Participants with episodic migraine (EM) or chronic migraine (CM) will be included in both Step 1 and Step 2. “Headache days” are when participants experience headaches that meet the criteria for a migraine or a headache without the additional migraine-specific symptoms. “Migraine days” occur when the headache displays clear migraine characteristics. This study aims to determine: The safety and efficacy of injecting IPN10200 directly into the muscles of the head and neck to prevent EM and CM, The right amount (dose) of IPN10200 to inject at each point, The total amount (dose) of IPN10200 that provides the best balance between safety and efficacy preventing migraines. Participants will need to complete a daily electronic migraine Diary (eDiary) and questionnaires throughout the study. The total study duration for a participant will be up to 44 weeks. The study will consist of 3 periods: A ‘screening period’ to assess whether the participant can take part in the study. Step 1 is divided in two cohorts. The study will assess sequentially the safety of two doses of IPN10200, a lower dose in the cohort 1 and a higher dose in cohort 2. Participants will be administered with the study drug or placebo. The treatment is injected in muscles of the head, face and neck. The safety of participants is monitored throughout the 36 weeks at each cohort. Step 2: In this step, new eligible participants will be divided into two groups based on their diagnosis (EM or CM). These groups will then be randomly assigned to one of three intervention groups: Dose A, Dose B, or a placebo. The intervention will be given in a series of injections in muscles of the head, face and neck. Participants will be monitored for both efficacy and safety until they complete the Week 36 visit (the end of study).

Primary Sclerosing Cholangitis

ritivixibat

Italy

France

Spain

Poland

Recruiting

Safety and Tolerability of A3907 in Primary Sclerosing Cholangitis

This study will test a drug called A3907 to see how safe and tolerated it is for treating people with Primary Sclerosing Cholangitis (PSC). Detailed Description: The primary goal of this study in participants with PSC with and without a Clinically Relevant Stricture (CRS) who are treated with A3907 is to assess the safety and tolerability of A3907 following repeat doses. Secondary goals include evaluation of the pharmacokinetic properties of A3907 (the study of how the body interacts with A3907 for the entire duration of exposure) and changes in safety parameters via laboratory testing such as liver enzymes, bile acid levels and markers of bile acid synthesis.

Alagille Syndrome

Bylvay | Kayfanda

Poland

Malaysia

Netherlands (Kingdom of the)

Belgium

Turkey

United Kingdom of Great Britain and Northern Ireland (the)

France

Italy

Germany

United States of America (the)

Recruiting

Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome

The purpose of this study is to assess the long-term safety and effectiveness of odevixibat in participants with Alagille syndrome (ALGS). The participants of this study will have ALGS a rare genetic disorder that can affect multiple organ systems of the body including the liver, heart, skeleton, eyes and kidneys. Common symptoms, which often develop during the first three months of life, include blockage of the flow of bile from the liver (cholestasis), yellowing of the skin and mucous membranes (jaundice), poor weight gain and growth and severe itching (pruritis). Detailed Description: This Phase 3, open-label, multi-center extension study will have two groups of participants: Cohort 1 (participants who participated in Study A4250-012 [NCT04674761; ASSERT] and meet the entry criteria for this study) and Cohort 2 (infants under 12 months of age) with ALGS. The study will consist of 2 or 3 periods: A ‘Treatment period’ of 72 weeks (cohort 1) or 12 weeks (cohort 2). Participants will visit the clinic every 4 to 12 weeks and will receive a dose of 120 ?g/kg odevixibat daily. An ‘Optional extension period’ where participants who wish to continue receiving odevixibat after the ‘treatment period’ will have the opportunity to remain on treatment with visits every 16 weeks until the drug is commercially available. The optional extension is available provided continued use is supported by the risk-benefit profile, the participant has not been previously withdrawn or discontinued from the study, and the study is not terminated by the Sponsor. A ‘Safety follow-up period’ of 4 weeks (cohort 1) or 2 weeks (cohort 2). The Safety Follow-up Period will not occur for those who remain on treatment in the optional extension period. Participants will need to complete an e-diary and questionnaires throughout the study (cohort 1 only). Participants will undergo blood samplings, urine collections (cohort 1 only), physical examinations, and clinical evaluations. They may continue some other medications, but the details need to be recorded.

