Hematologic MalignancyPhase 1/2NCT02668315
Expanded Cord Blood in Patients in Need of an Allogeneic Stem Cell Transplant
A Phase 1/2 study testing Transplantation of cord blood expanded with UM171 in people with Hematologic Malignancy, run by Maisonneuve-Rosemont Hospital at 1 site in Canada.
Trial summary
Allogeneic hematopoietic stem cell transplantation is a life-saving procedure in patients with blood cancers, but only 25% of transplant candidates have a sibling donor. A matched unrelated donor can be found for 60% of patients but this number is lower for non-Caucasians. Read more about this study
- Who is it for?
- Ages 3 to 64 years
- Conditions the study is for
- Hematologic Malignancy
- What is being tested?
- Transplantation of cord blood expanded with UM171
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 1 siteCanada
- Study dates
- 16 Dec 2015to Aug 2018
- Phase
- Phase 1/2
- 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 3 to 64 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 3-64 years old (Pediatric patients will become eligible only after 5 adult patients have been enrolled in the study and received the UM171 expanded product).
- Weight ≥ 12kg
- Underlying hematologic malignancy excluding primary myelofibrosis requiring an unrelated donor allogeneic HSC transplant (as defined by the transplant center) but patient lacks an 8/8 HLA identical donor or disease requires an urgent transplant and cannot wait the required time to identify an unrelated donor.
- Availability of at least 3 cord bloods with HLA match ≥ 4/6 and ≥4/8 and meeting the following requirements:
- CB to be expanded: CD34+ cell count 1.0-4.9 x 10E5/kg for cohort 1, 0.5-4.9 x 10E5/kg for cohort 2, and 0.25-4.9 x 10E5/kg for cohort 3; nucleated cell count ≥ 2.0 x 10E7/kg for cohort 1, ≥ 1.5 x 10E7/kg for cohort 2, and ≥ 1.25 x 10E7/kg for cohort 3.
- Non expanded cord: TNC count ≥ 2.0 x 10E7/kg with CD34 min of 1 x 10E5/kg or minimum of 1.5 x 10E7 TNC/kg with 1.8 x 10E5 CD34+ cells/kg.
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- Back up cord: TNC count ≥ 1.5 x 10E7/kg with CD34 min of 1 x 10E5/kg
- Left ventricular ejection fraction > 40%.
- Karnofsky score ≥ 70%
- Forced vital capacity (FVC), forced expiratory volume in 1 second (FEV1) and diffusing capacity corrected for hemoglobin (DLCOc) ≥ 50% of predicted
- Bilirubin < 2 x upper limit of normal (ULN) unless felt to be related to Gilbert's disease or hemolysis; AST (aspartate aminotransferase) and ALT (alanine aminotransférase) ≤ 2.5 x ULN; alkaline phosphatase ≤ 5 x ULN.
- Measured or estimated creatinine clearance ≥ 60 ml/min/1.73m2.
Who cannot join
- Myeloablative transplant within 24 months.
- Uncontrolled infection.
- Presence of a malignancy other than the one for which the UCB transplant is being performed and the expected survival related to the malignancy is estimated to be less than 75% at 5 years.
- HIV positivity.
- Hepatitis B or C infection with measurable viral load.
- Liver cirrhosis.
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- Availability of a cord with ≥ 5 x 105/kg CD34+ cells.
- Pregnancy, breastfeeding or unwillingness to use appropriate contraception.
- Participation in a trial with an investigational agent within 30 days prior to entry in the study.
- Patient unable to give informed consent or unable to comply with the treatment protocol including appropriate supportive care, follow-up, and tests.
