Acute Myelocytic LeukemiaPhase 1/2NCT01474681
Safety and Tolerability of HSC835 in Patients With Hematological Malignancies
A Phase 1/2 study testing HSC835 in people with Acute Myelocytic Leukemia and Acute Lymphocytic Leukemia, run by Novartis Pharmaceuticals at 1 site in United States.
Trial summary
This study evaluated the safety and tolerability of using HSC835 in patients with hematological malignancies. Read more about this study
- Who is it for?
- Ages 10 to 55 years
- Conditions the study is for
- Acute Myelocytic Leukemia, Acute Lymphocytic Leukemia, Chronic Myelogenous Leukemia
- What is being tested?
- HSC835
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 1 siteUnited States
- Study dates
- 9 Jan 2012to 3 Oct 2016
- 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 10 to 55 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients with a diagnosis that qualifies them for a DUCBT
- Absence of recent active mold infection
- Adequate organ function
- Availability of eligible donor material
Who cannot join
- Pregnancy or breastfeeding women and women of child-bearing potential unless two acceptable forms of contraception are being used
- Human immunodeficiency virus (HIV) infection
- Active infection
- Extensive prior chemotherapy
- Prior myeloablative allotransplantation or autologous transplant.
Trial locations and contacts
1 site in 1 country.
United States1 site
Novartis Investigative Site
Minneapolis, Minnesota, 55455
About this study
Official title: A First-in-human, Single-arm, Single-center, Open-label, Proof-of-concept Study to Evaluate the Safety and Tolerability of Infusing HSC835 (LFU835-expanded Umbilical Cord Blood Hematopoietic Stem Cells) in Patients With Hematological Malignancies
This study evaluated the safety and tolerability of using HSC835 in patients with hematological malignancies.
- Conditions
- Acute Myelocytic Leukemia, Acute Lymphocytic Leukemia, Chronic Myelogenous Leukemia, Myelodysplastic Syndrome, Chronic Lymphocytic Leukemia, Marginal Zone Lymphoma
- Being tested
- HSC835
- Keywords
- hematologic malignancies, leukemia, lymphoma
- Medical subject headings
- Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Myelodysplastic Syndromes, Leukemia, Lymphocytic, Chronic, B-Cell, Lymphoma, B-Cell, Marginal Zone, Lymphoma, Follicular, Dendritic Cell Sarcoma, Interdigitating
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HSC835Biological
| Study group | Role | What they receive |
|---|---|---|
| HSC835 | Experimental | HSC835 infusion Receives: Biological: HSC835 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Tolerability of HSC835 for Clinical Use Were Measured by Infusional Toxicity (Within First 48 Hours After Transplant) and Absence of Graft Failure After 32 Days in Excess of That Currently Observed With UCBT.
The safety and tolerability of HSC835 for clinical use were measured by infusional toxicity and absence of graft failure in excess of that currently observed with UCBT. Infusional toxicity - AE from transplant until first 48 hours. Administration of the HSC835 expanded CD34-positive cell product, infused over a period of approximately 15 minutes may theoretically cause adverse reactions based on hemodynamic effects, the release of factors like cytokines through administration into the systemic circulation, or acute hypersensitivity, among others.
Measured: 32 days
Secondary outcomes (8)
Incidence of Neutrophil Recovery Within 42 Days
Neutrophil recovery (engraftment) is defined as the first of three consecutive days with ANC > 0.5 x 109/L which occurred for all patients before 42 days post transplant.
Measured: 42 days
Incidence of Platelet Recovery Within Six Months
Incidence of platelet recovery within six months. Number of participants recovering platelet to ≥50,000 × 109/L for at least one week without transfusion in the prior 7 days to the first measurement.
