Vseobecna fakultni nemocnice v Praze Trials

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Region

Interventions

Sponsor

Conditions

Primary compl

Study compl

Viskochil, David

COMPASS, NCT05371613 / 2021-005200-35: A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) Vs Idursulfase in Pediatric and Young Adult Participants with Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Recruiting

2/3

Europe, Canada, US, RoW

tividenofusp alfa, idursulfase

Denali Therapeutics Inc.

Mucopolysaccharidosis II

12/25

NCT05005845: NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)

Completed

199

NFX-179 gel, NFX-179 topical gel, Vehicle gel, NFX-179 vehicle gel

NFlection Therapeutics, Inc.

Cutaneous Neurofibroma, Neurofibromatosis 1

10/23

NCT05238324: Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II

Withdrawn

Canada, US

Genetic HMI-203

Homology Medicines, Inc

Mucopolysaccharidosis II

10/24

01/29

NCT05795361: Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

Available

N/A

Europe, US, RoW

Idursulfase-IT, HGT-2310, TAK-609

Takeda

Hunter Syndrome

Cottin, Vincent

NCT03939520 / 2019-004326-19: Management of Progressive Disease in Idiopathic Pulmonary Fibrosis

Recruiting

378

Europe

pirfenidone and nintedanib, pirfenidone or nintedanib

Hospices Civils de Lyon, Ministry of Health

Progressive Idiopathic Pulmonary Fibrosis

01/26

NCT03626688 / 2018-001187-33: A Study Evaluating the Efficacy and Safety of Ralinepag to Improve Treatment Outcomes in PAH Patients

Recruiting

1000

Europe, Canada, US, RoW

Ralinepag, APD811, Placebo

United Therapeutics

PAH, Pulmonary Hypertension, Pulmonary Arterial Hypertension, Hypertension, Connective Tissue Diseases, Familial Primary Pulmonary Hypertension, Vascular Diseases, Cardiovascular Diseases, Hypertension, Pulmonary, Lung Diseases, Respiratory Tract Disease

12/25

IMPALA-2, NCT04544293 / 2020-001263-85: Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP)

Active, not recruiting

160

Europe, Canada, Japan, US, RoW

Molgramostim, Recombinant human granulocyte-macrophage colony stimulating factor (rhGM-CSF), Placebo

Savara Inc., Savara ApS,

Autoimmune Pulmonary Alveolar Proteinosis

11/23

11/24

TETON-2, NCT05255991 / 2021-005881-17: Multinational Study of Efficacy and Safety of Inhaled Treprostinil in Subjects With Idiopathic Pulmonary Fibrosis

Active, not recruiting

597

Europe, RoW

Placebo, Inhaled Treprostinil, Tyvaso, Treprostinil Ultrasonic Nebulizer

United Therapeutics

Idiopathic Pulmonary Fibrosis, Interstitial Lung Disease

07/25

EXAFIP2, NCT05674994: Glucocorticoids Versus Placebo for the Treatment of Acute Exacerbation of Idiopathic Pulmonary Fibrosis

Recruiting

110

Europe

Methylprednisone/Prednisone, Placebo

Fondation Hôpital Saint-Joseph

Acute Exacerbation of Idiopathic Pulmonary Fibrosis

10/25

12/26

IMPAHCT, NCT05036135 / 2021-001910-13: A Study of AV-101 (Dry Powder Inhaled Imatinib) in Patients With Pulmonary Arterial Hypertension (PAH)

Terminated

2/3

202

Europe, Canada, US, RoW

AV-101, Placebo

Aerovate Therapeutics, Aerovate Therapeutics, Inc.

