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A multi-center, randomized, double-blind, placebo controlled study of ligelizumab (QGE031) in the treatment of Chronic Inducible Urticaria (CINDU) in adolescents and adults inadequately controlled by H1-antihistamines

The purpose of this study is to establish efficacy and safety of ligelizumab (QGE031) versus placebo in participants with chronic inducible urticaria who remain symptomatic despite treatment with H1 antihistamine.

DAVINCY trial: optimal Duration of (fos)aprepitant prophylaxis for nausea and Vomiting INduced by ChemotherapY in children: a double-blind placebo-controlled crossover randomized phase III trial*

This study has been transitioned to CTIS with ID 2024-517846-32-00 check the CTIS register for the current data. To evaluate the effect of prolonged duration of (fos)aprepitant prophylaxis on the prevention of delayed CINV (complete remission in the…

A randomised, double-blind, placebo-controlled, Phase III trial to determine the efficacy and safety of inhaled SNG001 for the treatment of patients hospitalised due to moderate COVID-19

Primary Objective:To evaluate recovery in patients with moderate COVID-19 after administration of SNG001 compared to placebo.Secondary Objectives:a. To evaluate the efficacy of SNG001 compared to placebo in patients with moderate COVID-19, using a…

A 52 week, multi-center, randomized, double-blind placebo-controlled study to assess the clinical efficacy and safety of ligelizumab (QGE031) in decreasing the sensitivity to peanuts in patients with peanut allergy

The purpose of this Phase 3 study is to evaluate the safety and clinical efficacy of ligelizumab 240 mg and 120 mg given subcutaneously (s.c.) every 4 weeks (q4w) to ensure protection against allergic reaction by decreasing the sensitivity to oral…

Peppermint Oil for the treatment of Irritable Bowel Syndrome or Functional Abdominal Pain in Children:
the MINT study

The aim of the Mint study is to investigate the effectiveness of peppermint oil capsules compared to placebo capsules in reducing abdominal pain intensity in these children. In addition, we investigate the effect of peppermint oil capsules compared…

A Multi-Center, Randomized, Double-Blind, Placebo-Controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth type 1A (CMT1A)

Primary:To evaluate the efficacy of treatment with PXT3003 (a fixed-dose combination of (RS)-baclofen, naltrexone hydrochloride, and D-sorbitol) compared to placebo in subjects with CMT1A.Secondary: To evaluate the safety and tolerability of PXT3003…

A 6-month multicenter, randomized, double-blind, placebo-controlled study to evaluate the
efficacy, safety and PK/PD of an age-and body weight-adjusted oral finerenone regimen, in
addition to an ACEI or ARB, for the treatment of children, 6 months to <18 years of age, with
chronic kidney disease and proteinuria

This study has been transitioned to CTIS with ID 2023-504884-17-00 check the CTIS register for the current data. The main goal of this study is to find out whether finerenone in combination with either an ACE inhibitor or an ARB can reduce the…

A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated
Virus Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase
(OTC) in Patients with Late-Onset OTC Deficiency

This study has been transitioned to CTIS with ID 2024-514337-38-00 check the CTIS register for the current data. PrimaryTo evaluate the efficacy of DTX301 on the improvement of OTC function by maintaining safe plasma ammonia levelsSecondaryTo…

A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Mitapivat in Subjects With Sickle Cell Disease

This study has been transitioned to CTIS with ID 2024-515202-84-00 check the CTIS register for the current data. Phase 2:Primary ObjectivesTo determine the recommended Phase3 dose of mitapivat by evaluating the effect of 2 dose levels of mitapivat…

A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of NBI-921352 as Adjunctive Therapy in Subjects with hSCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE)

Main objective: To assess the efficacy of NBI-921352 as adjunctive therapy on the frequency of countable motor seizuresSecondary Objectives • To evaluate the efficacy of NBI-921352 using the Clinical and Parent/Caregiver Global Impression of Change…

Phase 3, Multicenter, Double-blind, Randomized, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Apremilast in Children From 5 to Less Than 18 Years of Age With Active Juvenile Psoriatic Arthritis (PEAPOD)

This study has been transitioned to CTIS with ID 2023-503435-17-00 check the CTIS register for the current data. Estimate the efficacy of apremilast compared with placebo in the treatment of Juvenile Psoriatic Arthritis (JPsA) in pediatric subjects…

A Randomised, Double-Blind, Placebo-Controlled Phase 3 Clinical Trial to Assess the Efficacy and
Safety of Lebrikizumab in Combination With Topical Corticosteroids in Adult and Adolescent Patients
With Moderate-To-Severe Atopic Dermatitis That Are Not Adequately Controlled With Cyclosporine or
For Whom Cyclosporine is Not Medically Advisable.

