OPKO

 

 

Completed

2

63

RoW

Dendritic Cells DCVAC/PCa, Leuprolide acetate, Lupron, Goserelin Acetate, Zoladex

SOTIO a.s., Ascend Biopharmaceuticals Ltd

Basal Cell Carcinoma, Basal Cell Nevus Syndrome (BCNS)

12/15

06/16

Not yet recruiting

2

50

Europe

Solaris, Infusion, Solaris

Newcastle Upon Tyne Hospitals NHS Foundation Trust, National Institute for Health Research

Atypical Haemolytic Uraemic Syndrome (aHUS), aHUS is a rare disease caused by a fault in the complement system. The complement system is part of your body’s immune response that attacks bugs., Diseases [C] - Immune System Diseases [C20]

 

 

Not yet recruiting

2

12

Europe

Amifampridina fosfato, [Amifampridina fosfato], Tablet, FIRDAPSE - 10 MG - COMPRESSE - USO ORALE - BLISTER(ALU/PVC/PVDC) 100 X 1 COMPRESSE

CATALYST PHARMACEUTICALS INC., Catalyst Pharmaceuticals, Inc.

Spinal Muscular Atrophy (SMA) Type 3 Atrofia Muscolare Spinale (SMA) tipo 3, Spinal Muscular Atrophy (SMA) Atrofia Muscolare Spinale (SMA), Diseases [C] - Nervous System Diseases [C10]

 

 

Not yet recruiting

2

16

Europe

INS1007, Film-coated tablet

Insmed Incorporated, Insmed Incorporated

Granulomatosis with Polyangiitis (GPA, Wegener’s granulomatosis, Diseases [C] - Immune System Diseases [C20]

 

 

Completed

2

62

Europe, Japan, US, RoW

Risdiplam, RO7034067, Evrysdi

Hoffmann-La Roche

Muscular Atrophy, Spinal

11/19

12/23

Not yet recruiting

2

26

Europe

ALXN2050, ACH-0145228, Capsule

Achillion Pharmaceuticals, Inc., a wholly owned subsidiary of Alexion Pharmaceuticals Inc., Alexion Pharmaceuticals Inc., Achillion Pharmaceuticals, Inc. a wholly owned subsidiary of Alexion Pharmaceuticals Inc., Alexion Pharmaceuticals Inc.

Paroxysmal Nocturnal Hemoglobinuria (PNH), Paroxysmal Nocturnal Hemoglobinuria (PNH), Diseases [C] - Immune System Diseases [C20]

 

 

Completed

2

58

Europe, US

SRK-015

Scholar Rock, Inc.

Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations

01/21

02/24

Not yet recruiting

2

63

Europe

Itacitinib, Tablet

ASSISTANCE PUBLIQUE HOPITAUX DE PARIS, INCYTE BIOSCIENCES INTERNATIONAL SARL Company,

Adults patients having non severe sporadic Hemophagocytosis Lymphohistiocytosis, Adults patients having non severe sporadic Hemophagocytosis Lymphohistiocytosis, Diseases [C] - Immune System Diseases [C20]

 

 

Active, not recruiting

2

10

Europe

OTL-200, Previously GSK2696274

Orchard Therapeutics, Ospedale San Raffaele

Lysosomal Storage Disease, Metachromatic Leukodystrophy

04/22

04/28

Active, not recruiting

2

20

US

Abatacept, Orencia, Placebo

Rohit Aggarwal, MD, Bristol-Myers Squibb

Myositis, Interstitial Lung Disease

05/22

07/23

Completed

2

15

RoW

FRSW117, PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection

Jiangsu Gensciences lnc.

Severe Hemophilia A

08/22

08/22

Recruiting

2

9

RoW

Recombinant Humanized Anti-interleukin-6 Receptor Monoclonal Antibody Injection 4mg/kg, Recombinant Humanized Anti-interleukin-6 Receptor Monoclonal Antibody Injection 6mg/kg, Recombinant Humanized Anti-interleukin-6 Receptor Monoclonal Antibody Injection 8mg/kg

Beijing VDJBio Co., LTD.

Idiopathic Multicentric Castleman's Disease

11/22

12/24

Ongoing

2

200

Europe

BCX9930, BCX9930, Tablet

BioCryst Pharmaceuticals Inc, BioCryst Pharmaceuticals Inc

Paroxysmal Nocturnal Hemoglobinuria, PNH, Diseases [C] - Immune System Diseases [C20]

 

 

Ongoing

2

90

Europe

SP-420, SP-420, Capsule, hard

Pharmacosmos A/S, Pharmacosmos A/S

Transfusion-dependent β-thalassemia, Beta-thalassemia is a rare disease that is inherited and leads to a reduced production of hemoglobin (anemia)., Diseases [C] - Blood and lymphatic diseases [C15]

 

 

Not yet recruiting

2

16

Europe

Velusetrag, [TD-5108 hydrochloride], Capsule, hard

ALFASIGMA S.P.A., Alfasigma S.p.A.