- Any abnormal condition or laboratory result that is considered by the principal investigator capable of altering patient condition or study outcome.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase I-II Open-label Study of UM171 Expanded Cord Blood in a Fed-batch Culture System (UFCB-001) in Patients Who Need an Allogeneic Hematopoietic Stem Cell Transplant But Lack a Suitable Donor
Allogeneic hematopoietic stem cell transplantation is a life-saving procedure in patients with blood cancers, but only 25% of transplant candidates have a sibling donor. A matched unrelated donor can be found for 60% of patients but this number is lower for non-Caucasians. Cord blood (CB), another source of stem cells, has major advantages over unrelated donors including immediate availability, better permissiveness in immune mismatches between donor and transplant recipient, better availability for non-Caucasians, and less graft versus host disease, a complication frequently seen after transplant which negatively affects quality of life. Unfortunately, the use of CB is still limited in adults because of the small number of stem cells. UM171, a molecule with hematopoietic stem cell expansion properties, has been shown to increase cord blood stem cells 13 fold. In this trial, Investigators will use UM171 treated CB in patients who need a transplant but lack an acceptable donor.This protocol seeks to test the safety of CB cells expanded with UM171, and to determine the kinetics of engraftment as well as the minimal cord blood unit cell dose that when expanded achieves prompt engraftment.
Read the detailed description
Investigators are proposing a phase I-II, Canadian multi-center, open-label study of UM171 ex vivo expanded CB transplant in 25 patients who need an allogeneic hematopoietic stem cell transplantation (HSCT) but lack a Human Leucocyte Antigen (HLA) matched donor.
Investigators key primary and secondary objectives include:
- To establish the feasibility of expanding cord blood units for allogeneic cord blood transplantation.
- To establish the safety and identify unexpected toxicities associated with the transplantation of cord blood cells expanded with UM171/fed-batch culture system.
- To measure kinetics of neutrophil and platelet recovery.
- To determine minimal cord blood unit cell dose (Total Nucleated Cell (TNC)/CD34+ cells) that when expanded achieves prompt engraftment as a single cord transplant.
Methodology:
Patients with a hematologic malignancy and an indication for allogeneic HSCT who lack a matched unrelated donor will receive a myeloablative or submyeloablative conditioning regimen followed by infusion of UM171 expanded CB graft. Accrual is expected to last 18 months and patients will be followed for 3 years.
Expected benefits:
Investigators expect that expansion with UM171/fed-batch will be safe and lead to both rapid and sustained engraftment. This will likely decrease the high early morbidity/mortality of CB HSCT and improve access to transplant, especially ethnic minorities. In addition, if low cell dose is solved, patients will benefit from CB's lower risk of chronic (graft versus host disease) GVHD, a major cause of morbidity.
.
- Conditions
- Hematologic Malignancy
- Being tested
- Transplantation of cord blood expanded with UM171
- Medical subject headings
- Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Transplantation of cord blood expanded with UM171Biological
Myeloablative or submyeloablative conditioning regimen followed by an expanded cord blood transplant
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Intervention name: Transplantation of cord blood expanded with UM171 Prethaw CB cell count prior to manipulation: CD34+ cell count 1.0-4.9 x 10E5/kg and TNC superior or equal to 2.0 x 10E7/kg Receives: Biological: Transplantation of cord blood expanded with UM171 |
| Cohort 2 | Experimental | Intervention name: Transplantation of cord blood expanded with UM171 Prethaw CB cell count prior to manipulation: CD34+ cell count 0.5-4.9 x 10E5/kg and TNC superior or equal to 1.5 x 10E7/kg Receives: Biological: Transplantation of cord blood expanded with UM171 |
| Cohort 3 | Experimental | Intervention name: Transplantation of cord blood expanded with UM171 Prethaw CB cell count prior to manipulation: CD34+ cell count 0.25-4.9 x 10E5/kg and TNC superior or equal to 1.25 x 10E7/kg Receives: Biological: Transplantation of cord blood expanded with UM171 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Monitoring adverse events, toxicities and medical evolution
To identify unexpected toxicities associated with transplantation using cord blood cells expanded with UM171/fed-batch culture system by means of history, physical examination and laboratory evaluation. All adverse events will be evaluated for duration, intensity, and causal relationship with the study medication and followed to the end of the study or until resolution.
