Hematological MalignanciesPhase 1/2NCT01816230
Transplantation of NiCord®, Umbilical Cord Blood-derived Ex Vivo Expanded Cells, in Patients With HM
A Phase 1/2 study testing NiCord® in people with Hematological Malignancies and Acute Lymphoblastic Leukemia (ALL), run by Gamida Cell ltd at 15 sites in 5 countries.
Trial summary
A Study Evaluating the Safety and Efficacy of Transplantation of a single cord blood unit (CBU) of NiCord®, umbilical cord blood-derived Ex Vivo Expanded Stem and Progenitor Cells in Patients with Hematological Malignancies. Read more about this study
- Who is it for?
- Ages 12 to 65 years
- Conditions the study is for
- Hematological Malignancies, Acute Lymphoblastic Leukemia (ALL), Acute Myeloid Leukemia (AML)
- What is being tested?
- NiCord®
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 15 sitesin 5 countries
- Study dates
- Apr 2013to Jun 2018
- Phase
- Phase 1/2
- 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 12 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Applicable disease and eligible for myeloablative SCT
- Patients must have two partially HLA-matched CBUs
- Back-up stem cell source
- Adequate Karnofsky Performance score or Lansky Play-Performance scale
- Sufficient physiological reserves
- Signed written informed consent
Who cannot join
- HLA-matched donor able to donate
- Prior allogeneic HSCT
- Other active malignancy
- Active or uncontrolled infection
- Active/symptoms of central nervous system (CNS) disease
- Pregnancy or lactation
Trial locations and contacts
15 sites in 5 countries.
15 sites in 5 countries
United States8 sites
Cleveland Clinic
Cleveland, Ohio, 44195
Duke University Medical Center
Durham, North Carolina, 27710
Cedars-Sinai Medical Center
Los Angeles, California, 90048
Loyola University, Cardinal Bernardin Cancer Center
Maywood, Illinois, 60153
University of Minnesota
Minneapolis, Minnesota, 55455
Vanderbilt
Nashville, Tennessee, 37240
Steven and Alexandra Cohen Children's Medical Center
New York, New York, 11040
Oregon Health & Science University
Portland, Oregon, 97239
Italy2 sites
Children Hospital Gaslini Scientific Institute
Genoa, 16147
University of Turin
Turin, 10043
Singapore2 sites
National University Cancer Institute
Singapore, 119074
Singapore General Hospital
Singapore, 169608
Spain2 sites
University Hospital Vall d´Hebron
Barcelona, 08035
Hospital Universitario La Fe
Valencia, 46009
About this study
Official title: Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-derived Ex Vivo Expanded Stem and Progenitor Cells, in Adolescent and Adult Patients With Hematological Malignancies
A Study Evaluating the Safety and Efficacy of Transplantation of a single cord blood unit (CBU) of NiCord®, umbilical cord blood-derived Ex Vivo Expanded Stem and Progenitor Cells in Patients with Hematological Malignancies.
Read the detailed description
Umbilical cord blood (UCB) is an alternative stem cell source for hematopoietic stem cell transplantations (HSCT) and can be used for the treatment of various life-threatening diseases, such as hematological malignancies or genetic blood disorders, in such cases where a matched related stem cell donor is not available. However, the major drawback of using this valuable stem cells source is the limited cell dose in a single cord blood unit (CBU), which was shown to be associated with inadequate hematopoietic reconstitution and high risk of transplant-related mortality. To improve outcomes and extend applicability of UCB transplantation, one potential solution is ex vivo expansion of UCB-derived stem and progenitor cells. NiCord® is a stem/progenitor cell based product composed of ex vivo expanded allogeneic UCB cells. NiCord® is based on a novel technology for the ex vivo cell expansion of cord blood derived hematopoietic progenitor cells. By increasing the number of the short and long-term reconstitution progenitor cells transplanted, NiCord® has the potential to enable the broader application of UCB transplantation, and improve the clinical outcomes of UCB transplantation.
The study is designed as a multi center, single arm study, evaluating the safety and efficacy of the transplantation of NiCord® to patients with hematological malignancies following myeloablative therapy.
Total study duration is approximately 400 days from the signing of informed consent to the last visit one year following transplantation
The overall study objective is to evaluate the safety and efficacy of NiCord®: single ex-vivo expanded cord blood unit transplantation in patients with hematological malignancies following myeloablative therapy as follows:
The main study objectives are to assess the cumulative incidence of patients with NiCord®-derived neutrophil engraftment at 42 days following transplantation and to assess the incidence of secondary graft failure at 180 days following transplantation of NiCord® Ten evaluable patients recruited for the study should be 12-65 years of age, up to a maximum of 15 treated patients.
- Conditions
- Hematological Malignancies, Acute Lymphoblastic Leukemia (ALL), Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS)
- Being tested
- NiCord®
- Medical subject headings
- Hematologic Neoplasms, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myeloid, Acute, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NiCord®Drug
| Study group | Role | What they receive |
|---|---|---|
| NiCord® | Experimental | NiCord® is a stem/progenitor cell based product composed of ex vivo expanded allogeneic UCB cells. Receives: Drug: NiCord® |
What is being measured
The primary outcome is the main question the study is designed to answer.
Engraftment
Assess the cumulative incidence of patients with NiCord®-derived neutrophil engraftment at 42 days following transplantation.