Chronic intestinal pseudo-obstruction (CIPO) is a rare, severe condition characterized by an impairment of coordinated propulsive activity in the intestinal tract resulting in a clinical picture similar to that of mechanical intestinal obstruction, although in the absence of any lesion occluding the gut. CIPO can be idiopathic, when no primary underlying disorder is demonstrated, or secondary, when related to systemic diseases. La pseudoobstrucción intestinal crónica (CIPO) es una enfermedad rara y grave, caracterizada por una alteración de la actividad de propulsión coordinada en los intestinos que se traduce en un cuadro clínico parecido al de la obstrucción intestinal mecánica, salvo por la ausencia de lesiones que ocluyen el intestino. La CIPO puede ser idiopática, cuando no hay un trastorno primario subyacente demostrado, o secundaria, cuando está relacionada con enfermedades sistémicas., Chronic intestinal pseudo-obstruction (CIPO) is a rare disease impacting the intestinal motility, potentially invalidating. La pseudoobstrucción intestinal crónica (CIPO) es una enfermedad rara que afecta a la motilidad intestinal, potencialmente invalidante., Diseases [C] - Digestive System Diseases [C06]

 

 

Recruiting

2

24

RoW

KN057 doseⅠ, KN057 dose Ⅱ, KN057 dose Ⅲ

Suzhou Alphamab Co., Ltd.

Hemophilia

02/24

06/24

Completed

2

32

RoW

STSP-0601 for Injection

Staidson (Beijing) Biopharmaceuticals Co., Ltd, Beijing Novikang Medical Technology Co., LTD

Hemophilia

08/23

08/23

Recruiting

2

63

Europe

Itacitinib

Assistance Publique - Hôpitaux de Paris, Incyte Corporation

Adults Patients Having Non Severe HLH

11/23

02/24

Active, not recruiting

2

12

US

Lenalidomide, Revlimid

Dana-Farber Cancer Institute, Celgene

Langerhans Cell Histiocytosis (LCH), Histiocytoses Erdheim-chester Disease, Histiocytic Sarcoma (HS)

02/24

08/24

Recruiting

2

24

RoW

HRS-5965 tablets

Chengdu Suncadia Medicine Co., Ltd.

Paroxysmal Nocturnal Hemoglobinuria

12/27

12/27

Terminated

2

12

RoW

BCX9930 monotherapy, Placebo

BioCryst Pharmaceuticals

Paroxysmal Nocturnal Hemoglobinuria (PNH)

09/23

09/23

Recruiting

2

25

RoW

STSP-0601 for Injection

Staidson (Beijing) Biopharmaceuticals Co., Ltd

Hemophilia

02/25

02/25

Enrolling by invitation

2

66

Europe

Tezepelumab, Placebo

Imperial College London, AstraZeneca

EGPA - Eosinophilic Granulomatosis With Polyangiitis

10/25

10/25

Withdrawn

2

12

US

NFX-179 Gel 1.50%

Albert Chiou, NFlection Therapeutics, Inc.

Epidermal Nevi, Nevus Sebaceus

06/24

12/24

Active, not recruiting

2

25

RoW

HLX208, BRAF V600E inhibitor

Shanghai Henlius Biotech

Langerhans Cell Histiocytosis, Erdheim-Chester Disease, LCH, ECD

06/24

10/24

Not yet recruiting

2

25

RoW

Dusquetide, SGX945, SGX942

Soligenix

Behçet Disease

02/25

03/25

Terminated

2

12

Europe

BCX9930, Eculizumab, Soliris, Ravulizumab, Ultomiris, ALXN1210, ravulizumab-cwvz

BioCryst Pharmaceuticals

Paroxysmal Nocturnal Hemoglobinuria (PNH)

09/23

09/23

Active, not recruiting

2

10

US

Sirolimus, Rapamycin, Rapamune

Ohio State University, PTEN Research, Pfizer

PTEN Gene Mutation, PTEN Hamartoma Tumor Syndrome, PTEN Hamartoma Syndrome, Cowden Syndrome, Bannayan Syndrome, Bannayan Zonana Syndrome, Polyposis

02/25

02/25

Not yet recruiting

2

30

NA

CBL-514 injection, 0.9% Sodium chloride

Caliway Biopharmaceuticals Co., Ltd.

