Hematologic MalignanciesPhase 2NCT01328496

Umbilical Cord Blood Transplantation In Patients With Hematologic Malignancies Using A Myeloablative Preparative Regimen

A Phase 2 study testing Preparative Regimen in people with Hematologic Malignancies and Disorder Related to Transplantation, run by St. Jude Children's Research Hospital at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

In this study, participants with high-risk hematologic malignancies undergoing hematopoietic cell transplantation (HCT), who do not have a suitable human leukocyte antigen (HLA)-matched related/sibling donor (MSD), matched unrelated donor (MURD) or killer-immunoglobulin receptors (KIR) ligand mismatched haploidentical… Read more about this study

Who is it for?: Ages Up to 21 years
Conditions the study is for: Hematologic Malignancies, Disorder Related to Transplantation, Hematopoietic Malignancy
What is being tested?: Preparative Regimen
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 14 participants
Where: 1 siteUnited States
Study dates: 15 Jun 2011to 31 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 Up to 21 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

Age less than or equal to 21 years old.
Has a partially HLA-matched single or double UCB product
High-risk hematologic malignancy.
High risk ALL in CR1, ALL in High risk CR2, ALL in CR3 or subsequent.
AML in high risk CR1, AML in CR2 or subsequent
AML in first relapse with < 25% blasts in BM

Show all 22Show fewer

Therapy related AML, with prior malignancy in CR > 12mo
MDS, primary or secondary
NK cell, biphenotypic, or undifferentiated leukemia in CR1 or subsequent.
CML in accelerated phase, or in chronic phase with persistent molecular positivity or intolerance to tyrosine kinase inhibitor.
Hodgkin lymphoma in CR2 or subsequent after failure of prior autologous HCT, or unable to mobilize stem cells for autologous HCT.
Non-Hodgkin lymphoma in CR2 or subsequent after failure of prior autologous HCT, or unable to mobilize stem cells for autologous HCT.
JMML
All patients with evidence of CNS leukemia must be treated and be in CNS CR to be eligible for study.
Patient must fulfill pre-transplant evaluation:
Cardiac shortening fraction ≥ 26%.
Creatinine clearance ≥ 70 ml/min/1.73m2.
Forced vital capacity (FVC) ≥ 50% of predicted value or pulse oximetry ≥ 92% on room air.
Karnofsky (≥ 16 years) or Lansky (<16 years) performance score ≥ 70
Bilirubin ≤ 2.5 mg/dL.
Alanine aminotransferase (ALT) ≤ 5 times the upper limit of normal for age.
Aspartate aminotransferase (AST) ≤ 5 times the upper limit of normal for age.

Who cannot join

Patient has a suitable MSD, volunteer MURD, or KIR mismatched haploidentical donor available in the necessary time for stem cell donation.
Patient has any other active malignancy other than the one for which HCT is indicated.
Patient had a prior allogeneic HCT
Patient had an autologous HCT within the previous 12 months.
Patient is pregnant as confirmed by positive serum or urine pregnancy test within 14 days prior to enrollment.
Patient is lactating
Patient has Down Syndrome
Patient has a current uncontrolled bacterial, fungal, or viral infection per the judgment of the PI.

Trial locations and contacts

1 site in 1 country.

United States1 site

St. Jude Children's Research Hospital
Memphis, Tennessee, 38105

Other trials in United States

About this study

The preparative regimen includes fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (10.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.

Read the detailed description

The primary objectives is to estimate the event-free survival (EFS) at one-year post-transplant for research participants with high-risk hematologic malignancies undergoing hematopoietic cell transplantation (HCT) using single unit umbilical cord blood (UCB).

Secondary objectives are:

Describe the clinical outcome of patients undergoing a double unit UCBT.
Estimate the incidence and severity of acute and chronic graft versus host disease (GVHD) of patients enrolled in the research arm.
Estimate the incidence and time to neutrophil and platelet engraftment among patients enrolled in the research arm.
Estimate the incidence of transplant related mortality (TRM) and transplant related morbidity in the first 100 days after transplantation among patients enrolled in the research

Exploratory Objectives are:

Assess the relationship between pre-transplant minimal residual disease (MRD) with transplant outcomes.
Record immune reconstitution parameters, including chimerism analysis, quantitative lymphocyte subsets, T cell receptor excision circle (TREC) and spectratyping. Immunophenotyping and functional assays of T, B and NK cells and lymphocytes will also be evaluated.
Evaluate the determinants of engraftment.

