University of Iowa Stead Family Children's Hospital
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 27 Trials 
210 Trials

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Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Mathews, Katherine
HOPE-3, NCT05126758: A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

Active, not recruiting
3
104
US
Deramiocel (CAP-1002), Cardiosphere-Derived Cells (CDCs), Placebo
Capricor Inc.
Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases, Neuromuscular Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn, Nervous System Diseases
12/25
12/27
ENVISION, NCT05881408 / 2020-002372-13: A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

Recruiting
3
148
Europe, Japan, US, RoW
delandistrogene moxeparvovec, SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS, placebo
Sarepta Therapeutics, Inc., Hoffmann-La Roche
Duchenne Muscular Dystrophy
05/27
06/28
ASCEND, NCT05067790 / 2021-001294-23: A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam

Recruiting
3
45
Europe, Japan, US
Nusinersen, BIIB058, Spinraza
Biogen
Spinal Muscular Atrophy
06/27
06/27
REACH CDM, NCT03692312: Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

Completed
2/3
56
Europe, Canada, US, RoW
Tideglusib, Placebo
AMO Pharma Limited
Congenital Myotonic Dystrophy
04/23
04/23
REACH CDM X, NCT05004129: Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy

Recruiting
2/3
76
Canada, US, RoW
Tideglusib
AMO Pharma Limited
Congenital Myotonic Dystrophy
03/25
03/25
MOMENTUM, NCT04004065 / 2019-000601-77: Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

Checkmark From MOMENTUM trial for DMD
May 2021 - May 2021: From MOMENTUM trial for DMD
Checkmark Interim data from the MOMENTUM study
Dec 2020 - Dec 2020: Interim data from the MOMENTUM study
Checkmark Data from MOMENTUM trial for Duchenne muscular dystrophy
More
Active, not recruiting
2
62
Europe, Canada, US
Vesleteplirsen, SRP-5051
Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
10/23
01/29
LION-CS101, NCT05230459: A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)

Recruiting
1/2
10
US
AB-1003 dose level 1, AB-1003 dose level 2, Placebo
Asklepios Biopharmaceutical, Inc.
Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2, LGMD2I, Muscular Dystrophy, LGMD2, LGMD, FKRP, FKRP Mutation, Fukutin Related Protein
12/28
12/28
MOVE FSHD, NCT04635891: Motor Outcomes to Validate Evaluations in FSHD

Recruiting
N/A
450
Europe, Canada, US
University of Kansas Medical Center, FSHD Society, Inc., Friends Research Institute, Inc., University of Rochester, University of Nevada, Reno, FSHD Canada, Avidity Biosciences, Inc., AMRA Medical, Seattle Children's Hospital, Dyne Therapeutics, Hoffmann-La Roche
FSHD
12/26
01/27
GRASP-01-002, NCT05257473: Defining Endpoints in Becker Muscular Dystrophy

Recruiting
N/A
80
Europe, US, RoW
Virginia Commonwealth University, Edgewise Therapeutics, Inc.
Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Becker
05/25
05/26
NCT04174157: Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)

Recruiting
N/A
700
Europe, Japan, US, RoW
Prospective observational registry, Zolgensma
Novartis Pharmaceuticals, United BioSource, LLC
Spinal Muscular Atrophy (SMA)
06/38
06/38
Harmatz, Paul
NCT05494593: A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)

Recruiting
4
5
US
ELAPRASE, Idursulfase, Rituximab, Methotrexate, Intravenous Immunoglobulin (IVIG)
Takeda, Takeda Development Center Americas, Inc.
Mucopolysaccharidosis (MPS), Hunter Syndrome
07/28
07/28
STARLIGHT, NCT04573023 / 2020-003200-14: A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II

Recruiting
3
80
Europe, US, RoW
JR-141, Idursulfase, JR-141 or Idursulfase
JCR Pharmaceuticals Co., Ltd., JCR Pharmaceuticals Co., Ltd.
Mucopolysaccharidosis II
01/26
01/26
NCT05594992: An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis Type II (Hunter Syndrome) Subjects

Enrolling by invitation
3
80
Europe, US, RoW
JR-141
JCR Pharmaceuticals Co., Ltd.
Mucopolysaccharidosis II
02/28
02/28
NCT03566043: CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

Active, not recruiting
2/3
48
US, RoW
RGX-121
REGENXBIO Inc.
Mucopolysaccharidosis Type II (MPS II)
11/23
08/25
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
54
Europe, Canada, US, RoW
tividenofusp alfa, idursulfase
Denali Therapeutics Inc.
Mucopolysaccharidosis II
12/25
12/25
PROPEL 2, NCT04265651 / 2019-002954-21: Study of Infigratinib in Children with Achondroplasia

