Malignant DiseasesNCT02162511
CD34+ Cell Enriched and T Cell Depleted Allogeneic Stem Cell Transplantation for Patients With Mismatched Related Donors or Borderline Organ Function
A study testing CliniMACS CD34+ cell enrichment and T-cell depletion in people with Malignant Diseases and Non-malignant Diseases, run by Rajni Agarwal at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages Up to 35 years
- Conditions the study is for
- Malignant Diseases, Non-malignant Diseases
- What is being tested?
- CliniMACS CD34+ cell enrichment and T-cell depletion
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- May 2014to Mar 2023
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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;
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- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Stanford Children's Hospital
Palo Alto, California, 94305
About this study
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
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.
- Conditions
- Malignant Diseases, Non-malignant Diseases
- Being tested
- CliniMACS CD34+ cell enrichment and T-cell depletion
- Keywords
- Haplocompatible, Stem cell transplant, Borderline organ function, Alternative donor stem cell transplant
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CliniMACS CD34+ cell enrichment and T-cell depletionDevice
| Study group | Role | What they receive |
|---|---|---|
| ARM A Malignant TBI | Experimental | Malignant diseases Conditioning including total body irradiation and chemotherapy Receives: Device: CliniMACS CD34+ cell enrichment and T-cell depletion |
| ARM B Malignant Non-TBI | Experimental | Malignant diseases chemotherapy based conditioning Receives: Device: CliniMACS CD34+ cell enrichment and T-cell depletion |
| ARM C Non-malignant | Experimental | Non-malignant diseases Chemotherapy based conditioning Receives: Device: CliniMACS CD34+ cell enrichment and T-cell depletion |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Day +100
Secondary outcomes (10)
Number of Participants With Graft Failure
Failure of donor stem cells to make neutrophils
Measured: Up to Day +42 after stem cell transplant
Length of Time to Engraftment
Absolute neutrophil count (ANC) >500 for 3 consecutive days and >80% donor cells in blood.
Measured: up to +1 year post-transplant
Chimerism of Donor Cells
The percentage of donor cells for all evaluable (without disease progression) patients
Measured: Day +100 post-transplant
Immune Recovery (CD4)
The time to CD4 count >100
Measured: up to +1 year post-transplant
Number of Participants With Immune Recovery (CD4 >200) by Year 1
Measured: up to +1 year post-transplant
Immune Recovery Shown as Phytohemagglutin (PHA)
Immune recovery defined as achieving normal levels of PHA (53,000-200,000 CPM)
Measured: 6 months and 1 year post-transplant
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).
Measured: up to +1 year post-transplant
Number of Patients With Severe Toxicities
Incidence of transplant-related toxicities
Measured: up to +1 year post-transplant
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.
Measured: up to +1 year post-transplant
Transplant-related Mortality (TRM)
Death related to transplant
Measured: at Day +100 and +1 year post-transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jun 2014 (estimated)
- Study startMay 2014
- Primary completionJul 2019
- Study completionMar 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Malignant DiseasesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Rajni Agarwal
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Rajni Agarwal, Stanford University
- Investigators
- Rajni Agarwal, MD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02162511
- Sponsor's protocol ID
- 28663
- Status last verified
- May 2023
- First submitted
- 10 Jun 2014
- Last updated
- 26 Jun 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 26 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02162511
- Acronym / sponsor ID
- 28663
- Conditions
- Malignant Diseases, Non-malignant Diseases
- Keywords
- Haplocompatible, Stem cell transplant, Borderline organ function, Alternative donor stem cell transplant
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Rajni AgarwalUniversity, hospital or other
- Study start
- May 2014
- Primary completion
- Jul 2019 (actual)
- Study completion
- Mar 2023 (actual)
- First posted
- 12 Jun 2014 (estimated)
- Results
- Posted 26 Jun 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| ARM A Malignant TBI | Experimental | Device: CliniMACS CD34+ cell enrichment and T-cell depletionMalignant diseases Conditioning including total body irradiation and chemotherapy |
| ARM B Malignant Non-TBI | Experimental | Device: CliniMACS CD34+ cell enrichment and T-cell depletionMalignant diseases chemotherapy based conditioning |
| ARM C Non-malignant | Experimental | Device: CliniMACS CD34+ cell enrichment and T-cell depletionNon-malignant diseases Chemotherapy based conditioning |
Who may qualify
- Ages
- Up to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- 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:
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CliniMACS CD34+ cell enrichment and T-cell depletion | Device | ARM A Malignant TBI, ARM B Malignant Non-TBI, ARM C Non-malignant |
Primary outcome
Number of Patients With Severe (Grade III/IV) Acute Graft vs Host Disease (GVHD)
Time frame: Day +100
GVHD is a condition that occurs when donor bone marrow or stem cells attack the recipient.
10 secondary outcomes
Number of Participants With Graft Failure
Time frame: Up to Day +42 after stem cell transplant
Length of Time to Engraftment
Time frame: up to +1 year post-transplant
Chimerism of Donor Cells
Time frame: Day +100 post-transplant
Immune Recovery (CD4)
Time frame: up to +1 year post-transplant
Number of Participants With Immune Recovery (CD4 >200) by Year 1
Time frame: up to +1 year post-transplant
Immune Recovery Shown as Phytohemagglutin (PHA)
Time frame: 6 months and 1 year post-transplant
Number of Patients With Post-transplant Lymphoproliferative Disease (PTLD)
Time frame: up to +1 year post-transplant
Number of Patients With Severe Toxicities
Time frame: up to +1 year post-transplant
Number of Participants Experiencing Post-transplant Infections
Time frame: up to +1 year post-transplant
Transplant-related Mortality (TRM)
Time frame: at Day +100 and +1 year post-transplant
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jun 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Stanford Stem Cell Transplantation websiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Rajni Agarwal, MD principal investigator
Stanford University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing CliniMACS CD34+ cell enrichment and T-cell depletion in people with Malignant Diseases and Non-malignant Diseases, run by Rajni Agarwal at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 26 Jun 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Patients With Severe (Grade III/IV) Acute Graft vs Host Disease (GVHD) (time frame: Day +100). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Jun 2023.