A Phase II Study of Perifosine in Patients With Relapsed/Refractory Waldenström's Macroglobulinemia

NCT00398710 | Completed Phase 2

• 1 other identifier: Perifosine 221
• Study Type: interventional
• Target: 37
• Locations: 1 country
• Sites: 1

Brief Summary

This is a phase II study in relapsed/refractory WM patients treated with perifosine. It is designed to assess the proportion of overall confirmed responses (CR + PR + MR) using a two-stage phase II study design to permit early stopping of the trial if there is strong evidence that the study regimen is inactive. In addition, it will assess toxicity of this drug in patients with WM. Patients will receive perifosine 150 mg qhs daily. Patients will be assessed by serum immunoelectrophoresis and IgM level at least every 4 weeks.

Conditions

Waldenström's Macroglobulinemia

Keywords

Waldenström's Macroglobulinemia; Perifosine

Outcome Measures

Primary Outcomes (1)

• Response rate

  Response will include complete remission, partial remission (PR), and minimal response (MR) using serum protein electrophoresis. Response will also be assessed by IgM using nephelometry.

  Every 4 weeks

Secondary Outcomes (4)

• Safety

  Every 4 weeks

• Time to progression

  Every 4 weeks

• Progression free survival

  Every 4 weeks

• Duration of response

  Every 4 weeks

Study Arms (1)

Perifosine

EXPERIMENTAL

Patients will receive perifosine orally at 150 mg daily after food for 28-d cycles.

Drug: Perifosine

Interventions

Perifosine DRUG

150 mg daily (100 mg daily in case of dose reduction)

Also known as: D-21266, KRX-0401

Perifosine

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age \>= 18 years.
• Must have received prior therapy for their WM and have relapsed or refractory WM. Any number of prior therapies is acceptable.
• Measurable disease, defined as presence of immunoglobulin M (IgM) paraprotein with a minimum IgM level of \> 2 times the upper limit of each institution's normal value is required and over 10% of lymphoplasmacytic cells in the bone marrow.
• ECOG Performance Status (PS) 0, 1, or 2.
• The following laboratory values obtained 14 days prior to registration
• ANC \>= 1 x109/L
• PLT \>= 75 x109/L
• Total bilirubin ≤ 2.0 mg/dL (If total is elevated check direct and if normal patient is eligible.)
• AST \<= 3 x upper limit of normal (ULN)
• Creatinine \<= 2 x ULN
• Ability to provide informed consent.
• Life expectancy \>= 12 weeks.

You may not qualify if:

• Uncontrolled infection.
• Other active malignancies.
• CNS involvement.
• Cytotoxic chemotherapy ≤ 3 weeks, or biologic therapy ≤ 2 weeks, or corticosteroids ≤ 2 weeks, prior to registration. Patients may be receiving chronic corticosteroids if they are being given for disorders other than WM such as auto-immune diseases. Plasmapheresis is not considered as an active therapy and can be used at the physician's discretion.
• Other concurrent chemotherapy, immunotherapy, radiotherapy, or any ancillary therapy considered investigational.
• Any of the following:
• Pregnant women
• Nursing women
• Men or women of childbearing potential who are unwilling to employ adequate contraception (condoms, diaphragm, birth control pills, injections, intrauterine device (IUD), or abstinence, etc.)
• Known to be HIV positive.
• Radiation therapy ≤ 2 weeks prior to registration.

Sponsors & Collaborators

• AEterna Zentaris: lead
• Dana-Farber Cancer Institute: collaborator

Study Sites (1)

Dana-Farber Cancer Institute

Boston, Massachusetts, 02115, United States

Location

Related Publications (1)

• Ghobrial IM, Roccaro A, Hong F, Weller E, Rubin N, Leduc R, Rourke M, Chuma S, Sacco A, Jia X, Azab F, Azab AK, Rodig S, Warren D, Harris B, Varticovski L, Sportelli P, Leleu X, Anderson KC, Richardson PG. Clinical and translational studies of a phase II trial of the novel oral Akt inhibitor perifosine in relapsed or relapsed/refractory Waldenstrom's macroglobulinemia. Clin Cancer Res. 2010 Feb 1;16(3):1033-41. doi: 10.1158/1078-0432.CCR-09-1837. Epub 2010 Jan 26.

  PMID: 20103671 RESULT

MeSH Terms

Conditions

Waldenstrom Macroglobulinemia

Interventions

perifosine

Condition Hierarchy (Ancestors)

Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemorrhagic Disorders; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Study Officials

• Irene M Ghobrial, MD

  Dana-Farber Cancer Institute

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 9, 2006
• First Posted: November 14, 2006
• Study Start: October 1, 2006
• Primary Completion: August 1, 2011
• Study Completion: October 1, 2011
• Last Updated: March 15, 2018

Record last verified: 2011-11

Locations

US (1)