Malignant Diseases Clinical Trial
Official title:
An Expanded Access Study Using the CliniMACS System to Offer Therapeutic Manipulated Grafts That Are CD34 Cell Enriched and T Cell Depleted for Allogeneic Stem Cell Recipients With Mismatched Related Donors or Borderline Organ Function
| NCT number | NCT02162511 |
| Other study ID # | 28663 |
| Secondary ID | |
| Status | Completed |
| Phase | N/A |
| First received | |
| Last updated | |
| Start date | May 2014 |
| Est. completion date | March 2023 |
| Verified date | May 2023 |
| Source | Stanford University |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
The purpose of this protocol is to provide access to the CliniMACS® System to hematopoietic cell transplant (HSCT) patients who do not have a matched related donor. The CliniMACS system is currently approved for use in patients who have AML, and a genetically matched sibling donor. Through this protocol, the investigators will be able to offer potentially life-saving transplants to patients who have genetically mis-matched donor, who have no other options for treatment.
| Status | Completed |
| Enrollment | 3 |
| Est. completion date | March 2023 |
| Est. primary completion date | July 2019 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | N/A to 35 Years |
| Eligibility | Inclusion Criteria: - Participant age is 0 (newborn) to 35 years-old. - Participant has a disorder affecting the hematopoietic system that are inherited, acquired, or a result from the myeloablative treatment that can benefit from alternative stem cell transplantation according to standard practice guidelines for including patients for transplant. - Participant's medical screening clears s/he for allogeneic transplantation as per current institutional SOP based on standards of foundation for accreditation of cellular therapy and stem cell transplantation (FACT); - Participant must lack a healthy, HLA-identical related or unrelated donor unless s/he has a borderline organ function that will preclude the recipient from receiving a curative therapy due to the need of post-HSCT immunosuppressive therapy. - Participant must have a matched or mismatched-related donor who is: - Able to receive granulocyte colony-stimulating factor (G-CSF) and undergo apheresis either through placement of catheters in antecubital veins or a temporary central venous catheter OR agrees on a bone marrow harvest; - Healthy as per donor selection screening (following current SOP based on standards of foundation for accreditation of cellular therapy and stem cell transplantation - FACT); - Willing to participate and sign consent. - Participant or Legal Authorized Representative is able to sign informed consent (and signed assent, if applicable) for transplant. Exclusion Criteria: - Participant does not qualify for an allogeneic transplant due to medical screening, underlying disease, or lack of alternative donors. - Any condition that compromises compliance with the procedures of this protocol, as judged by the principal investigator. |
| Country | Name | City | State |
|---|---|---|---|
| United States | Stanford Children's Hospital | Palo Alto | California |
| Lead Sponsor | Collaborator |
|---|---|
| Rajni Agarwal |
United States,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Number of Patients With Severe (Grade III/IV) Acute Graft vs Host Disease (GVHD) | GVHD is a condition that occurs when donor bone marrow or stem cells attack the recipient. | Day +100 | |
| Secondary | Number of Participants With Graft Failure | Failure of donor stem cells to make neutrophils | Up to Day +42 after stem cell transplant | |
| Secondary | Length of Time to Engraftment | Absolute neutrophil count (ANC) >500 for 3 consecutive days and >80% donor cells in blood. | up to +1 year post-transplant | |
| Secondary | Chimerism of Donor Cells | The percentage of donor cells for all evaluable (without disease progression) patients | Day +100 post-transplant | |
| Secondary | Immune Recovery (CD4) | The time to CD4 count >100 | up to +1 year post-transplant | |
| Secondary | Number of Participants With Immune Recovery (CD4 >200) by Year 1 | up to +1 year post-transplant | ||
| Secondary | Immune Recovery Shown as Phytohemagglutin (PHA) | Immune recovery defined as achieving normal levels of PHA (53,000-200,000 CPM) | 6 months and 1 year post-transplant | |
| Secondary | Number of Patients With Post-transplant Lymphoproliferative Disease (PTLD) | Post-transplant lymphoproliferative disorder (PTLD) is a well-known, life-threatening complication of organ transplantation, predominantly occurring after solid organ transplantation (SOT) and hematopoietic stem cell transplantation (HSCT). | up to +1 year post-transplant | |
| Secondary | Number of Patients With Severe Toxicities | Incidence of transplant-related toxicities | up to +1 year post-transplant | |
| Secondary | Number of Participants Experiencing Post-transplant Infections | Post-transplant infections will be described by incidence and type. Participants may have had more than one type of infection. | up to +1 year post-transplant | |
| Secondary | Transplant-related Mortality (TRM) | Death related to transplant | at Day +100 and +1 year post-transplant |