Alpha 1 Anti-Trypsin (AAT) in Treating Patients With Acute Graft-Versus-Host Disease GVHD)

Browse trials for

• Graft vs Host Disease 806

Graft vs Host Disease

806 studies on the registry are indexed under Graft vs Host Disease; 138 are open to participants now.

This study's enrollment of 20 is below the median of 35 across 637 interventional studies indexed under Graft vs Host Disease.

Lead sponsor

Fred Hutchinson Cancer Center is the lead sponsor of 537 studies on the registry; 79 are open to participants now.

Of its 57 completed or terminated interventional studies of FDA-regulated products, 45 (79%) have results posted.

Study arms

• Other
  Cohort 1 (30 mg/kg)

  Alpha 1 anti-trypsin (AAT) will be administered intravenously at a dose of 90 mg/kg (loading dose) on day 1 followed by 30mg/kg (maintenance dose) every other day (QOD) on days 3, 5, 7, 9, 11, 13 \& 15.

  Drug: Alpha 1-Proteinase Inhibitor, Human 1 MG [Glassia]

• Other
  Cohort 2 (60 mg/kg)

  AAT will be administered intravenously at a dose of 90 mg/kg (loading dose) on day 1 followed by 60 mg/kg (maintenance dose) QOD on days 3, 5, 7, 9, 11, 13 \& 15.

  Drug: Alpha 1-Proteinase Inhibitor, Human 1 MG [Glassia]

• Other
  Cohort 3 (90 mg/kg)

  AAT will be administered intravenously at a dose of 90 mg/kg on days 1, 3, 5, 7, 9, 11, 13 \& 15.

  Drug: Alpha 1-Proteinase Inhibitor, Human 1 MG [Glassia]

Interventions

• DrugAlpha 1-Proteinase Inhibitor, Human 1 MG [Glassia]

  Alpha 1-Proteinase Inhibitor, Human 1 MG \[Glassia\] at various levels over different days

Primary outcomes

1. Number (Percentage) of Patients at Each Dosing Cohort Who Experience no Toxicity and in Whom Graft Versus Host Disease (GVHD) is Stable or Improved

   Toxicity and adverse events were assessed using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v4.0. All adverse events were reported regardless of attribution to alpha 1 anti-trypsin (AAT). GVHD response was defined per standard criteria for improvement, no change or progression of signs/symptoms in skin rash (% body surface area), GI (nausea, vomiting, anorexia, diarrhea, GI bleeding, abdominal cramping) and hepatic function (serum bilirubin levels). For this outcome measure, the requirement for additional GVHD treatment beyond AAT was not included in the criteria for response (i.e patients who may have required additional GVHD treatment before study day 28 were not automatically categorized as non-responders or as having progressive GVHD).

   Time frame: Adverse events were reported through 15 days after the last dose of AAT. GVHD response assessed at study day 28.

Secondary outcomes

1. Number (Percentage) of Patients at Each Dosing Cohort Experiencing an Unexpected Serious Adverse Event (SAE)

   Serious adverse events included death, a life-threatening adverse drug experience, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant disability/ incapacity, or congenital anomaly/birth defect. Significant events that do not meet these criteria may be considered serious if they jeopardize the patient and require a medical intervention to prevent one of the outcomes above. An "unexpected" adverse event is defined as an event that is not identified in nature, severity or frequency in the current investigator brochure/package insert/product information.

   Time frame: SAEs were reported through 30 days after the last dose of alpha 1 anti-trypsin (AAT).

2. Number (Percentage) of Patients at Each Dosing Cohort Who Experience One or More Suspected Serious Adverse Reactions (Infusion Related Reactions)

   Serious adverse reactions were assessed by the NCI CTCAE v4.0.

   Time frame: Within 48 hours after each infusion

3. Number (Percentage) of Patients at Each Dosing Cohort Who Experience One or More Thrombotic or Thrombo-embolic Events

   Events were assessed using the NCI CTCAE v4.0.

   Time frame: Events were reported through 15 days after the last dose of AAT.

4. Number (Percentage) of Patients at Each Dosing Cohort With Occurrence of Infections

   Infections were assessed using NCI CTCAE v4.0.

   Time frame: Infections were reported through 15 days after the last dose of AAT.

5. Number (Percentage) of Patients at Each Dosing Cohort With Progression of GVHD

   GVHD responses were assessed using criteria established by the Center for International Blood and Marrow Transplant Research and criteria from the Acute GVHD Activity Index. Patients who required additional systemic GVHD treatment beyond AAT before study day 28 were defined as having progressive GVHD.

   Time frame: GVHD responses were assessed on day 28 after starting AAT therapy or at time of death if patient died before study day 28.

Baseline characteristics

Individual participant data

Plan to share: No