Lucille Packard Children's Hospital, Stanford University
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 4 Trials 
10 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Agarwal-Hashmi, Rajni
NCT02963064: JSP191 Antibody Targeting Conditioning in SCID Patients

Terminated
1/2
23
US
Humanized anti-CD117 Monoclonal Antibody (JSP191)
Jasper Therapeutics, Inc.
SCID
07/25
07/25
FusuciA, NCT04522375: A Dose Escalation Study of FP-045 in Patients With Fanconi Anemia

Withdrawn
1/2
16
NA
FP-045
Foresee Pharmaceuticals Co., Ltd.
Fanconi Anemia
06/25
12/25
Porter, Brenda
NCT05219617 / 2022-001256-42: Investigate Efficacy and Safety of Carisbamate as Adjunctive Treatment for Seizures Associated With LGS in Children and Adults

Recruiting
3
252
Europe, US, RoW
Carisbamate
SK Life Science, Inc., SK Life Science, Inc
Seizures, Lennox Gastaut Syndrome
02/27
12/28
TSC-STEPS, NCT05104983: Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study

Recruiting
2
64
US
Sirolimus, Placebo
Darcy Krueger
Tuberous Sclerosis Complex, Epilepsy
06/25
06/26
NCT06144957: SLC13A5 Deficiency Natural History Study - United States Only

Enrolling by invitation
N/A
17
US
TESS Research Foundation, Stanford University, Brown University, University of Texas Southwestern Medical Center
Citrate Transporter Deficiency, Epilepsy, Rare Diseases, Movement Disorders, Genetic Disorder, SLC13A5 Deficiency, EIEE25, Kohlschutter-Tonz Syndrome (Non-ROGDI), Citrate Transporter Disorder, DEE25
09/25
09/25
NCT04681781: SLC13A5 Deficiency Natural History Study - Remote Only

Enrolling by invitation
N/A
20
US
TESS Research Foundation, Stanford University
Citrate Transporter Deficiency, Epilepsy, Rare Diseases, Movement Disorders, Genetic Disorder, SLC13A5 Deficiency, EIEE25, Kohlschutter-Tonz Syndrome (non-ROGDI), 17p13.1 Deletions Confined to SLC13A5 Gene, Citrate Transporter Disorder
12/23
09/25
NCT02461459: Autism Spectrum Disorder (ASD) and Intellectual Disability (ID) Determinants in Tuberous Sclerosis Complex (TSC)

Active, not recruiting
N/A
205
US
Boston Children's Hospital, National Institutes of Health (NIH), National Institute of Neurological Disorders and Stroke (NINDS), Tuberous Sclerosis Alliance, National Center for Advancing Translational Sciences (NCATS), Office of Rare Diseases (ORD), Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Tuberous Sclerosis, Autism Disorder, Intellectual Disability
12/25
12/26
Chromik, Lindsay
NCT04681781: SLC13A5 Deficiency Natural History Study - Remote Only

Enrolling by invitation
N/A
20
US
TESS Research Foundation, Stanford University
Citrate Transporter Deficiency, Epilepsy, Rare Diseases, Movement Disorders, Genetic Disorder, SLC13A5 Deficiency, EIEE25, Kohlschutter-Tonz Syndrome (non-ROGDI), 17p13.1 Deletions Confined to SLC13A5 Gene, Citrate Transporter Disorder
12/23
09/25
Solidum, Rayann
MEND-PNPO, NCT04706013: Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency

Recruiting
3
15
US, RoW
Pyridoxal Phosphate
Medicure
Pyridox(am)Ine 5'-Phosphate Oxidase Deficiency
11/26
11/26
NCT06144957: SLC13A5 Deficiency Natural History Study - United States Only

Enrolling by invitation
N/A
17
US
TESS Research Foundation, Stanford University, Brown University, University of Texas Southwestern Medical Center
Citrate Transporter Deficiency, Epilepsy, Rare Diseases, Movement Disorders, Genetic Disorder, SLC13A5 Deficiency, EIEE25, Kohlschutter-Tonz Syndrome (Non-ROGDI), Citrate Transporter Disorder, DEE25
09/25
09/25
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Agarwal-Hashmi, Rajni
NCT02963064: JSP191 Antibody Targeting Conditioning in SCID Patients

