University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh
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 15 Trials 
23 Trials

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Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Davis, Peter
NCT05546359 / 2022-002947-23: Study of Intravenous Amisulpride for Prophylaxis of Post-operative Nausea and Vomiting (PONV) in Pediatric Patients

Recruiting
2/3
410
Europe, Canada, US
Amisulpride Injection, Barhemsys, Dexamethasone, Decadron
Acacia Pharma Ltd, Amicus CD LLC, Premier Research Group plc
Nausea and Vomiting, Postoperative
06/25
12/25
Vockley, Gerard
NCT03335488 / 2015-000075-27: Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)

Completed
4
16
Europe, US
RAVICTI, Glycerol phenylbutyrate, GPB, HPN-100, NaPBA, Sodium phenylbutyrate
Amgen
Urea Cycle Disorder
07/22
12/22
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
SYNPHENY-3, NCT05764239: Efficacy and Safety of SYNB1934 in Patients With PKU

Terminated
3
35
Canada, US, RoW
SYNB1934v1, Placebo
Synlogic
Phenylketonuria
03/24
03/24
NCT05166161 / 2021-000497-28: A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria

Recruiting
3
200
Europe, Canada, Japan, US, RoW
PTC923, Sepiapterin
PTC Therapeutics
Phenylketonuria
04/26
04/26
STRIDE, NCT04535609 / 2020-002855-40: An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients

Completed
2
213
Europe, Canada, US, RoW
Mavodelpar, REN001, Placebo
Reneo Pharma Ltd
Primary Mitochondrial Myopathy
09/23
10/23
NCT06069375: Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Recruiting
2
24
US
Sodium phenylbutyrate, Olpruva
Jerry Vockley, MD, PhD, Acer Therapeutics Inc.
Medium-chain Acyl-CoA Dehydrogenase Deficiency
06/25
12/25
NCT06067802: Study of Triheptanoin for the Prevention of Hypoglycemia in Patients with Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Recruiting
2
8
US
Triheptanoin, Dojolvi
Jerry Vockley, MD, PhD, Ultragenyx Pharmaceutical Inc
Medium-chain Acyl-CoA Dehydrogenase Deficiency
03/26
07/26
NCT06773026: Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients with Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Recruiting
2
24
US
Sodium phenylbutyrate, Olpruva
Jerry Vockley, MD, PhD, Zevra Therapeutics
Medium-chain Acyl-CoA Dehydrogenase Deficiency
04/26
07/26
NCT05222178: Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency

Terminated
1
3
US
HMI-103
Homology Medicines, Inc
Phenylketonurias, PAH Deficiency, Phenylketonuria
09/23
09/23
NCT02968953: Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome

No Longer Available
N/A
US
Triheptanoin, UX007
Jerry Vockley, MD, PhD, Ultragenyx Pharmaceutical Inc
Glucose Transporter 1 Deficiency Syndrome
 
 
NCT05998109: PheCheck Feasibility Study

Recruiting
N/A
30
US
PheCheck™, HPLC Amino Acid Analyzer, Dried Blood Spot Cards
Lumos Diagnostics, Aptatek Biosciences
Phenylketonurias
01/24
03/24
NCT02927158: Exome and Genome Analysis to Elucidate Genetic Etiologies and Population Characteristics in the Plain Community

Recruiting
N/A
300
US
University of Pittsburgh, Horizon Pharma USA, Inc.
Undiagnosed Disease
08/26
08/30
Baker, Jennifer
pheNIX, NCT03952156: Gene Therapy Clinical Study in Adult PKU

Terminated
1/2
10
US
HMI-102
Homology Medicines, Inc
Phenylketonurias, PAH Deficiency
01/23
08/23
NCT05998109: PheCheck Feasibility Study

Recruiting
N/A
30
US
PheCheck™, HPLC Amino Acid Analyzer, Dried Blood Spot Cards
Lumos Diagnostics, Aptatek Biosciences
Phenylketonurias
01/24
03/24
NCT02927158: Exome and Genome Analysis to Elucidate Genetic Etiologies and Population Characteristics in the Plain Community

Recruiting
N/A
300
US
University of Pittsburgh, Horizon Pharma USA, Inc.
Undiagnosed Disease
08/26
08/30
Bukowinski, Andrew
AOST-2121, NCT04974008: Osteosarcoma Maintenance Therapy With OST31-164

Completed
2
39
US
OST31-164
George Clinical Pty Ltd
Bone Cancer
09/23
09/23
OPTIMUM, NCT03561259: A Study of Therapeutic Iobenguane (131-I) and Vorinostat for Recurrent or Progressive High-Risk Neuroblastoma Subjects

Recruiting
2
60
US
131I-MIBG, I-131 meta-iodobenzylguanidine, Iobenguane I-131 MIBG Injection, 131-MIBG + Vorinostat
Jubilant DraxImage Inc.
Neuroblastoma, Neuroectodermal Tumors, Neoplasms
12/23
04/25
CAMPFIRE, NCT05999994: A Master Protocol (LY900023) That Includes Several Clinical Trials of Drugs for Children and Young Adults With Cancer

Recruiting
2
105
Europe, Japan, US, RoW
Ramucirumab, LY3009806, Cyclophosphamide, Vinorelbine, Gemcitabine, LY188011, Docetaxel, Abemaciclib, LY2835219, Irinotecan, Temozolomide
Eli Lilly and Company
Neoplasms, Child, Adolescent
05/27
05/27
NCT05093322 / 2021-003602-41: A Study of Surufatinib in Combination With Gemcitabine in Pediatric, Adolescent, and Young Adult Patients With Recurrent or Refractory Solid Tumors

