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NCT ID: NCT05020873 Terminated - Sickle Cell Disease Clinical Trials

Prospective, Observational Study in Sickle Cell Disease Patients on Crizanlizumab Treatment in Middle East Countries and India

SPOTLIGHT
Start date: November 4, 2021
Phase:
Study type: Observational

This is a multicenter, prospective, single-arm observational non-interventional study (NIS), which will be conducted in various countries in the Middle East and India.

NCT ID: NCT04927247 Terminated - Sickle Cell Disease Clinical Trials

A Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants With Sickle Cell Disease and Recurrent Vaso-occlusive Crises

Start date: December 13, 2021
Phase: Phase 3
Study type: Interventional

This Phase 3 study will assess the safety and efficacy of a single dose of inclacumab, a P-selectin inhibitor, for a vaso-occlusive crisis (VOC) after an index VOC in participants with sickle cell disease (SCD). Participants will be randomized to receive either inclacumab or placebo.

NCT ID: NCT04732429 Terminated - Propionic Acidemia Clinical Trials

Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia

HERO
Start date: March 15, 2021
Phase: Phase 2
Study type: Interventional

This is an interventional study to assess the safety, PK, and efficacy of HST5040 in 12 subjects - 6 with Methylmalonic Acidemia (MMA) and 6 with Propionic Acidemia (PA). The study consists of 3 parts: - Part A: Open-label, within-subject, dose escalation study in PA and MMA subjects ≥ 2 years old to identify a safe and pharmacologically active (optimal) dose of HST5040 for use in Part B. Subjects will continue in a Part A open-label extension until all subjects complete Part A and the optimal dose of HST5040 is identified for use in Part B. - Part B: 6-month, randomized, double-blind, placebo-controlled, 2-period crossover in the same subjects from Part A to evaluate safety and efficacy of the optimal dose of HST5040 in addition to standard of care (SoC). - Part C: open-label long-term extension study in PA and MMA subjects ≥ 2 years old (N = approximately 12, 6 each) to evaluate the long-term safety and efficacy of the optimal dose of HST5040. This study will determine whether HST5040 can improve levels of disease-associated toxins that accumulate in patients with PA and MMA.

NCT ID: NCT04435782 Terminated - Clinical trials for Pulmonary Arterial Hypertension

A Study of Selexipag Assessing Right Ventricular Remodeling in Pulmonary Arterial Hypertension by Cardiac Magnetic Resonance Imaging

RESTORE
Start date: July 7, 2021
Phase: Phase 4
Study type: Interventional

The purpose of the study is to assess the effects of selexipag on right ventricular (RV) function in participants with Pulmonary arterial hypertension (PAH).

NCT ID: NCT04271475 Terminated - Clinical trials for Chronic Thromboembolic Pulmonary Hypertension

A Study to Evaluate Efficacy and Safety of Macitentan 75 mg in Inoperable or Persistent/Recurrent Chronic Thromboembolic Pulmonary Hypertension

MACiTEPH
Start date: July 7, 2020
Phase: Phase 3
Study type: Interventional

The purpose of the study is to evaluate the effect of macitentan 75 mg versus placebo on exercise capacity at Week 28 in participants with chronic thromboembolic pulmonary hypertension (CTEPH).

NCT ID: NCT03870763 Terminated - Clinical trials for Multiple Sclerosis, Relapsing-Remitting

Study to Evaluate the Efficacy and Safety of Dimethyl Fumarate (Tecfidera) and Peginterferon Beta-1a (Plegridy) for the Treatment of Relapsing-Remitting Multiple Sclerosis in Pediatric Participants

Start date: March 19, 2019
Phase: Phase 3
Study type: Interventional

The main objective of the study is to evaluate the efficacy of dimethyl fumarate (Tecfidera) and peginterferon beta-1a (Plegridy), both compared with placebo, in pediatric participants with RRMS. The other objectives of this study are to evaluate the safety and tolerability of dimethyl fumarate and peginterferon beta-1a and to assess the effect of dimethyl fumarate and peginterferon beta-1a, both compared with placebo, on additional clinical and radiological measures of disease activity.

NCT ID: NCT03291899 Terminated - Clinical trials for Biliary Tract Cancer

Trial of Infusional FOLFIRINOX in First Line Treatment of Advanced Biliary Tract Cancers

FBI-TRAC
Start date: January 3, 2017
Phase: Phase 2
Study type: Interventional

This study is to evaluate the response rate and toxicity profile of infusional 5 fluorouracil, leucovorin, oxaliplatin and irinotecan (FOLFIRINOX) in first-line treatment of advanced biliary tract cancers and also to assess progression free survival and overall survival of FOLFIRINOX in first line treatment of advanced biliary tract cancers

NCT ID: NCT03269695 Terminated - Ulcerative Colitis Clinical Trials

Efficacy, Safety and Tolerability of PF-06687234 as Add-on Therapy to Infliximab in Active UC Subjects Not in Remission.

Start date: December 20, 2017
Phase: Phase 2
Study type: Interventional

The purpose of this study is to determine if PF-06687234 is effective and safe as add-on therapy to infliximab in subjects with active ulcerative colitis who are not in remission.

NCT ID: NCT03189667 Terminated - Clinical trials for Dysfunctional Dialysis Arteriovenous Fistula

Drug Coated Balloons vs Plain Balloons for the Management of Dysfunctional Dialysis Fistula

Start date: October 15, 2017
Phase: N/A
Study type: Interventional

Compare the effectiveness of drug-coated balloons to plain balloon angioplasty in reducing stenosis rates in dialysis arteriovenous fistulas (AVFs).

NCT ID: NCT03052517 Terminated - Asthma Clinical Trials

Study of Safety of QAW039 in Patients With Asthma Inadequately Controlled on Standard-of-care Asthma Treatment

Start date: March 21, 2017
Phase: Phase 3
Study type: Interventional

This study was a 2-treatment period, randomized, multicenter parallel-group study. The overall purpose of this study was to provide long- term safety data for fevipiprant (QAW039) (Dose 1 and Dose 2), compared with placebo, when added to the Global Initiative for Asthma (GINA) steps 3, 4, and 5 standard-of-care (SoC) asthma therapy (GINA 2016), in patients with moderate-to- severe asthma. The purpose of this study was to provide long-term safety data for QAW039 150 mg once daily and 450 mg once daily, compared with placebo, when added to GINA steps 3, 4, and 5 standard-of-care asthma therapy (GINA 2020) in adult and adolescent (≥12 years) patients with moderate-to-severe asthma. The study included 2 cohorts of patients: 1. Rollover patients who had completed any of the four Phase 3 pivotal efficacy studies with QAW039 (QAW039A2307, QAW039A2314, QAW039A2316, or QAW039A2317, hereafter referred to as Studies A2307, A2314, A2316, and A2317), thus providing data for a longer duration of exposure, and 2. New patients who had not previously participated in a study of QAW039, permitting an increase in the number of patients with long-term exposure to QAW039. By including these 2 categories of patients, the total number of patients treated with QAW039 as well as the duration of exposure to QAW039 treatment was substantially increased, supporting evaluation of the safety profile of QAW039.