Outreach & Inclusion of Underserved Populations (up to 2 pages, excess pages will be discarded)

Outreach is a critical function of Stanford Health centers ensuring people who are low-income, uninsured,
and/or members of underserved populations such as migrant and seasonal farmworkers, people
experiencing homelessness and racial and ethnic minorities have adequate access. A strong outreach
program offers the most vulnerable populations connection and engagement with appropriate medical
opportunities.
Local Catchment Area: Stanford Children’s Health is one of the major medical networks in the Bay Area—
and one of the few in the country—
exclusively dedicated to children and
expectant mothers. With over 60 locations
throughout Northern California, and a
growing network of affiliates beyond this area
(see map), Stanford Children’s Health
makes accessing premier health care for all,
a priority. The Bay Area comprises
enormous diversity: 6.7% Black or African
Americans, 24.6% Asians or Pacific
Islanders or Native Americans and 23%
Latinos and 17.2% from two or more, or other
races. The region is the second most diverse
among the largest 150 metro areas in the
United States. This diversity has led to
Stanford’s commitment to providing
healthcare across all demographics. Indeed,
The Lucile Packard Children’s Hospital
(LPCH) Stanford annually invests more than
$244 million in community benefit services
and activities. Through this funding, Stanford
Children’s Health provides expert care for
our community’s high-risk kids and young
adults ages 10 to 25 through the Mobile Adolescent Health Services program. The multidisciplinary staff
provide custom-designed care for those who rely exclusively on the “Teen Van” as their only link to a
network of services and knowledge they urgently need. The Stanford Pediatrics Advocacy Program is a
joint initiative between Stanford Medicine and LPCH that advocates for improving the health status of
children while reducing health disparities in the Bay Area, California and nationally.
Expansive Recruitment Strategy: Alongside the local and Statewide LPCH outreach efforts, the rarity of
IPEX syndrome has meant the research team, led by Dr. Bacchetta, have initiated active outreach programs
whereby strategic alliances with physicians, their patients and families outside of California and the United
States have been developed. As a world renown expert in IPEX syndrome Dr. Bacchetta has extensive
access to potential patients, as exemplified by the letters we include from families in England, Italy, Canada
and Uruguay all of whom support her efforts developing a cure for this devastating disease. Within North
America, Dr. Bacchetta (the PI on this CIRM proposal) and Dr. Roncarolo (the Medical Director of the
CD4LVFOXP3 trial) are both members of the NIH sponsored Primary Immune Deficiency Treatment
Consortium (PIDTC; see letter of support). The PIDTC includes 47 academic centers of excellence in the
USA and Canada whose shared goal is to improve the outcomes of patients with rare, life threatening,
inherited disorders of the immune system. Dr. Bacchetta is currently a Co-PI on the PIDTC study,
responsible for the mechanistic studies on the immune dysregulation in these patients. In the current
granting period (2019-2024) 300 patients are expected to be recruited for a retrospective study and 240 for
a prospective study. All patients will be assessed for their genetic abnormality. Patients identified with
FOXP3 mutations will be referred to Dr. Bacchetta and her team for this Phase 1 study. In addition, 17
patients are presently enrolled in the PIDTC research protocol at Stanford and currently in contact with Dr.
Bacchetta, all of whom will be invited to participate in this clinical trial provided they fulfill eligibility criteria;
these patients are indicated in the table below (enrollment of the 8 patients labeled “undetermined” is
pending). The PIDTC has shown strong support for patient recruitment to this trial. Further, based on
historical data, we anticipate our referral network to bring ~ 2 newly diagnosed patients per year to Stanford.

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Finally, as discussed above, patients and physicians from outside the USA do contact the Investigators;
where appropriate these patients will be offered inclusion (see letter from Dr. Aiuti, Deputy Director, San
Raffaele Telethon Institute for Gene Therapy (SR-TIGET) Italy).
Thus, our active outreach programs have helped in both identifying patients globally with this extremely
rare disease, AND in reaching these patients from all geographic areas, ethnicities, and education levels.
We are very excited to have the opportunity to now offer these patients the prospect of participating in a
new and innovative clinical trial directly aimed at treating their disease.
Study Population: Aside from the fact that IPEX, as a monogenic X-linked disease, manifests exclusively
in males, the disease shows no ethnic or socioeconomic bias. Of the 25 patients associated with our
program (17 of whom are enrolled in our research protocol) listed in the table below, 12% are Hispanic,
16% are African American/Black, 32% are White, 4% are Asian, 8% are Mixed race and 28% are unknown.
Thus, given the rarity of IPEX syndrome, a
diverse population of patients have sought
the expertise of Dr. Bacchetta and her team.
In the new trial administering CD4LVFOXP3, we
expect to include many of these patients
currently in our care, as well as new patients
we recruit through our expansive recruitment
network; we anticipate we will achieve
similar diversity in race and ethnicity. Given
our success networking amidst numerous
North American and International Hospitals,
we are confident we will continue to recruit
patients with high representation from
underserved communities.
Mitigating Social Economic Barriers: Our
treatment goes beyond socio-economic
barriers because the policy at Stanford is
that any new interventional therapy is
accessible to any patient. Our global patient
catchment area serves diverse,
underrepresented patients including those
with traditionally limited access to state-of-the-art modes of cell therapy such as the FTiH CD4LVFOXP3. Our
success is exemplified in the demographics Table above. LPCH services include social work and an
international office (International Patient Services), which assist with logistics and socioeconomic barriers
for patients and families traveling from out of state, or country, or who lack insurance or financial resources
to cover the ancillary costs of coming for medical treatment. We are committed to ensuring that no patient
in need of treatment is turned away by providing equitable coverage for study-related procedures and
reimbursement for travel for participants and caregivers during their participation in the clinical trial. All travel
reimbursements and financial coverage of study procedures will be IRB approved [21 CFR 50.20].
Exclusion Justification: No patient will be excluded from the trial on the basis of socioeconomic, ethnicity
or language barriers. At LPCH, all children diagnosed with IPEX, the population in this study, are treated
regardless of their ability to pay. With respect to IPEX patients, the initial treatment modalities vary
depending on the severity and specific disease manifestations at diagnoses; enrollment in the current trial
will be confirmed through eligibility criteria, ensuring patients from all socioeconomic areas can benefit. In
addition, we recognize that language can pose a barrier. At LPCH we have an active interpreter and
translation services program and language bank where high caliber interpreters can work with non-English
speaking families in at least 40 different languages.
Finally, our team’s reputation attracts patients from around the world. Referring physicians, patients and
families reach out because of our expertise. Our physicians bring uniquely valuable relationships, including
strong ties to advocacy groups, enabling our therapies to quickly reach the intended patient populations.
Our Rationale is our Vision: That with our help, all children in the communities we serve are able to reach
their maximum health potential.

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