Measured: 42 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Mar 2013 (estimated)
- Study startApr 2013
- Primary completionJun 2017
- Study completionJun 2018
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hematological MalignanciesStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gamida Cell ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mitchell Horwitz, MD, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01816230
- Sponsor's protocol ID
- GC P#03.01.020
- Status last verified
- Oct 2020
- First submitted
- 19 Mar 2013
- Last updated
- 22 Oct 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Horwitz ME, Wease S, Blackwell B, Valcarcel D, Frassoni F, Boelens JJ, Nierkens S, Jagasia M, Wagner JE, Kuball J, Koh LP, Majhail NS, Stiff PJ, Hanna R, Hwang WYK, Kurtzberg J, Cilloni D, Freedman LS, Montesinos P, Sanz G. Phase I/II Study of Stem-Cell Transplantation Using a Single Cord Blood Unit Expanded Ex Vivo With Nicotinamide. J Clin Oncol. 2019 Feb 10;37(5):367-374. doi: 10.1200/JCO.18.00053. Epub 2018 Dec 4.
PubMed 30523748
Study at a glance
From the registry record, last updated by the sponsor on 22 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-derived Ex Vivo Expanded Stem and Progenitor Cells, in Adolescent and Adult Patients With Hematological Malignancies
- ClinicalTrials.gov ID
- NCT01816230
- Acronym / sponsor ID
- GC P#03.01.020
- Conditions
- Hematological Malignancies, Acute Lymphoblastic Leukemia (ALL), Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS)
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Gamida Cell ltdIndustry (pharma, biotech, devices)
- Study start
- Apr 2013
- Primary completion
- Jun 2017 (actual)
- Study completion
- Jun 2018 (actual)
- First posted
- 22 Mar 2013 (estimated)
- Results
- Posted 21 Sep 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A Study Evaluating the Safety and Efficacy of Transplantation of a single cord blood unit (CBU) of NiCord®, umbilical cord blood-derived Ex Vivo Expanded Stem and Progenitor Cells in Patients with Hematological Malignancies.
Detailed description
Umbilical cord blood (UCB) is an alternative stem cell source for hematopoietic stem cell transplantations (HSCT) and can be used for the treatment of various life-threatening diseases, such as hematological malignancies or genetic blood disorders, in such cases where a matched related stem cell donor is not available. However, the major drawback of using this valuable stem cells source is the limited cell dose in a single cord blood unit (CBU), which was shown to be associated with inadequate hematopoietic reconstitution and high risk of transplant-related mortality. To improve outcomes and extend applicability of UCB transplantation, one potential solution is ex vivo expansion of UCB-derived stem and progenitor cells. NiCord® is a stem/progenitor cell based product composed of ex vivo expanded allogeneic UCB cells. NiCord® is based on a novel technology for the ex vivo cell expansion of cord blood derived hematopoietic progenitor cells. By increasing the number of the short and long-term reconstitution progenitor cells transplanted, NiCord® has the potential to enable the broader application of UCB transplantation, and improve the clinical outcomes of UCB transplantation.
The study is designed as a multi center, single arm study, evaluating the safety and efficacy of the transplantation of NiCord® to patients with hematological malignancies following myeloablative therapy.
Total study duration is approximately 400 days from the signing of informed consent to the last visit one year following transplantation
The overall study objective is to evaluate the safety and efficacy of NiCord®: single ex-vivo expanded cord blood unit transplantation in patients with hematological malignancies following myeloablative therapy as follows:
The main study objectives are to assess the cumulative incidence of patients with NiCord®-derived neutrophil engraftment at 42 days following transplantation and to assess the incidence of secondary graft failure at 180 days following transplantation of NiCord® Ten evaluable patients recruited for the study should be 12-65 years of age, up to a maximum of 15 treated patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| NiCord® | Experimental | Drug: NiCord®NiCord® is a stem/progenitor cell based product composed of ex vivo expanded allogeneic UCB cells. |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Applicable disease and eligible for myeloablative SCT
- Patients must have two partially HLA-matched CBUs
- Back-up stem cell source
- Adequate Karnofsky Performance score or Lansky Play-Performance scale
- Sufficient physiological reserves
Key exclusion criteria (6 listed)
- HLA-matched donor able to donate
- Prior allogeneic HSCT
- Other active malignancy
- Active or uncontrolled infection
- Active/symptoms of central nervous system (CNS) disease
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 |
|---|---|---|
| NiCord® | Drug | NiCord® |
Primary outcome
Engraftment
Time frame: 42 days
Assess the cumulative incidence of patients with NiCord®-derived neutrophil engraftment at 42 days following transplantation.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Oct 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 30523748Horwitz ME, Wease S, Blackwell B, Valcarcel D, Frassoni F, Boelens JJ, Nierkens S, Jagasia M, Wagner JE, Kuball J, Koh LP, Majhail NS, Stiff PJ, Hanna R, Hwang WYK, Kurtzberg J, Cilloni D, Freedman LS, Montesinos P, Sanz G. Phase I/II Study of Stem-Cell Transplantation Using a Single Cord Blood Unit Expanded Ex Vivo With Nicotinamide. J Clin Oncol. 2019 Feb 10;37(5):367-374. doi: 10.1200/JCO.18.00053. Epub 2018 Dec 4.
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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.
Mitchell Horwitz, MD principal investigator
Duke University
15 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing NiCord® in people with Hematological Malignancies and Acute Lymphoblastic Leukemia (ALL), run by Gamida Cell ltd at 15 sites in 5 countries.
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 6 inclusion and 6 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 22 Oct 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States, Italy, Singapore, Spain, Netherlands. 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?
Yes. Results are posted in the registry (first posted 21 Sep 2020). 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?
Engraftment (time frame: 42 days). 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 NCT01816230, which also lists 1 linked publication.
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 22 Oct 2020.