Active, not recruiting
2
84
Europe, Canada, US, RoW
Infigratinib 0.016 mg/kg, Infigratinib 0.032 mg/kg, Infigratinib 0.064 mg/kg, Infigratinib 0.128 mg/kg, Infigratinib 0.25 mg/kg
QED Therapeutics, Inc.
Achondroplasia
10/24
12/24
NCT04571970: RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

Completed
1/2
6
Canada, US
RGX-121
REGENXBIO Inc.
Mucopolysaccharidosis Type II (MPS II)
05/23
05/24
NCT06181136: Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

Recruiting
1/2
20
US
DNL126
Denali Therapeutics Inc.
Mucopolysaccharidosis Type IIIA
08/28
08/28
NCT04783181: A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)

Active, not recruiting
1/2
8
US
AAV BBP-631
Adrenas Therapeutics Inc
Congenital Adrenal Hyperplasia
02/29
02/29
NCT04251026 / 2019-004909-27: A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

Active, not recruiting
1/2
47
Europe, Canada, US
tividenofusp alfa
Denali Therapeutics Inc.
Mucopolysaccharidosis II
07/27
07/27
NCT05682144: ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I

Recruiting
1
2
US
Autologous Plasmablasts (B cells)
Immusoft of CA, Inc.
Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS
06/25
06/39
NCT05238324: Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II

Withdrawn
1
9
Canada, US
Genetic HMI-203
Homology Medicines, Inc
Mucopolysaccharidosis II
10/24
01/29
NCT06567769: Phase 1 Study of GC1130A in Pediatric Patients with Sanfilippo Syndrome Type a (MPS IIIA)

Recruiting
1
9
US, RoW
GC1130A
GC Biopharma Corp, Novel Pharma Inc.
Sanfilippo Syndrome Type a
06/27
06/27
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
 
 
NCT04007536: A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome

Completed
N/A
18
Europe, US
No Intervention
Denali Therapeutics Inc.
Mucopolysaccharidosis II
03/24
03/24
PRONTO, NCT05109793: GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study

Active, not recruiting
N/A
31
Europe, US, RoW
Azafaros A.G.
GM1 Gangliosidosis, Sandhoff Disease, Tay-Sachs Disease
05/26
05/26
PROPEL, NCT04035811: Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

Recruiting
N/A
250
Europe, Canada, US, RoW
QED Therapeutics, Inc.
Achondroplasia
06/26
06/26
NCT04041102: Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

Recruiting
N/A
40
Europe, Canada, US, RoW
University of Pennsylvania, Passage Bio, Inc.
GM1 Gangliosidosis
06/26
12/26
Stephan, Carrie
MOVE FSHD, NCT04635891: Motor Outcomes to Validate Evaluations in FSHD

Recruiting
N/A
450
Europe, Canada, US
University of Kansas Medical Center, FSHD Society, Inc., Friends Research Institute, Inc., University of Rochester, University of Nevada, Reno, FSHD Canada, Avidity Biosciences, Inc., AMRA Medical, Seattle Children's Hospital, Dyne Therapeutics, Hoffmann-La Roche
FSHD
12/26
01/27
GRASP-01-002, NCT05257473: Defining Endpoints in Becker Muscular Dystrophy

Recruiting
N/A
80
Europe, US, RoW
Virginia Commonwealth University, Edgewise Therapeutics, Inc.
Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Becker
05/25
05/26
NCT04174157: Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)

Recruiting
N/A
700
Europe, Japan, US, RoW
Prospective observational registry, Zolgensma
Novartis Pharmaceuticals, United BioSource, LLC
Spinal Muscular Atrophy (SMA)
06/38
06/38
Bernat, John
LEAP2MONO, NCT05222906 / 2021-005402-10: Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

Active, not recruiting
3
43
Europe, Canada, Japan, US, RoW
Venglustat, imiglucerase, Cerezyme®
Sanofi, Sanofi-aventis recherche & développement
Gaucher's Disease Type III
09/25
10/26
FLY, NCT06328608: A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease

Recruiting
2/3
22
Europe, US
PRX-102 1 mg/kg every two weeks, pegunigalsidase alfa, Recombinant human alpha galactosidase-A
Chiesi Farmaceutici S.p.A., ICON plc
Fabry Disease
12/27
03/28
NCT04145037: Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease

Terminated
1/2
8
Canada, US
AVR-RD-02
AVROBIO
Gaucher Disease
08/23
08/23
STAAR, NCT04046224 / 2019-000667-24: Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease

