Mifamurtide (L-MTP-PE) for High-Risk Osteosarcoma

• ClinicalTrials.gov Identifier: NCT00631631
• Recruitment Status: Completed
• First Posted: March 10, 2008
• Last Update Posted: May 14, 2014
• Sponsor: Millennium Pharmaceuticals, Inc.
• Information provided by (Responsible Party): Millennium Pharmaceuticals, Inc.

Tracking Information

• First Submitted Date: February 29, 2008
• First Posted Date: March 10, 2008
• Last Update Posted Date: May 14, 2014
• Study Start Date: January 2008
• Actual Primary Completion Date: October 2012 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: May 13, 2014): Number of participants with adverse events [ Time Frame: 12 months or disease progression, whichever occurs first ]
• Original Primary Outcome Measures (submitted: February 29, 2008): To provide an investigational drug, liposomal muramyl tripeptide phosphatidyl ethanolamine (L-MTP-PE) alone or combined with recommended approved therapy for treatment of osteosarcoma. [ Time Frame: 4 Years ]

Current Secondary Outcome Measures (submitted: May 13, 2014)

• Serum concentration-time profiles of free and total mifamurtide in 15-20 patients [ Time Frame: Just before the start of the first infusion of mifamurtide and at 0.5, 1, 2, 4, 6 and, 24 hours following the start of the first infusion and just prior to the 2nd dose of mifamurtide ]
• Overall survival [ Time Frame: From date of enrollment to date of death ]
• Progression-free survival [ Time Frame: From date of enrollment to date of first documented disease progression or death ]

• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Mifamurtide (L-MTP-PE) for High-Risk Osteosarcoma
• Official Title: Liposomal Muramyl Tripeptide Phosphatidyl Ethanolamine (L-MTP-PE) for High-risk Osteosarcoma
• Brief Summary: The purpose of this study was to collect information regarding the safety and tolerability of mifamurtide (liposomal muramyl tripeptide phosphatidyl ethanolamine; L-MTP-PE).

Detailed Description

The drug being tested in this study is called mifamurtide (L-MTP-PE; liposomal muramyl tripeptide phosphatidyl ethanolamine). Mifamurtide is being used to treat people with osteosarcoma, a form of cancer.

This was a patient-access study that looked at adverse events, disease progression, and overall survival in study participants.

The study enrolled 205 patients, of whom 204 were treated with mifamurtide intravenously at a dose of 2 mg/m2 twice weekly (at least 3 days apart) for 12 weeks, and then weekly for an additional 24 weeks, for a total of 48 doses in 36 weeks (following surgery for primary or metastatic disease).

This study was conducted in the United States. Participants could receive treatment for up to 9 months. This study was previously mis-categorized as an interventional study.

• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• Sampling Method: Non-Probability Sample
• Study Population: Had diagnosis of high grade osteosarcoma with relapsed or recurrent disease, locally or metastatic, with disease not completely resectable or who were unable to complete recommended chemotherapy due to toxicity: relapse, recurrence local or metastatic; unable to have standard surgical resection; abbreviated chemotherapy regimen secondary to toxicity.
• Condition: Osteosarcoma

Intervention

Drug: Mifamurtide (L-MTP-PE)

Solution for intravenous infusion

Other Name: Liposomal Muramyl Tripeptide Phosphatidyl Ethanolamine

Study Groups/Cohorts

Mifamurtide (L-MTP-PE)

Mifamurtide (L-MTP-PE), intravenous, at a dose of 2 mg/m^2 twice weekly (at least 3 days apart) for 12 weeks, and then weekly for an additional 24 weeks, for a total of 48 doses in 36 weeks.

Intervention: Drug: Mifamurtide (L-MTP-PE)

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: June 21, 2013): 205
• Original Estimated Enrollment (submitted: February 29, 2008): 100
• Actual Study Completion Date: October 2012
• Actual Primary Completion Date: October 2012 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

1. Had signed informed consent/assent. Voluntary participation in the pharmacokinetic portion of the compassionate access protocol was included in the informed consent but not required for compassionate use participation.
2. Had diagnosis of high grade osteosarcoma with relapsed or recurrent disease, locally or metastatic, with disease not completely resectable or who were unable to complete recommended chemotherapy due to toxicity: relapse, recurrence local or metastatic; unable to have standard surgical resection; abbreviated chemotherapy regimen secondary to toxicity (e.g. hypophosphatemia from ifosfamide, cardiotoxicity from doxorubicin, renal dysfunction from methotrexate, ifosfamide, or cisplatin.)
3. Aged 2 ≤ 50 years.
4. Had adequate hematopoietic function as demonstrated by: 1) Absolute Neutrophil Count (ANC) > 750/microL; Hemoglobin (Hb) > 8 g/dL; Platelets > 30,000/microL.
5. Had adequate hepatic function as documented by 1) ALT < 2.5 x upper limit of normal (ULN) for age; 2) total bilirubin ≤ 1.5 x ULN for age.
6. Had adequate renal function as demonstrated by: 1) Creatinine clearance or radioisotope glomerular filtration rate > 70 mL/min/1.73 m^2; OR, 2) Serum creatinine ≤ 2x ULN for age.
7. Had absence of concurrent active acute infection (i.e., afebrile).
8. In females of child bearing potential (not menopausal for 12 months or no previous surgical sterilization), had a negative pregnancy test. All sexually active participants used an effective means of contraception. Such means included oral contraceptives, Lupron Depot, DepoProvera, and condom with diaphragm and spermicidal jelly.
9. Performance status: Lansky 50-100% (≤ 16 years of age); OR, Eastern Cooperative Oncology Group (ECOG) 0-2 or Karnofsky 50-100% (>16 years of age).

Exclusion Criteria:

1. Had chronic use of corticosteroids or other immunosuppressive agents.
2. Was pregnant or breast-feeding.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 2 Years to 50 Years (Child, Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT00631631
• Other Study ID Numbers: MTP-OS-403
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: Millennium Pharmaceuticals, Inc.
• Study Sponsor: Millennium Pharmaceuticals, Inc.
• Collaborators: Not Provided

Investigators

• Principal Investigator: Peter M. Anderson, MD, PhD; M.D. Anderson Cancer Center

• PRS Account: Millennium Pharmaceuticals, Inc.
• Verification Date: May 2014