Acute Graft Versus Host DiseasePhase 1NCT04006652
ApoGraft for the Prevention of Acute Graft Versus Host Disease in Haploidentical Hematopoietic Cell Transplant Recipients
A Phase 1 study testing ApoGraft in people with Acute Graft Versus Host Disease and Haploidentical Hematopoietic Stem Cell Transplant, run by Washington University School of Medicine at 1 site in United States.
Trial summary
Finding a donor remains a challenge for patients in need of an urgent hematopoietic stem cell transplantation (HSCT). The ability to obtain half matched stem cells from any family member represents a significant breakthrough in the field. Read more about this study
- Who is it for?
- Ages 18 to 70 yearsHealthy volunteers can take part
- Conditions the study is for
- Acute Graft Versus Host Disease, Haploidentical Hematopoietic Stem Cell Transplant
- What is being tested?
- ApoGraft
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- 16 Dec 2020to 19 Dec 2022
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 18 to 70 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Recipient Inclusion Criteria:
- Adult male or female subjects, 18-70 years of age.
- Availability of an HLA-haploidentical-HSCT related donor with a minimum match of 5/10 at the HLA A, B, C, DR and DQ loci.
- Hematologic malignancy in remission or controlled as below:
- Acute myelogenous leukemia (AML) and Acute lymphoblastic leukemia (ALL) in 1st or subsequent complete remission (CR)
- Non-Hodgkin's Lymphoma (NHL) in CR by CT or PET/CT
- Hodgkin's disease (HD) in 1st or subsequent CR by CT or PET/CT
- Intermediate or high risk Myelodysplastic syndrome (MDS) (IPSS-R criteria)
- ECOG performance status score 0-1 at time of the screening visit
- Cardiac left ventricular ejection fraction ≥ 40% in adults within 90 days of start of lymphodepleting chemotherapy
- Pulmonary function test with DLCO, FEV1 and FVC of ≥ 50% within 90 days of start of lymphodepleting chemotherapy.
- Oxygen saturation ≥ 90% on room air at screening visit.
- Subjects must have adequate organ function as defined below within 2 weeks of Day 0:
- AST (SGOT)/ALT (SGPT) ≤ 2.5 × upper limit of normal (ULN).
- Serum bilirubin <3 mg/dL.
- Estimated creatinine clearance >50
- If female of childbearing potential, agree to use an acceptable method of birth control or be surgically sterile, and have a negative pregnancy test.
- Available HLA-haploidentical donor
- Must be able to receive GvHD prophylaxis with tacrolimus, mycophenolate mofetil, and cyclophosphamide
- Able to understand and willing to sign an IRB approved written informed consent document (or that of legally authorized representative, if applicable). Subjects requiring a guardian to sign informed consent will not be included.
Recipient Exclusion Criteria:
- If a matched related donor is available and able to donate
- Participation in an interventional investigational trial within 30 days of Day 0.
- Subject suffering from any active or ongoing malignancy (other than the reason for HSCT) in the last 5 years prior to baseline; excluding basal cell carcinoma, in situ malignancy, low-risk prostate cancer, cervix cancer after curative therapy.
- Uncontrolled infections (bacterial, viral or fungal including sepsis, pneumonia with hypoxemia, persistent bacteremia, or meningitis within two weeks of the screening visit).
- Current known active acute or chronic infection with HBV or HCV.
- Known human immunodeficiency virus (HIV) infection or AIDS.
- Subjects with severe or symptomatic restrictive or obstructive lung disease or respiratory failure requiring ventilator support.
- Subjects with other concurrent severe and/or uncontrolled medical condition, which could compromise participation in the study (i.e. active infection, uncontrolled diabetes, uncontrolled hypertension, congestive cardiac failure, ventricular arrhythmias, and chronic liver or renal disease)
- History of any of the following within 12 months prior to screening: Uncontrolled congestive heart failure (New York Heart Association Classification 3 or 4), unstable angina, myocardial infarction, cerebrovascular accident, coronary/peripheral artery bypass graft surgery, transient ischemic attack, or pulmonary embolism.
- Any form of substance abuse (including drug or alcohol abuse), psychiatric disorder or any chronic condition susceptible, in the opinion of the investigator, of interfering with the conduct of the study.
- Organ allograft transplant recipient.
- If female of childbearing potential, agree to use an acceptable method of birth control or be surgically sterile, and have a negative pregnancy test.
- Pregnancy or lactation
- Must not have undergone a prior allogeneic donor (related, unrelated, or cord) transplant. Prior autologous transplant is not exclusionary.
- Presence of donor-specific anti-HLA antibodies.
- Must not receive antithymocyte globulin as part of pre-transplant conditioning regimen, plan to receive a T-cell depleted product, or have planned donor leukocyte infusions post-transplant.
- Immunosuppressive doses of steroids. Subjects with steroids for adrenal insufficiency will not be excluded.
Donor Inclusion Criteria
- Adult male or female subjects, 18-65 years of age.
- Donor criteria according to standard NMDP criteria for donor selection.