Conditions: Hematologic Malignancies, Disorder Related to Transplantation, Hematopoietic Malignancy
Being tested: Preparative Regimen
Keywords: Hematologic Malignancies, Umbilical Cord Blood transplantation, Hematopoietic Cell Transplantation
Medical subject headings: Hematologic Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Preparative RegimenDrug
Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2.
Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.

Study groups in NCT01328496
Study group	Role	What they receive
Research Arm	Other	Participant with high-risk hematologic malignancies undergoing Hematopoietic Cell Transplantation, who do not have a suitable Human Leukocyte Antigen -matched related/sibling donor, Matched Unrelated Donor or Killer immunoglobulin receptors ligand mismatched haploidentical donor identified, will receive a single UCB unit. Intervention: Preparative Regimen Receives: Drug: Preparative Regimen
Observation Arm	Other	Patients requiring two UCB units will be eligible for UCBT01 on the observational arm. Intervention: Preparative Regimen Receives: Drug: Preparative Regimen

What is being measured

The primary outcome is the main question the study is designed to answer.

Event Free Survival (EFS) for Research Participants
Estimate EFS for research participants at one-year post transplant by using single unit umbilical cord blood. The event is defined as relapse, graft failure, death due to any cause. The number of participants who did not experience any of those events (relapse, graft failure, death due to any cause) at year 1 post-transplant was given.
Measured: 1 year post-transplant

Secondary outcomes (9)

Number of Observational Arm Participants Engrafted
For patients enrolled in the observational arm (undergoing a double unit UCBT), the number of patients engrafted was given.
Measured: 1 year
Number of Observational Arm Patients Who Relapsed
The number of observational arm patients who relapsed was given.
Measured: 1 year
Number of Deaths of Observational Arm Patients
The number of observational arm patients who died was given.
Measured: 1 year
Number of Observational Arm Patients With Transplant-related Mortality (TRM)
The number of patients with TRM within the first 100 days post transplant was given.
Measured: First 100 days
Number of Participants With Acute GVHD
The number of participants with incidence of acute GVHD by grade was given. Participants are graded on a scale from 1 to 4, with 1 being mild and 4 being severe.
Measured: 1 year
Number of Participants With Chronic GVHD
Due to the small sample size, cumulative incidence analysis was not done. The incidence of chronic GVHD was evaluated using NIH Consensus Global Severity Scoring. The number of patients with incidence of chronic GVHD by severity was provided.
Measured: 1 year
Time to Engraftment of Research Arm Participants
Platelet engraftment was defined as platelet count ≥20,000/mm^3 for 3 consecutive tests performed on different days with no platelet transfusions in the preceding 7 days. Neutrophil engraftment will be defined as achieving ANC ≥ 500/mm3 for 3 consecutive tests performed on different days with evidence of donor cell engraftment. Descriptive statistics are provided.
Measured: first 100 days post transplant
Incidence of Transplant-related Mortality (TRM)
TRM is death occurring in patients in continuous complete remission. The numbers of patients with TRM was given.
Measured: first 100 days post transplant
The Number of Participants With Transplant-related Morbidity
Any patient who had adverse events listed either as probable or definite in the first 100 days post-transplant are counted as transplant related morbidity. The number of patients with transplant-related morbidity was given.
Measured: first 100 days post transplant

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

15 Jun 201131 Oct 2016

First posted4 Apr 2011 (estimated)
Study start15 Jun 2011
Primary completion31 Oct 2016
Study completion31 Oct 2016

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 Hematologic MalignanciesStudies of the same condition, open and under way.
More trials run by St. Jude Children's Research HospitalThe 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 2
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Enrolment: 14 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01328496
Sponsor's protocol ID: UCBT01
Other IDs: NCI-2011-03700
Status last verified: Mar 2017
First submitted: 31 Mar 2011
Last updated: 5 Jun 2017
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No

Study at a glance

From the registry record, last updated by the sponsor on 5 Jun 2017. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Umbilical Cord Blood Transplantation In Patients With Hematologic Malignancies Using A Myeloablative Preparative Regimen
ClinicalTrials.gov ID: NCT01328496
Acronym / sponsor ID: UCBT01
Conditions: Hematologic Malignancies, Disorder Related to Transplantation, Hematopoietic Malignancy
Keywords: Hematologic Malignancies, Umbilical Cord Blood transplantation, Hematopoietic Cell Transplantation
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 14 participants (actual)
Lead sponsor: St. Jude Children's Research HospitalUniversity, hospital or other
Collaborators: The Hartwell Foundation, Assisi Foundation
Study start: 15 Jun 2011 (actual)
Primary completion: 31 Oct 2016 (actual)
Study completion: 31 Oct 2016 (actual)
First posted: 4 Apr 2011 (estimated)
Results: Posted 5 May 2017