Pulmonary Arterial Hypertension

08/24

ELEVATE 2, NCT04712669 / 2020-004971-42: A Study of Rodatristat Ethyl in Patients With Pulmonary Arterial Hypertension

Completed

108

Europe, Canada, US, RoW

rodatristat ethyl 300 mg tablet BID, rodatristat ethyl 600 mg BID, Placebo

Altavant Sciences GmbH

Pulmonary Arterial Hypertension

06/23

08/23

RaDiCo PID, NCT04238871: Cohort (RaDiCo-ILD Cohort in English)

Recruiting

N/A

2500

Europe

Institut National de la Santé Et de la Recherche Médicale, France

Idiopathic Interstitial Pneumopathy/Pneumopathy Interstitial Diffuse, Paediatric and Adult Patients

03/21

09/21

HPI-COVID-19, NCT04376476: Host-pathogen Interactions During SARS-CoV-2 Infection

Completed

N/A

140

Europe

Blood sample, Low or upper respiratory tract sample, Stool collection or fecal swab, phone call

Hospices Civils de Lyon

Infection, Coronavirus, Severe Acute Respiratory Syndrome Coronavirus 2

05/22

I-FILE, NCT04304898: An International Patient-led Registry in Fibrotic Interstitial Lung Diseases Using eHealth Technology

Recruiting

N/A

700

Europe

Home spirometry

Erasmus Medical Center

Pulmonary Fibrosis

07/25

07/26

RE-LAM-CE, NCT01484236: National Lymphangioleiomyomatosis Registry, France

Recruiting

N/A

200

Europe

Vincent COTTIN, Hospices Civils de Lyon

Lymphangioleiomyomatosis

12/24

12/25

HEMA-HTP, NCT02800941: Bleeding Frequency Under Anticoagulant Treatment in Pulmonary Hypertension

Completed

N/A

203

Europe

Oral anticoagulant treatment, warfarin (Coumadine®), fluindione (Préviscan®), acenocoumarol (Sintrom®)

Centre Hospitalier Universitaire de Saint Etienne

Pulmonary Arterial Hypertension

09/22

09/23

ILDnose, NCT04680832: Exhaled Breath Analysis Using eNose Technology as a Biomarker for Diagnosis and Disease Progression in Fibrotic ILD

Recruiting

N/A

600

Europe

Electronic nose, SpiroNose, eNose

Erasmus Medical Center

Pulmonary Fibrosis

12/25

12/26

FONCE-HTAP, NCT02579954: Cardiac Function and Exercise Capacity in Pulmonary Arterial Hypertension

Completed

N/A

Europe

Rehabilitation, Supervised rehabilitation

Centre Hospitalier Universitaire de Saint Etienne, Ministry of Health, France

Pulmonary Arterial Hypertension

12/22

01/24

MAINEPSAN, NCT03290456 / 2018-001215-69: Evaluate the Remission MAINtenance Using Extended Administration of Prednisone in Systemic Anti-neutrophil Cytoplasmic Antibodies (ANCA)-Associated Vasculitis.

Recruiting

N/A

146

Europe

Prednisone 5mg/day extended of 12 additional months, Placebo 5mg/day extended of 12 additionnal months.

Hospices Civils de Lyon, Ministry of health

Granulomatosis With Polyangitis

06/24

MEDICO-LIBS, NCT03901196: Multi-elemental Imaging of Lung Tissues With LIBS (Laser-induced Breakdown Spectroscopy)

Completed

N/A

Europe

Elemental analysis

University Hospital, Grenoble

Interstitial Lung Disease, Sarcoidosis, Idiopathic Pulmonary Fibrosis

04/24

DCP, NCT05951478: (RaDiCo Cohort) (RaDiCo-)

Recruiting

N/A

300

Europe

Institut National de la Santé Et de la Recherche Médicale, France

Primary Ciliary Dyskinesia

12/28

RaDiCo-ILD 2, NCT06036719: Interstitial Lung Disease: a Study from Infancy to Elderly Including Relatives

Recruiting

N/A

3000

Europe

Institut National de la Santé Et de la Recherche Médicale, France

Interstitial Lung Diseases

03/31

Tesar, Vladimir

FALCON, NCT03918447 / 2018-004651-20: A Trial of Bardoxolone Methyl in Patients With ADPKD -

Terminated

667

Europe, Japan, US, RoW

Bardoxolone methyl oral capsule, RTA 402, Placebo oral capsule

Reata, a wholly owned subsidiary of Biogen, Reata Pharmaceuticals, Inc., REATA PHARMACEUTICALS, INC

Autosomal Dominant Polycystic Kidney, ADPKD

08/23

ALIGN, NCT04573478: Atrasentan in Patients With IgA Nephropathy

Active, not recruiting

404

Europe, Canada, Japan, US, RoW

Atrasentan, CHK-01, Atrasentan Hydrochloride, ABT-627, Placebo

Chinook Therapeutics, Inc.