Measured: up to 36 months post transplant
Secondary outcomes (15)
Feasibility of cord blood expansion using UM171 (transplant success)
The feasibility of expanding cord blood units for allogeneic cord blood transplantation in adults will be evaluated by determining the percentage of selected grafts that will be successfully expanded in the absence of technical hurdles such as contamination or unexpected technical failure, and that will meet release criteria and be successfully infused in patients.
Measured: 18 months
Kinetics of hematopoietic recovery
Time to neutrophil engraftment is defined as the first day of attainment of an absolute neutrophil count (ANC) ≥ 0.5 x 109/L for 3 consecutive days as per Center for International Blood and Marrow Transplant Research (CIBMTR) standards.
Platelet engraftment is defined as the first day of a sustained platelet count ≥ 20 x 109/L with no platelet transfusion in the preceding 7 days as per CIBMTR standards.
Measured: 42 days post transplant
Minimal cord blood cell dose that when expanded achieves prompt engraftment
The minimal cord blood unit cell dose (TNC/CD34+) that, once expanded, achieves prompt engraftment as a single cord transplant will be defined as the lower cord blood cell dose that satisfies all of the following conditions for umbilical cord blood (UCB) dose reduction , i.e.:
- Minimum of 3 patients who will have received a single cord transplant (no back-up cord),
- Cord to be expanded contains less than 2.0 x 105 CD34+/kg at thaw pre-expansion in cohort 1 and less than 1.0 x 105 CD34+/kg at thaw pre-expansion in cohort 2
- Engraftment must have occurred within 18 days.
Measured: up to 12 months
Correlation between neutrophil and platelet engraftment and CD34+ and CD34+CD45RA- dose
Time to neutrophil and platelet engraftment will be correlated to the CD34+ and the CD34+CD45RA- dose (which includes all human hematopoietic stem cells (HSCs): long term and short term repopulating cells) contained in the expanded graft. Expansion and cultures might change the characteristics and behaviour of CD34+ cells, hence the need to look at the correlation between primitive CD34+CD45RA- subpopulation and engraftment.
Measured: 42 days post transplant
Incidence of primary and late graft failure
The patient will be declared as having developed primary graft failure if:
- Lack of neutrophil engraftment by day 42.
- Infusing another stem cell source prior to day 42 (other than the nonmanipulated cord and back up cord).
- < 5% donor chimerism after day +14 up to and including day +42 after transplant.
- Death from day 28 to 42 without neutrophil engraftment.
Secondary or late graft failure when:
- ANC drops below 0.5 x 109/L for 14 consecutive days unresponsive to growth colony stimulating factor (G-CSF) witout identifiable cause (medication, viral infection, vitamin deficience or other)
Measured: 42 days post transplant
Incidence of backup cord infusion and graft dominance
This evaluation will be performed in lymphoid and myeloid cells to determine the expanded cord's role in short and long term engraftment in patients. Cell sorting to examine chimerism of different cell subsets will be performed with complex of differentiation (CD) such as CD3, CD33, CD56, CD14 and CD19 to determine the proportions in which donor's immune cells are represented in the graft recipient.
Donor cell engraftment is defined as donor cell (lymphoid or myeloid) chimerism ≥ 5% by day +42 post transplant. Full donor chimerism is defined as ≥ 95% donor myeloid and lymphoid chimerism.
Measured: 7, 14, 21, 28, 56, 100 days and at 6 and 12 months
Incidence of acute and chronic GVHD
The incidence of acute and chronic GVHD will be evaluated throughout the study using NIH criteria. Acute GVHD will be defined as classic acute (time of onset of symptoms ≤ 100 days) or persistent, recurrent, or late onset acute GVHD (time of onset of symptoms > 100 days) as defined by the NIH.
Patients will be assessed for the presence of chronic GVHD at each medical evaluation. Data will be collected directly from providers and chart review according to the recommendations of the NIH Consensus Conference.
