Reconstitution of a Human Immune System in a Patient Derived Xenograft (PDX) Model of Genitourinary (GU) Cancers (Immune PDX)

• ClinicalTrials.gov Identifier: NCT03134027
• Recruitment Status: Suspended
• First Posted: April 28, 2017
• Last Update Posted: December 21, 2020
• Sponsor: Duke University
• Information provided by (Responsible Party): Duke University

Tracking Information

• First Submitted Date: April 25, 2017
• First Posted Date: April 28, 2017
• Last Update Posted Date: December 21, 2020
• Actual Study Start Date: October 19, 2017
• Estimated Primary Completion Date: November 2021 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: April 25, 2017)

Reconstitution of a matched human immune system in patient derived xenografts (PDXs) [ Time Frame: approximately 4 months ]

Number of samples yielding a matched human immune system in immunodeficient mice resulting in a PDX with a humanized immune system.

• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Reconstitution of a Human Immune System in a Patient Derived Xenograft (PDX) Model of Genitourinary (GU) Cancers
• Official Title: Reconstitution of a Human Immune System in a Patient Derived Xenograft (PDX) Model of Genitourinary (GU) Cancers

Brief Summary

The objective of this study is to obtain human blood CD34+ hematopoietic stem/progenitor cells (HSPCs) to reconstitute a match human immune system in our PDX model. The hypothesis is that by using matched leukocytes and PDX from the same patient, rejection of the PDX by the host immune system will not be observed and therefore a preclinical model to study immunotherapy can be developed to study, understand and improve upon our current therapies. HSPCs will be collected from bone marrow aspirate obtained from a bone marrow biopsy.

The secondary objective is to use patient tumor biopsy samples or circulating tumor cell samples to develop additional preclinical models of GU cancers, particularly prostate cancer, that are clinically relevant by generating additional PDXs.

• Detailed Description: Not Provided
• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• Sampling Method: Non-Probability Sample
• Study Population: Duke Cancer Institute Patients

Condition

• Genito Urinary Cancer
• Bladder Cancer
• Kidney Cancer
• Prostate Cancer

Intervention

• Procedure: Bone marrow biopsy
  Subjects will undergo a bone marrow biopsy to obtain aspirate for stem cell collection.
• Procedure: Tumor biopsy
  Subjects will undergo a tumor biopsy to obtain tissue for generation of a PDX.

Study Groups/Cohorts

• Subjects from which PDXs have been generated.
  Subjects will be identified from which PDXs have been generated from an already approved IRB protocol.
  Intervention: Procedure: Bone marrow biopsy
• Subjects without an existing PDX
  Subjects with prostate cancer amenable to a tumor biopsy.
  Intervention: Procedure: Tumor biopsy

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Suspended
• Estimated Enrollment (submitted: December 17, 2020): 30
• Original Estimated Enrollment (submitted: April 25, 2017): 5
• Estimated Study Completion Date: November 2021
• Estimated Primary Completion Date: November 2021 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria for Bone Marrow Biopsy:

1. Patients with Genitourinary (GU) Cancers and presence of existing patient derived xenograft or planned collection of tumor for PDX generation
2. Age ≥ 18 years.
3. Ability to understand and provide signed informed consent that fulfills Institutional Review Board guidelines.
4. Ability to undergo bone marrow biopsy
5. Laboratory requirements (Hematocrit 30%, Platelets 75,000 + 10^6/l and WBC 4000 X 10^6/l)
6. Karnofsky performance status >70%

Exclusion Criteria for Bone Marrow Biopsy:

1. Participants with serious concurrent chronic or acute illness that would affect the safety of a bone marrow biopsy.

Inclusion Criteria for Tumor Biopsy

1. Patients with prostate cancer

   a. Biopsies in this study for diseases other than prostate cancer are not permitted.

2. Age ≥ 18 years.
3. Ability to understand and provide signed informed consent that fulfills Institutional Review Board guidelines.

4. Planned or willing to undergo a tumor biopsy. Biopsies may be for research or clinical purposes depending on the treating provider for men with prostate cancer. Biopsies in this study for diseases other than prostate cancer are not permitted

   1. For research only biopsies: At least one lymph node or bone metastatic amenable to a minimum risk biopsy in the opinion of the treating physician.
   2. For clinical purpose biopsies: There are no location or risk requirements.
5. Laboratory requirements (Hematocrit 30%, Platelets 75,000 X 10^6/l and WBC 4000 X 10^6/l)
6. Karnofsky performance status >70%

Exclusion Criteria for Tumor Biopsy:

1. Participants with serious concurrent chronic or acute illness that would affect the safety of a tumor biopsy.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT03134027
• Other Study ID Numbers: Pro00082398
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

• IPD Sharing Statement: Not Provided
• Responsible Party: Duke University
• Study Sponsor: Duke University
• Collaborators: Not Provided

Investigators

• Principal Investigator: Andrew Armstrong, MD, ScM; Duke Cancer Institute

• PRS Account: Duke University
• Verification Date: December 2020