Active, not recruiting
1/2
34
Europe, Canada, US, RoW
ST-920
Sangamo Therapeutics
Fabry Disease
04/25
09/25
NCT06181136: Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

Recruiting
1/2
20
US
DNL126
Denali Therapeutics Inc.
Mucopolysaccharidosis Type IIIA
08/28
08/28
NCT04552691: Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients

Approved for marketing
N/A
US
Pegunigalsidase Alfa
Chiesi Farmaceutici S.p.A., Chiesi USA, Inc.
Fabry Disease
 
 
Sharathkumar, Anjali
NCT04154488: A Study of Mavorixafor in Participants With Congenital Neutropenia and Chronic Idiopathic Neutropenia Disorders

Completed
1/2
32
US
Mavorixafor, X4P-001
X4 Pharmaceuticals
Neutropenia
08/24
08/24
NCT04478227: TPO-Mimetic Use in Children for Hematopoietic Failure

Completed
1
15
US
Romiplostim, Nplate
Anjali Sharathkumar, Amgen
Bone Marrow Failure Disorders, Aplastic Anemia, Thrombocytopenia, Refractory Cytopenia of Childhood, Myelodysplastic Syndrome(MDS)
01/24
01/24
Contact, Site Public
NCT04985682 / 2022-004149-11: A Study of ADVATE in People With Hemophilia A in India

Completed
4
50
RoW
ADVATE
Baxalta now part of Shire, Takeda Development Center Americas, Inc.
Hemophilia A
02/23
02/23
SWITCH, NCT04718779: A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy

Completed
4
4
US
Digital Engagement Application (GD App), No Intervention
Takeda
Gaucher Disease
02/23
02/23
COMPASS-CD, NCT04809363: A Study of CDPATH™ to Help Manage and Treat Crohn's Disease

Active, not recruiting
4
200
US
CDPATH™, PROSPECT, Blood Draw
Takeda
Crohn's Disease
10/25
06/26
NCT05341115 / 2022-002471-11: A Study of Leuprolide Acetate Depot in Children With Central Precocious Puberty

Recruiting
4
80
RoW
Leuprorelin Acetate Depot 3M, Leuprolide acetate Depot 3M
Takeda, Takeda
Central Precocious Puberty
05/25
05/25
TAK-665-4001, NCT05058391 / 2022-004193-39: A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India

Completed
4
5
RoW
Elaprase, Idursulfase
Takeda, Takeda Biopharmaceuticals India Pvt. Ltd.
Hunter Syndrome
04/24
04/24
NCT05442554: A Study of Brentuximab Vedotin Treatment in Chinese Adults With CD30-Positive Cutaneous T-Cell Lymphoma

Completed
4
10
RoW
Brentuximab vedotin, SGN-35
Takeda
T-Cell Lymphoma
08/24
08/24
NCT05067868: A Study of Replagal in Children and Adults With Fabry Disease in India

Active, not recruiting
4
5
RoW
Replagal
Shire
Fabry Disease
10/26
11/26
TAK-113-4002, NCT06562543: A Trial to Evaluate the Safety and Activity of Fruquintinib in Minority Populations With Advanced, Previously Treated Colorectal Cancer

Recruiting
4
78
US
Fruquintinib, Fruzaqla™
Takeda
Colorectal Cancer
02/28
02/28
NCT04085172 / 2018-000821-29: A Study of TAK-503 in Children and Teenagers With Attention Deficit Hyperactivity Disorder (ADHD)

Active, not recruiting
4
288
Europe, US
Guanfacine hydrochloride (TAK-503), Intuniv, SPD503, Atomoxetine hydrochloride, Placebo
Shire, Takeda Development Center Americas, Inc.
Attention Deficit Hyperactivity Disorder
06/27
06/27
ASPIRE, NCT04118088 / 2017-002491-10: A Study of Darvadstrocel in Adults With Crohn's Disease and Complex Perianal Fistula

Active, not recruiting
4
50
Europe, RoW
Darvadstrocel, Alofisel, Cx601
Takeda, Takeda Development Center Americas, Inc.
Crohn's Disease, Complex Perianal Fistula
03/26
03/26
NCT05494593: A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)

Recruiting
4
5
US
ELAPRASE, Idursulfase, Rituximab, Methotrexate, Intravenous Immunoglobulin (IVIG)
Takeda, Takeda Development Center Americas, Inc.
Mucopolysaccharidosis (MPS), Hunter Syndrome
07/28
07/28
NCT06045754: A Study of Vedolizumab Intravenous (IV) and Adalimumab or Vedolizumab and Ustekinumab in Adults With Crohn's Disease