Terminated
1/2
23
US
Humanized anti-CD117 Monoclonal Antibody (JSP191)
Jasper Therapeutics, Inc.
SCID
07/25
07/25
FusuciA, NCT04522375: A Dose Escalation Study of FP-045 in Patients With Fanconi Anemia

Withdrawn
1/2
16
NA
FP-045
Foresee Pharmaceuticals Co., Ltd.
Fanconi Anemia
06/25
12/25
Porter, Brenda
NCT05219617 / 2022-001256-42: Investigate Efficacy and Safety of Carisbamate as Adjunctive Treatment for Seizures Associated With LGS in Children and Adults

Recruiting
3
252
Europe, US, RoW
Carisbamate
SK Life Science, Inc., SK Life Science, Inc
Seizures, Lennox Gastaut Syndrome
02/27
12/28
TSC-STEPS, NCT05104983: Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study

Recruiting
2
64
US
Sirolimus, Placebo
Darcy Krueger
Tuberous Sclerosis Complex, Epilepsy
06/25
06/26
NCT06144957: SLC13A5 Deficiency Natural History Study - United States Only

Enrolling by invitation
N/A
17
US
TESS Research Foundation, Stanford University, Brown University, University of Texas Southwestern Medical Center
Citrate Transporter Deficiency, Epilepsy, Rare Diseases, Movement Disorders, Genetic Disorder, SLC13A5 Deficiency, EIEE25, Kohlschutter-Tonz Syndrome (Non-ROGDI), Citrate Transporter Disorder, DEE25
09/25
09/25
NCT04681781: SLC13A5 Deficiency Natural History Study - Remote Only

Enrolling by invitation
N/A
20
US
TESS Research Foundation, Stanford University
Citrate Transporter Deficiency, Epilepsy, Rare Diseases, Movement Disorders, Genetic Disorder, SLC13A5 Deficiency, EIEE25, Kohlschutter-Tonz Syndrome (non-ROGDI), 17p13.1 Deletions Confined to SLC13A5 Gene, Citrate Transporter Disorder
12/23
09/25
NCT02461459: Autism Spectrum Disorder (ASD) and Intellectual Disability (ID) Determinants in Tuberous Sclerosis Complex (TSC)

Active, not recruiting
N/A
205
US
Boston Children's Hospital, National Institutes of Health (NIH), National Institute of Neurological Disorders and Stroke (NINDS), Tuberous Sclerosis Alliance, National Center for Advancing Translational Sciences (NCATS), Office of Rare Diseases (ORD), Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Tuberous Sclerosis, Autism Disorder, Intellectual Disability
12/25
12/26
Chromik, Lindsay
NCT04681781: SLC13A5 Deficiency Natural History Study - Remote Only

Enrolling by invitation
N/A
20
US
TESS Research Foundation, Stanford University
Citrate Transporter Deficiency, Epilepsy, Rare Diseases, Movement Disorders, Genetic Disorder, SLC13A5 Deficiency, EIEE25, Kohlschutter-Tonz Syndrome (non-ROGDI), 17p13.1 Deletions Confined to SLC13A5 Gene, Citrate Transporter Disorder
12/23
09/25
Solidum, Rayann
MEND-PNPO, NCT04706013: Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency

Recruiting
3
15
US, RoW
Pyridoxal Phosphate
Medicure
Pyridox(am)Ine 5'-Phosphate Oxidase Deficiency
11/26
11/26
NCT06144957: SLC13A5 Deficiency Natural History Study - United States Only

Enrolling by invitation
N/A
17
US
TESS Research Foundation, Stanford University, Brown University, University of Texas Southwestern Medical Center
Citrate Transporter Deficiency, Epilepsy, Rare Diseases, Movement Disorders, Genetic Disorder, SLC13A5 Deficiency, EIEE25, Kohlschutter-Tonz Syndrome (Non-ROGDI), Citrate Transporter Disorder, DEE25
09/25
09/25

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