Completed
1/2
13
US
Surufatinib in combination with Gemcitabine, HMPL-012, sulfatinib in combination with Gemcitabine
Hutchmed, HUTCHMED Limited
Solid Tumor, Lymphoma, Osteosarcoma, Ewing Sarcoma, Rhabdomyosarcoma, Non-rhabdomyosarcoma Soft Tissue Sarcoma (NRSTS)
04/23
04/23
AAML1921, NCT04258943 / 2015-002916-34: Bosutinib in Pediatric Patients With Newly Diagnosed Chronic Phase or Resistant/Intolerant Ph + Chronic Myeloid Leukemia

Active, not recruiting
1/2
60
US
Bosutinib
Children's Oncology Group, Erasmus Medical Center, Dutch Childhood Oncology Group, Innovative Therapies for Children with Cancer, Pfizer
Philadelphia Chromosome Positive CML, Accelerated Phase Chronic Myelogenous Leukemia, Blastic Phase Chronic Myelogenous Leukemia, Chronic Phase Chronic Myelogenous Leukemia
07/28
07/28
NCT06247787: A Study to Find the Highest Dose of Imetelstat in Combination With Fludarabine and Cytarabine for Patients With AML, MDS or JMML That Has Come Back or Does Not Respond to Therapy

Recruiting
1
36
US
Biospecimen Collection, Biological Sample Collection, Biospecimen Collected, Specimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Biopsy of Bone Marrow, Biopsy, Bone Marrow, Cytarabine, .beta.-Cytosine arabinoside, 1-.beta.-D-Arabinofuranosyl-4-amino-2(1H)pyrimidinone, 1-.beta.-D-Arabinofuranosylcytosine, 1-Beta-D-arabinofuranosyl-4-amino-2(1H)pyrimidinone, 1-Beta-D-arabinofuranosylcytosine, 1.beta.-D-Arabinofuranosylcytosine, 2(1H)-Pyrimidinone, 4-Amino-1-beta-D-arabinofuranosyl-, 2(1H)-Pyrimidinone, 4-amino-1.beta.-D-arabinofuranosyl-, Alexan, Ara-C, ARA-cell, Arabine, Arabinofuranosylcytosine, Arabinosylcytosine, Aracytidine, Aracytin, Aracytine, Beta-Cytosine Arabinoside, CHX-3311, Cytarabinum, Cytarbel, Cytosar, Cytosine Arabinoside, Cytosine-.beta.-arabinoside, Cytosine-beta-arabinoside, Erpalfa, Starasid, Tarabine PFS, U 19920, U-19920, Udicil, WR-28453, Echocardiography, EC, Fludarabine, Fluradosa, Hydrocortisone Sodium Succinate, (11beta)-21-(3-Carboxy-1-oxopropyl)-11,17-dihydroxypregn-4-ene-3,20-dione, Monosodium Salt, A-Hydrocort, Buccalsone, Corlan, Cortisol Sodium Succinate, Cortop, Efcortelan, Emergent-EZ, Flebocortid, Hidroc Clora, Hycorace, Hydro-Adreson, Hydrocort, Hydrocortisone 21-Sodium Succinate, Hydrocortisone Na Succinate, Kinogen, Nordicort, Nositrol, Sinsurrene, Sodium hydrocortisone succinate, Solu-Cortef, Solu-Glyc, Imetelstat, GRN 163L, GRN-163L, Leucovorin Calcium, Adinepar, Calcifolin, Calcium (6S)-Folinate, Calcium Folinate, Calcium Leucovorin, Calfolex, Calinat, Cehafolin, Citofolin, Citrec, Citrovorum Factor, Cromatonbic Folinico, Dalisol, Disintox, Divical, Ecofol, Emovis, Factor, Citrovorum, Flynoken A, Folaren, Folaxin, FOLI-cell, Foliben, Folidan, Folidar, Folinac, Folinate Calcium, folinic acid, Folinic Acid Calcium Salt Pentahydrate, Folinoral, Folinvit, Foliplus, Folix, Imo, Lederfolat, Lederfolin, Leucosar, leucovorin, Rescufolin, Rescuvolin, Tonofolin, Wellcovorin, Lumbar Puncture, LP, Spinal Tap, Methotrexate, Abitrexate, Alpha-Methopterin, Amethopterin, Brimexate, CL 14377, CL-14377, Emtexate, Emthexat, Emthexate, Farmitrexat, Fauldexato, Folex, Folex PFS, Lantarel, Ledertrexate, Lumexon, Maxtrex, Medsatrexate, Metex, Methoblastin, Methotrexate LPF, Methotrexate Methylaminopterin, Methotrexatum, Metotrexato, Metrotex, Mexate, Mexate-AQ, MTX, Novatrex, Rheumatrex, Texate, Tremetex, Trexeron, Trixilem, WR-19039
Children's Oncology Group, Geron Corporation, National Cancer Institute (NCI)
Recurrent Childhood Acute Myeloid Leukemia, Recurrent Childhood Myelodysplastic Syndrome, Recurrent Juvenile Myelomonocytic Leukemia, Refractory Childhood Acute Myeloid Leukemia, Refractory Childhood Myelodysplastic Syndrome, Refractory Juvenile Myelomonocytic Leukemia
06/26
06/26
McCraken, Elizabeth
HERO, NCT04732429 / 2023-000785-33: Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia

Terminated
2
26
US, RoW
HST5040, Placebo
HemoShear Therapeutics, HemoShear Therapeutics
Methylmalonic Acidemia, Propionic Acidemia
10/23
10/23

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