Clinical trial NCT00003512: Antineoplaston Therapy in Treating Patients With Recurrent or Refractory Waldenstrom's Macroglobulinemia
Official title: Phase II Study of Antineoplastons A10 and AS2-1 in Patients With Macroglobulinemia of Waldenstrom
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE2
Conditions: Waldenstrom Macroglobulinemia
Keywords: Waldenstrom Macroglobulinemia Recurrent
Waldenstrom Macroglobulinemia Refractory
Lead sponsor: Burzynski Research Institute
Sponsor class: OTHER
First posted: 2003-01-27
Enrollment: 0
Sex: ALL
Minimum age (years): 18
Maximum age (years): 99
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Current therapies for Recurrent or Refractory Waldenstrom's Macroglobulinemia provide very limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of Recurrent or Refractory Waldenstrom's Macroglobulinemia.

PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on patients with Recurrent or Refractory Waldenstrom's Macroglobulinemia.

Detailed description: Recurrent or Refractory Waldenstrom's Macroglobulinemia patients receive gradually escalating doses of intravenous Antineoplaston therapy (Atengenal + Astugenal) until the maximum tolerated dose is reached. Treatment continues up to 12 months in the absence of disease progression or unacceptable toxicity.

OBJECTIVES:

* To determine the efficacy of Antineoplaston therapy in patients with Recurrent or Refractory Waldenstrom's Macroglobulinemia, as measured by an objective response to therapy (complete response, partial response or stable disease).
* To determine the safety and tolerance of Antineoplaston therapy in patients with Recurrent or Refractory Waldenstrom's Macroglobulinemia.
* To determine objective response, tumor size is measured utilizing physical examination, radiologic studies, and bone marrow biopsies as necessary, performed every 8 weeks for the first two years, every 3 months for the third and fourth years, every 6 months for the 5th and sixth years, and annually thereafter.

Eligibility criteria: DISEASE CHARACTERISTICS:

* Histologically confirmed recurrent or progressive Waldenstrom's macroglobulinemia that is unlikely to respond to existing therapy, including surgery, radiotherapy, and chemotherapy

  * Evidence of tumor by MRI, CT scan, chest x-ray, or radionuclide scan
  * Must have received and failed at least one standard first-line therapy (e.g., corticosteroid/alkylator combination)
* Biochemical evidence of Waldenstrom's macroglobulinemia

  * Abnormal proteins in serum and urine

PATIENT CHARACTERISTICS:

Age:

* 18 and over

Performance status:

* Karnofsky 60-100%

Life expectancy:

* At least 2 months

Hematopoietic:

* WBC at least 2000/mm3
* Platelet count at least 50,000/mm3

Hepatic:

* No hepatic insufficiency
* Bilirubin no greater than 2.5 mg/dL
* SGOT and SGPT no greater than 5 times upper limit of normal

Renal:

* No renal insufficiency
* Creatinine no greater than 2.5 mg/dL
* No history of renal conditions that contraindicate high dosages of sodium

Cardiovascular:

* No uncontrolled hypertension
* No history of congestive heart failure
* No history of other cardiovascular conditions that contraindicate high dosages of sodium

Pulmonary:

* No serious lung disease, such as chronic obstructive pulmonary disease

Other:

* Not pregnant or nursing
* Fertile patients must use effective contraception during and for 4 weeks after study
* No active infection
* No non-malignant systemic disease
* Not a high medical or psychiatric risk

PRIOR CONCURRENT THERAPY:

Biologic therapy:

* At least 4 weeks since prior immunotherapy
* No concurrent immunomodulating agent

Chemotherapy:

* See Disease Characteristics
* At least 4 weeks (6 weeks since nitrosoureas) since prior chemotherapy
* No concurrent antineoplastic agents

Endocrine therapy:

* See Disease Characteristics
* Concurrent corticosteroids allowed

Radiotherapy:

* See Disease Characteristics
* At least 8 weeks since prior radiotherapy

Surgery:

* See Disease Characteristics
* Recovered from prior surgery

Other:

* No prior antineoplastons
* Prior cytodifferentiating agents allowed

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Houston
country: United States
facility: Burzynski Clinic
state: Texas
Study officials:
affiliation: Burzynski Research Institute
name: Stanislaw R. Burzynski, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: