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Scholars @ Weill Cornell Medicine

Help & supportPhase 1 prototype

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Scholars8,803 Publications201,993 Funding7,268 Clinical research6

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Scholars8,803 Publications201,993 Funding7,268 Clinical research6

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Status

Recruiting2
Active, not recruiting1
Completed3

Study type

Interventional6
Observational1

Phase

Phase 1/23
Phase 21
Phase 2/31
Not applicable1

Investigator

Harini Sarva2
Alana Nguyen1
Catherine McGuinn1
Melissa Frey1
Roger N Pearse1

Department

Medicine2
Neurology2
Obstetrics and Gynecology1
Pediatrics1

Condition

Neoplasms2
Parkinson Disease2
Blood Coagulation Disorders1
Blood Coagulation Disorders, Inherited1
Coagulation Protein Disorders1

Intervention type

Genetic6
Drug788
Other176
Device166
Biological150
Procedure119

Sponsor type

Industry5
WCM (investigator-initiated)1

Start date

Has results posted3

Interventional×Genetic×Clear all

Showing 1–6 of 6 studiesSort:

RecruitingInterventional · Phase 2/3NCT03435796 ↗
Long-Term Follow-up Protocol for Subjects Treated With Gene-Modified T Cells

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Status

Recruiting2
Active, not recruiting1
Completed3

Study type

Interventional6
Observational1

Phase

Phase 1/23
Phase 21
Phase 2/31
Not applicable1

Investigator

Harini Sarva2
Alana Nguyen1
Catherine McGuinn1
Melissa Frey1
Roger N Pearse1

Department

Medicine2
Neurology2
Obstetrics and Gynecology1
Pediatrics1

Condition

Neoplasms2
Parkinson Disease2
Blood Coagulation Disorders1
Blood Coagulation Disorders, Inherited1
Coagulation Protein Disorders1

Intervention type

Genetic6
Drug788
Other176
Device166
Biological150
Procedure119

Sponsor type

Industry5
WCM (investigator-initiated)1

Start date

Has results posted3

Interventional×Genetic×Clear all

Showing 1–6 of 6 studiesSort:

RecruitingInterventional · Phase 2/3NCT03435796 ↗
Long-Term Follow-up Protocol for Subjects Treated With Gene-Modified T Cells

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Genetic Diseases, Inborn1
Genetic Diseases, X-Linked1
Hematologic Diseases1
Hemophilia A1
Hemorrhagic Disorders1
Neoplasm Metastasis1
Neoplastic Syndromes, Hereditary1

Combination product11

Roger N Pearse

Intervention: Genetic: Gene-modified (GM) T cell therapy
Dates: Jul 2018 – May 2043 (est.)
Sponsor: Celgene Corporation

Neoplasms

Active, not recruitingInterventional · Phase 1/2NCT05894343 ↗

Long-term Follow-up of Glutamic Acid Decarboxylase Gene Transfer to the Subthalamic Nuclei in Participants With Parkinson's Disease

Harini SarvaPI

Intervention: Genetic: AAV-GAD
Dates: May 2023 – Oct 2029 (est.)
Sponsor: MeiraGTx, LLC

Parkinson's Disease

RecruitingInterventional · Not applicableNCT07052266 ↗

Feasibility of Obstetric and Cancer Universal Screening

Melissa FreyPI

Intervention: Genetic: MyRisk Hereditary Cancer Test
Dates: Sep 2025 – Jun 2027 (est.)
Sponsor: Weill Medical College of Cornell University

Hereditary Cancer Syndromes

CompletedInterventional · Phase 1/2NCT03734588 ↗

Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors

Catherine McGuinnPI

Intervention: Genetic: SPK-8016
Dates: Jan 2019 – Oct 2020· Results posted
Sponsor: Spark Therapeutics

Adeno-Associated Virus (AAV)Blood Coagulation DisorderBlood Coagulation Disorders, Inherited+14 more

CompletedInterventional · Phase 1/2NCT05603312 ↗

A Randomized, Double-Blind, Sham-Controlled Study to Evaluate the Safety and Tolerability of Glutamic Acid Decarboxylase Gene Transfer to the Subthalamic Nuclei in Participants With Parkinson's Disease

Harini SarvaPI

Intervention: Genetic: AAV-GAD Low Dose · Genetic: AAV-GAD High Dose · Procedure: Sham Surgery
Dates: Oct 2022 – Sep 2024· Results posted
Sponsor: MeiraGTx, LLC

Parkinson's Disease

CompletedInterventional · Phase 2NCT05673811 ↗

A Phase IIb, Open-label, Randomized Study of Nab-Paclitaxel and Gemcitabine and Plus/Minus VCN-01 in Patients With Metastatic Pancreatic Cancer

Alana NguyenPI

Intervention: Drug: Nab-paclitaxel · Drug: Gemcitabine · Genetic: VCN-01
Dates: Jan 2023 – Mar 2025· Results posted
Sponsor: Theriva Biologics, Inc.

Pancreatic AdenocarcinomaMetastatic

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Long-Term Follow-up Protocol for Subjects Treated With Gene-Modified T Cells

Long-Term Follow-up Protocol for Subjects Treated With Gene-Modified T Cells

Long-term Follow-up of Glutamic Acid Decarboxylase Gene Transfer to the Subthalamic Nuclei in Participants With Parkinson's Disease

Feasibility of Obstetric and Cancer Universal Screening

Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors

A Randomized, Double-Blind, Sham-Controlled Study to Evaluate the Safety and Tolerability of Glutamic Acid Decarboxylase Gene Transfer to the Subthalamic Nuclei in Participants With Parkinson's Disease

A Phase IIb, Open-label, Randomized Study of Nab-Paclitaxel and Gemcitabine and Plus/Minus VCN-01 in Patients With Metastatic Pancreatic Cancer