Recruiting
4
150
Canada, US
Vedolizumab, Entyvio, Adalimumab, Humira, Ustekinumab, Stelara
Takeda
Crohn's Disease
06/27
06/27
NCT06095128: A Study of Vedolizumab With Tofacitinib in Adults With Ulcerative Colitis (UC)

Recruiting
4
65
Canada, US
Vedolizumab, Entyvio, MLN0002, Tofacitinib, Xeljanz, CP-690, CP-550
Takeda
Ulcerative Colitis
07/27
07/27
PhALLCON, NCT03589326 / 2018-000397-30: A Study of Ponatinib Versus Imatinib in Adults With Acute Lymphoblastic Leukemia

Checkmark Data from PhALLCON Study for newly diagnosed Ph+ ALL at SOHO 2020
Sep 2019 - Sep 2019: Data from PhALLCON Study for newly diagnosed Ph+ ALL at SOHO 2020
Checkmark Initiation of new trial in Ph+ ALL
Jul 2018 - Jul 2018: Initiation of new trial in Ph+ ALL
Active, not recruiting
3
245
Europe, Canada, Japan, US, RoW
Ponatinib, Iclusig, Imatinib, Gleevec, Vincristine, Dexamethasone, Cytarabine, Methotrexate, Prednisone
Takeda
Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia (Ph+ALL)
08/22
07/27
NCT04840667 / 2018-004689-32: A Study of Replagal in Treatment-naïve Adults With Fabry Disease

Terminated
3
17
Europe, Canada
REPLAGAL, SHP675
Shire
Fabry Disease
12/22
12/22
NCT04444895 / 2019-004823-20: A Study of Long-Term Safety and Efficacy of Lanadelumab for Prevention of Acute Attacks of Non-histaminergic Angioedema With Normal C1-Inhibitor

Completed
3
73
Europe, Canada, Japan, US
Lanadelumab, DX-2930, SHP643, TAK-743
Shire, Takeda Development Center Americas, Inc.
Angioedema
05/23
05/23
NCT03393975 / 2017-000858-18: A Study of BAX 930 in Children, Teenagers, and Adults Born With Thrombotic Thrombocytopenic Purpura (TTP)

Completed
3
51
Europe, Japan, US
BAX930, rADAMTS13, SHP-655, TAK-755, recombinant ADAMTS13, BAX 930, Standard of care
Baxalta now part of Shire, Takeda Development Center Americas, Inc.
Thrombotic Thrombocytopenic Purpura (TTP)
12/23
05/24
NCT03879135 / 2018-003453-16: A Study of Recombinant Von Willebrand Factor (rVWF) in Pediatric and Adult Participants With Severe Von Willebrand Disease (VWD)

Active, not recruiting
3
38
Europe, US, RoW
rVWF, Vonvendi, Vonicog alfa, rFVIII, Octocog alfa, ADVATE
Baxalta now part of Shire, Takeda Development Center Americas, Inc.
Von Willebrand Disease (VWD)
03/25
03/25
SKYWAY, NCT04938427 / 2021-002481-40: A Study of Soticlestat as an Add-on Therapy in Children, Teenagers, and Adults With Lennox-Gastaut Syndrome

Completed
3
270
Europe, Canada, Japan, US, RoW
Placebo, Soticlestat, TAK-935
Takeda
Lennox Gastaut Syndrome (LGS)
01/24
01/24
NCT06165341: Study to Learn About the Safety of Fazirsiran and if it Can Help People With Alpha-1 Antitrypsin Liver Disease With Mild Liver Scarring (Fibrosis)

Recruiting
3
50
Europe, Canada, US
Fazirsiran Injection, TAK-999, ARO-AAT, ADS-001, Placebo
Takeda
Alpha1-Antitrypsin Deficiency
08/28
08/28
NCT02932618 / 2016-001477-33: A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)

Recruiting
3
34
Europe, US, RoW
von Willebrand factor (Recombinant), VONVENDI, rVWF, BAX111, BAX 111, Antihemophilic Factor (Recombinant), ADVATE, Recombinant Factor VIII, rFVIII
Baxalta now part of Shire, Takeda Development Center Americas, Inc.
Von Willebrand Disease
01/25
01/25
SKYLINE, NCT04940624 / 2021-002480-22: A Study of Soticlestat as an Add-on Therapy in Children and Young Adults With Dravet Syndrome

Completed
3
144
Europe, Canada, Japan, US, RoW
Soticlestat, TAK-935, Placebo
Takeda
Dravet Syndrome (DS)
04/24
04/24
TAK-935-3004, NCT06422377: A Study Evaluating Soticlestat in Participants With Dravet Syndrome or Lennox-Gastaut Syndrome Who Have Been Exposed to Fenfluramine