IgA Nephropathy, Immunoglobulin A Nephropathy

12/26

NCT05123586: A IMMA Master Protocol: A Study of LY3361237 in Participants With at Least Moderately Active Systemic Lupus Erythematosus

Terminated

Europe, US, RoW

LY3361237, Placebo

Eli Lilly and Company

Systemic Lupus Erythematosus

12/23

NCT06274489: A Study to Evaluate Setanaxib in Patients With Alport Syndrome

Recruiting

1/2

Europe, RoW

Setanaxib, Placebo

Calliditas Therapeutics AB

Alport Syndrome

01/25

REYNAUD-GAUBERT, Martine

NCT03939520 / 2019-004326-19: Management of Progressive Disease in Idiopathic Pulmonary Fibrosis

Recruiting

378

Europe

pirfenidone and nintedanib, pirfenidone or nintedanib

Hospices Civils de Lyon, Ministry of Health

Progressive Idiopathic Pulmonary Fibrosis

01/26

BOSTON-2, NCT03656926 / 2018-003205-25: Efficacy + Safety of Liposome Cyclosporine a to Treat Bronchiolitis Obliterans Post Single Lung Transplant

Completed

220

Europe, US, RoW

Liposomal Cyclosporine A, L-CsA, Standard of Care, SoC

Zambon SpA, BREATH Therapeutics Inc.

Bronchiolitis Obliterans, Chronic Rejection of Lung Transplant, Lung Transplant Rejection, Lung Transplant; Complications, Lung Transplant Failure and Rejection, Chronic Lung Allograft Dysfunction

03/24

TETON-2, NCT05255991 / 2021-005881-17: Multinational Study of Efficacy and Safety of Inhaled Treprostinil in Subjects With Idiopathic Pulmonary Fibrosis

Active, not recruiting

597

Europe, RoW

Placebo, Inhaled Treprostinil, Tyvaso, Treprostinil Ultrasonic Nebulizer

United Therapeutics

Idiopathic Pulmonary Fibrosis, Interstitial Lung Disease

07/25

IMPAHCT, NCT05036135 / 2021-001910-13: A Study of AV-101 (Dry Powder Inhaled Imatinib) in Patients With Pulmonary Arterial Hypertension (PAH)

Terminated

2/3

202

Europe, Canada, US, RoW

AV-101, Placebo

Aerovate Therapeutics, Aerovate Therapeutics, Inc.

Pulmonary Arterial Hypertension

08/24

NCT05032066: A Multicenter Trial to Evaluate the Efficacy, Safety and Tolerability of HZN-825 in Subjects With Idiopathic Pulmonary Fibrosis

Jan 2024 - Dec 2024: Data from trial for diffuse cutaneous systemic sclerosis

Terminated

153

Europe, Canada, Japan, US, RoW

HZN-825, Placebo

Amgen

Idiopathic Pulmonary Fibrosis

07/24

01/25

DCP, NCT05951478: (RaDiCo Cohort) (RaDiCo-)

Recruiting

N/A

300

Europe

Institut National de la Santé Et de la Recherche Médicale, France

Primary Ciliary Dyskinesia

12/28

Cibula, David

innovaTV 301, NCT04697628 / 2019-001655-39: Tisotumab Vedotin vs Chemotherapy in Recurrent or Metastatic Cervical Cancer

Active, not recruiting

502

Europe, Canada, Japan, US, RoW

tisotumab vedotin, TIVDAK, topotecan, vinorelbine, gemcitabine, irinotecan, pemetrexed

Seagen Inc., Genmab

Cervical Cancer

07/23

08/25

PORTEC-4a, NCT03469674: Molecular Profile-based Versus Standard Adjuvant Radiotherapy in Endometrial Cancer

