Immuno-ablation With Chemoimmunoradiation and Autologous Stem Cell Transplant for Churg-Strauss Syndrome

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT02728271
• Date of registration: 14/03/2016
• Prospective Registration: Yes
• Primary sponsor: Mounzer Agha
• Public title: Immuno-ablation With Chemoimmunoradiation and Autologous Stem Cell Transplant for Churg-Strauss Syndrome
• Scientific title: A Pilot Study of Immuno-ablation With Chemoimmunoradiation Followed by Autologous Hematopoietic Progenitor Cell (HPC) Transplant for Adult Subjects With Churg-Strauss Syndrome
• Date of first enrolment: April 2016
• Target sample size: 1
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT02728271
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Early Phase 1

Contacts

• Name: Mounzer Agha, MD
• Affiliation: University of Pittsburgh

Key inclusion & exclusion criteria

Inclusion Criteria:

- Age 18-60, inclusive

- Subjects carry a diagnosis of Churg-Strauss syndrome, with typical clinical,
pathologic, and/or radiological appearances.

- Must have a pulmonologist/immunologist providing the primary care for the
Churg-Strauss syndrome and be willing to be evaluated for the Churg-Strauss syndrome
who is the co-investigator in the protocol.

- Must be documented to be HIV negative.

- Subjects must be able to give written consent.

- Subjects with abscesses are eligible to enroll once the abscesses or any other
significant infection has resolved.

- Subjects must not be pregnant and will undergo a pregnancy test prior to starting the
study treatment. The subjects should also be willing to take the appropriate
contraception starting at least three months prior to the transplant.

- All eligible subjects will need the approval of the insurance company for the coverage
of the study treatment.

- Life expectancy of more than 6 months. ECOG performance status of 0 or 1.

- No evidence of myelodysplastic on peripheral blood smear

- Baseline serum creatinine must be <1.5 mg/dL, left ventricular ejection fraction >55%,
adequate pulmonary functions (oxygen saturation at room air of >90%), and AST and ALT
not > 2x upper limits of normal, and no history of previous or active malignancy,
except for localized cutaneous basal or squamous cell carcinoma in situ of the cervix.

- Evidence for life threatening disease, including FEV1 <50% predicted (on therapy)
and/or cardiac involvement (arrhythmias, failure)

- Failure to stabilize in response to prednisone (or equivalent) at doses of <20 mg per
day

- Failure of at least 3 other immunosuppressives to stabilize disease, including drugs
like cyclophosphamide, rituximab, mepolizumab, azathioprine.

Exclusion Criteria:

- Failure to accept or comprehend irreversible sterility as a potential side effect of
therapy.

- Previous allergy to cyclophosphamide, rituximab, mepolizumab, azathioprine.



Age minimum: 18 Years
Age maximum: 60 Years
Gender: All

Health Condition(s) or Problem(s) studied

Churg-Strauss Syndrome

Intervention(s)

Biological: HPC cell infusion

Primary Outcome(s)

number of patients with adverse events during treatment [Time Frame: change from baseline at 3, 6, 9, 12, 18, 24, 30, 36, 42, 48 months and then every 12 months, up to 100 months or if the patient dies, whichever occurs first.]

Secondary Outcome(s)

hematologic recovery [Time Frame: change from baseline at 3, 6, 9, 12, 18, 24, 30, 36, 42, 48 months and then every 12 months, up to 100 months or until the patient dies, whichever occurs first.]

graft failure rate [Time Frame: change from baseline at 3, 6, 9, 12, 18, 24, 30, 36, 42, 48 months and then every 12 months, up to 100 months or until the patient dies, whichever occurs first.]

resolution of eosinophilia [Time Frame: change from baseline at 3, 6, 9, 12, 18, 24, 30, 36, 42, 48 months and then every 12 months, up to 100 months or until the patient dies, whichever occurs first.]

Secondary ID(s)

15-146

Source(s) of Monetary Support

Please refer to primary and secondary sponsors