Measured: through study completion (3 years follow-up)
Incidence of severe infectious complications
any grade 3 infection will be tabulated as well as any of the following infections requiring systemic therapy will be captured: invasive candidiasis, aspergillus, other invasive fungi, cytomegalovirus (CMV), adenovirus, Epstein-Barr virus (EBV), Human herpes virus (HHV-6), herpes simplex (HSV), varicella-zoster virus (VZV), Pneumocystis carinii pneumonitis (PCP), toxoplasmosis and mycobacterium. Severity of infections will be graded according to the Technical Manual of Procedure of the Blood and Marrow Transplant Clinical Trials Network.
Measured: through study completion (3 years follow-up)
T cell evaluation (Cluster of differentiation (CD) 4+ and CD8+)
Multiparametric flow cytometry to quantify the proportion of naïve (CD45RA+/CD27+) and memory (CD45RA-/CD27+) cells.
Measured: 3, 6, 12, and 18 months
B cell evaluation
The outcome (success or failure) in B cell recovery will be evaluated by flow cytometry, immunoglobulin measurements and PCR for donor cell chimerism.
Measured: 3, 6, 12, and 18 months
Natural Killer (NK) cell evaluation
The outcome (success or failure) in NK cell recovery will be evaluated by flow cytometry and PCR for donor cell chimerism.
Measured: 3, 6, 12, and 18 months
Transplant related mortality (TRM)
Incidence of TRM will be monitored throughout the study and in particular at 100 days, 1, 2, and 3 years post transplantation. If it should exceed 30% at day 100, stopping rules may apply that could prematurely terminate the trial.
Measured: 100 days and 1,2, 3 years post transplant
progression free survival
Progression free survival (PFS) will be monitored throughout the study and in particular at 1, 2, and 3 years post-transplant.
PFS will be measured from time of transplant until progression, death or last follow-up.
PFS: an event is defined as relapse/progression or death.
Measured: 100 days an 1,2, 3 years post transplant
Overall survival
Overall survival (OS) will be monitored throughout the study and in particular at 1, 2, and 3 years post-transplant.
OS will be measured from time of transplant until progression, death or last follow-up.
OS: an event is defined as death.
Measured: 100 days an 1,2, 3 years post transplant
Incidence of engraftment syndrome requiring therapy
Patients will be monitored for the occurrence of engraftment and preengraftment syndromes (ES and PES) as well as the necessity for treatment. Diagnostic criteria for ES are noninfectious fever plus any of the following: skin rash, pulmonary infiltrates, hepatic dysfunction, or diarrhea.
Measured: 24 hours post transplant and during follow-up (3 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jan 2016 (estimated)
- Study start16 Dec 2015
- Primary completionAug 2018
- Study completionAug 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Hematologic MalignancyStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Factorial
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Maisonneuve-Rosemont Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Canadian Cancer Society (CCS), Canadian Institutes of Health Research (CIHR), Hopital de l'Enfant-Jesus, Vancouver General Hospital, St. Justine's Hospital, Stem Cell Network
- Responsible party
- Principal investigator, Sandra Cohen, Maisonneuve-Rosemont Hospital
- Investigators
- Sandra Cohen, MD, Principal investigator, Maisonneuve-Rosemont Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02668315
- Sponsor's protocol ID
- UM0128171
- Status last verified
- Aug 2019
- First submitted
- 15 Jan 2016
- Last updated
- 5 Aug 2019
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided
Publications
Liu B, Klatt D, Zhou Y, Manis JP, Sauvageau G, Pellin D, Brendel C, Williams DA. UM171 enhances fitness and engraftment of gene-modified hematopoietic stem cells from patients with sickle cell disease. Blood Adv. 2024 Nov 26;8(22):5885-5895. doi: 10.1182/bloodadvances.2024013932.
PubMed 39293082Cohen S, Bambace N, Ahmad I, Roy J, Tang X, Zhang MJ, Burns L, Barabe F, Bernard L, Delisle JS, Kiss T, Lachance S, Roy DC, Veilleux O, Sauvageau G. Improved outcomes of UM171-expanded cord blood transplantation compared with other graft sources: real-world evidence. Blood Adv. 2023 Oct 10;7(19):5717-5726. doi: 10.1182/bloodadvances.2023010599.
