FDA's New Rule For Compassionate Use

[CourtneyL]

Since you ladies and gentlemen are the smartest, most well -informed group of people on the planet, I thought there might be some of you familiar with the FDA's process for obtaining access to investigational drugs outside of the clinical trial setting.

The background on this is that I have some progression of disease in my lungs now and I am not super excited about going on yet another chemo. I'd much rather try out a HER2+ targeted therapy such as Neratinib - made by Wyeth. As most of you know, it is in clinical trial phase right now and showing great promise. It also crosses the blood brain barrier which is huge news for me since I've had brain mets.

The problem is that the exclusion criteria for the clinical trials is too strict as it doesn't allow prior Tykerb/Xeloda usage.

So I am investigating the possibility of applying for emergency use via the FDA's new rule regarding such use. The new rule was just passed in August 2009 and take effect 60 days later - conveniently that is right now! Here is a link to some information on the rule if you are not familiar. It may come in handy for some of you down the road:

http://www.fda.gov/ForConsumers/ConsumerUpdates/ucm176845.htm

My question is - has anyone here every applied to the FDA for such use? They call the form an IND for "Investigational New Drug". Any advice, insite, would be greatly appreciated.

Cheers!

[Carolyns]

Hi Courtney,

I have not gone this route yet although I have investigated it a little. Women with mets and the general patient population with advanced disease find out about these challenges at their most difficult time. Someone here shared with me a link to this website: http://abigail-alliance.org/

This group has been fighting for expanded access and compassionate care for a very long time.

I wish you well in your efforts.

Take care, Carolyn

[Cal-Gal]

Thanks for posting this Courtney and good luck--I have no experience w/this as I am fairly new to bc-Good Luck again--and my thoughts and prayers are with you---

[Darlene Denise]

Courtney: Did you consult with MD Anderson? Interested in what doctors might think this is a good choice. Also if you did consult at MD, please share what they had to say. You kow I'm in the same boat with you and always interested in new therapies.

Pasting part of a trial that indicates they will accept 20 additional patients in part 2 with prior Tykerb. It looks like you might squeeze in. Not sure about your other antineoplastic drugs.

Let me know if this could work and keep me in your loop.

May the force be with us...Darlene

Study Evaluating HKI-272 in Combination With Vinorelbine in Subjects With Solid Tumors and Metastatic Breast Cancer
This study is currently recruiting participants.
Verified by Wyeth, October 2009
First Received: June 25, 2008 Last Updated: October 7, 2009 History of Changes (http://her2support.org/ct2/archive/NCT00706030)
Sponsored by: Wyeth
Information provided by: WyethClinicalTrials.gov Identifier: NCT00706030
http://her2support.org/ct2/html/images/frame/triangle.gif Purpose The purposes of this study are to identify the highest tolerable dose of HKI-272 in combination with vinorelbine and to assess the safety of the combination of the two drugs as well as to obtain preliminary information on whether the combination of the two drugs has any effect on solid tumors.


Condition (http://her2support.org/ct2/help/conditions_desc) Intervention (http://her2support.org/ct2/help/interventions_desc) Phase (http://her2support.org/ct2/help/phase_desc) Advanced Breast Cancer
Advanced Malignant Solid Tumors
Drug: HKI-272 in combination with vinorelbine
Phase I
Phase II


Study Type:InterventionalStudy Design:Treatment, Randomized, Open Label, Single Group Assignment, Safety/Efficacy StudyOfficial Title:A Phase 1/2 Study of HKI-272 in Combination With Vinorelbine in Subjects With Solid Tumors and Metastatic Breast Cancer
Resource links provided by NLM:

Genetics Home Reference (http://ghr.nlm.nih.gov/) related topics: breast cancer (http://her2support.org/ct2/bye/mQoPWw4lZXcilwpxudhWudNzlXNiZip90dcx5Q1PedcO9BUyzB 1gWBcGuBcHS.)
MedlinePlus (http://www.nlm.nih.gov/medlineplus/) related topics: Breast Cancer (http://her2support.org/ct2/bye/WQoPWw4lZX-i-iSxudhWudNzlXNiZip9m67PvQ7xzwhaLwS9pwNHkw-P0B7x061nuQoPmdt.) Cancer (http://her2support.org/ct2/bye/uQoPWw4lZX-i-iSxudhWudNzlXNiZip9m67PvQ7xzwhaLwS90B7x061nuQoPmdt .)
Drug Information (http://druginfo.nlm.nih.gov/drugportal/drugportal.jsp) available for: Vinorelbine (http://her2support.org/ct2/bye/jQoPWw4lZXcPSi7iedN6ZXNxvdDxuQ7Ju6c9cXcPSi7iEd-yWB7EZ6o35Q1yzB-VuQUgEscxkd789HNzud-yzdhyedN8.) Vinorelbine tartrate (http://her2support.org/ct2/bye/gQoPWw4lZXcPSi7iedN6ZXNxvdDxuQ7Ju6c9cXcPSi7iEd-yWB7EZ6o35Q1yzB-VuQUgEscxkd789HNzud-yzdhyedN8fihGSioykih8.) HKI-272 (http://her2support.org/ct2/bye/tQoPWw4lZXcPSi7iedN6ZXNxvdDxuQ7Ju6c9cXcPSi7iEd-yWB7EZ6o35Q1yzB-VuQUgEscxkd789DjmyXCn-RJ.)
U.S. FDA Resources (http://her2support.org/ct2/info/fdalinks)


Further study details as provided by Wyeth:

Primary Outcome Measures:

Physical Exams, Adverse Event Assessment, Tumor Assessments (CT, MRI, bone scan) including independent radiologic review for Part 2 [ Time Frame: 10 months (estimated average participation) ] [ Designated as safety issue: Yes ]


Secondary Outcome Measures:

Electrocardiograms (ECGs), QTCLeft Ventricular Ejection Fraction, (LVEF [ Time Frame: 10 months (estimated average participation) ] [ Designated as safety issue: No ]


Estimated Enrollment:95Study Start Date:April 2008Estimated Study Completion Date:January 2011Estimated Primary Completion Date:January 2011 (Final data collection date for primary outcome measure)Arms (http://her2support.org/ct2/help/arm_group_desc)Assigned Interventions (http://her2support.org/ct2/help/interventions_desc)A: Experimental Drug: HKI-272 in combination with vinorelbine

Detailed Description: The study will be conducted in two parts. In the first part, testing will be done on up to 12 subjects to determine the highest tolerable dose of HKI-272 and vinorelbine in patients with advanced solid tumors. In the second part of the study, approximately 60 additional subjects with metastatic ErbB-2-positive breast cancer, with no prior exposure to lapatinib (Tykerb®), are planned to be added to better define the tolerability and preliminary activity of HKI-272 in combination with vinorelbine. Up to 20 additional subjects with ErbB-2-positive breast cancer with prior lapatinib (Tykerb®) exposure are also planned to be enrolled in part 2 for exploratory analyses.




http://her2support.org/ct2/html/images/frame/triangle.gif Eligibility

Ages Eligible for Study: 18 Years and olderGenders Eligible for Study: BothAccepts Healthy Volunteers: NoCriteria
Inclusion Criteria:

Confirmed pathologic diagnosis of a solid tumor that is not curable with available therapies for which HKI-272 plus vinorelbine is a reasonable treatment option (part 1 only) or Confirmed pathologic diagnosis of ErbB-2-positive breast cancer (current stage IV) in female subjects for which vinorelbine plus HKI-272 is a reasonable treatment option (part 2 only).
At least 1 prior antineoplasic chemotherapy treatment regimen for metastatic disease and at least 1 prior treatment with a trastuzumab-containing regimen for at least 6 weeks, for metastatic disease or subject relapsing under adjuvant treatment(part 2 only).
At least 1 measurable lesion as defined by Response Evaluation Criteria in Solid Tumors (RECIST).
Exclusion Criteria:

More than 2 prior antineoplasic chemotherapy treatment regimens for metastatic disease. Subjects who relapsed under adjuvant treatment shouldn't have received more than one line of chemotherapy for metastatic disease(part 2 only).
Prior treatment with vinorelbine for metastatic setting, or prior treatment with any ErbB-2 targeted agents except trastuzumab (part 2 only). Up to 20 subjects with ErbB-2-overexpressing metastatic breast cancer who have been previously exposed to lapatinib but are not refractory to lapatinib may be enrolled in part 2.
Prior treatment with anthracyclines with a cumulative dose of doxorubicin of greater than 400 mg/m2, or of epirubicin dose of greater than 800 mg/m2, or the equivalent dose for other anthracyclines or derivatives (part 2 only).



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