Dercum's Disease

Active, not recruiting

2

26

RoW

HRS-5965 tablets

Chengdu Suncadia Medicine Co., Ltd.

Paroxysmal Nocturnal Hemoglobinuria

10/24

10/24

Recruiting

2

35

RoW

KP104

Kira Pharmacenticals (US), LLC.

Paroxysmal Nocturnal Hemoglobinuria

11/24

02/25

Completed

2

2

US

Daratumumab Injection, Darzalex

University of Arkansas, Janssen Scientific Affairs, LLC

POEMS Syndrome

08/23

10/23

Not yet recruiting

2

40

NA

MY008211A tablets, MY008211A

Wuhan Createrna Science and Technology Co., Ltd

Paroxysmal Nocturnal Hemoglobinuria (PNH)

07/25

12/25

Terminated

2

6

US

Obinutuzumab, Rituximab

Mayo Clinic, Genentech, Inc.

Granulomatosis With Polyangiitis, Microscopic Polyangiitis, ANCA Associated Vasculitis

05/24

05/24

Recruiting

2

45

Japan, US

NS-229, Placebo

NS Pharma, Inc., Nippon Shinyaku Co., Ltd.

Eosinophilic Granulomatosis With Polyangiitis, Churg-Strauss Syndrome

04/25

05/25

Recruiting

2

150

US

Durvalumab, MEDI4736, Tremelimumab, CP-675,206

Prisma Health-Upstate, AstraZeneca, MedImmune LLC

Cancer, Rare Disease

02/26

06/27

Not yet recruiting

2

12

NA

NM8074, Soliris, Eculizumab

NovelMed Therapeutics

Paroxysmal Nocturnal Hemoglobinuria

08/26

01/27

Recruiting

2

12

Europe, US, RoW

SerpinPC, Activated Protein C (APC) inhibitor

ApcinteX Ltd, Centessa Pharmaceuticals plc

Hemophilia B With Inhibitor, Hemophilia B

09/25

10/25

Recruiting

2

120

Europe, Canada, US, RoW

SerpinPC, Activated Protein C (APC) inhibitor

ApcinteX Ltd, Centessa Pharmaceuticals plc

Hemophilia A, Hemophilia B

03/26

06/26

Not yet recruiting

2

6

NA

NM8074

NovelMed Therapeutics

PNH - Paroxysmal Nocturnal Hemoglobinuria

08/26

10/26

Recruiting

2

190

Europe, US

Tabelecleucel, tab-cel®, ATA129, EBV-CTLs

Atara Biotherapeutics

Epstein-Barr Virus (EBV)-Associated Diseases, EBV+ Lymphoproliferative Disease With Primary Immunodeficiency (EBV+ PID LPD), EBV+ Lymphoproliferative Disease With Acquired (Non-congenital) Immunodeficiency (EBV+ AID LPD), EBV+ Posttransplant Lymphoproliferative Disease in Central Nervous System (EBV+ CNS PTLD), EBV+ Post-transplant Lymphoproliferative Disease (EBV+ PTLD), Solid Organ Transplant Complications, Lymphoproliferative Disorders, Allogeneic Hematopoietic Cell Transplant, Stem Cell Transplant Complications, EBV+ Sarcomas, Leiomyosarcoma

06/27

05/29

Ongoing

1/2

6

Europe

Vector viral adenoasociado de serotipo 9 que contiene el gen de la sulfamidasa humana, AAV9-CAG-coh-SGSH, Suspension for injection

Laboratorios del Dr. Esteve, S.A., Laboratorios del Dr. Esteve,S.A.

Mucopolysaccharidosis type IIIA (Sanfilippo A syndrome) is an inherited lysosomal storage disease caused by a specific lysosomal enzyme deficiency that leads to intracellular accumulation of the GAG heparan sulphate (HS). It is caused by a deficiency of one of the four enzymes involved in the lysosomal degradation of HS. In the case of subtype A is the heparan N-sulfatase (SGSH). La Mucopolisacaridosis tipo IIIA (síndrome de Sanfilippo A) es una enfermedad hereditaria de almacenamiento lisosomal causada por una deficiencia de la enzima lisosomal específica que conduce a la acumulación intracelular del Heparán Sulfato GAG (HS). Es causada por una deficiencia de una de las cuatro enzimas implicadas en la degradación lisosomal de HS. En el caso del subtipo A es el heparán N-sulfatasa (SGSH)., Inherited disease caused by the deficiency of Sulfamidase, the enzime that degrades Heparan Sulfate, leading to its accumulation in the cells causing the severe signs and symptoms of MPSIIIA Enfermedad hereditaria causada por la deficiencia de Sulfamidasa, enzima que degrada el Heparan Sulfato,conduciendo a su acumulación en las células causando los graves signos y síntomas de la MPS IIIA, Diseases [C] - Nutritional and Metabolic Diseases [C18]

 

 

Not yet recruiting

1/2

10

Europe

AAV5-hFVIII-SQ, BMN 270, Solution for infusion

BioMarin Pharmaceutical Inc., BioMarin Pharmaceutical Inc.