Active, not recruiting

550

Europe, RoW

Vaginal brachytherapy, External beam radiotherapy, Observation

Leiden University Medical Center, Dutch Cancer Society, Comprehensive Cancer Centre The Netherlands

Endometrial Cancer Stage I, Endometrial Cancer Stage II

12/24

12/28

ENGOT-EN20, NCT05611931 / 2022-002540-42: Selinexor in Maintenance Therapy After Systemic Therapy for Participants with P53 Wild-Type, Advanced or Recurrent Endometrial Carcinoma

Jul 2024 - Dec 2024: Top-line data from XPORT-EC trial in participants with TP53 wild type endometrial cancer

Recruiting

220

Europe, Canada, US, RoW

Selinexor, KPT-330, Matching Placebo for selinexor

Karyopharm Therapeutics Inc, GOG Foundation, European Network of Gynaecological Oncological Trial Groups (ENGOT), Belgium and Luxembourg Gynaecological Oncology Group, North Eastern German Society of Gynaecological Oncology, Multicentre Italian Trials in Ovarian Cancer and Gynecologic Malignancies, Spanish Research Group in Ovarian Cancer, The Central and Eastern European Gynecologic Oncology Group, Israeli Society of Gynecologic Oncology, Australia New Zealand Gynaecological Oncology Group

Endometrial Cancer

01/25

01/28

PHOCUS, NCT04789694: Prehabilitation in Gynaecological Cancer Patients

Recruiting

RoW

Physiological intervention, Psychological intervention, Nutritional intervention

Charles University, Czech Republic, University Hospital Ostrava, University Hospital Olomouc, University Hospital Pilsen, The Faculty Hospital Na Bulovce

Ovarian Cancer, Ovarian Cancer Stage III, Ovarian Cancer Stage IV, Endometrial Cancer, Frailty

06/25

06/26

FLORA-4, NCT04498117 / 2020-002270-26: Oregovomab Plus Chemo in Newly Diagnosed Patients With Advanced Epithelial Ovarian Cancer Following Optimal Debulking Surgery

Active, not recruiting

615

Europe, Canada, US, RoW

Oregovomab, MAb-B43.13, Paclitaxel, Taxol, Carboplatin, Paraplatin, Placebo

CanariaBio Inc., Gynecologic Oncology Group, Iqvia Pty Ltd

Carcinoma, Ovarian Epithelial, Ovarian Neoplasms, Ovarian Cancer, Ovarian Serous Adenocarcinoma, Fallopian Tube Neoplasms, Fallopian Tube Adenocarcinoma, Fallopian Tube Serous Adenocarcinoma, Peritoneal Cancer, Peritoneal Carcinoma, Peritoneal Neoplasms

09/25

08/27

CERVANTES, NCT04989647: Intermediate Risk Cervical Cancer: Radical Surgery +/- Adjuvant Radiotherapy

Recruiting

514

RoW

No adjuvant therapy, Adjuvant radiotherapy

The Central and Eastern European Gynecologic Oncology Group

Cervical Cancer

12/29

12/32

SENTIX, NCT02494063: Sentinel Lymph Node Biopsy in Patients With Early Stages Cervical Cancer

Completed

N/A

600

RoW

Sentinel lymph node biopsy, At least unilateral systematic pelvic lymph node dissection

Charles University, Czech Republic

Cervical Cancer

12/22

02/24

TESLA-1, NCT04665544: Tracers for Endometrial Cancer Sentinel Node Labeling

Recruiting

N/A

Europe, RoW

SLN side-specific detection rate using radioactive tracer with/without blue dye and ICG tracer.

University Medical Centre Maribor

Endometrial Cancer, Sentinel Lymph Node

01/23

01/24

SENTICOL III, NCT03386734: International Validation Study of Sentinel Node Biopsy in Early Cervical Cancer

Active, not recruiting

N/A

989

Europe, Canada, Japan, RoW

SLN biopsy only, SLN biopsy + PLN dissection

Centre Hospitalier Universitaire de Besancon, ARCAGY/ GINECO GROUP, European Network of Gynaecological Oncological Trial Groups (ENGOT)

Cervical Cancer

05/27

05/29

Rajan, Deepa Soundara

Recruiting

2/3

Europe, Canada, US, RoW

tividenofusp alfa, idursulfase

Denali Therapeutics Inc.