PubMed 37467030Dumont-Lagace M, Li Q, Tanguay M, Chagraoui J, Kientega T, Cardin GB, Brasey A, Trofimov A, Carli C, Ahmad I, Bambace NM, Bernard L, Kiss TL, Roy J, Roy DC, Lemieux S, Perreault C, Rodier F, Dufresne SF, Busque L, Lachance S, Sauvageau G, Cohen S, Delisle JS. UM171-Expanded Cord Blood Transplants Support Robust T Cell Reconstitution with Low Rates of Severe Infections. Transplant Cell Ther. 2021 Jan;27(1):76.e1-76.e9. doi: 10.1016/j.bbmt.2020.09.031. Epub 2020 Oct 3.
PubMed 33022376Cohen S, Roy J, Lachance S, Delisle JS, Marinier A, Busque L, Roy DC, Barabe F, Ahmad I, Bambace N, Bernard L, Kiss T, Bouchard P, Caudrelier P, Landais S, Larochelle F, Chagraoui J, Lehnertz B, Corneau S, Tomellini E, van Kampen JJA, Cornelissen JJ, Dumont-Lagace M, Tanguay M, Li Q, Lemieux S, Zandstra PW, Sauvageau G. Hematopoietic stem cell transplantation using single UM171-expanded cord blood: a single-arm, phase 1-2 safety and feasibility study. Lancet Haematol. 2020 Feb;7(2):e134-e145. doi: 10.1016/S2352-3026(19)30202-9. Epub 2019 Nov 6.
PubMed 31704264
Study at a glance
From the registry record, last updated by the sponsor on 5 Aug 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I-II Open-label Study of UM171 Expanded Cord Blood in a Fed-batch Culture System (UFCB-001) in Patients Who Need an Allogeneic Hematopoietic Stem Cell Transplant But Lack a Suitable Donor
- ClinicalTrials.gov ID
- NCT02668315
- Acronym / sponsor ID
- UM0128171
- Conditions
- Hematologic Malignancy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Maisonneuve-Rosemont HospitalUniversity, hospital or other
- Collaborators
- Canadian Cancer Society (CCS), Canadian Institutes of Health Research (CIHR), Hopital de l'Enfant-Jesus, Vancouver General Hospital, St. Justine's Hospital, Stem Cell Network
- Study start
- 16 Dec 2015 (actual)
- Primary completion
- Aug 2018 (actual)
- Study completion
- Aug 2018 (actual)
- First posted
- 29 Jan 2016 (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.
Allogeneic hematopoietic stem cell transplantation is a life-saving procedure in patients with blood cancers, but only 25% of transplant candidates have a sibling donor. A matched unrelated donor can be found for 60% of patients but this number is lower for non-Caucasians. Cord blood (CB), another source of stem cells, has major advantages over unrelated donors including immediate availability, better permissiveness in immune mismatches between donor and transplant recipient, better availability for non-Caucasians, and less graft versus host disease, a complication frequently seen after transplant which negatively affects quality of life. Unfortunately, the use of CB is still limited in adults because of the small number of stem cells. UM171, a molecule with hematopoietic stem cell expansion properties, has been shown to increase cord blood stem cells 13 fold. In this trial, Investigators will use UM171 treated CB in patients who need a transplant but lack an acceptable donor.This protocol seeks to test the safety of CB cells expanded with UM171, and to determine the kinetics of engraftment as well as the minimal cord blood unit cell dose that when expanded achieves prompt engraftment.
Detailed description
Investigators are proposing a phase I-II, Canadian multi-center, open-label study of UM171 ex vivo expanded CB transplant in 25 patients who need an allogeneic hematopoietic stem cell transplantation (HSCT) but lack a Human Leucocyte Antigen (HLA) matched donor.
Investigators key primary and secondary objectives include:
- To establish the feasibility of expanding cord blood units for allogeneic cord blood transplantation.
- To establish the safety and identify unexpected toxicities associated with the transplantation of cord blood cells expanded with UM171/fed-batch culture system.