Advanced Solid Tumours

IPN01195

United States of America (the)

Recruiting

A Study to Assess a New Medicine Called IPN01195 When Administered Alone in Adults With Advanced Solid Tumours

The purpose of this study is to determine the appropriate dosage, safety and effectiveness of a new study drug IPN01195 in adults with advanced solid tumours. The participants in this study will have advanced solid tumours. ‘Advanced solid tumours’ refers to cancers that can occur in several places, including cancers in organs or tissues that have spread from their original site to nearby tissues or other parts of the body. The study consists of two phases, called phase I and phase II. Phase I will be conducted in two parts: Part A: Phase I Part A study (dose escalation) is designed to find the dose range showing activity on the tumour that can be tolerated by the participants by testing different doses of IPN01195. Part B: Phase I Part B of the study (dose confirmation) will assess the ability of study drug to prevent, slow down, or stop the growth of tumours (abnormal cell growths that can lead to cancer) and how the body processes and responds to the study drug when administered in a “low dose” or “high dose” and further explore the safety and tolerability. These parts will consist of the following periods: A period to assess eligibility (screening period). A treatment period that will require at least two visits for the first month followed by one visit every month. There will be also one visit, at the end of treatment, at 30 days after the last administration of study drug. An assessment visit will be required every 6 weeks up to Week 24 and every 12 weeks thereafter to measure the tumour again and to assess how it is evolving, whether it is getting bigger, smaller, is stable or has gone away. Based on the results obtained from phase I, a phase II extension study will be included through to an updated study plan, to further evaluate the study drug. In both study phases, participants will undergo blood samplings, urine collections, physical examinations and clinical evaluations. They may continue some other medications, but the details need to be recorded.

Colorectal Cancer

Head And Neck Squamous Cell Carcinoma

Melanoma

Pancreatic Ductal Adenocarcinoma

Solid Tumor

IPN01194

United States of America (the)

Spain

France

Recruiting

A Study to Assess IPN01194 When Administered Alone in Adults With Advanced Solid Tumours

The purpose of this study is to determine the appropriate dosage, safety and effectiveness of the study drug, IPN01194 in adults with advanced solid tumours. The participants in this study will have advanced solid tumours. ‘Advanced solid tumours’ refers to cancers that can occur in several places, including cancers in organs or tissues that have spread from their original site to nearby tissues or other parts of the body. In this study, all participants will receive the study drug, which will be taken by mouth (orally). Detailed Description: The study consists of two parts, called Phase I and Phase IIa. Phase I is designed to assess the safety of increasing doses of IPN01194 in participants with specific types of advanced solid tumours. The aim of this “dose escalation” phase is to find the dose range showing activity on the tumor that can be tolerated by the participants, and to determine the two doses for further testing in Phase IIa. Phase I will assess how the body processes and responds to the study drug when administered with and without food. In Phase IIa, participants with selected single tumour type will be invited to take part. During this phase, the two dose levels of the study drug identified from Phase I will be tested. Participants will take the study drug one of the two dose levels. Each participant will be assigned to a dose level at random (by chance). Each phase will consist of three periods: 1- A period to assess eligibility (screening period) that will take up to 28 days. 2- A treatment period of at least 28 days that will require at least two visits for the first month followed by one visit every month. There will be also one visit, at the end of treatment, at least 30 days after the last administration of study drug. 3- A follow-up period (Phase IIa participants only), where every 3 months, participants will be contacted by phone, until death or the study cut-off date, whichever comes first. Participants will undergo blood samplings, urine collections, physical examinations, and clinical evaluations. They may continue some other medications, but the details need to be recorded. If in the opinion of the investigator a participant is continuing to experience clinical benefit after the cut-off date, the participant may remain in the study and continue to receive the study drug until either disease progression, unacceptable toxicity or other withdrawal criteria are met.