Mucopolysaccharidosis II

12/25

NCT04251026 / 2019-004909-27: A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

Active, not recruiting

1/2

Europe, Canada, US

tividenofusp alfa

Denali Therapeutics Inc.

Mucopolysaccharidosis II

07/27

NCT04007536: A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome

Completed

N/A

Europe, US

No Intervention

Denali Therapeutics Inc.

Mucopolysaccharidosis II

03/24

NCT06776341: Natural History Study of GEMIN-5 Related Neurodevelopmental Disorder

Not yet recruiting

N/A

500

GEMIN5-Related Neurodevelopmental Disorder

University of Pittsburgh

SMN Complex Proteins, GEMIN5 Protein, Human, Neurodevelopmental Disorders

12/50

NCT02995538: Neurogenetics Patient Registry

Recruiting

N/A

1000

University of Pittsburgh

Neurogenetic Disorders

01/27

01/28

NCT00787865: Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease

Active, not recruiting

N/A

100

University of Pittsburgh, National Institute of Neurological Disorders and Stroke (NINDS)

Krabbe Disease

04/27

NCT03333200: Longitudinal Study of Neurodegenerative Disorders

Recruiting

N/A

1500

Palliative Care, Hematopoetic Stem Cell Transplantation

University of Pittsburgh

MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease

01/30

01/35

NCT03047369: The Myelin Disorders Biorepository Project

Recruiting

N/A

12000

Children's Hospital of Philadelphia, National Institutes of Health (NIH), National Institute of Neurological Disorders and Stroke (NINDS), National Center for Advancing Translational Sciences (NCATS), Biogen, Eli Lilly and Company, Myrtelle Inc., Orchard Therapeutics Ltd., Passage Bio, Inc., Synaptix Biotherapeutics Ltd., Takeda, Boehringer Ingelheim, Ionis Pharmaceuticals, Inc., Sanofi Winthrop Industrie, Sana Biotechnology, Yaya Foundation for 4H Leukodystrophy, University of Pennsylvania, United MSD Foundation, Foundation to Fight H-ABC, Calliope Joy Foundation, Don't Forget Me Foundation

Leukodystrophy, White Matter Disease, Leukoencephalopathies, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD Gene Mutation, ALD (Adrenoleukodystrophy), X-linked Adrenoleukodystrophy, X-ALD, Adrenomyeloneuropathy, Aicardi Goutieres Syndrome, AGS, Alexander Disease, Alexanders Leukodystrophy, AxD, ADLD, Canavan Disease, CTX, Cerebrotendinous Xanthomatoses, Krabbe Disease, GALC Deficiency, Globoid Leukodystrophy, TUBB4A-Related Leukodystrophy, H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum, HBSL, HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, ALSP, CSF1R Gene Mutation, HCC - Hypomyelination and Congenital Cataract, MLC1, Megalencephalic Leukoencephalopathy With Subcortical Cysts, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication | Blood or Tissue | Mutations, Pelizaeus Merzbacher Like Disease, Peroxisomal Biogenesis Disorder, Zellweger Syndrome, Refsum Disease, Salla Disease, Sialic Storage Disease, Sjögren, Sjogren-Larsson Syndrome, Van Der Knapp Disease, Vanishing White Matter Disease, Charcot-Marie-Tooth, CMT, Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency, Allan-Herndon-Dudley Syndrome, Cadasil, Cockayne Syndrome, Multiple Sulfatase Deficiency, Gangliosidoses, GM2 Gangliosidosis, BPAN, Labrune Syndrome, LCC, Mucopolysaccharidoses, TBCK-Related Intellectual Disability Syndrome

12/30

Bailey, Carrie

Recruiting

2/3

Europe, Canada, US, RoW

tividenofusp alfa, idursulfase

Denali Therapeutics Inc.

Mucopolysaccharidosis II

12/25

NCT05005845: NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)

Completed

199

NFX-179 gel, NFX-179 topical gel, Vehicle gel, NFX-179 vehicle gel

NFlection Therapeutics, Inc.

