Exceptional Responder Study – NCI

This is handled by the physician however; you the patient, may want to pass on the information

“Phenotype to Genotype” trial:

The “Exceptional Responders” study
•Brief description of Exceptional Responders study
•How to inform NCI of a potential “Exceptional Responder” case
•Slide set describing Exceptional Responders study
•Protocol document for Exceptional Responders study
•NCI Press Release – Exceptional Responders
•Exceptional Responders Frequently Asked Questions (FAQs)
•Exceptional Responders on ClinicalTrials.gov

Rarely, a patient has all tumor disappear, or has a long term response to a drug that doesn’t usually cause such responses to happen. In the Exceptional Responders study, investigators will study the molecular characteristics of tumors of patients who had an exceptional response to a cancer therapy. In doing this, they hope to discover molecular features in the tumors that may predict benefit to a particular drug or type of drug. Where possible, non-tumor specimens from the patients will also be examined, as these help in determining which mutations are present only in the tumor, rather than in normal cells/tissue.

The Exceptional Responders Initiative is a pilot study to determine if information can be obtained from archived samples that will enable hypotheses to be made about the reasons for exceptional responses to cancer treatment.

The molecular information will be placed into a large database, along with clinical information, and shared with other approved researchers so that they can help determine why the patient(s) had such an exceptional response, and if this knowledge can be applied to other cancer patients. (Information will be de-identified.)

The Exceptional Responders study will be led by NCI Division of Cancer Treatment and Diagnosis and Center for Cancer Genomics, with genetic sequencing performed at Baylor. The NCI will collect up to 300 samples to successfully analyze 100 cases.

Clinical providers and investigators are invited to send NCI information on patients they believe may be “exceptional responders” by following the instructions below (NCI will provide reimbursement for each accepted case):

How to inform NCI of cases you believe are Exceptional Responders

Definition of “Exceptional Responder”:

In a setting where the overall response rate is typically < 10%, the patient has:
a.a complete response
or
b.a partial response of at least 6 months duration

The response can be to an experimental systemic therapy or to a standard systemic therapy, in a trial setting or in a clinical setting.

Providers or investigators who know of a potential “Exceptional Responder” can propose cases by sending an email NCIExceptionalResponders@mail.nih.gov.

In this email, please:

1.Provide a short description of the case, without patient identifiers. The description should include the patient’s disease, length of response, and treatment. No Personal Health information should be included in the description.

2.Note whether tissue is available from the time before the exceptional response occurred. (This is required.). Additional information on the type of tissue (Fresh frozen or formalin fixed paraffin embedded) is appreciated.

3.Note whether informed consent was given to use tissue for research. (The study has a process to address cases without informed consent.)

4.Note the patient’s vital status.

5.Provide any additional information you wish.

Within 2 weeks, NCI will email you informing you whether the case appears eligible. If the case appears eligible, the site that treated the patient will be asked to provide clinical information, informed consent, tissue samples, and a pathology report. The site will also execute a material transfer agreement (MTA) with the biospecimen facility for the tissue transfer, and a contract with the CTSU for reimbursement for the provision of samples and data.

You may also email NCIExceptionalResponders@mail.nih.gov if you have any questions about the Exceptional Responders study.