Official Title
Managed Access Program (MAP) to Provide Access to Alpelisib (BYL719) for Patients With PIK3CA-Related Overgrowth Spectrum (PROS)
Brief Summary

The purpose of this Cohort Treatment Plan is to allow access to alpelisib for patients diagnosed with PIK3CA-Related Overgrowth Spectrum (PROS) who fulfill certain eligibility criteria as specified in this document. The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations

Available
Treatment IND/Protocol
PIK3CA-Related Overgrowth Spectrum (PROS)

Drug: alpelisib
Alpelisib will be provided as 50 mg, 125mg and 200 mg film coated tablets as individual patient supply, and will be dosed on a flat scale of mg/day not to be adjusted to body weight or surface area.
In Adult patients, alpelisib will be administered at a starting dose of 250 mg orally once daily on a continuous dosing schedule and can be adjusted for toxicity per the recommendations in this treatment plan.
In Pediatric patients, alpelisib will be administered at a maximum starting dose of 50 mg orally once daily on a continuous dosing schedule and can be interrupted for toxicity per the recommendations in this treatment plan; no dose reductions are allowed.

Eligibility Criteria

Patients eligible for inclusion in this Treatment Plan have to meet all of the following
criteria: 1. Adult or pediatric patients ≥ 2 years of age, with a diagnosis of PROS preferably with evidence of a mutation in the PIK3CA gene 2. The treating physician has determined that the patient's condition is severe or life threatening, treatment is necessary and there are no other feasible alternatives for the patient. 3. Confirmed adequate bone marrow function Written patient informed consent must be obtained prior to start of treatment

Exclusion criteria

Patients eligible for this Treatment Plan must not meet any of the following criteria: 1. Patient has history of hypersensitivity to any drugs or metabolites of PI3K inhibitor or any of the excipients of alpelisib. 2. Patient with uncontrolled diabetes mellitus type I or not controlled type II (based on FPG and HbA1c, see inclusion criterion 2) 3. Patient who has other concurrent severe and/or uncontrolled medical conditions that would, in the Treating Physician's judgment, contraindicate administration of alpelisib (eg. active or uncontrolled severe infection, chronic active hepatitis, immuno-compromised, acute or chronic pancreatitis, uncontrolled high blood pressure, interstitial lung disease, etc.) 4. Patient has a known history of Severe Cutaneous Adverse Reactions (SCAR) like Steven Johnson's syndrome (SJS), Erythema Multiforme (EM), Toxic Epidermal Necrolysis (TEN), or Drug Reaction with Eosinophilia and Systemic Symptoms (DRESS). 5. History of pancreatitis within 1 year of screening or past medical history of chronic pancreatitis 6. Subject with Child Pugh score B or C 7. Subjects with unresolved osteonecrosis of the jaw 8. Subject is currently receiving any of the following medications and cannot be discontinued 7 days prior to the start of the treatment: - Strong inducers of CYP3A4 - Inhibitors of BCRP 9. Patient has a known history of Human Immunodeficiency Virus (HIV) infection (testing not mandatory unless required by local regulations or requirements).

10. Patient who is concurrently being treated with drugs known to be strong inhibitors or inducers of the isoenzyme CYP3A; switching to different medications prior to start of program treatment is allowed within the last 5 days prior to starting program treatment

11. Patient is currently receiving or has received systemic corticosteroids ≤ 2 weeks prior to start of program treatment, or who have not fully recovered from side effects of such treatment. Note: The following uses of corticosteroids are permitted: single doses, topical applications (e.g., for rash), inhaled sprays (e.g., for obstructive airways diseases), eye drops or local injections (e.g., intra-articular).

