Phase II Trial of Natalizumab + Prednisone for Initial Therapy of Acute GI GVHD
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|ClinicalTrials.gov Identifier: NCT02176031|
Recruitment Status : Completed
First Posted : June 26, 2014
Results First Posted : April 17, 2020
Last Update Posted : April 17, 2020
|Condition or disease||Intervention/treatment||Phase|
|Graft Versus Host Disease||Drug: Natalizumab Drug: Methylprednisolone||Phase 2|
Natalizumab is a drug that was initially discovered as a treatment for autoimmune conditions. Natalizumab has been approved for use in patients with Multiple Sclerosis and Crohn's disease. In these diseases, the drug works to inhibit dysfunctional immune cells that are responsible for the symptoms seen in these diseases. Acute graft versus host disease is caused by a similar dysfunction of immune cells; Natalizumab is thought to inhibit these immune cells, similarly to how it does in Multiple Sclerosis and Crohn's disease. In this research study,the investigators are looking to see whether Natalizumab provides additional benefit to patients receiving standard treatment for acute graft versus host disease of the gastrointestinal tract.
Participants who fulfill eligibility criteria will be entered into the trial to receive Natalizumab.
- Participant will receive a dose of the natalizumab through intravenous infusion. Participants may receive a second dose at Day 28 if they experience a partial response or very good partial response.
- Scheduled Physical Examination at screening, during the week of first dose and at 28 days, 56 days, 100 days, 180 days and one year.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||21 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase II Trial of Natalizumab (Tysabri®) Plus Prednisone for Initial Therapy of Acute Graft Versus Host Disease (aGVHD) of the Gastrointestinal Tract|
|Study Start Date :||January 2015|
|Actual Primary Completion Date :||February 2019|
|Actual Study Completion Date :||February 2019|
Other Name: Tysabri®
Other Name: Steroid
- GVHD-free Survival Rate [ Time Frame: Day 56 ]Graft-versus-host disease (GVHD) free survival is defined as achieving complete response without death or relapse or requiring secondary immunosuppressive therapy . Proportions are reported descriptively. GVHD-free survival was assessed using the Kaplan-Meier method.
- Graft-verus-host Disease (GVHD) Response Rate [ Time Frame: 28 Days, 56 Days ]
- Complete Response (CR) is defined as resolution of all signs and symptoms of acute GVHD.
- Very Good Partial Response (VGPR) is defined by no rash or residual erythematous rash involving less than 25% of the body surface, and total serum bilirubin concentration less than 2 mg/dL or less than 25% of baseline at enrollment and tolerating food or enteral feeding, predominantly formed stools, no overt gastrointestinal bleeding or abdominal cramping, and no more than occasional nausea and vomiting.
- Partial Response (PR) is defined as an improvement of one stage in one or more organs without progression in any other organ.
- Non-response (NR) is defined as no reduction in any GVHD organ staging.
- Progression is defined as either new organ involvement on day +8 or thereafter, or increased organ specific symptoms sufficient to increase the organ stage by one or more or the initiation of an additional GVHD agent.
- Overall Response Rate (ORR) is the sum of CR, VGPR, and PR.
- GI aGVHD Response Rate [ Time Frame: Day 28, Day 56 ]Gastrointestinal (GI) acute graft-versus-host disease (GVHD) Response is defined by complete response, very good partial response, or partial response in signs and symptoms of GI aGVHD.
- Overall Survival (OS) Rate [ Time Frame: 2 years ]Overall survival (OS) is defined from the date of natalizumab infusion to death or censored at last clinical evaluation. OS was estimated using the Kaplan-Meier method.
- Rate of GVHD Flares [ Time Frame: by Day 28 ]Number of subjects who experienced graft-versus-host disease (GVHD) flares requiring therapy after initial complete response (CR) or partial response (PR) by day 28 after the first dose of Natalizumab.
- Percentage Steroid Dose Was Reduced at Day 28, 56, and 100 in Comparison to Steroid Dose at First Administration of Natalizumab. [ Time Frame: Day 28, 56, and 100 ]Median percentage steroid dose was reduced at Day 28, Day 56, and Day 100 in comparison to steroid dose at first administration of Natalizumab.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02176031
|United States, Massachusetts|
|Massachusetts General Hospital|
|Boston, Massachusetts, United States, 02114|
|Dana-Farber Cancer Institute|
|Boston, Massachusetts, United States, 02115|
|Principal Investigator:||Corey Cutler, MD, MPH||Dana-Farber Cancer Institute|