GVHD,AcutePhase 1NCT05443464
Mesenchymal Stem Cells (MSCs) for Steroid Refractory Acute GVHD (SR-aGVHD)
A Phase 1 study testing OSSM-001 in people with GVHD,Acute, run by Ossium Health, Inc..
Trial summary
The objective of this clinical study is to demonstrate safety and feasibility of single-dose infusion of Ossium MSCs product (OSSM-001) to treat steroid refractory acute GVHD (SR-aGVHD). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- GVHD,Acute
- What is being tested?
- OSSM-001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 5 Jun 2023to 14 Oct 2024
- Phase
- Phase 1
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Minimum 18 years of age who have undergone hematopoietic cell transplantation (HCT), from any donor source donor (including HLA-matched related and unrelated, haploidentical and umbilical cord donors) with any conditioning regimen
- Clinically diagnosed Grades II to IV acute GVHD as per standard criteria occurring within 100 days of HCT
- Confirmed diagnosis of steroid refractory aGVHD defined as patients administered high-dose systemic corticosteroids (methylprednisolone 2 mg/kg/day [or equivalent prednisone dose 2.5 mg/kg/day]), given alone or combined with calcineurin inhibitors (CNI) and either:
- Progressing based on organ assessment after at least 3 days at the time of initiation of high-dose systemic corticosteroid +/- CNI for the treatment of Grade II-IV aGVHD, OR
- Failure to achieve at a minimum partial response based on organ assessment after 7 days compared to organ stage at the time of initiation of high-dose systemic corticosteroid +/- CNI for the treatment of Grade II-IV aGVHD, OR
- Patients who fail corticosteroid taper defined as fulfilling either one of the following criteria:
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- Requirement for an increase in the corticosteroid dose to methylprednisolone ≥2 mg/kg/day (or equivalent prednisone dose ≥2.5 mg/kg/day), OR
- Failure to taper the methylprednisolone dose to <0.5 mg/kg/day (or equivalent prednisone dose <0.6 mg/kg/day) for a minimum 7 days.
- Minimum Karnofsky Performance Level of at least 30 or higher at the time of study entry.
Who cannot join
- Has received more than one systemic treatment for steroid refractory aGVHD in addition to steroids.
- Received stem cell therapy in the past
- Presence of an active uncontrolled infection including significant bacterial, fungal, viral, or parasitic infection requiring treatment
- Presence of relapsed primary malignancy, or who have been treated for relapse after the HCT was performed, or who may require rapid immune suppression withdrawal as pre-emergent treatment of early malignancy relapse.
- Evidence of pulmonary infiltrate or hemorrhage based on imaging or requiring high flow oxygen via face mask
- Patients who have had treatment with any other investigational agent, device, or procedure within 30days (or 5 half-lives, whichever is greater) prior to enrollment.
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- Patients who have received more than one HCT
- Any medical or psychological condition or situation deemed by the Investigators to put the patient at increased risk of complications or non-compliance.
- Unresolved veno-occlusive disease
- HLA antibody screen positive for HLA antibodies specific against the MSCs products
- ALT or AST > 5X of upper limit of normal
- Serum Bilirubin >2 X of upper limit of normal
- GFR <50 ml/min
- SpO2 <94% despite being on continuous supplemental oxygen
- Patients requiring continuous >4L/minute of supplemental oxygen (irrespective of oxygen saturation)
About this study
Official title: A Phase I Safety Study of Single Dose Allogeneic Bone Marrow Derived MSCs for Steroid Refractory Acute Graft vs. Host Disease
The objective of this clinical study is to demonstrate safety and feasibility of single-dose infusion of Ossium MSCs product (OSSM-001) to treat steroid refractory acute GVHD (SR-aGVHD).
Read the detailed description
The dose escalation will be used to establish a maximum tolerated dose, starting from 2M cells/kg. If no safety issues are seen, then MSCs dose maybe escalated to 6M cells/kg, 12M cells/kg and 24M cell/kg based on data review from each cohort. Additional studies will be conducted to establish a dosing regimen and to evaluate the safety and efficacy of OSSM-001 for SR-aGVHD patients.