Terminated
3
1
Europe
Soticlestat, TAK-935
Takeda
Dravet Syndrome (DS), Lennox-Gastaut Syndrome (LGS)
08/24
08/24
NCT04974749 / 2022-004246-35: A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease

Completed
3
20
RoW
REPLAGAL, Agalsidase Alfa, TAK-675
Takeda, Takeda Development Center Americas, Inc
Fabry Disease
01/24
01/24
NCT05460325 / 2023-001105-31: A Study of Lanadelumab (SHP643) in Chinese Participants With Hereditary Angioedema (HAE)

Completed
3
20
RoW
Lanadelumab, SHP643, TAKHZYRO, TAK-743, DX-2930, Lanadelumab Injection
Takeda, Takeda
Hereditary Angioedema (HAE)
11/23
11/23
TAK-279-PsO-3003, NCT06550076: A Study of TAK-279 in Participants With Moderate-to-Severe Plaque Psoriasis

Recruiting
3
1300
Europe, Canada, Japan, US, RoW
TAK-279
Takeda
Plaque Psoriasis
05/26
05/26
Vedolizumab-3041, NCT06443502: A Study to Learn About the Safety of Vedolizumab and How Well it Works in Children and Teenagers With Active Chronic Pouchitis

Recruiting
3
30
Europe, RoW
Vedolizumab, Entyvio, MLN0002, Kynteles, Concomitant Antibiotic Therapy
Takeda
Pouchitis
12/29
12/29
NCT06060067 / 2023-000134-15: A Study of Dengue Vaccine in Healthy Children, Teenagers and Adults in India

Active, not recruiting
3
480
RoW
TDV, TAK-003, Placebo
Takeda, Takeda
Healthy Volunteers
05/25
05/25
NCT04779320 / 2020-004301-31: A Study of Vedolizumab in Children and Teenagers With Moderate to Severe Crohn's Disease (CD)

Recruiting
3
120
Europe, Canada, Japan, US, RoW
Vedolizumab IV, ENTYVIO, KYNTELES, MLN0002
Takeda, Takeda Development Center Americas, Inc.
Crohn's Disease (CD)
11/24
11/24
NCT05529992 / 2022-002323-35: A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease

Completed
3
20
RoW
Velaglucerase Alfa, VPRIV
Takeda, Takeda
Gaucher Disease
08/24
08/24
NCT05707351: A Study of Adynovate in Previously Treated Chinese Teenagers and Adults With Severe Hemophilia A

Completed
3
37
RoW
Adynovate, Antihemophilic Factor (recombinant) PEGylated, Rurioctocog Alfa Pegol
Takeda
Hemophilia A
09/24
09/24
VICTRIVA, NCT06227910: A Study of Vedolizumab With and Without Upadacitinib in Adults With Crohn's Disease

Recruiting
3
396
Canada, US
Vedolizumab, Entyvio, MLN0002, KYNTELES, Upadacitinib, Rinvoq, Placebo
Takeda
Crohn's Disease
06/27
08/28
NCT06439342: A Study of Maribavir in Chinese Adults With Cytomegalovirus (CMV) Infections

Not yet recruiting
3
20
RoW
Maribavir, TAK-620
Takeda
Cytomegalovirus (CMV)
02/27
02/27
SWOG S2302, NCT05633602: Ramucirumab Plus Pembrolizumab vs Usual Care for Treatment of Stage IV or Recurrent Non-Small Cell Lung Cancer Following Immunotherapy, Pragmatica-Lung Study

Recruiting
3
700
US
Chemotherapy, Chemo, Chemotherapy (NOS), Chemotherapy, Cancer, General, Pembrolizumab, Keytruda, Lambrolizumab, MK-3475, SCH 900475, Ramucirumab, Anti-VEGFR-2 Fully Human Monoclonal Antibody IMC-1121B, Cyramza, IMC-1121B, LY3009806
SWOG Cancer Research Network, National Cancer Institute (NCI), Eli Lilly and Company, Merck Sharp & Dohme LLC
Recurrent Lung Non-Small Cell Carcinoma, Stage IV Lung Cancer AJCC v8
03/25
03/28
NCT04701411 / 2020-003193-48: A Study of Darvadstrocel for Treating Complex Perianal Fistulas in Children and Teenagers With Crohn's Disease

