Malignant NeoplasmPhase 2NCT01871441
Haploidentical Donor Hematopoietic Stem Cell Transplant in Treating Patients With Hematologic Malignancies
A Phase 2 study testing Total-body irradiation, Donor lymphocytes infusion (DLI) and Cyclophosphamide in people with Malignant Neoplasm, run by Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University at 1 site in United States.
Trial summary
This phase II trial studies how well haploidentical donor hematopoietic stem cell transplant works in treating patients with hematologic malignancies. Giving chemotherapy and total-body irradiation before a donor stem cell transplant helps stop the growth of cancer cells. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Malignant Neoplasm
- What is being tested?
- Total-body irradiation, Donor lymphocytes infusion (DLI) and Cyclophosphamide
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- 17 May 2013to 20 Oct 2016
- Phase
- Phase 2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Any patient with a hematologic or oncologic diagnosis without morphological evidence of disease in which allogeneic HSCT is thought to be beneficial.
- Patients must have a related donor who is a two or more allele mismatch at the HLA-A; B; C; DR loci.
- Patients must have adequate organ function:
- LVEF (Left ventricular ejection fraction) of >50%
- Diffusion Capacity for Carbon Monoxide (DLCO) >50% of predicted corrected for hemoglobin
- Adequate liver function as defined by a serum bilirubin <1.8, Aspartate Aminotransferase (AST) or alanine aminotransferase (ALT) < 2.5X upper limit of normal
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- Creatinine clearance of > 60 ml/min
- Performance status > 80% (TJU Karnofsky)
- Hematopoietic Comorbidity Index (HCT-CI) Score < 5 Points
- Patients must be willing to use contraception if they have childbearing potential
- Able to give informed consent, or if decisionally impaired, have a legal next of kin or guardian that can give informed consent
Who cannot join
- Performance status < 80 % (TJU Karnofsky)
- HCT-CI Score > 5 Points
- Combination of Performance status of < 80% (TJU Karnofsky) and an HCT-CI of 4 points or more.
- HIV positive
- Active involvement of the central nervous system with malignancy
- Psychiatric disorder that would preclude patients from signing an informed consent
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- Pregnancy
- Patients with life expectancy of < 6 months for reasons other than their underlying hematologic/oncologic disorder
- Patients who have received alemtuzumab within 8 weeks of the transplant admission, or who have recently received horse or rabbit anti-thymocyte globulin and have an ATG level of > 2 ugm/ml
- Patients who cannot receive cyclophosphamide
- Patients with evidence of another malignancy, exclusive of a skin cancer that requires only local treatment, should not be enrolled on this protocol
Trial locations and contacts
1 site in 1 country.
United States1 site
Thomas Jefferson University
Philadelphia, Pennsylvania, 19107
About this study
Official title: A Two Step Approach to Haploidentical Hematopoietic Stem Cell Transplantation for Patients in Remission From HLA Partially-Matched Related Donors-Effect of Maternal Donors on Outcomes
This phase II trial studies how well haploidentical donor hematopoietic stem cell transplant works in treating patients with hematologic malignancies. Giving chemotherapy and total-body irradiation before a donor stem cell transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. Giving an infusion of the donor's T cells (donor lymphocyte infusion) may replace the patient's immune cells and help destroy any remaining cancer cells. When the stem cells from a related donor, that closely matches the patient's blood, are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets.
Read the detailed description
PRIMARY OBJECTIVES:
- Examine the 1 year disease free survival (DFS) rate of patients with maternal donors or sibling donors who share the maternal haplotype (maternal group) and compare them to patients receiving cells from donors who have points from other characteristics such as killer immunoglobulin-like receptor (KIR) ligand mismatching, minor histocompatibility antigen (MHag) differences, or number of human leukocyte antigen (HLA) mismatches (non-maternal group).
SECONDARY OBJECTIVES:
- Assess the incidences of relapse and graft-versus-host disease (GVHD) in maternal recipients whose only eligible donors are offspring.
II. Assess the incidence of grades III-IV GVHD in female recipients with male donors.
III. Compare the rates of DFS in recipient-donor combinations in which there is at least 1 KIR ligand mismatch versus those without a KIR ligand mismatch.
OUTLINE:
Patients undergo total body irradiation (TBI) twice daily (BID) on days -9 to -6, undergo donor lymphocyte infusion (DLI) on day -6, and receive cyclophosphamide intravenously (IV) over 2 hours on days -3 and -2.
TRANSPLANT: Patients undergo haploidentical allogeneic hematopoietic stem cell transplant on day 0.
GVHD PROPHYLAXIS: Patients receive tacrolimus IV beginning on day -1 with taper beginning on day 42, and mycophenolate mofetil IV BID from day -1 to day 28.