Cutaneous Neurofibroma, Neurofibromatosis 1

10/23

NF111, NCT04544007: A Phase II Trial of Poly-ICLC for Low-Grade Gliomas

Recruiting

Poly ICLC

University of Alabama at Birmingham, Children's Healthcare of Atlanta, Children's Hospital Los Angeles

NF1, Low-grade Glioma

02/26

02/27

pheNIX, NCT03952156: Gene Therapy Clinical Study in Adult PKU

Terminated

1/2

HMI-102

Homology Medicines, Inc

Phenylketonurias, PAH Deficiency

01/23

08/23

NCT05781399: First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria

Recruiting

112

US, RoW

JNT-517 Suspension, Placebo Suspension, JNT-517 Tablet, Placebo Tablet

Jnana Therapeutics

Phenylketonuria

12/24

03/25

ENERGY, NCT05734196: The Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency

Recruiting

Europe, US

INZ-701, (rhENPP1-Fc).

Inozyme Pharma

Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy, ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum

03/26

04/26

NCT06247553: To Develop a Walking Exercise Program for Non-ambulatory Stroke Survivors

Recruiting

N/A

Gait Training, Leg Cycling Exercise

University of Kansas Medical Center

Stroke, Walking, Difficulty, Cardiovascular Injury

09/25

Magner, Martin

Recruiting

2/3

Europe, Canada, US, RoW

tividenofusp alfa, idursulfase

Denali Therapeutics Inc.

Mucopolysaccharidosis II

12/25

Hillman, Paul

Recruiting

2/3

Europe, Canada, US, RoW

tividenofusp alfa, idursulfase

Denali Therapeutics Inc.

Mucopolysaccharidosis II

12/25

OR-ARI-EAP-NPC, NCT04316637: Early Access Program With Arimoclomol in US Patients With NPC

Available

N/A

Arimoclomol

ZevraDenmark

Niemann-Pick Disease, Type C

Jansa, Pavel

TripleTRE, NCT06317805: Initial Triple Therapy Including Parenteral Treprostinil vs Initial Double Oral Therapy in PAH Group I Patients

Recruiting

110

Europe, RoW

Generic treprostinil sodium + Standard of Care (Double Oral), Trisuva, Tresuvi, Treposa, Treprostinil Orpha-Devel, Treprostinil Amomed, Treprostinil OrPha, Treposuvi, Standard of Care - Double Oral

AOP Orphan Pharmaceuticals AG, ANOVA CRO s.r.o., PharmaLex Belgium, Aixial s.r.o., GCP-Service International Ltd. & Co. KG

Pulmonary Arterial Hypertension

06/27

NCT03626688 / 2018-001187-33: A Study Evaluating the Efficacy and Safety of Ralinepag to Improve Treatment Outcomes in PAH Patients

Recruiting

1000

Europe, Canada, US, RoW

Ralinepag, APD811, Placebo

United Therapeutics

12/25

IMPAHCT, NCT05036135 / 2021-001910-13: A Study of AV-101 (Dry Powder Inhaled Imatinib) in Patients With Pulmonary Arterial Hypertension (PAH)

Terminated

2/3

202

Europe, Canada, US, RoW

AV-101, Placebo

Aerovate Therapeutics, Aerovate Therapeutics, Inc.

Pulmonary Arterial Hypertension

08/24

ELEVATE 2, NCT04712669 / 2020-004971-42: A Study of Rodatristat Ethyl in Patients With Pulmonary Arterial Hypertension

Completed

108

Europe, Canada, US, RoW

rodatristat ethyl 300 mg tablet BID, rodatristat ethyl 600 mg BID, Placebo

Altavant Sciences GmbH

Pulmonary Arterial Hypertension

06/23

08/23

Spicka

MajesTEC-4, NCT05243797 / 2021-002531-27: Phase 3 Study of Teclistamab in Combination With Lenalidomide and Teclistamab Alone Versus Lenalidomide Alone in Participants With Newly Diagnosed Multiple Myeloma as Maintenance Therapy Following Autologous Stem Cell Transplantation