Recruiting
3
20
Europe, Japan, RoW
Darvadstrocel, Cx601
Takeda, Takeda Development Center Americas, Inc.
Crohn's Disease, Complex Perianal Fistula
06/25
12/25
ACTION-1, NCT05477576 / 2022-000507-12: Study of RYZ101 Compared with SOC in Pts W Inoperable SSTR+ Well-differentiated GEP-NET That Has Progressed Following 177Lu-SSA Therapy

Recruiting
3
288
Europe, Canada, US, RoW
RYZ101, Everolimus, Sunitinib, Octreotide, Lanreotide
RayzeBio, Inc., RayzeBio, Inc.
GEP-NET, Gastroenteropancreatic Neuroendocrine Tumor, Gastroenteropancreatic Neuroendocrine Tumor Disease, Neuroendocrine Tumors, Carcinoid, Carcinoid Tumor, Pancreatic NET
12/25
07/28
TAK-279-3002, NCT06108544: A Study About How Well TAK-279 Works and Its Safety in Participants With Moderate-to-severe Plaque Psoriasis During 60 Weeks of Treatment With a Withdrawal and Retreatment Period

Active, not recruiting
3
1108
Europe, Canada, US, RoW
TAK-279, Placebo, Apremilast
Takeda
Plaque Psoriasis
12/24
11/25
NCT06088043: A Study About How Well TAK-279 Works and Its Safety in Participants With Moderate-to-severe Plaque Psoriasis During 52 Weeks of Treatment

Active, not recruiting
3
693
Europe, Canada, Japan, US, RoW
TAK-279, Placebo, Apremilast
Takeda
Plaque Psoriasis
08/25
04/26
MLN0002-3024, NCT04779307 / 2020-004300-34: A Study of Vedolizumab in Children and Teenagers with Moderate to Severe Ulcerative Colitis (UC)

Active, not recruiting
3
121
Europe, Canada, Japan, US, RoW
Vedolizumab, MLN0002, ENTYVIO, KYNTELES
Takeda, Takeda Development Center Americas, Inc.
Colitis, Ulcerative
08/25
08/25
NCT04759833 / 2022-003221-22: A Study of Prucalopride For Functional Constipation in Children and Teenagers

Terminated
3
175
US
Prucalopride, TAK-555, Prucalopride succinate, Placebo
Takeda, Takeda Development Center Americas, Inc.
Constipation
11/23
11/23
NCT05156983 / 2021-004138-12: A Study of TAK-330 to Reverse the Effects of Factor Xa Inhibitors For Adults Needing Urgent Surgery

Recruiting
3
328
Europe, Canada, US
TAK-330, SOC 4F-PCC
Takeda, Takeda Development Center Americas, Inc.
Coagulation Disorder
04/28
04/28
ENDYMION 2, NCT05163314 / 2021-002482-17: A Study of Soticlestat as an Add-on Therapy in Children and Adults With Dravet Syndrome or Lennox-Gastaut Syndrome

Recruiting
3
400
Europe, Canada, Japan, US, RoW
Soticlestat, TAK-935
Takeda
Dravet Syndrome (DS), Lennox Gastaut Syndrome (LGS)
05/26
05/26
NCT04683003: A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura

Recruiting
3
77
Europe, Japan, US, RoW
TAK-755, rADAMTS13; recombinant ADAMTS13; SHP-655; BAX 930
Takeda, Takeda Development Center Americas, Inc., Shire
Thrombotic Thrombocytopenic Purpura (TTP)
03/27
03/27
TAK-620-2004, NCT05319353 / 2021-004279-15: A Study to Evaluate the Safety and Tolerability, Pharmacokinetics, and Antiviral Activity of Maribavir for the Treatment of Cytomegalovirus (CMV) Infection in Children and Adolescents Who Have Received a Hematopoietic Stem Cell Transplant (HSCT) or a Solid Organ Transplant (SOT)

Active, not recruiting
3
80
Europe, Japan, US, RoW
Maribavir, TAK-620
Takeda, Takeda Development Center Americas, Inc.
Cytomegalovirus (CMV)
01/27
01/27
NCT04969731: Safety and Efficacy of Immuncell-LC With Gemcitabine in Resectable Pancreatic Cancer

Recruiting
3
408
RoW
Immuncell-LC, Autologous activated T lymphocyte, Gemcitabine
GC Cell Corporation
Pancreatic Ductal Adenocarcinoma
12/26
06/27
PACES, NCT01349881: S0820, Adenoma and Second Primary Prevention Trial