- To measure kinetics of neutrophil and platelet recovery.
- To determine minimal cord blood unit cell dose (Total Nucleated Cell (TNC)/CD34+ cells) that when expanded achieves prompt engraftment as a single cord transplant.
Methodology:
Patients with a hematologic malignancy and an indication for allogeneic HSCT who lack a matched unrelated donor will receive a myeloablative or submyeloablative conditioning regimen followed by infusion of UM171 expanded CB graft. Accrual is expected to last 18 months and patients will be followed for 3 years.
Expected benefits:
Investigators expect that expansion with UM171/fed-batch will be safe and lead to both rapid and sustained engraftment. This will likely decrease the high early morbidity/mortality of CB HSCT and improve access to transplant, especially ethnic minorities. In addition, if low cell dose is solved, patients will benefit from CB's lower risk of chronic (graft versus host disease) GVHD, a major cause of morbidity.
.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Factorial
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Biological: Transplantation of cord blood expanded with UM171Intervention name: Transplantation of cord blood expanded with UM171 Prethaw CB cell count prior to manipulation: CD34+ cell count 1.0-4.9 x 10E5/kg and TNC superior or equal to 2.0 x 10E7/kg |
| Cohort 2 | Experimental | Biological: Transplantation of cord blood expanded with UM171Intervention name: Transplantation of cord blood expanded with UM171 Prethaw CB cell count prior to manipulation: CD34+ cell count 0.5-4.9 x 10E5/kg and TNC superior or equal to 1.5 x 10E7/kg |
| Cohort 3 | Experimental | Biological: Transplantation of cord blood expanded with UM171Intervention name: Transplantation of cord blood expanded with UM171 Prethaw CB cell count prior to manipulation: CD34+ cell count 0.25-4.9 x 10E5/kg and TNC superior or equal to 1.25 x 10E7/kg |
Who may qualify
- Ages
- 3 to 64 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (12 listed)
- 3-64 years old (Pediatric patients will become eligible only after 5 adult patients have been enrolled in the study and received the UM171 expanded product).
- Weight ≥ 12kg
- Underlying hematologic malignancy excluding primary myelofibrosis requiring an unrelated donor allogeneic HSC transplant (as defined by the transplant center) but patient lacks an 8/8 HLA identical donor or disease requires an urgent transplant and cannot wait the required time to identify an unrelated donor.
- Availability of at least 3 cord bloods with HLA match ≥ 4/6 and ≥4/8 and meeting the following requirements:
- Left ventricular ejection fraction > 40%.
Key exclusion criteria (11 listed)
- Myeloablative transplant within 24 months.
- Uncontrolled infection.
- Presence of a malignancy other than the one for which the UCB transplant is being performed and the expected survival related to the malignancy is estimated to be less than 75% at 5 years.
- HIV positivity.
- Hepatitis B or C infection with measurable viral load.
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 |
|---|---|---|
| Transplantation of cord blood expanded with UM171Myeloablative or submyeloablative conditioning regimen followed by an expanded cord blood transplant | Biological | Cohort 1, Cohort 2, Cohort 3 |
Primary outcome
Monitoring adverse events, toxicities and medical evolution
Time frame: up to 36 months post transplant
To identify unexpected toxicities associated with transplantation using cord blood cells expanded with UM171/fed-batch culture system by means of history, physical examination and laboratory evaluation. All adverse events will be evaluated for duration, intensity, and causal relationship with the study medication and followed to the end of the study or until resolution.