Recruiting

1594

Europe, Canada, US, RoW

Teclistamab, JNJ-64007957, Lenalidomide

Stichting European Myeloma Network, Janssen Pharmaceutica

Multiple Myeloma

04/28

04/32

Flekac, Milan

NCT06383390: The Effect of Retatrutide Once Weekly on Cardiovascular Outcomes and Kidney Outcomes in Adults Living With Obesity (TRIUMPH-Outcomes)

Recruiting

10000

Europe, Canada, US, RoW

Retatrutide, LY3437943, Placebo

Eli Lilly and Company

Atherosclerotic Cardiovascular Disease (ASCVD), Chronic Kidney Disease (CKD)

02/29

ACHIEVE-4, NCT05803421: A Study of Daily Oral Orforglipron (LY3502970) Compared With Insulin Glargine in Participants With Type 2 Diabetes and Obesity or Overweight at Increased Cardiovascular Risk

Active, not recruiting

2749

Europe, US, RoW

Orforglipron, LY3502970, Insulin Glargine

Eli Lilly and Company

Type 2 Diabetes, Obesity, Overweight or Obesity, Overweight, Cardiovascular Diseases, Chronic Kidney Disease

09/25

01/26

NCT06037252: A Study of Investigational Tirzepatide (LY3298176) Doses in Participants With Type 2 Diabetes and Obesity

Recruiting

350

US, RoW

Tirzepatide, LY3298176, Placebo

Eli Lilly and Company

Type 2 Diabetes, Obesity

01/26

10/26

NCT06074562: A Study of LY3556050 in Adult Participants With Diabetic Peripheral Neuropathic Pain

Recruiting

410

Europe, Japan, US, RoW

LY3556050, Placebo

Eli Lilly and Company

Diabetic Peripheral Neuropathy

07/25

Kolar, Dawn

Recruiting

2/3

Europe, Canada, US, RoW

tividenofusp alfa, idursulfase

Denali Therapeutics Inc.

Mucopolysaccharidosis II

12/25

NCT04251026 / 2019-004909-27: A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

Active, not recruiting

1/2

Europe, Canada, US

tividenofusp alfa

Denali Therapeutics Inc.

Mucopolysaccharidosis II

07/27

PROVIDE, NCT04411654: Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease

Active, not recruiting

1/2

Europe, US

LY3884961, Methylprednisolone, Sirolimus, Prednisone

Prevail Therapeutics, Eli Lilly and Company

Gaucher Disease, Type 2

05/28

NCT04007536: A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome

Completed

N/A

Europe, US

No Intervention

Denali Therapeutics Inc.

Mucopolysaccharidosis II

03/24

Dolezalova

EMERALD, NCT05001737 / 2021-001577-24: Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE

Active, not recruiting

Europe, Canada, Japan, US, RoW

Emapalumab, NI-0501, emapalumab-lzsg, ATC code: L04AA39 (WHO)

Swedish Orphan Biovitrum

Macrophage Activation Syndrome, Secondary Hemophagocytic Lymphohistiocytosis, Still Disease, Systemic Lupus Erythematosus, SJIA, AOSD, MAS

06/24

09/25

Hachulla

EMERALD, NCT05001737 / 2021-001577-24: Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE

Active, not recruiting

Europe, Canada, Japan, US, RoW

Emapalumab, NI-0501, emapalumab-lzsg, ATC code: L04AA39 (WHO)

Swedish Orphan Biovitrum

Macrophage Activation Syndrome, Secondary Hemophagocytic Lymphohistiocytosis, Still Disease, Systemic Lupus Erythematosus, SJIA, AOSD, MAS

06/24

09/25

Linkova, Lenka

Recruiting

2/3

Europe, Canada, US, RoW

tividenofusp alfa, idursulfase

Denali Therapeutics Inc.

