Mesenchymal Stem Cells (MSCs) for Steroid Refractory Acute GVHD (SR-aGVHD)

NCT05443464 | Withdrawn Phase 1 DMC FDA Drug

• 1 other identifier: OSSM-001-006-01
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief 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).

Conditions

GVHD,Acute

Outcome Measures

Primary Outcomes (1)

• Safety events

  OSSM-001 related safety events

  112 days (16 weeks)

Secondary Outcomes (1)

• Maximum tolerated dose (MTD)

  112 days (16 weeks)

Other Outcomes (5)

• Overall survival (OS) from initiation of therapy

  112 days (16 weeks)

• Overall survival (OS) by baseline GVHD grading

  112 days (16 weeks)

• Overall survival (OS) stratified by organ involvement

  112 days (16 weeks)

Study Arms (4)

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.

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.

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.

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.

Biological: OSSM-001

Interventions

OSSM-001 BIOLOGICAL

Direct IV infusion of OSSM-001

12M cells/kg; 24M cells/kg; 2M cells/kg; 6M cells/kg

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• i. Requirement for an increase in the corticosteroid dose to methylprednisolone ≥2 mg/kg/day (or equivalent prednisone dose ≥2.5 mg/kg/day), OR
• ii. 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.

You may not qualify if:

• 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.
• 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)

Sponsors & Collaborators

• Ossium Health, Inc.: lead

Study Officials

• Sagar Munjal

  Ossium Health, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 29, 2022
• First Posted: July 5, 2022
• Study Start: June 5, 2023
• Primary Completion: June 10, 2024
• Study Completion: October 14, 2024
• Last Updated: December 23, 2022

Record last verified: 2022-12

Data Sharing

• IPD Sharing: Will not share