Stanford APBI Trial

Clinical Trial

Overview

Intraoperative Radiotherapy (IORT) is one of three approaches used for accelerated, partial breast irradiation at Stanford.

Accelerated, partial breast irradiation (APBI) is a potentially important new way to incorporate radiotherapy in the treatment of women with breast cancer.

Currently, women with breast cancer who undergo a lumpectomy  typically have 6 1/2 weeks of radiation to the entire affected breast after surgery.  Accelerated, partial breast irradiation (APBI) changes this approach in two ways. It shortens the treatment time from 6 1/2 weeks to between 1 to 5 days, and reduces the treatment area from the entire breast to the area of the breast immediately around the lumpectomy site. This is the part of the breast where most cancers are likely to recur.

In many ways APBI is to current whole breast radiotherapy what a lumpectomy is to a mastectomy. The goal is to use a less invasive more focused treatment without compromising survival.

APBI has been used in limited trials in several hundred patients over the last 10 years. These trials show that in properly selected breast cancer patients APBI  worked just as well as whole breast radiotherapy. In the initial studies, investigators relied on the placement of many catheters in the breast tissue (interstial brachytherapy). Newer techniques will hopefully provide the same good results but will deliver the radiation in faster and/or more convenient ways. This could increase interest in APBI and allow additional clinical trials that test the safety and effectiveness of the newer approaches. These newer approaches could increase quality of life for many women with breast cancer.

Investigators at Stanford University Medical Center are currently offering an IRB approved clinical trial that uses three new approaches for APBI. These three approaches are:

    Intraoperative Radiotherapy (IORT) - 1 day

    Intracavitary Brachytherapy (MammoSite) - 5 days

    3-D Conformal/External Beam Radiotherapy - 5 days

The Stanford trial is led by Dr. Frederick Dirbas, Assistant Professor of Surgery, and by Dr. Donald Goffinet, Professor of Radiation Oncology. For further information about the trial please contact Janelle Maxwell or Triona Dolphin at (650) 498-7740.

Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Dystrophinopathy is a disease continuum that includes Duchenne muscular dystrophy, which develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walking ability. A specific type of mutation, called a nonsense (premature stop codon) mutation is the cause of dystrophinopathy in approximately 10-15 percent (%) of boys with the disease. Ataluren is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. The main goal of this Phase 3 study is to evaluate the effect of ataluren on walking ability. The effect of ataluren on physical function, quality of life, and activities of daily living will be evaluated. This study will also provide additional information on the long-term safety of ataluren.

Stanford is currently accepting patients for this trial.

Intervention(s):

  • drug: Ataluren
  • drug: Placebo

Eligibility


Inclusion Criteria:

   - Ability to provide written informed consent (parental/guardian consent if
   applicable)/assent per local requirements.

   - Phenotypic evidence of dystrophinopathy based on the onset of characteristic clinical
   symptoms or signs (for example; proximal muscle weakness, waddling gait, and Gowers'
   maneuver) by 6 years of age, an elevated serum creatinine kinase level, and ongoing
   difficulty with walking.

   - Documentation of the presence of a nonsense point mutation in the dystrophin gene as
   determined by gene sequencing from a laboratory certified by the College of American
   Pathologists (CAP), the Clinical Laboratory Improvement Act/Amendment (CLIA) or an
   equivalent organization.

   - Documentation that a blood sample has been drawn for confirmation of the presence of a
   nonsense mutation in the dystrophin gene.

   - Use of systemic corticosteroids (prednisone, prednisolone, or deflazacort) for a
   minimum of 6 months immediately prior to start of study treatment, with no significant
   change in dosage or dosing regimen (not related to body weight change) for a minimum
   of 3 months immediately prior to start of study treatment and a reasonable expectation
   that dosage and dosing regimen will not change significantly for the duration of the
   study.

   - Valid Screening 6-minute walk distance (6MWD) greater than or equal to (≥) 150 meters.
   Valid Screening 6MWD must have been less than or equal to (≤) 80% of predicted for age
   and height.

   - Results of the 2 Baseline 6MWD results must be determined as valid and results of the
   Day 2 Baseline 6MWD must be within 20% of the Day 1 Baseline 6MWD.

   - Baseline 6MWD (mean of valid Day 1 and Day 2 values) must be no more than a 20% change
   from the valid Screening 6MWD.

   - Confirmed screening laboratory values within the central laboratory ranges (hepatic,
   renal, and serum electrolyte parameters).

   - Willingness to abstain from sexual intercourse or employ an approved method of
   contraception during the period of study drug administration and 6-week follow-up
   period.

   - Willingness and ability to comply with scheduled visits, drug administration plan,
   study procedures, laboratory tests, and study restrictions.

Exclusion Criteria:

   - Treatment with systemic aminoglycoside antibiotics within 3 months prior to start of
   study treatment.

   - Initiation of systemic corticosteroids therapy within 6 months prior to start of study
   treatment.

   - Change in systemic corticosteroid therapy (for example; change in type of drug, dose
   modification not related to body weight change, schedule modification, interruption,
   or reinitiation) within 3 months prior to start of study treatment.

   - Any change (initiation, change in type of drug, dose modification, schedule
   modification,interruption, discontinuation, or reinitiation) in prophylaxis/treatment
   for congestive heart failure (CHF) within 3 months prior to start of study treatment.

   - Ongoing use of coumarin-based anticoagulants (for example; warfarin), phenytoin,
   tolbutamide, or paclitaxel.

   - Prior therapy with ataluren.

   - Known hypersensitivity to any of the ingredients or excipients of the study drug.

   - Exposure to another investigational drug within 3 months prior to start of study
   treatment.

   - History of major surgical procedure within 6 weeks prior to start of study treatment.

   - Ongoing immunosuppressive therapy (other than corticosteroids).

   - Ongoing participation in any clinical trial (except for studies specifically approved
   by PTC Therapeutics).

   - Expectation of major surgical procedure (for example; scoliosis surgery) during the
   12-month treatment period of the study.

   - Requirement for daytime ventilator assistance.

   - Uncontrolled clinical symptoms and signs of CHF (American College of
   Cardiology/American Heart Association Stage C or Stage D).

   - Prior or ongoing medical condition (for example; concomitant illness, psychiatric
   condition, behavioral disorder, alcoholism, drug abuse), medical history, physical
   findings (for example; lower limb injury that may affect 6MWT performance),
   electrocardiogram (ECG) findings, or laboratory abnormality that, in the
   investigator's opinion, could adversely affect the safety of the participant, makes it
   unlikely that the course of treatment or follow-up would be completed, or could impair
   the assessment of study results.

Ages Eligible for Study

7 Years - 16 Years

Genders Eligible for Study

Male

Now accepting new patients

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Shirley Paulose
650-724-3792
I'm interested

What's New

Stanford’s APBI trial has now been expanded to include women with  ductal carcinoma in situ (DCIS). Please call 650-498-7740 for more information.