To evaluate the efficacy of lebrikizumab compared with placebo in patients not adequately controlled with cyclosporine or for whom cyclosporine is not medically advisable up to Week 16

A Phase III, Randomized, Double-blind, Placebo-controlled, Multicenter Trial to Evaluate the Efficacy and Safety of Diamyd® to Preserve Endogenous Beta Cell Function in Adolescents and Adults with Recently Diagnosed Type 1 Diabetes, Carrying the Genetic HLA DR3-DQ2 Haplotype

This study has been transitioned to CTIS with ID 2024-513304-33-00 check the CTIS register for the current data. The primary objective is to assess the effect of three doses of Diamyd compared to a placebo in terms of (1) beta cell function; and (2…

EPIK-P2: A Phase II double-blind study with an upfront, 16-week randomized, placebo-controlled period, to assess the efficacy, safety and pharmacokinetics of alpelisib (BYL719) in pediatric and adult patients with PIK3CA-related overgrowth spectrum (PROS)

This study has been transitioned to CTIS with ID 2023-508530-34-00 check the CTIS register for the current data. Primary objective:* To demonstrate the efficacy of alpelisib as measured by the proportion of participants randomized to alpelisib with…

A Phase 2 study to assess the efficacy and safety of 2 dosage regimens of oral fidrisertib (IPN60130) for the treatment of fibrodysplasia ossificans progressiva in male and female paediatric and adult participants

This study has been transitioned to CTIS with ID 2024-511469-13-00 check the CTIS register for the current data. Main objective:• Evaluate the efficacy of IPN60130 monotherapy compared with placebo recipients in inhibiting new HO volume in adult and…

Phase 1-3, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients with Alexander Disease

This study has been transitioned to CTIS with ID 2024-510603-11-00 check the CTIS register for the current data. Primary Objective: To evaluate the efficacy of ION373 in improving or stabilizing gross motor function in patients with Alexander…

A PHASE III, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTICENTER STUDY TO EVALUATE EFFICACY, SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF SATRALIZUMAB IN PATIENTS WITH GENERALIZED MYASTHENIA GRAVIS

This study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of satralizumab compared with placebo in patients with gMG on stable background therapy. In addition, the study will assess the long-term safety and efficacy of…

Transvamix (10% THC / 5% CBD) to treat chronic pain in Epidermolysis Bullosa: An explorative randomized, placebo-controlled and double-blind intervention crossover study

The primary objective is to determine the effect of Transvamix on the pain quality item *unpleasantness*, relative to placebo. Secondary objectives include the assessment of general pain qualities, pain self-efficacy, general pain and pruritus…

Efficacy and safety of the SQ tree sublingual immunotherapy tablet in children and adolescents (5 through 17 years of age) with moderate to severe allergic rhinitis and/or conjunctivitis induced by pollen from birch and trees belonging to the birch homologous group

To compare the efficacy of the SQ tree SLIT-tablet to placebo in the treatment of moderate to severe allergic rhinitis and/or conjunctivitis induced by pollen from birch and trees belonging to the birch homologous group in children and adolescents (…

A Phase 3, Randomized, Double-blind, Placebo-Controlled, Multicenter Study of Ravulizumab in Adult and Adolescent Participants who have Thrombotic Microangiopathy (TMA) after Hematopoietic Stem Cell Transplant (HSCT)

This study has been transitioned to CTIS with ID 2023-510107-22-00 check the CTIS register for the current data. To assess the efficacy of ravulizumab versus placebo in the treatment of adult and adolescent participants with HSCT-TMA.