Phase 2
N=76
Multicenter Study Of Natalizumab Plus Standard Steroid Treatment For High Risk Acute Graft-Versus-Host Disease
Acute Graft Versus Host Disease
Bottom Line
View on ClinicalTrials.gov: NCT02133924 ↗Enrolled (actual)
76
Serious AEs
68.0%
Results posted
Oct 2022
Primary outcome: Primary: Number of Participants With Complete Response (CR) — 34 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- natalizumab (Drug); steroids (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- John Levine
- Primary completion
- Dec 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Complete Response (CR) |
34 | — |
| SECONDARY Number of Participants With Overall Survival (OS) |
37 | — |
| SECONDARY Number of Participants With Non-Relapse Mortality (NRM) |
25; 28 | — |
| SECONDARY Number of Participants With SR GVHD |
30 | — |
| SECONDARY Time to Discontinuation of Steroid Therapy |
108 | — |
| SECONDARY Number of Participants Who Received Additional GVHD Therapies |
33 | — |
| SECONDARY Number of Serious Infections |
52 | — |
| SECONDARY Number of Participants With Overall Response Rate (CR + PR) |
45 | — |
Summary
This research trial is designed to study the safety and effectiveness of combining the study drug, Natalizumab (Tysabri®) with the standard treatment, the use of steroids, as a new treatment for acute graft versus host disease (acute GVHD). GVHD is the most common serious complication, after bone marrow transplant. GVHD occurs when the donor cells (the graft), treat the recipient's body as "foreign" and attack the cells in the recipient's body. During this immune system response, donor cells damage body tissues, such as the skin, liver, stomach, and/or intestines. Acute GVHD can be severe and if severe, potentially fatal to the transplant recipient. Acute GVHD usually happens within the first several months after transplant.
The goal of this research is to develop a safer and more effective treatment for acute GVHD, and particularly for acute GVHD that affects the gastrointestinal (or GI) tract, with the ultimate goal being safer and more effective transplant therapies for blood cancers such as leukemia, lymphoma, and multiple myeloma.
Eligibility Criteria
Inclusion Criteria
- New onset high risk acute GVHD (Ann Arbor score 2 or3 as defined in Appendix C of the protocol) following allogeneic bone marrow transplantation. Any clinical severity (Glucksberg grade I-IV) is eligible. Patients with prior or existing diagnosis of GVHD without any treatment are eligible. Patients given only topical corticosteroids for skin GVHD are eligible.
- Any donor type (e.g., related, unrelated) or stem cell source (bone marrow, peripheral blood, cord blood). Recipients of non-myeloablative and myeloablative transplants are eligible.
- No prior systemic treatment for acute GVHD except for a maximum of 3 days of prednisone ≤2 mg/kg/day (or IV methylprednisolone). Topical skin steroid treatment, non-absorbable oral steroid treatment for GI GVHD, and resumption of GVHD prophylaxis agents (e.g., calcineurin inhibitors) are permissible. Patients enrolled in BMT CTN 1501 who randomized to sirolimus are also eligible.
- Age 18 years or older.
- Direct bilirubin must be 0.5 mg/kg/d of methylprednisolone or equivalent within 7 days prior to initiation of GVHD treatment
- Patients on dialysis
- Patients requiring ventilator support
- Investigational agent within 30 days of enrollment without approval from the Sponsor-Investigator
Data sourced from ClinicalTrials.gov (NCT02133924). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.