NCT02338050

A Phase II Study of the Anti-CD22 Recombinant Immunotoxin Moxetumomab Pasudotox (CAT-8015, HA22) in Children With B-lineage Acute Lymphoblastic Leukemia and Minimal Residual Disease Prior to Allogeneic Hematopoietic Stem Cell Transplantation

Terminated Phase 2 Last updated 15 September 2015

What this trial tests

Phase 2 trial testing Moxetumomab Pasudotox in Acute Lymphoblastic Leukemia (ALL) in 1 participant. Terminated before completion.

Timeline

1 May 2015

Primary endpoint
1 May 2020

Quick facts

Lead sponsor	Center for International Blood and Marrow Transplant Research
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	1
Start date	1 May 2015
Primary completion	1 May 2020
Sites	4 locations across United States

Drugs / interventions tested

• Moxetumomab Pasudotox (MOXETUMOMAB PASUDOTOX) — full drug profile →

Conditions studied

• Acute Lymphoblastic Leukemia (ALL) — all drugs for Acute Lymphoblastic Leukemia (ALL) →

Sponsor

Center for International Blood and Marrow Transplant Research — full company profile →

Who can join

Adults 6 Months to 24, any sex, with Acute Lymphoblastic Leukemia (ALL). Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• MRD negativity
  Time frame: change in MRD levels between baseline and 3-10 days post last Moxe dose
  The primary endpoint is the event of whether patients successfully achieved either MRD negativity (defined as \<0.01%) or at least 1 log10 reduction in MRD levels using flow cytometry via central laboratory testing after moxetumomab treatment but prior to HCT relative to pre-moxetumomab MRD measurement (baseline). The proportion of patients who become MRD negative or who have at least a 1-log10 re

Sponsor's own description

This is a phase II, open-label, nonrandomized, prospective study to evaluate the activity, safety, and feasibility of administration of moxetumomab pasudotox in the pre-allogeneic hematopoietic cell transplantation (HCT) setting to patients with B-lineage Acute Lymphoblastic Leukemia (ALL) who are in a morphologic complete remission and have pre-transplant minimal residual disease (MRD) \> 0.01% (detected by flow cytometry). The primary objective of this study is to determine if treatment with moxetumomab pasudotox in the MRD positive setting is able to lead to MRD negativity (\< 0.01% by flow cytometry) or at least a 1-log10 reduction in MRD prior to allogeneic HCT.

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

1. Therapeutic Targeting of Siglecs using Antibody- and Glycan-Based Approaches.
   Angata T, Nycholat CM, Macauley MS. · · 2015 · cited 83× · PMID 26435210 · DOI 10.1016/j.tips.2015.06.008
2. Moxetumomab pasudotox for hairy cell leukemia: preclinical development to FDA approval.
   Lin AY, Dinner SN. · · 2019 · cited 35× · PMID 31594764 · DOI 10.1182/bloodadvances.2019000507
3. Fatal capillary leak syndrome in a child with acute lymphoblastic leukemia treated with moxetumomab pasudotox for pre-transplant minimal residual disease reduction.
   Shah NN, Schneiderman J, Kuruvilla D, Bhojwani D, et al · · 2021 · cited 3× · PMID 32959985 · DOI 10.1002/pbc.28574