- Blood-related family member (sibling (full or half), offspring, parent, cousin, niece or nephew, aunt or uncle, or grandparent).
- HLA-haploidentical donor/recipient match by at least low-resolution typing per institutional standards.
- In the investigator's opinion, donor is in general good health, and medically able to tolerate leukapheresis required for harvesting HSC.
- Fit to receive G-CSF and donate peripheral blood stem cells.
- Able to sign written informed consent including consent for 2nd donation in the event of graft failure in the recipient.
Donor Exclusion Criteria
- HIV, HBV or HCV positive subjects within 30 days prior to day 0.
- Pregnant or lactating women.
- Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
- Graft donation with less than 0.3% CD34+ cells
Trial locations and contacts
1 site in 1 country.
United States1 site
Washington University School of Medicine
St Louis, Missouri, 63110
About this study
Official title: A Phase I Study of ApoGraft for the Prevention of Acute Graft Versus Host Disease in Haploidentical Hematopoietic Cell Transplant Recipients
Finding a donor remains a challenge for patients in need of an urgent hematopoietic stem cell transplantation (HSCT). The ability to obtain half matched stem cells from any family member represents a significant breakthrough in the field. Haploidentical haplo-HSCT is characterized by the nearly uniform and immediate availability of a donor and the availability of the donor for post-transplant cellular immunotherapy. However, haplo-HSCT has a high risk of Graft versus Host Disease (GvHD) and poor immune reconstitution when GvHD is prevented by all existing methods of vigorous ex vivo or in vivo T-cell depletion. Different treatment approaches are currently being explored to mitigate complications such as graft rejection, severe GvHD, and prolonged immune suppression. Novel experimental utilization of T regulatory cells, alloreactive natural killer (NK) cells, and other T cell subsets hold great promise. Cellect Biotherapeutics' platform technology, ApoGraft, is based on the findings that GvHD can be prevented by Fas receptor mediated selective depletion of T cell subsets, ex vivo. The investigators hypothesize that the use of ApoGrafts for haplo-HSCT will be safe, and reduce rates of GVHD without affecting Graft-versus-Leukemia (GvL).
- Being tested
- ApoGraft
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ApoGraftDrug
ApoGraft is a cell based product, manufactured with mobilized peripheral blood.
| Study group | Role | What they receive |
|---|---|---|
| Recipient | Experimental | * Will undergo institutionally standard myeloablative or reduced intensity chemotherapy or chemoradiotherapy which will be administered at the discretion of the treating physician
* Recipients will undergo a single fresh ApoGraft transplant as per standard clinical site guidelines Receives: Drug: ApoGraft |
| Donor | No intervention | -Donors will undergo apheresis from peripheral blood after daily G-CSF administration (for up to 5 days prior to Day -1) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability of ApoGraft as measured by adverse events related to ApoGraft product
-The descriptions and grading scales found in the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 will be utilized for all toxicity reporting.
Measured: From day 0 to 1 year post-transplantation of ApoGraft product
Secondary outcomes (10)
Cumulative incidence of graft failure
-Failure to engraft will be defined as failure to achieve absolute neutrophil count > 500 for 3 days by Day 35
Measured: 35 days post haplo-HCT
Treatment related mortality
-Death that results from a transplant procedure-related complication (e.g. infection, organ failure, hemorrhage, GVHD) rather than from relapse of the underlying disease or an unrelated cause.
Measured: Through 1 year post-transplantation of ApoGraft product
Time of neutrophil engraftment as determined by number of days for reaching first of 3 consecutive days with ANC ≥ 500/mm3
Measured: 35 days post haplo-HCT
Rate of neutrophil engraftment as determined by number of days for reaching first of 3 consecutive days with ANC ≥ 500/mm3
Measured: 35 days post haplo-HCT
Time of platelet engraftment determined by number of days for reaching first measurement of 3 consecutive measurements with platelets ≥ 20,000/mm3 in the absence of platelet administration during the prior 7 days
Measured: 35 days post haplo-HCT
Rate of platelet engraftment determined by number of days for reaching first measurement of 3 consecutive measurements with platelets ≥ 20,000/mm3 in the absence of platelet administration during the prior 7 days
Measured: 35 days post haplo-HCT
Incidence of Grade 2-4 acute GVHD
-Acute GVHD will be assessed using MAGIC criteria
Measured: Day 180
Time to development of Grade 2-4 acute GVHD
-Acute GVHD will be assessed using MAGIC criteria
Measured: Day 180
Incidence of Grade 3-4 acute GVHD
-Acute GVHD will be assessed using MAGIC criteria
Measured: Day 180
Time to development of Grade 3-4 acute GVHD
-Acute GVHD will be assessed using MAGIC criteria
Measured: Day 180
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jul 2019
- Study start16 Dec 2020
- Primary completion19 Dec 2022
- Study completion19 Dec 2022
Why it stopped: Lost funding
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Graft Versus Host DiseaseStudies of the same condition, open and under way.
- More trials run by Washington University School of MedicineThe sponsor's other registered studies.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- The first three recipients will be treated in a sequential manner meaning that subjects will be transplanted with ApoGraft only after the engraftment of the previous subject has occurred.