After completion of study treatment, patients are followed up at 90, 180, and 270 days, and 1 year.
- Conditions
- Malignant Neoplasm
- Being tested
- Total-body irradiation, Donor lymphocytes infusion (DLI), Cyclophosphamide, Allogeneic hematopoietic stem cell transplantation (HSCT), Tacrolimus
- Medical subject headings
- Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Total-body irradiationRadiation
Also called TBI
Undergo TBI
Donor lymphocytes infusion (DLI)Biological
Also called Allogeneic Lymphocytes, ALLOLYMPH
Undergo DLI
CyclophosphamideDrug
Also called Endoxan, Cytoxan, Neosar, Procytox
Given IV
Allogeneic hematopoietic stem cell transplantation (HSCT)Procedure or surgery
Undergo haploidentical allogeneic HSCT
TacrolimusDrug
Also called FK-506, fujimycin, Prograf, Advagraf
Given IV
Also called CellCept, Myfortic, MMF
Given IV
| Study group | Role | What they receive |
|---|---|---|
| Treatment (haploidentical allogeneic HSCT) | Experimental | Patients undergo TBI BID on days -9 to -6, undergo DLI on day -6, and receive cyclophosphamide IV over 2 hours on days -3 and -2.
TRANSPLANT: Patients undergo haploidentical allogeneic hematopoietic stem cell transplant on day 0.
GVHD PROPHYLAXIS: Patients receive tacrolimus IV beginning on day -1 with taper beginning on day 42, and mycophenolate mofetil IV BID from day -1 to day 28. Receives: Radiation: Total-body irradiation; Biological: Donor lymphocytes infusion (DLI); Drug: Cyclophosphamide; Procedure: Allogeneic hematopoietic stem cell transplantation (HSCT); Drug: Tacrolimus; Drug: Mycophenolate mofetil |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Disease-free Survival (DFS)
Disease free survival (DFS), defined as the time to death, relapse or disease progression.
Measured: 1 year
Secondary outcomes (3)
Number of Participants With Relapse of Disease
Relapse of Disease is defined as the return of a disease or the signs and symptoms of a disease after a period of improvement. Relapse is almost always associated with the immunological failure of the donor immune system to recognize and/or respond to reemergence of a tumor. The number of participants with relapse of disease will be collected.
Measured: Up to 1 year
Rate of Grade III-IV GVHD in Female Recipients With Male Donors
The rates of grade III-IV GVHD in female recipients with male donors will be computed with corresponding exact binomial 95% confidence intervals.
Measured: Up to 1 year
The Rates of Grade III-IV GVHD in Female Recipients With Male Donors Will be Computed With Corresponding Exact Binomial 95% Confidence Intervals.
The difference in DFS in recipient-donor combinations in which there is at least 1 KIR ligand mismatch versus those without a KIR ligand mismatch will be tested using log-rank test.
Measured: Up to 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jun 2013 (estimated)
- Study start17 May 2013
- Primary completion12 Dec 2013
- Study completion20 Oct 2016
Why it stopped: Trial was closed due to poor accrual.
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 NeoplasmStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Dolores Grosso, DNP, CRNP, Principal investigator, Thomas Jefferson University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01871441
- Sponsor's protocol ID
- 13D.127
- Other IDs
- 2012-104, JT 3048
- Status last verified
- May 2025
- First submitted
- 4 Jun 2013
- Last updated
- 16 May 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 16 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Two Step Approach to Haploidentical Hematopoietic Stem Cell Transplantation for Patients in Remission From HLA Partially-Matched Related Donors-Effect of Maternal Donors on Outcomes
- ClinicalTrials.gov ID
- NCT01871441
- Acronym / sponsor ID
- 13D.127
- Conditions
- Malignant Neoplasm
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Trial was closed due to poor accrual.
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson UniversityUniversity, hospital or other
- Study start
- 17 May 2013 (actual)
- Primary completion
- 12 Dec 2013 (actual)
- Study completion
- 20 Oct 2016 (actual)
- First posted
- 6 Jun 2013 (estimated)
- Results
- Posted 9 Jan 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase II trial studies how well haploidentical donor hematopoietic stem cell transplant works in treating patients with hematologic malignancies. Giving chemotherapy and total-body irradiation before a donor stem cell transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. Giving an infusion of the donor's T cells (donor lymphocyte infusion) may replace the patient's immune cells and help destroy any remaining cancer cells. When the stem cells from a related donor, that closely matches the patient's blood, are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets.
Detailed description
PRIMARY OBJECTIVES:
- Examine the 1 year disease free survival (DFS) rate of patients with maternal donors or sibling donors who share the maternal haplotype (maternal group) and compare them to patients receiving cells from donors who have points from other characteristics such as killer immunoglobulin-like receptor (KIR) ligand mismatching, minor histocompatibility antigen (MHag) differences, or number of human leukocyte antigen (HLA) mismatches (non-maternal group).