Active, not recruiting
3
354
US
Eflornithine placebo & sulindac placebo, eflornithine & sulindac placebo, Eflornithine placebo & sulindac, Eflornithine plus sulindac
SWOG Cancer Research Network, National Cancer Institute (NCI), Cancer Prevention Pharmaceuticals, Inc.
Colorectal Neoplasms
02/27
02/32
NCT05582993 / 2020-003304-13: A Study of Recombinant Von Willebrand Factor (rVWF) (TAK-577) in Children With Severe Von Willebrand Disease (vWD)

Recruiting
3
24
Europe, US
Recombinant von Willebrand Factor (rVWF), TAK-577, Vonicog Alfa, ADVATE, Recombinant Factor VIII (rFVIII), Octocog Alfa
Takeda, Takeda Development Center Americas, Inc.
Von Willebrand Disease (VWD)
04/30
04/30
NCT05677971: Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein

Recruiting
3
160
Europe, Canada, US, RoW
Fazirsiran Injection, TAK-999, ARO-AAT, ADS-001, Placebo, Sodium chloride
Takeda, Takeda Development Center Americas, Inc.
Alpha1-Antitrypsin Deficiency
03/27
03/29
TAK-881-3002, NCT06076642: A Study About the Long-Term Safety of TAK-881 in People With Primary Immunodeficiency Diseases

Recruiting
3
39
US
TAK-881, Immune Globulin Subcutaneous (Human), 20% Solution with Recombinant Human Hyaluronidase (rHuPH20)., SC Investigational Needle Sets
Takeda, Takeda Development Center Americas, Inc., Baxalta Innovations GmbH, now part of Takeda
Primary Immunodeficiency Diseases (PID)
01/29
01/29
CompassHER2 RD, NCT04457596: T-DM1 and Tucatinib Compared with T-DM1 Alone in Preventing Relapses in People with High Risk HER2-Positive Breast Cancer, the Trial

Calendar Jan 2028 - Dec 2028: From CompassHER2 RD trial in combination with Kadcyla for HER2+ breast cancer
Recruiting
3
1031
Canada, US
Trastuzumab Emtansine, Placebo Administration, Tucatinib, Questionnaire Administration, Quality-of-Life Assessment
Alliance for Clinical Trials in Oncology, National Cancer Institute (NCI), Seagen Inc.
Anatomic Stage IA Breast Cancer AJCC V8, Anatomic Stage II Breast Cancer AJCC V8, Anatomic Stage IIA Breast Cancer AJCC V8, Anatomic Stage IIB Breast Cancer AJCC V8, Anatomic Stage III Breast Cancer AJCC V8, Anatomic Stage IIIA Breast Cancer AJCC V8, Anatomic Stage IIIB Breast Cancer AJCC V8, Anatomic Stage IIIC Breast Cancer AJCC V8, HER2 Positive Breast Carcinoma, Invasive Breast Carcinoma, Multifocal Breast Carcinoma, Prognostic Stage I Breast Cancer AJCC V8, Prognostic Stage IA Breast Cancer AJCC V8, Prognostic Stage IB Breast Cancer AJCC V8, Prognostic Stage II Breast Cancer AJCC V8, Prognostic Stage IIA Breast Cancer AJCC V8, Prognostic Stage IIB Breast Cancer AJCC V8, Prognostic Stage III Breast Cancer AJCC V8, Prognostic Stage IIIA Breast Cancer AJCC V8, Prognostic Stage IIIB Breast Cancer AJCC V8, Prognostic Stage IIIC Breast Cancer AJCC V8, Synchronous Bilateral Breast Carcinoma
01/28
05/35
NCT03221036: Efficacy and Safety of Vedolizumab IV in Chinese Participants With Ulcerative Colitis

Recruiting
3
402
RoW
Vedolizumab IV, Placebo
Takeda
Moderately to Severely Active Ulcerative Colitis
05/28
07/28
DRAMMATIC, NCT04071457: S1803, Lenalidomide +/- Daratumumab/rHuPh20 as Post-ASCT Maintenance for MM w/MRD to Direct Therapy Duration

Hourglass Jan 2023 - Jun 2023 : From trial for patients with r/r multiple myeloma treated with an IMiD and PI
Recruiting
3
1100
Canada, US
Lenalidomide, CC-5013, Revlimid, Daratumumab/rHuPH20
SWOG Cancer Research Network, National Cancer Institute (NCI), Janssen, LP
Multiple Myeloma
07/29
07/40
NCT05837897: A Study of Vedolizumab in Adult Participants With Moderate to Severe Crohn's Disease

Recruiting
3
408
RoW
Vedolizumab IV, Placebo
Takeda
Crohn's Disease
03/30
05/31
NCT05442567 / 2021-000630-34: A Study of Vedolizumab in Children With Ulcerative Colitis (UC) or Crohn's Disease (CD)

