Official Title
An Early Access Programme for Moxetumomab Pasudotox in Relapsed/Refractory Hairy Cell Leukemia
Brief Summary

Early Access Programme to provide treatment access to moxetumomab pasudotox for eligible patients with relapsed/refractory hairy cell leukemia

Detailed Description

Multicentre Early Access Programme, designed to provide treatment access to moxetumomab
pasudotox for eligible patients with relapsed/refractory hairy cell leukemia who are at least
18 years of age, have adequate organ function and are Pseudomonas-immunotoxin naïve. Patients
will be administered moxetumomab pasudotox on Days 1, 3 and 5 of each 28-day cycle until
documentation of CR or for up to 6 cycles, progressive disease (PD), initiation of alternate
therapy or unacceptable toxicity.

Approved for marketing
Individual Patients
Relapsed/Refractory Hairy Cell Leukemia

Drug: Moxetumomab Pasudotox

A recombinant conjugated immunotoxin composed of a disulfide-stabilized anti-CD22 Ig Fv genetically fused to a truncated form of Pseudomonas exotoxin, PE38
Other Name: CAT-8015, HA22

Eligibility Criteria

Inclusion Criteria:

1. Histologically confirmed HCL or HCL variant with a need for therapy based on at least
one of the following criteria:

a neutrophils < 1.0x 109/L b platelets < 100 x 109/L c hemoglobin < 10 g/dL d
symptomatic splenomegaly

2. Pseudomonas-immunotoxin naïve

3. At least 2 prior systemic therapies, including 2 courses of a PNA, or 1 course of
either rituximab or BRAF inhibitor following a single prior course of PNA.

4. Age ≥ 18 years.

5. ECOG performance status ≤ 2

6. Adequate organ function as defined below:

1. total bilirubin ≤ 1.5 mg/dL, unless consistent with Gilbert's (ratio between
total and direct bilirubin > 5)

2. AST and ALT ≤ 3x upper limit of normal (ULN)

3. alkaline phosphatase < 2.5 ULN

4. serum creatinine ≤ 1.5 mg/dL or creatinine clearance ≥ 60 mL/min as estimated by
the Cockcroft-Gault equation

7. Prothrombin time (PT)/international normalized ratio (INR) or partial thromboplastin
time (PTT) < 2.5 ULN, fibrinogen ≥ 0.5 lower limit of normal; if on warfarin, INR <
3.5, if on any other anticoagulation, PT < 2.5 x baseline

8. Ability to understand and the willingness to sign a written informed consent document.

9. Life expectancy ≥ 6 months.

10. Females of childbearing potential who are sexually active with a nonsterilized male
partner must use a highly effective method of contraception prior to study entry and
for the duration of study participation, and must agree to continue using such
precautions for 4 months after completion of moxetumomab pasudotox administration;
cessation of contraception after this point should be discussed with a responsible
physician. Periodic abstinence, the rhythm method, and the withdrawal method are not
acceptable methods of contraception.

Exclusion Criteria:

1. Have had chemotherapy, immunotherapy or radiotherapy within 4 weeks prior to initiation
of treatment. 2. Receiving any other investigational agents. 3. Known brain metastases 4.
Retinal or choroidal detachment identified during the screening ophthalmologic evaluation.
5. Pregnant or breastfeeding females. . 6. Positive for Hepatitis B core antibody or
surface antigen unless the patient is on Lamivudine or Entecavir and Hepatitis B Viral
deoxyribonucleic acid (DNA) load is < 2000 IU/mL. 7. Active second malignancy requiring
treatment other than minor resection of indolent cancers like basal cell and squamous skin
cancers. 8. Uncontrolled intercurrent illness including but not limited to ongoing or
active infection, Symptomatic congestive heart failure, unstable angina pectoris,
uncontrolled hypertension, cardiac arrhythmia, malaria infection, or psychiatric
illness/social situations that would limit compliance with study requirements. 9. Known
human immunodeficiency virus (HIV)-positive patients unless taking appropriate anti-HIV
medications with a CD4 count of > 200. 10.History of allogeneic bone marrow transplant.
11.History of both thromboembolism and known congenital hypercoagulable conditions.
12.Uncontrolled pulmonary infection, pulmonary edema 13.Oxygen saturation at rest < 88%
measured by pulse oximetry or PaO2

- 55 mm Hg 14.Serum albumin < 2 g/dL 15.Radioimmunotherapy within 2 years prior to
enrollment in the study. 16.Absolute neutrophil count (ANC) < 1.0 x 109/L, or platelet
count < 50 x 109/L, unless judged by the investigator to be due to underlying disease
A patient will not be excluded because of pancytopenia ≥ Grade 3, or erythropoietin
dependence, if due to disease, based on the results of bone marrow studies.
17.Patients with < 50% of predicted forced expiratory volume or < 50% of predicted
diffusing capacity for carbon monoxide, corrected for hemoglobin concentration and
alveolar volume 18.History of thrombotic microangiopathy or thrombotic microangiopathy
/ HUS. 19.Corrected QT interval (Frederica) elevation > 500 msec (manually over-read
by medically qualified person) based on at least two separate 12-lead ECGs. 20.High
dose estrogen (defined as > 0.625 mg/day of an estrogen compound). 21.Clinical
evidence of disseminated intravascular coagulation (Grade 3- 4).

Eligibility Gender
Eligibility Age
Minimum: N/A ~ Maximum: N/A

Nai Shun (Nancy) Yao, MD, Study Chair
MedImmune LLC

NCT Number
Early Access
Hairy cell
Moxetumomab Pasudotox
Purine analog
BRAF Inhibitor
Pseudomonas-immunotoxin naïve
MeSH Terms
Leukemia, Hairy Cell
Immunotoxin HA22