Measured: 6 months
Frequency of Expanded Unit Predominance at Day 100 (DUCBT Recipients Only)
Frequency of expanded unit predominance at day 100 (DUCBT recipients only) unit predominance was assessed by differences in microsatellite patterns between the recipient, HSC835 and the unmanipulated cord blood unit. Evaluation of sorted CD15-positive/CD33-positive myeloid and CD3-positive T cells in the peripheral blood, revealed three patterns: predominance of HSC835, Mixed dominance an unique chimerism pattern was observed with the CD15/CD33 population predominantly derived from HSC835 and the CD3 population almost exclusively derived from the unmanipulated unit, and predominance of the unmanipulated unit
Measured: Day 100
Incidence of Transplant Related Mortality (TRM) Within 100 Days and One Year
Number of participants with incidence of transplant related mortality (TRM) within 100 days and one year
Measured: Day 100 and Month 12
Incidence of Acute Graft Versus Host Disease (aGVHD) Within 100 Days and Chronic Graft Versus Host Disease (cGVHD) Within 1 Year
Number of participants with incidence of Acute Graft Versus Host Disease (aGVHD) within 100 days and Chronic Graft Versus Host Disease (cGVHD) within 1 year
Measured: Day 100 and Monnth 12
Incidence of Relapse Within One Year
Number of participants with Incidence of relapse within one year
Measured: Month 12
Overall Survival (OS) Within One Year
Number of participants with Overall survival (OS) within one year
Measured: Month 12
Disease Free Survival (DFS) Within One Year
Number of participants with Disease Free Survival (DFS) within one year. Patients are considered to have achieved DFS or relapse-free survival if they had not experienced either relapse or death (of any cause)
Measured: Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2011 (estimated)
- Study start9 Jan 2012
- Primary completion3 Oct 2016
- Study completion3 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 Acute Myelocytic LeukemiaStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01474681
- Sponsor's protocol ID
- CHSC835X2201
- Status last verified
- Mar 2019
- First submitted
- 15 Nov 2011
- Last updated
- 30 Dec 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 30 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A First-in-human, Single-arm, Single-center, Open-label, Proof-of-concept Study to Evaluate the Safety and Tolerability of Infusing HSC835 (LFU835-expanded Umbilical Cord Blood Hematopoietic Stem Cells) in Patients With Hematological Malignancies
- ClinicalTrials.gov ID
- NCT01474681
- Acronym / sponsor ID
- CHSC835X2201
- Conditions
- Acute Myelocytic Leukemia, Acute Lymphocytic Leukemia, Chronic Myelogenous Leukemia, Myelodysplastic Syndrome, Chronic Lymphocytic Leukemia, Marginal Zone Lymphoma
- Keywords
- hematologic malignancies, leukemia, lymphoma
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 9 Jan 2012 (actual)
- Primary completion
- 3 Oct 2016 (actual)
- Study completion
- 3 Oct 2016 (actual)
- First posted
- 18 Nov 2011 (estimated)
- Results
- Posted 12 May 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study evaluated the safety and tolerability of using HSC835 in patients with hematological malignancies.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HSC835 | Experimental | Biological: HSC835HSC835 infusion |
Who may qualify
- Ages
- 10 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Patients with a diagnosis that qualifies them for a DUCBT
- Absence of recent active mold infection
- Adequate organ function
- Availability of eligible donor material
Key exclusion criteria (5 listed)
- Pregnancy or breastfeeding women and women of child-bearing potential unless two acceptable forms of contraception are being used
- Human immunodeficiency virus (HIV) infection
- Active infection
- Extensive prior chemotherapy
- Prior myeloablative allotransplantation or autologous transplant.
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 |
|---|---|---|
| HSC835 | Biological | HSC835 |
Primary outcome
Safety and Tolerability of HSC835 for Clinical Use Were Measured by Infusional Toxicity (Within First 48 Hours After Transplant) and Absence of Graft Failure After 32 Days in Excess of That Currently Observed With UCBT.
Time frame: 32 days
The safety and tolerability of HSC835 for clinical use were measured by infusional toxicity and absence of graft failure in excess of that currently observed with UCBT. Infusional toxicity - AE from transplant until first 48 hours. Administration of the HSC835 expanded CD34-positive cell product, infused over a period of approximately 15 minutes may theoretically cause adverse reactions based on hemodynamic effects, the release of factors like cytokines through administration into the systemic circulation, or acute hypersensitivity, among others.
8 secondary outcomes
Incidence of Neutrophil Recovery Within 42 Days
Time frame: 42 days
Incidence of Platelet Recovery Within Six Months
Time frame: 6 months
Frequency of Expanded Unit Predominance at Day 100 (DUCBT Recipients Only)
Time frame: Day 100
Incidence of Transplant Related Mortality (TRM) Within 100 Days and One Year
Time frame: Day 100 and Month 12
Incidence of Acute Graft Versus Host Disease (aGVHD) Within 100 Days and Chronic Graft Versus Host Disease (cGVHD) Within 1 Year
Time frame: Day 100 and Monnth 12
Incidence of Relapse Within One Year
Time frame: Month 12
Overall Survival (OS) Within One Year
Time frame: Month 12
Disease Free Survival (DFS) Within One Year
Time frame: Month 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Dec 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- A Plain Language Trial Summary is available on novartisclinicaltrials.comLink 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.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
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 HSC835 in people with Acute Myelocytic Leukemia and Acute Lymphocytic Leukemia, run by Novartis Pharmaceuticals 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 4 inclusion and 5 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 30 Dec 2020. 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 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 12 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?
Safety and Tolerability of HSC835 for Clinical Use Were Measured by Infusional Toxicity (Within First 48 Hours After Transplant) and Absence of Graft Failure After 32 Days in Excess of That Currently Observed With UCBT. (time frame: 32 days). 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 30 Dec 2020.