This trial is active, not recruiting.

Condition multiple myeloma
Treatments sirolimus, tacrolimus, lenalidomide
Phase phase 1/phase 2
Targets mTOR, FKBP-12
Sponsor Sherif S. Farag
Collaborator Celgene Corporation
Start date February 2011
End date December 2016
Trial size 53 participants
Trial identifier NCT01303965, 1012-24; IUCRO-0307


One of the complications that can occur after a stem cell transplant is called graft versus host disease (GVHD). Another complication is that multiple myeloma may come back (relapse). In this study, a drug called lenalidomide will be started 1-2 months after a transplant, or possibly later depending on recovery of your side effects. Lenalidomide and sirolimus have been shown to work together against multiple myeloma. Therefore, lenalidomide will be combined with sirolimus with the hope that this will help prolong the amount of time the disease is in remission. Researchers hope these steps will help prolong the amount of time the multiple myeloma is in remission and will decrease the chance of GvHD.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Intervention model single group assignment
Masking open label
Primary purpose treatment
Use sirolimus and tacrolimus as GvHD prophylaxis with sirolimus and lenalidomide as post-transplant maintenance
Start on Day -3 and continue for 1 year
Start on Day -3 and begin tapering on Day +100 until Day +180.
Start between Day +30 and +120 and continue for 1 year.

Primary Outcomes

Number of Participants with Dose Limiting Toxicities as a Measure of Safety of the combination of sirolimus, tacrolimus and lenalidomide
time frame: 1 year post transplantation
Percent of patients alive and free of progression at 12 months.
time frame: baseline through 1 year post-transplant

Secondary Outcomes

Number of subjects with acute and chronic Graft versus Host Disease (GvHD)
time frame: Day 0 through 1 year post transplantation
Number of treatment-related deaths at 100 days and 1 year
time frame: Day +100 and 1 year post transplant
Percentage of donor chimerism as assessed at days +30, +90, +180 and at 12 months post transplant
time frame: +30, +90, +180 and 12 months post transplant
Number of toxicities, frequency and type of infections occurring within the first year
time frame: Day 0 through 1 year post transplantation
Time to engraftment of neutrophils and platelets
time frame: baseline through engraftment
Time from day 0 to disease progression or death (regardless of cause of death), whichever comes first.
time frame: Day 0 until disease progression
Number of patients with disease progression
time frame: Day 0 until disease progression
Time from day 0 to death from any cause
time frame: Day 0 until death
Number of complete responses (CR), stringent complete responses (sCR), near complete responses (nCR), very good partial responses (VGPR), partial responses (PR) and minimal responses (MR).
time frame: Day 0 until disease progression

Eligibility Criteria

Male or female participants at least 18 years old.

Recipient Inclusion Criteria: - 1. Understand and voluntarily sign an informed consent form. - 2. Age 18-70 years at the time of signing the informed consent form. - 3. Able to adhere to the study visit schedule and other protocol requirements. - 4. Previously documented multiple myeloma (MM) with measurable monoclonal protein by either serum/urine protein electrophoresis or serum free light chains, or measurable plasmacytomas. - 5. ECOG performance status of 0-2 at study entry (see Appendix 2). - 6. Acceptable organ function as outlined in the protocol. - 7. Otherwise fitting institutional criteria for allogeneic stem cell transplantation. - 8. Presence of an HLA-matched (5/6 or 6/6 matched for HLA-A, B, and DR) sibling donor, or a HLA-matched (matched for at least HLA-A, B, C, and DRB1) unrelated donor by high-resolution testing. - 9. Disease free of prior malignancies for >/= 5 years with exception of currently treated basal cell, squamous cell carcinoma of the skin, or carcinoma "insitu" of the cervix or breast. - 10. All study participants must be registered into the mandatory RevAssist® program, and be willing and able to comply with the requirements of RevAssist®. - 11. Females of childbearing potential (FCBP) must have a negative serum or urine pregnancy test Recipient Exclusion Criteria: - 1. Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form. - 2. Pregnant or breast feeding females. - 3. Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study. - 4. Known hypersensitivity to thalidomide or Lenalidomide. - 5. The development of erythema nodosum if characterized by a desquamating rash while taking thalidomide or similar drugs. - 6. Known seropositive for or active viral infection with human immunodeficiency virus (HIV), hepatitis B virus (HBV) or hepatitis C virus (HCV). Patients who are seropositive because of hepatitis B virus vaccine or prior infection to which they are now immune (i.e., not carriers) are eligible. Donor Inclusion Criteria: The following categories of donor will be acceptable: - 1. HLA-matched related donor (5/6 or 6/6 match): Minimal typing necessary is serologic typing for class I (A, B) and molecular typing for class II (DRB1). - 2. HLA-matched Unrelated Donor (MUD): Molecular identity at least at HLA A, B, C, and DRB1 and DQB1 (8/10 match) by high resolution typing is required. - 3. Syngeneic donors are not eligible. - 4. The donor must be healthy and must be an acceptable donor as per institutional standards for marrow or stem cell donation. - 5. Age ≥ 18 years

Additional Information

Official title Phase I/II Trial of Allogeneic Peripheral Blood Stem Cell Transplantation Followed by Maintenance Therapy With Lenalidomide and Sirolimus in Patients With High-Risk Multiple Myeloma
Principal investigator Sherif Farag, MD, PhD
Trial information was received from ClinicalTrials.gov and was last updated in March 2016.
Information provided to ClinicalTrials.gov by Indiana University.