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Washington University School of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- Cellect Biotechnology
- Responsible party
- Sponsor
- Investigators
- Mark Schroeder, M.D., Principal investigator, Washington University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04006652
- Sponsor's protocol ID
- 201911131
- Status last verified
- May 2023
- First submitted
- 1 Jul 2019
- Last updated
- 19 May 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Study of ApoGraft for the Prevention of Acute Graft Versus Host Disease in Haploidentical Hematopoietic Cell Transplant Recipients
- ClinicalTrials.gov ID
- NCT04006652
- Acronym / sponsor ID
- 201911131
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Lost funding
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Washington University School of MedicineUniversity, hospital or other
- Collaborators
- Cellect Biotechnology
- Study start
- 16 Dec 2020 (actual)
- Primary completion
- 19 Dec 2022 (actual)
- Study completion
- 19 Dec 2022 (actual)
- First posted
- 5 Jul 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Finding a donor remains a challenge for patients in need of an urgent hematopoietic stem cell transplantation (HSCT). The ability to obtain half matched stem cells from any family member represents a significant breakthrough in the field. Haploidentical haplo-HSCT is characterized by the nearly uniform and immediate availability of a donor and the availability of the donor for post-transplant cellular immunotherapy. However, haplo-HSCT has a high risk of Graft versus Host Disease (GvHD) and poor immune reconstitution when GvHD is prevented by all existing methods of vigorous ex vivo or in vivo T-cell depletion. Different treatment approaches are currently being explored to mitigate complications such as graft rejection, severe GvHD, and prolonged immune suppression. Novel experimental utilization of T regulatory cells, alloreactive natural killer (NK) cells, and other T cell subsets hold great promise. Cellect Biotherapeutics' platform technology, ApoGraft, is based on the findings that GvHD can be prevented by Fas receptor mediated selective depletion of T cell subsets, ex vivo. The investigators hypothesize that the use of ApoGrafts for haplo-HSCT will be safe, and reduce rates of GVHD without affecting Graft-versus-Leukemia (GvL).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- The first three recipients will be treated in a sequential manner meaning that subjects will be transplanted with ApoGraft only after the engraftment of the previous subject has occurred.
| Arm | Type | Receives |
|---|---|---|
| Recipient | Experimental | Drug: ApoGraft* Will undergo institutionally standard myeloablative or reduced intensity chemotherapy or chemoradiotherapy which will be administered at the discretion of the treating physician * Recipients will undergo a single fresh ApoGraft transplant as per standard clinical site guidelines |
| Donor | No intervention | –-Donors will undergo apheresis from peripheral blood after daily G-CSF administration (for up to 5 days prior to Day -1) |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
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 |
|---|---|---|
| ApoGraftApoGraft is a cell based product, manufactured with mobilized peripheral blood. | Drug | Recipient |
Primary outcome
Safety and tolerability of ApoGraft as measured by adverse events related to ApoGraft product
Time frame: From day 0 to 1 year post-transplantation of ApoGraft product
-The descriptions and grading scales found in the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 will be utilized for all toxicity reporting.
10 secondary outcomes
Cumulative incidence of graft failure
Time frame: 35 days post haplo-HCT
Treatment related mortality
Time frame: Through 1 year post-transplantation of ApoGraft product
Time of neutrophil engraftment as determined by number of days for reaching first of 3 consecutive days with ANC ≥ 500/mm3
Time frame: 35 days post haplo-HCT
Rate of neutrophil engraftment as determined by number of days for reaching first of 3 consecutive days with ANC ≥ 500/mm3
Time frame: 35 days post haplo-HCT
Time of platelet engraftment determined by number of days for reaching first measurement of 3 consecutive measurements with platelets ≥ 20,000/mm3 in the absence of platelet administration during the prior 7 days
Time frame: 35 days post haplo-HCT
Rate of platelet engraftment determined by number of days for reaching first measurement of 3 consecutive measurements with platelets ≥ 20,000/mm3 in the absence of platelet administration during the prior 7 days
Time frame: 35 days post haplo-HCT
Incidence of Grade 2-4 acute GVHD
Time frame: Day 180
Time to development of Grade 2-4 acute GVHD
Time frame: Day 180
Incidence of Grade 3-4 acute GVHD
Time frame: Day 180
Time to development of Grade 3-4 acute GVHD
Time frame: Day 180
Results and safety
The study was stopped early (reason given: Lost funding). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 May 2023.
- Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of MedicineLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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.
Mark Schroeder, M.D. principal investigator
Washington University School of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ApoGraft in people with Acute Graft Versus Host Disease and Haploidentical Hematopoietic Stem Cell Transplant, run by Washington University School of Medicine 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?
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: stopped early, as of the sponsor's last update on 19 May 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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Lost funding). Results are not available in the source record.
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?
Safety and tolerability of ApoGraft as measured by adverse events related to ApoGraft product (time frame: From day 0 to 1 year post-transplantation of ApoGraft product). 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 19 May 2023.