Mucopolysaccharidosis II

12/25

Costello

PREVAIL, NCT05202509 / 2021-005092-39: Cardiovascular Outcome Study to Evaluate the Effect of Obicetrapib in Patients With Cardiovascular Disease

Active, not recruiting

9541

Europe, Canada, Japan, US, RoW

Obicetrapib, Placebo

NewAmsterdam Pharma, Monash University

Atherosclerotic Cardiovascular Disease

11/26

CARTITUDE-6, NCT05257083 / 2021-003284-10: A Study of Daratumumab, Bortezomib, Lenalidomide and Dexamethasone (DVRd) Followed by Ciltacabtagene Autoleucel Versus Daratumumab, Bortezomib, Lenalidomide and Dexamethasone (DVRd) Followed by Autologous Stem Cell Transplant (ASCT) in Participants With Newly Diagnosed Multiple Myeloma

Recruiting

750

Europe, Canada, Japan, US, RoW

Daratumumab, Bortezomib, Lenalidomide, Dexamethasone, Cilta-cel, JNJ-68284528, Cyclophosphamide, Fludarabine

Stichting European Myeloma Network, Janssen Research & Development, LLC

Multiple Myeloma

06/33

08/40

Ceska

PREVAIL, NCT05202509 / 2021-005092-39: Cardiovascular Outcome Study to Evaluate the Effect of Obicetrapib in Patients With Cardiovascular Disease

Active, not recruiting

9541

Europe, Canada, Japan, US, RoW

Obicetrapib, Placebo

NewAmsterdam Pharma, Monash University

Atherosclerotic Cardiovascular Disease

11/26

Rojas, Carolina

Recruiting

2/3

Europe, Canada, US, RoW

tividenofusp alfa, idursulfase

Denali Therapeutics Inc.

Mucopolysaccharidosis II

12/25

NCT06397573: Ablative Stereotactic MR-Guided Adaptive Reirradiation

Recruiting

N/A

Reirradiation

Baptist Health South Florida, Baptist Health Foundation

Abdominal Cancer, Pelvic Cancer

06/27

NCT05553392: StrataXRT Versus Aquaphor for the Prevention and Management of Radiation Dermatitis

Terminated

N/A

StrataXRT, Aquaphor

Stratpharma AG

Radiation Dermatitis

05/24

Jungova

Recruiting

1594

Europe, Canada, US, RoW

Teclistamab, JNJ-64007957, Lenalidomide

Stichting European Myeloma Network, Janssen Pharmaceutica

Multiple Myeloma

04/28

04/32

Recruiting

750

Europe, Canada, Japan, US, RoW

Daratumumab, Bortezomib, Lenalidomide, Dexamethasone, Cilta-cel, JNJ-68284528, Cyclophosphamide, Fludarabine

Stichting European Myeloma Network, Janssen Research & Development, LLC

Multiple Myeloma

06/33

08/40

Garcia, Marilyn

Recruiting

2/3

Europe, Canada, US, RoW

tividenofusp alfa, idursulfase

Denali Therapeutics Inc.

Mucopolysaccharidosis II

12/25

HERO, NCT04732429 / 2023-000785-33: Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia

Terminated

US, RoW

HST5040, Placebo

HemoShear Therapeutics, HemoShear Therapeutics

Methylmalonic Acidemia, Propionic Acidemia

10/23

OR-ARI-EAP-NPC, NCT04316637: Early Access Program With Arimoclomol in US Patients With NPC

Available

N/A

Arimoclomol

ZevraDenmark

Niemann-Pick Disease, Type C

Fainboim, Alejandro

Recruiting

2/3

Europe, Canada, US, RoW

tividenofusp alfa, idursulfase

Denali Therapeutics Inc.

Mucopolysaccharidosis II

12/25

EMERALD, NCT05001737 / 2021-001577-24: Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE

Active, not recruiting

Europe, Canada, Japan, US, RoW

Emapalumab, NI-0501, emapalumab-lzsg, ATC code: L04AA39 (WHO)

Swedish Orphan Biovitrum

Macrophage Activation Syndrome, Secondary Hemophagocytic Lymphohistiocytosis, Still Disease, Systemic Lupus Erythematosus, SJIA, AOSD, MAS

06/24

09/25

Pachmanova, Barbora

NCT06274489: A Study to Evaluate Setanaxib in Patients With Alport Syndrome

Recruiting

1/2

Europe, RoW

Setanaxib, Placebo

Calliditas Therapeutics AB

Alport Syndrome

01/25



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