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Secondary objectives are:

Describe the clinical outcome of patients undergoing a double unit UCBT.
Estimate the incidence and severity of acute and chronic graft versus host disease (GVHD) of patients enrolled in the research arm.
Estimate the incidence and time to neutrophil and platelet engraftment among patients enrolled in the research arm.
Estimate the incidence of transplant related mortality (TRM) and transplant related morbidity in the first 100 days after transplantation among patients enrolled in the research

Exploratory Objectives are:

Assess the relationship between pre-transplant minimal residual disease (MRD) with transplant outcomes.
Record immune reconstitution parameters, including chimerism analysis, quantitative lymphocyte subsets, T cell receptor excision circle (TREC) and spectratyping. Immunophenotyping and functional assays of T, B and NK cells and lymphocytes will also be evaluated.
Evaluate the determinants of engraftment.

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Research Arm	Other	Drug: Preparative RegimenParticipant with high-risk hematologic malignancies undergoing Hematopoietic Cell Transplantation, who do not have a suitable Human Leukocyte Antigen -matched related/sibling donor, Matched Unrelated Donor or Killer immunoglobulin receptors ligand mismatched haploidentical donor identified, will receive a single UCB unit. Intervention: Preparative Regimen
Observation Arm	Other	Drug: Preparative RegimenPatients requiring two UCB units will be eligible for UCBT01 on the observational arm. Intervention: Preparative Regimen

Who may qualify

Ages: Up to 21 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (22 listed)

Age less than or equal to 21 years old.
Has a partially HLA-matched single or double UCB product
High-risk hematologic malignancy.
High risk ALL in CR1, ALL in High risk CR2, ALL in CR3 or subsequent.
AML in high risk CR1, AML in CR2 or subsequent

Key exclusion criteria (8 listed)

Patient has a suitable MSD, volunteer MURD, or KIR mismatched haploidentical donor available in the necessary time for stem cell donation.
Patient has any other active malignancy other than the one for which HCT is indicated.
Patient had a prior allogeneic HCT
Patient had an autologous HCT within the previous 12 months.
Patient is pregnant as confirmed by positive serum or urine pregnancy test within 14 days prior to enrollment.

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
Preparative RegimenFludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.	Drug	Observation Arm, Research Arm

Primary outcome

Event Free Survival (EFS) for Research Participants
Time frame: 1 year post-transplant
Estimate EFS for research participants at one-year post transplant by using single unit umbilical cord blood. The event is defined as relapse, graft failure, death due to any cause. The number of participants who did not experience any of those events (relapse, graft failure, death due to any cause) at year 1 post-transplant was given.

9 secondary outcomes

Number of Observational Arm Participants Engrafted
Time frame: 1 year
Number of Observational Arm Patients Who Relapsed
Time frame: 1 year
Number of Deaths of Observational Arm Patients
Time frame: 1 year
Number of Observational Arm Patients With Transplant-related Mortality (TRM)
Time frame: First 100 days
Number of Participants With Acute GVHD
Time frame: 1 year
Number of Participants With Chronic GVHD
Time frame: 1 year
Time to Engraftment of Research Arm Participants
Time frame: first 100 days post transplant
Incidence of Transplant-related Mortality (TRM)
Time frame: first 100 days post transplant
The Number of Participants With Transplant-related Morbidity
Time frame: first 100 days post transplant

Results and safety

Results posted to the registry on 5 May 2017, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Research Arm	10	9
Observational Arm	4	4

Primary outcome: Event Free Survival (EFS) for Research Participants

Time frame: 1 year post-transplant. Count of participants. Unit: Participants

Measure	Research Arm n = 9
Value	4

9 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.. Other adverse events reported at 00% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Research Arm	7 of 9 (77.8%)	9 of 9 (100%)	4 of 9
Observation Arm	3 of 4 (75%)	4 of 4 (100%)	2 of 4

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 5 Jun 2017.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
St. Jude Children's Research HospitalLink provided by the sponsor.
Clinical Trials Open at St. JudeLink 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.

Amr Qudeimat, MD principal investigator

St. Jude Children's Research Hospital

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 Preparative Regimen in people with Hematologic Malignancies and Disorder Related to Transplantation, run by St. Jude Children's Research Hospital 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 22 inclusion and 8 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 5 Jun 2017. 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 5 May 2017). 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?

Event Free Survival (EFS) for Research Participants (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?

On the ClinicalTrials.gov record for NCT01328496.

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 5 Jun 2017.