SECONDARY OBJECTIVES:
- Assess the incidences of relapse and graft-versus-host disease (GVHD) in maternal recipients whose only eligible donors are offspring.
II. Assess the incidence of grades III-IV GVHD in female recipients with male donors.
III. Compare the rates of DFS in recipient-donor combinations in which there is at least 1 KIR ligand mismatch versus those without a KIR ligand mismatch.
OUTLINE:
Patients undergo total body irradiation (TBI) twice daily (BID) on days -9 to -6, undergo donor lymphocyte infusion (DLI) on day -6, and receive cyclophosphamide intravenously (IV) over 2 hours on days -3 and -2.
TRANSPLANT: Patients undergo haploidentical allogeneic hematopoietic stem cell transplant on day 0.
GVHD PROPHYLAXIS: Patients receive tacrolimus IV beginning on day -1 with taper beginning on day 42, and mycophenolate mofetil IV BID from day -1 to day 28.
After completion of study treatment, patients are followed up at 90, 180, and 270 days, and 1 year.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (haploidentical allogeneic HSCT) | Experimental | Radiation: Total-body irradiation, Biological: Donor lymphocytes infusion (DLI), Drug: Cyclophosphamide, Procedure: Allogeneic hematopoietic stem cell transplantation (HSCT), Drug: Tacrolimus, Drug: Mycophenolate mofetilPatients undergo TBI BID on days -9 to -6, undergo DLI on day -6, and receive cyclophosphamide IV over 2 hours on days -3 and -2. TRANSPLANT: Patients undergo haploidentical allogeneic hematopoietic stem cell transplant on day 0. GVHD PROPHYLAXIS: Patients receive tacrolimus IV beginning on day -1 with taper beginning on day 42, and mycophenolate mofetil IV BID from day -1 to day 28. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Any patient with a hematologic or oncologic diagnosis without morphological evidence of disease in which allogeneic HSCT is thought to be beneficial.
- Patients must have a related donor who is a two or more allele mismatch at the HLA-A; B; C; DR loci.
- Patients must have adequate organ function:
- Performance status > 80% (TJU Karnofsky)
- Hematopoietic Comorbidity Index (HCT-CI) Score < 5 Points
Key exclusion criteria (11 listed)
- Performance status < 80 % (TJU Karnofsky)
- HCT-CI Score > 5 Points
- Combination of Performance status of < 80% (TJU Karnofsky) and an HCT-CI of 4 points or more.
- HIV positive
- Active involvement of the central nervous system with malignancy
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 |
|---|---|---|
| Total-body irradiationAlso: TBIUndergo TBI | Radiation | Treatment (haploidentical allogeneic HSCT) |
| Donor lymphocytes infusion (DLI)Also: Allogeneic Lymphocytes, ALLOLYMPHUndergo DLI | Biological | Treatment (haploidentical allogeneic HSCT) |
| CyclophosphamideAlso: Endoxan, Cytoxan, Neosar, ProcytoxGiven IV | Drug | Treatment (haploidentical allogeneic HSCT) |
| Allogeneic hematopoietic stem cell transplantation (HSCT)Undergo haploidentical allogeneic HSCT | Procedure or surgery | Treatment (haploidentical allogeneic HSCT) |
| TacrolimusAlso: FK-506, fujimycin, Prograf, AdvagrafGiven IV | Drug | Treatment (haploidentical allogeneic HSCT) |
| Mycophenolate mofetilAlso: CellCept, Myfortic, MMFGiven IV | Drug | Treatment (haploidentical allogeneic HSCT) |
Primary outcome
Number of Participants With Disease-free Survival (DFS)
Time frame: 1 year
Disease free survival (DFS), defined as the time to death, relapse or disease progression.
3 secondary outcomes
Number of Participants With Relapse of Disease
Time frame: Up to 1 year
Rate of Grade III-IV GVHD in Female Recipients With Male Donors
Time frame: Up to 1 year
The Rates of Grade III-IV GVHD in Female Recipients With Male Donors Will be Computed With Corresponding Exact Binomial 95% Confidence Intervals.
Time frame: Up to 1 year
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 May 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Thomas Jefferson University HospitalsLink 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.
Dolores Grosso, DNP, CRNP principal investigator
Thomas Jefferson University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Total-body irradiation, Donor lymphocytes infusion (DLI) and Cyclophosphamide in people with Malignant Neoplasm, run by Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University 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 11 inclusion and 11 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: stopped early, as of the sponsor's last update on 16 May 2025. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 9 Jan 2018). 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 Participants With Disease-free Survival (DFS) (time frame: 1 year). 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 16 May 2025.