Carfilzomib for the Prevention of Graft Versus Host Disease

Sheba Medical Center

Status and phase: Unknown; Phase 1

Conditions: Graft-versus-host Disease

Treatments: Drug: carfilzumib

Study type: Interventional

Funder types: Other

Identifiers

NCT01991301

SHEBA-12-9997-AN-CTIL

Details and patient eligibility

About

The aim of this study is to evaluate the safety and efficacy of Carfilzumib, which is a novel biological agent used in the treatment of multiple myeloma in preventing graft-versus-host disease, after stem cells transplantation from unrelated donors.

Enrollment: 30 estimated patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Patients with MDS/AML
2. 18 years or older and willing and able to comply with the protocol requirements.
3. LVEF ≥ 40%. 2-D transthoracic echocardiogram (ECHO) is the preferred method of evaluation. Multigated Acquisition Scan (MUGA) is acceptable if ECHO is not available.
4. Patients undergoing 8-10/10 HLA matched unrelated and unmanipulated PBSC transplantation
5. Patients conditioned with reduced intensity or reduced toxicity conditioning i.e. Fludarabine combine with Treosulfan or 2-4 days of I.V Busulfan.
6. Patients must sign written informed consent.
7. Adequate birth control in fertile patients.

Exclusion criteria

1. Patients undergoing other type of transplantation or with other type of basic disease other than AML or MDS.
2. Patients with respiratory failure (DLCO < 30%).
3. Active congestive heart failure (New York Heart Association [NYHA] Class III to IV), symptomatic ischemia, or conduction abnormalities uncontrolled by conventional intervention.
4. Patients with > grade II liver renal toxicity.
5. Psychiatric conditions/disease that impair the ability to give informed consent or to adequately co-operate
6. Bilirubin > 3.0 mg/dl, transaminases > 3 times upper normal limit
7. Creatinine > 2.0 mg/dl
8. ECOG-Performance status > 2
9. Uncontrolled infection
10. Pregnancy or lactation
11. CNS disease involvement
12. Pleural effusion or ascites > 1 liter.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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30 participants in 1 patient group

carfilzumib

Experimental group

Description:

Carfilzomib at a dose of 20mg/m2 I.V. will be administrated at day 1, 2 post infusion of the stem cell (SC) graft to the first 10 patients. If no \> grade II toxicity\* the next 10 patients will receive Carfilzomib (27 mg/m2) at day 1,2 and 8, 9, 15 and 16. If no \> grade II toxicity\* the next 10 patients will receive 2-3 cycles of Carfilzomib (27 mg/m2) administered at day 1,2 8,9,15 and 16 post SC graft infusion.

Treatment:

Drug: carfilzumib