15 secondary outcomes
Feasibility of cord blood expansion using UM171 (transplant success)
Time frame: 18 months
Kinetics of hematopoietic recovery
Time frame: 42 days post transplant
Minimal cord blood cell dose that when expanded achieves prompt engraftment
Time frame: up to 12 months
Correlation between neutrophil and platelet engraftment and CD34+ and CD34+CD45RA- dose
Time frame: 42 days post transplant
Incidence of primary and late graft failure
Time frame: 42 days post transplant
Incidence of backup cord infusion and graft dominance
Time frame: 7, 14, 21, 28, 56, 100 days and at 6 and 12 months
Incidence of acute and chronic GVHD
Time frame: through study completion (3 years follow-up)
Incidence of severe infectious complications
Time frame: through study completion (3 years follow-up)
T cell evaluation (Cluster of differentiation (CD) 4+ and CD8+)
Time frame: 3, 6, 12, and 18 months
B cell evaluation
Time frame: 3, 6, 12, and 18 months
Natural Killer (NK) cell evaluation
Time frame: 3, 6, 12, and 18 months
Transplant related mortality (TRM)
Time frame: 100 days and 1,2, 3 years post transplant
progression free survival
Time frame: 100 days an 1,2, 3 years post transplant
Overall survival
Time frame: 100 days an 1,2, 3 years post transplant
Incidence of engraftment syndrome requiring therapy
Time frame: 24 hours post transplant and during follow-up (3 years)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Aug 2019.
- Derived from this study: PubMed 39293082Liu B, Klatt D, Zhou Y, Manis JP, Sauvageau G, Pellin D, Brendel C, Williams DA. UM171 enhances fitness and engraftment of gene-modified hematopoietic stem cells from patients with sickle cell disease. Blood Adv. 2024 Nov 26;8(22):5885-5895. doi: 10.1182/bloodadvances.2024013932.
- Derived from this study: PubMed 37467030Cohen S, Bambace N, Ahmad I, Roy J, Tang X, Zhang MJ, Burns L, Barabe F, Bernard L, Delisle JS, Kiss T, Lachance S, Roy DC, Veilleux O, Sauvageau G. Improved outcomes of UM171-expanded cord blood transplantation compared with other graft sources: real-world evidence. Blood Adv. 2023 Oct 10;7(19):5717-5726. doi: 10.1182/bloodadvances.2023010599.
- Derived from this study: PubMed 33022376Dumont-Lagace M, Li Q, Tanguay M, Chagraoui J, Kientega T, Cardin GB, Brasey A, Trofimov A, Carli C, Ahmad I, Bambace NM, Bernard L, Kiss TL, Roy J, Roy DC, Lemieux S, Perreault C, Rodier F, Dufresne SF, Busque L, Lachance S, Sauvageau G, Cohen S, Delisle JS. UM171-Expanded Cord Blood Transplants Support Robust T Cell Reconstitution with Low Rates of Severe Infections. Transplant Cell Ther. 2021 Jan;27(1):76.e1-76.e9. doi: 10.1016/j.bbmt.2020.09.031. Epub 2020 Oct 3.
- Derived from this study: PubMed 31704264Cohen S, Roy J, Lachance S, Delisle JS, Marinier A, Busque L, Roy DC, Barabe F, Ahmad I, Bambace N, Bernard L, Kiss T, Bouchard P, Caudrelier P, Landais S, Larochelle F, Chagraoui J, Lehnertz B, Corneau S, Tomellini E, van Kampen JJA, Cornelissen JJ, Dumont-Lagace M, Tanguay M, Li Q, Lemieux S, Zandstra PW, Sauvageau G. Hematopoietic stem cell transplantation using single UM171-expanded cord blood: a single-arm, phase 1-2 safety and feasibility study. Lancet Haematol. 2020 Feb;7(2):e134-e145. doi: 10.1016/S2352-3026(19)30202-9. Epub 2019 Nov 6.
- Individual participant dataSharing planned: Undecided
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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.
Sandra Cohen, MD principal investigator
Maisonneuve-Rosemont Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Transplantation of cord blood expanded with UM171 in people with Hematologic Malignancy, run by Maisonneuve-Rosemont Hospital at 1 site in Canada.
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 12 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: finished, as of the sponsor's last update on 5 Aug 2019. 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 Canada. 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 combined 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.
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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Monitoring adverse events, toxicities and medical evolution (time frame: up to 36 months 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 NCT02668315, which also lists 4 linked publications.
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 Aug 2019.