Haemophilia A, Bleeding Disorder, Diseases [C] - Blood and lymphatic diseases [C15]

 

 

Not yet recruiting

1/2

27

Europe

rAAV2/6 Left ZFN Vector, rAAV2/6 Right ZFN Vector, SB-A6P-HRL Donor Vector, SB-47171, SB-47898, SB-IDUA, Solution for infusion

Sangamo Therapeutics, Inc., Sangamo Therapeutics, Inc.

Mucopolysaccharidosis type I (MPS I), Hurler/Hurler-Scheie/Scheie syndrome, a recessive lysosomal storage disorder caused by a deficiency in iduronidase (IDUA) enzyme., Diseases [C] - Nutritional and Metabolic Diseases [C18]

 

 

Not yet recruiting

1/2

100

Europe

BMN 307, Solution for infusion

BioMarin Pharmaceutical Inc., BioMarin Pharmaceutical Inc.

Phenylketonuria, metabolic disorder, Diseases [C] - Nutritional and Metabolic Diseases [C18]

 

 

Not yet recruiting

1/2

48

Europe

PPSGG solution for infusion, PPSGG, Solution for infusion

Polyneuron Pharmaceuticals AG, Polyneuron Pharmaceuticals AG

anti-MAG neuropathy, Anti-MAG peripheral neuropathy is a rare disease. It occurs when the body’s own immune system develops antibodies against myelin (protein of the nerves, myelin-associated glycoprotein, MAG)., Diseases [C] - Nervous System Diseases [C10]

 

 

Ongoing

1/2

10

Europe

AAV5-hFIX, AMT-060, Solution for infusion

uniQure biopharma B.V., uniQure biopharma B.V.

Haemophilia B, Haemophilia B - Bleeding disorder, Diseases [C] - Blood and lymphatic diseases [C15]

 

 

Not yet recruiting

1/2

21

Europe

SHP648 (TAK748), Solution for injection

Baxalta Innovations GmbH, Baxalta Innovations GmbH

Hemophilia B is a X-linked recessive bleeding disorder caused by mutations in the gene encoding clotting factor IX (FIX) that result in disruption of the normal clotting pathway. Hemophilia B affects 1 in 25,000 male births. Disease severity correlates directly with the concentration of functional FIX protein in the plasma. Severe disease is characterized as having <1% of normal plasma levels of FIX (100% = 1 IU activity/mL or approximately 5000 ng protein/mL)., Hemophilia B is an inherited bleeding disorder caused by a lack of the blood clotting factor IX (9) in your blood. Without enough factor IX, the blood cannot clot properly to stop bleeding., Diseases [C] - Blood and lymphatic diseases [C15]

 

 

Not yet recruiting

1/2

12

Europe

scAAV9.U1A.SGSH, ABO-102, Suspension for injection

Abeona Therapeutics Inc, Abeona Therapeutics Inc

MPS IIIA is a devastating lysosomal storage disease, caused by a Nsulfoglucosamine sulfohydrolase gene defect. Infants with MPS IIIA appear normal at birth, but the disease is relentlessly progressive, withdeterioration of social and adaptive abilities, neurocognitive decline, and premature death. Death typically occurs by end of the second or beginning of the third decade. Quite importantly, there is no treatmentcurrently available for the disease. MPS IIIA es una enfermedad de depósito lisosomal, causada por un defecto genético de la enzima N-sulfoglucosamina sulfohidrolasa. Los niños parecen normales al nacer, pero la enfermedad es progresiva, conel deterioro de las habilidades sociales y de adaptación, la disminución neurocognitiva y la muerte prematura. La muerte se produce normalmente a finales de la segunda o principios de la tercera década. Es de destacar que no existe un tratamiento disponible actualmente para la enfermedad., Mucopolysaccharidosis type IIIA is a genetic disease in children, caused by the toxicity of an accumulation of substances in the body that generate a progressive deterioration. La mucopolisacaridosis tipo IIIA es una enfermedad genética en niños, causada por la toxicidad de un acúmulo de sustancias en el organismo que generan un deterioro progresivo., Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]