LeukemiaEarly Phase 1ExpMACsNCT02356653
Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC
A Early Phase 1 study testing Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs) in people with Leukemia and Inborn Errors of Metabolism, run by Children's Hospital of Philadelphia at 1 site in United States.
Trial summary
The goal of this protocol is to expand access for patients who lack a fully HLA (Human leukocyte antigen) matched sibling donor and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT). These patients have a serious or immediately life-threatening disease for which HSCT is indicated. Read more about this study
- Who is it for?
- Ages Up to 30 years
- Conditions the study is for
- Leukemia, Inborn Errors of Metabolism, Bone Marrow Failure Syndromes
- What is being tested?
- Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2013to Jan 2030
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
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 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients who lack a fully HLA matched sibling and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT) but are not deemed suitable candidates per their treating clinical team for current open institutional protocols using ClinMACs device for CD3+/CD19+ depletion.
- Patients with the following transplantable diseases:
- Non-malignant diseases:
- Metabolic storage diseases correctable by HSCT, Bone marrow failure syndromes, Immunodeficiencies/immune dysregulation syndromes/including HLH, Hemoglobinopathies correctable and requiring HSCT, and Other diseases treated with HSCT/Other non-malignant blood, metabolic, or immune disorders for which HSCT has been recommended
- Malignant diseases:
- Acute leukemias, Chronic leukemias, Lymphomas, Myelodyplastic syndrome
Show all 19Show fewer
- Signed informed consent
- Lansky or Karnofsky performance ≥60
- Hematologic and Organ Function per current institutional SOP.
- Infectious Evaluation as per current institutional SOP.
- Participants of childbearing potential must have a negative pregnancy test as per institutional SOP
- In cases that are deemed clinical emergencies (primary or secondary graft failure, severe marrow suppression), the above status criteria will be waived.
- Patients must have an identified living donor
- Donor selection will comply with 21 CFR 1271
- Unrelated donor that meets the matching criteria of the NMDP with allele matching at HLA -A, -B, -C, -DRB1, and -DQB1: Unrelated donors may be a 10/10 match, a 9/10 match, or an 8/10 match if one of the mismatches is at DQB1
- Related donor suitable for mobilization infectious disease criteria as per SOP, including HIV, HepB, HepC PCR negative.
- CHOP BMT procedures apply for determining donor eligibility, including donor screening and testing for relevant communicable disease agents and diseases. Our donor collection program is FACT accredited.
- Unrelated donor identified through the National Marrow Donor Program (NMDP) and fulfills the NMDP criteria for donation. Unrelated donor willing and able to undergo mobilization of peripheral stem cells and apheresis.
- The donors selected for this IND will either be unrelated donors identified through the National Marrow Donor Program (NMDP) or related donors. Regarding the unrelated donors; NMDP procedures for determining donor eligibility include donor screening and testing for relevant communicable disease agents and diseases
Who cannot join
- Uncontrolled bacterial, viral or fungal infections
- Suitable, fully HLA matched sibling donor
- Donor unable to donate peripheral stem cells
- Pregnant participants
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Megan Atkinson, 215-590-2820, cttsbmtintake@chop.edu
Patricia Hankins, BSN, RN, CCRC, 215-590-5168, hankinsp@chop.edu
About this study
Official title: Expanded Access Protocol Using CD3+/CD19+ Depleted Unrelated Donor or Related Donor Peripheral Stem Cells
The goal of this protocol is to expand access for patients who lack a fully HLA (Human leukocyte antigen) matched sibling donor and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT). These patients have a serious or immediately life-threatening disease for which HSCT is indicated. These patients are not eligible for other Children's Hospital of Philadelphia IRB approved protocols that utilize CliniMACs technology for T depletion.
Read the detailed description
Only 25-30% of patients who may benefit from HSCT have a matched related donor. There is a higher rate of complications using cells from an unrelated or partially matched related donor. T cells within the donor cells may cause a complication called graft vs. host disease (GVHD). The goal of this study is to use the CliniMACs device to remove the T cells that cause GVHD, called T cell depletion.
- Conditions
- Leukemia, Inborn Errors of Metabolism, Bone Marrow Failure Syndromes, Immunodeficiencies, Immunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome
- Being tested
- Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)
- Medical subject headings
- Leukemia, Metabolism, Inborn Errors, Bone Marrow Failure Disorders, Immunologic Deficiency Syndromes, Immune Dysregulation, Polyendocrinopathy, Enteropathy, X-Linked Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)Biological
Also called CliniMACs
Processing of stem cells using the CliniMACs device to selectively deplete specific T cells to decrease risk of graft versus host disease when using donor stem cells which are not fully matched.
| Study group | Role | What they receive |
|---|---|---|
| Expanded access to CliniMACs device for T cell depletion | Experimental | access for patients who lack a fully HLA matched sibling, and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT). These patients have a serious or immediately life-open protocols that utilize CliniMACs technology for T depletion. Subjects will undergo transplant of stem cells with CD3+/CD19+ depletion. Receives: Biological: Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Survival
Number of participants who remain alive.