Recruiting
3
240
Europe, Canada, Japan, US, RoW
Vedolizumab IV, MLN0002, ENTYVIO, KYNTELES, No Intervention
Takeda, Takeda Development Center Americas, Inc.
Ulcerative Colitis, Crohn's Disease
08/31
08/31
NCT05755035: A Study About How TAK-881 is Processed by the Body and Side Effects in People With Primary Immunodeficiency Diseases

Recruiting
2/3
75
Europe, US, RoW
TAK-881, Immune Globulin Subcutaneous (Human), 20% Solution with Recombinant Human Hyaluronidase (rHuPH20)., HYQVIA, Immune Globulin Infusion (Human), 10% Solution with rHuPH20.
Takeda, Takeda Development Center Americas, Inc.
Primary Immunodeficiency Diseases (PID)
02/26
09/26
CIRCULATE-US, NCT05174169: Colon Adjuvant Chemotherapy Based on Evaluation of Residual Disease

Recruiting
2/3
1912
Canada, US
Signatera test, mFOLFOX6 3-6 month, CAPOX 3 month, mFOLFIRINOX, mFOLFOX6 6 month, CAPOX 6 month
NRG Oncology, Natera, Inc., National Cancer Institute (NCI)
Stage III Colon Cancer
03/29
03/30
NCT06031259: Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment

Recruiting
2/3
8
Europe, Canada
Idursulfase-IT, Elaprase
Takeda
Hunter Syndrome
01/34
06/34
NCT05000216: COVID-19 Booster Vaccine in Autoimmune Disease Non-Responders

Completed
2
257
US
Moderna mRNA-1273, mRNA-1273 vaccine (Moderna), Moderna COVID-19 Vaccine, SARS-CoV-2 RNA vaccine, COVID-19 vaccine, BNT162b2, mRNA-1273 vaccine (Pfizer/BioNTech), Pfizer-BioNTech COVID-19 vaccine, Ad26.COV2.S, Janssen COVID-19 Vaccine, JNJ-78436735, Continue IS (MMF or MPA), immunosuppressive medication, mycophenolate mofetil, MMF, CellCept®, mycophenolic acid, MPA, Continue IS (MTX), methotrexate, MTX, Continue IS (B cell depletion therapy), mAbs targeting CD19 or CD20, anti-BAFF mAb, Monovalent [B.1.351] CoV2 preS dTM-AS03, Sanofi-GSK COVID-19 Vaccine, Withhold IS (MMF or MPA), Withhold IS (MTX), Withhold IS (B cell depletion therapy), Moderna mRNA-1273, Bivalent, mRNA-1273 vaccine (Moderna), Bivalent, Moderna COVID-19 Vaccine, Bivalent, SARS-CoV-2 RNA vaccine, Bivalent, COVID-19 vaccine, Bivalent, BNT162b2, Bivalent, mRNA-1273 vaccine (Pfizer/BioNTech), Bivalent, Pfizer-BioNTech COVID-19 vaccine, Bivalent
National Institute of Allergy and Infectious Diseases (NIAID), Autoimmunity Centers of Excellence, Rho Federal Systems Division, Inc.
Rheumatoid Arthritis (RA), Systemic Lupus Erythematosus (SLE), Pemphigus Vulgaris, Multiple Sclerosis (MS), Systemic Sclerosis (SSc), Pediatric SLE, Juvenile Idiopathic Arthritis (JIA), Juvenile Dermatomyositis (JDM), Pediatric-Onset Multiple Sclerosis (POMS)
06/23
03/24
NCT04530123: Dose-Ranging Study of the Efficacy and Safety of TAK-101 for Prevention of Gluten-Specific T Cell Activation in Participants With Celiac Disease on a Gluten-Free Diet

Recruiting
2
90
Canada, US, RoW
Placebo, TAK-101, Gluten
Takeda
Celiac Disease
10/25
10/25
ALTITUDE-AD, NCT06335173: A Study to Evaluate Efficacy and Safety of Intravenous Sabirnetug in Participants with Early Alzheimer's Disease

Active, not recruiting
2
540
Europe, Canada, US
sabirnetug, Placebo
Acumen Pharmaceuticals
Alzheimer Disease
10/26
10/26
NCT06233461: A Study on the Safety of TAK-279 and Whether it Can Reduce Inflammation in the Bowel of Participants With Moderately to Severely Active Crohn's Disease

Recruiting
2
268
Europe, Canada, Japan, US, RoW
TAK-279, Zasocitinib, Placebo
Takeda
Crohn's Disease
09/26
07/27
 

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