- Conditions
- GVHD,Acute
- Being tested
- OSSM-001
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OSSM-001Biological
Direct IV infusion of OSSM-001
| Study group | Role | What they receive |
|---|---|---|
| 2M cells/kg | Experimental | Dose level 1. 3 subjects will receive OSSM-001 at 2M cells/kg and followed for 28 days post dose to observe for DLT. Receives: Biological: OSSM-001 |
| 6M cells/kg | Experimental | Dose level 2. If no DLTs are observed in the previous dose level, 3 subjects will receive OSSM-001 at 6M cells/kg and followed for 28 days post dose to observe for DLT. Receives: Biological: OSSM-001 |
| 12M cells/kg | Experimental | Dose level 3. If no DLTs are observed in the previous dose level, 3 subjects will receive OSSM-001 at 12M cells/kg and followed for 28 days post dose to observe for DLT. Receives: Biological: OSSM-001 |
| 24M cells/kg | Experimental | Dose level 4. If no DLTs are observed in the previous dose level, 3 subjects will receive OSSM-001 at 24M cells/kg and followed for 28 days post dose to observe for DLT. Receives: Biological: OSSM-001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety events
OSSM-001 related safety events
Measured: 112 days (16 weeks)
Secondary outcomes (1)
Maximum tolerated dose (MTD)
Determine MTD of OSSM-001
Measured: 112 days (16 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jul 2022
- Study start5 Jun 2023 (estimated)
- Primary completion10 Jun 2024 (estimated)
- Study completion14 Oct 2024 (estimated)
Why it stopped: OSSM-001 will not be used in this program moving forward.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for GVHD,AcuteStudies 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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ossium Health, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sagar Munjal, Study director, Ossium Health, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05443464
- Sponsor's protocol ID
- OSSM-001-006-01
- Status last verified
- Dec 2022
- First submitted
- 29 Jun 2022
- Last updated
- 23 Dec 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Safety Study of Single Dose Allogeneic Bone Marrow Derived MSCs for Steroid Refractory Acute Graft vs. Host Disease
- ClinicalTrials.gov ID
- NCT05443464
- Acronym / sponsor ID
- OSSM-001-006-01
- Conditions
- GVHD,Acute
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: OSSM-001 will not be used in this program moving forward.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Ossium Health, Inc.Industry (pharma, biotech, devices)
- Study start
- 5 Jun 2023 (estimated)
- Primary completion
- 10 Jun 2024 (estimated)
- Study completion
- 14 Oct 2024 (estimated)
- First posted
- 5 Jul 2022 (actual)
- 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.
The objective of this clinical study is to demonstrate safety and feasibility of single-dose infusion of Ossium MSCs product (OSSM-001) to treat steroid refractory acute GVHD (SR-aGVHD).
Detailed description
The dose escalation will be used to establish a maximum tolerated dose, starting from 2M cells/kg. If no safety issues are seen, then MSCs dose maybe escalated to 6M cells/kg, 12M cells/kg and 24M cell/kg based on data review from each cohort. Additional studies will be conducted to establish a dosing regimen and to evaluate the safety and efficacy of OSSM-001 for SR-aGVHD patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 2M cells/kg | Experimental | Biological: OSSM-001Dose level 1. 3 subjects will receive OSSM-001 at 2M cells/kg and followed for 28 days post dose to observe for DLT. |
| 6M cells/kg | Experimental | Biological: OSSM-001Dose level 2. If no DLTs are observed in the previous dose level, 3 subjects will receive OSSM-001 at 6M cells/kg and followed for 28 days post dose to observe for DLT. |
| 12M cells/kg | Experimental | Biological: OSSM-001Dose level 3. If no DLTs are observed in the previous dose level, 3 subjects will receive OSSM-001 at 12M cells/kg and followed for 28 days post dose to observe for DLT. |
| 24M cells/kg | Experimental | Biological: OSSM-001Dose level 4. If no DLTs are observed in the previous dose level, 3 subjects will receive OSSM-001 at 24M cells/kg and followed for 28 days post dose to observe for DLT. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Minimum 18 years of age who have undergone hematopoietic cell transplantation (HCT), from any donor source donor (including HLA-matched related and unrelated, haploidentical and umbilical cord donors) with any conditioning regimen
- Clinically diagnosed Grades II to IV acute GVHD as per standard criteria occurring within 100 days of HCT
- Confirmed diagnosis of steroid refractory aGVHD defined as patients administered high-dose systemic corticosteroids (methylprednisolone 2 mg/kg/day [or equivalent prednisone dose 2.5 mg/kg/day]), given alone or combined with calcineurin inhibitors (CNI) and either:
- Minimum Karnofsky Performance Level of at least 30 or higher at the time of study entry.
Key exclusion criteria (15 listed)
- Has received more than one systemic treatment for steroid refractory aGVHD in addition to steroids.
- Received stem cell therapy in the past
- Presence of an active uncontrolled infection including significant bacterial, fungal, viral, or parasitic infection requiring treatment
- Presence of relapsed primary malignancy, or who have been treated for relapse after the HCT was performed, or who may require rapid immune suppression withdrawal as pre-emergent treatment of early malignancy relapse.
- Evidence of pulmonary infiltrate or hemorrhage based on imaging or requiring high flow oxygen via face mask
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 |
|---|---|---|
| OSSM-001Direct IV infusion of OSSM-001 | Biological | 12M cells/kg, 24M cells/kg, 2M cells/kg, 6M cells/kg |
Primary outcome
Safety events
Time frame: 112 days (16 weeks)
OSSM-001 related safety events
1 secondary outcome
Maximum tolerated dose (MTD)
Time frame: 112 days (16 weeks)
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Dec 2022.
- Individual participant dataSharing planned: No
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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.
Sagar Munjal study director
Ossium Health, Inc.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing OSSM-001 in people with GVHD,Acute, run by Ossium Health, Inc..
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 9 inclusion and 15 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: withdrawn, as of the sponsor's last update on 23 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.
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 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.
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 was withdrawn before enrolling participants, so there are no results.
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 events (time frame: 112 days (16 weeks)). 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 23 Dec 2022.