Brentuximab Vedotin in Treating Patients With Advanced Systemic Mastocytosis or Mast Cell Leukemia
|Aggressive Systemic Mastocytosis Mast Cell Leukemia Systemic Mastocytosis||Drug: brentuximab vedotin||Phase 2|
|Study Design:||Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
|Official Title:||A Study of Brentuximab Vedotin (SGN-35) in CD30-Positive Systemic Mastocytosis With or Without an Associated Hematological Clonal Non-Mast Cell Lineage Disease (AHNMD)|
- Overall response rate per consensus international response criteria (rate of complete or partial remissions or clinical improvement) [ Time Frame: Up to 1 year ]Will be estimated and its 95% confidence interval will be provided.
- Immunohistochemical expression of CD30 on neoplastic mast cells in core biopsy samples by flow cytometry [ Time Frame: Up to 1 year ]The expression of CD30+ neoplastic mast cells in core biopsy samples compared between pre and post brentuximab vedotin treatment.
- Total symptom score using a modified Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) [ Time Frame: Up to 1 year ]Total symptom score will be calculated using a modified Myeloproliferative Neoplasm Symptom Assessment Form by recording mean and median scores compared to pre-treatment score.
- Duration of response [ Time Frame: Time from first onset of confirmed response to the date of first documented and confirmed progression or death, assessed up to 1 year ]The Kaplan-Meier product-limit method will be used to summarize.
- Time to response [ Time Frame: Time from start of treatment until the date of onset of a confirmed response, assessed up to 1 year ]The Kaplan-Meier product-limit method will be used to summarize.
- Progression-free survival (PFS) [ Time Frame: Time from start of treatment to the date of the first documented and confirmed progression or death or institution of new therapy, assessed up to 1 year ]The Kaplan-Meier product-limit method will be used to summarize.
- Type of adverse events measured by National Cancer Institute Common Toxicity Criteria (NCI CTC) version 4.03 [ Time Frame: Up to 1 year ]Toxicity to be assessed as the type and relatedness of adverse events to brentuximab vedotin.
- Quality of life (QOL) score using a modified Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) [ Time Frame: Up to 1 year ]Quality of life score will be calculated using a modified Myeloproliferative Neoplasm Symptom Assessment Form and will be compared to pre-treatment score.
|Study Start Date:||September 2013|
|Estimated Study Completion Date:||December 2017|
|Estimated Primary Completion Date:||December 2017 (Final data collection date for primary outcome measure)|
Experimental: Brentuximab vedotin
Subjects receive a 30-minute IV infusion of brentuximab vedotin once every 21 days for 8 courses, in the absence of disease progression or unacceptable toxicity.
Drug: brentuximab vedotin
Brentuximab vedotin is an antibody with a covalently attached toxin. The antibody portion targets the protein CD30 on the surface of cells, and the toxin acts against those cells.
I. To evaluate the response rate to SGN-35 (brentuximab vedotin) in patients with tumor necrosis factor receptor superfamily, member 8 (CD30+) advanced systemic mastocytosis (SM) (ASM or mast cell leukemia [MCL] with or without an associated hematological clonal non-mast cell lineage disease [AHNMD]).
I. To evaluate the tolerability and safety profile of SGN-35 in patients with SM.
II. To evaluate expression of CD30 on neoplastic mast cells before and during therapy with SGN-35.
III. To evaluate changes in mastocytosis related symptom scores and quality of life (QOL) using a modified Myeloproliferative Neoplasm Symptom Assessment Form (MPNSAF).
IV. To evaluate the duration of response (DoR) and time to response (TTR). V. To evaluate progression-free survival (PFS).
Patients receive brentuximab vedotin intravenously (IV) over 30 minutes on day 1. Treatment repeats every 21 days for 8 courses in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up every 6 weeks for 1 year and then every 12 weeks thereafter.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01807598
|Contact: Isabel Reyesfirstname.lastname@example.org|
|United States, California|
|Stanford University, School of Medicine||Recruiting|
|Stanford, California, United States, 94305|
|Contact: Isabel Reyes 650-725-4047 email@example.com|
|Principal Investigator: Jason R. Gotlib|
|United States, Texas|
|MD Anderson Cancer Center||Completed|
|Houston, Texas, United States, 77030|
|Principal Investigator:||Jason Gotlib||Stanford University Hospitals and Clinics|