Measured: 1 year post transplant
Secondary outcomes (2)
Graft versus Host Disease
Number of participants who had grades II-IV acute graft versus host disease and/or limited or extensive chronic gvhd
Measured: 1 year post transplant
Graft Failure
Number of participants who experienced primary or secondary graft failure or autologous reconstitution.
Measured: 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 posted5 Feb 2015 (estimated)
- Study startDec 2013
- Primary completionJan 2027 (estimated)
- Study completionJan 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for LeukemiaStudies of the same condition, open and under way.
- More trials run by Children's Hospital of PhiladelphiaThe 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs) Processing of stem cells using the CliniMACs device to selectively deplete specific T cells to decrease risk of graft versus host disease when using donor stem cells which are not fully matched.
- Masking
- None (open label)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Philadelphia
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Timothy Olson, Children's Hospital of Philadelphia
- Investigators
- Timothy Olson, MD, PhD, Principal investigator, Children's Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02356653
- Sponsor's protocol ID
- 13-010286
- Other IDs
- 12BT125
- Acronym
- ExpMACs
- Status last verified
- Feb 2026
- First submitted
- 22 Oct 2014
- Last updated
- 20 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 20 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access Protocol Using CD3+/CD19+ Depleted Unrelated Donor or Related Donor Peripheral Stem Cells
- ClinicalTrials.gov ID
- NCT02356653
- Acronym / sponsor ID
- ExpMACs / 13-010286
- Conditions
- Leukemia, Inborn Errors of Metabolism, Bone Marrow Failure Syndromes, Immunodeficiencies, Immunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Children's Hospital of PhiladelphiaUniversity, hospital or other
- Study start
- Dec 2013
- Primary completion
- Jan 2027 (estimated)
- Study completion
- Jan 2030 (estimated)
- First posted
- 5 Feb 2015 (estimated)
- 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.
The goal of this protocol is to expand access for patients who lack a fully HLA (Human leukocyte antigen) matched sibling donor and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT). These patients have a serious or immediately life-threatening disease for which HSCT is indicated. These patients are not eligible for other Children's Hospital of Philadelphia IRB approved protocols that utilize CliniMACs technology for T depletion.
Detailed description
Only 25-30% of patients who may benefit from HSCT have a matched related donor. There is a higher rate of complications using cells from an unrelated or partially matched related donor. T cells within the donor cells may cause a complication called graft vs. host disease (GVHD). The goal of this study is to use the CliniMACs device to remove the T cells that cause GVHD, called T cell depletion.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs) Processing of stem cells using the CliniMACs device to selectively deplete specific T cells to decrease risk of graft versus host disease when using donor stem cells which are not fully matched.
| Arm | Type | Receives |
|---|---|---|
| Expanded access to CliniMACs device for T cell depletion | Experimental | Biological: Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)access for patients who lack a fully HLA matched sibling, and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT). These patients have a serious or immediately life-open protocols that utilize CliniMACs technology for T depletion. Subjects will undergo transplant of stem cells with CD3+/CD19+ depletion. |
Who may qualify
- Ages
- Up to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (19 listed)
- Patients who lack a fully HLA matched sibling and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT) but are not deemed suitable candidates per their treating clinical team for current open institutional protocols using ClinMACs device for CD3+/CD19+ depletion.
- Patients with the following transplantable diseases:
- Metabolic storage diseases correctable by HSCT, Bone marrow failure syndromes, Immunodeficiencies/immune dysregulation syndromes/including HLH, Hemoglobinopathies correctable and requiring HSCT, and Other diseases treated with HSCT/Other non-malignant blood, metabolic, or immune disorders for which HSCT has been recommended
- Acute leukemias, Chronic leukemias, Lymphomas, Myelodyplastic syndrome
- Signed informed consent
Key exclusion criteria (4 listed)
- Uncontrolled bacterial, viral or fungal infections
- Suitable, fully HLA matched sibling donor
- Donor unable to donate peripheral stem cells
- Pregnant participants
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 |
|---|---|---|
| Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)Also: CliniMACsProcessing of stem cells using the CliniMACs device to selectively deplete specific T cells to decrease risk of graft versus host disease when using donor stem cells which are not fully matched. | Biological | Expanded access to CliniMACs device for T cell depletion |
Primary outcome
Overall Survival
Time frame: 1 year post transplant
Number of participants who remain alive.
2 secondary outcomes
Graft versus Host Disease
Time frame: 1 year post transplant
Graft Failure
Time frame: 1 year post transplant
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Feb 2026.
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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.
Timothy Olson, MD, PhD principal investigator
Children's Hospital of Philadelphia
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs) in people with Leukemia and Inborn Errors of Metabolism, run by Children's Hospital of Philadelphia 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 19 inclusion and 4 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: looking for participants now, as of the sponsor's last update on 20 Feb 2026. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Megan Atkinson (215-590-2820, cttsbmtintake@chop.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Survival (time frame: 1 year post transplant). 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 20 Feb 2026.