12. Male patient who does not apply highly effective contraception during the treatment with alpelisib and through the duration as defined below after the final dose of alpelisib. Sexually active males should use a condom during intercourse while taking drug and for at least 4 weeks after stopping alpelisib and should not father a child in this period. A condom is required to be used also by vasectomized men in order to prevent delivery of the drug via seminal fluid

13. Subject is not able to understand and to comply with treatment instructions and requirements

14. Subject is a nursing (lactating) or pregnant woman as confirmed by a positive serum (hCG) test prior to initiating study treatment

15. Subject is a woman of child-bearing potential defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during study treatment and at least for 1 week after the last dose of any study treatment. Highly effective contraception methods include: - Total abstinence (when this is in line with the preferred and usual lifestyle of the subject ). Periodic abstinence (e.g., calendar, ovulation, symptom-thermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception. Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy), total hysterectomy or bilateral tubal ligation at least 6 weeks before taking study treatment. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment - Male sterilization (at least 6 months prior to screening). For female subjects on the study the vasectomized male partner should be the sole partner for that subject - Use of oral (estrogen and progesterone), injected or implanted combined hormonal method of contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS), or forms of hormonal contraception that have comparable efficacy (failure rate

Eligibility Gender
All
Eligibility Age
Minimum: 2 Years~Maximum: 99 Years
Countries
Argentina
Australia
Belgium
Canada
Croatia
Denmark
Finland
Germany
Greece
India
Ireland
Israel
Korea, Republic of
Malaysia
Mexico
Netherlands
Poland
Russian Federation
Singapore
Slovenia
Spain
Sweden
Switzerland
Taiwan
United Arab Emirates
United States
Locations

Arkansas Children's Hospital
Little Rock, Arkansas, 72202

Available

Contact: 501-320-3755

Investigator: Janna Journeycake

Arkansas Childrens Hospital Dept. Pediatric/Div Hem/Onc
Little Rock, Arkansas, 72202

Available

Investigator: Joana Mack

Children's Hospital Los Angeles Childen's Center for Cancer
Los Angeles, California, 90027

Available

Contact: 323-361-2212

Investigator: Fariba Navid

Children's Hospital Los Angeles
Los Angeles, California, 90027

Available

Contact: 323-660-2450

Investigator: Diana Moke

Children's Hospital Los Angeles
Los Angeles, California, 90027

Available

Contact: 323-669-2501

Investigator: Fariba Navid

Children's Hospital Los Angeles
Los Angeles, California, 90027

Available

Contact: 323-660-2450

Investigator: Fariba Navid

UCLA Medical Center
Los Angeles, California, 90095

Available

Investigator: Vivian Y Chang

University of California at Los Angeles
Los Angeles, California, 90095

Available

Investigator: Vivian Y Chang

Stanford University Medical Center Div. of Pediatric Rheumatology
Palo Alto, California, 94304-1509

Available

Investigator: Joyce Teng

Lucile Packard Children s Hospital Stanford University
Palo Alto, California, 94304

Available

Contact: 650-723-4000

Investigator: Joyce Teng

Children's Specialists of San Diego Medical Group
San Diego, California, 92123

Available

Contact: 858-966-8153

Investigator: Deborah Schiff

Rady Children s Hospital Dept of Oncology
San Diego, California, 92123

Available

Contact: 858-966-5118

Investigator: Deborah Schiff

Rady Children s Hospital Sickle Cell Disease Center
San Diego, California, 92123

Available

Contact: 858-966-5811

Investigator: Deborah Schiff

Rady Childrens Hospital
San Diego, California, 92123

Available

Contact: 858-246-2905

Investigator: Deborah Schiff

AI Dupont Children Hospital
Wilmington, Delaware, 19803

Available

Contact: 302-651-4888

Investigator: Andrew Walter

Alfred I DuPont Hospital for Children
Wilmington, Delaware, 19899

Available

Contact: 302-651-5804

Investigator: Andrew Walter

Children's National Med Center StudyCoord:ACZ886G2301/01E1/05
Washington, District of Columbia, 20010

Available

Contact: 202-476-5533

Childrens National Hospital Ctr - Cancer & Blood Disorders
Washington, District of Columbia, 20010

Available

Contact: 202-476-3656

Childrens National Hospital
Washington, District of Columbia, 20010

Available

Contact: 202-476-5000

Nicklaus Childrens Hospital
Miami, Florida, 33155

Available

Contact: 786-624-4908

Investigator: Ziad Khatib

Children s Healthcare of Atlanta Aflac Blood Cancer Center
Atlanta, Georgia, 30322

Available

Contact: 404-778-3126

Investigator: Michael Briones D O

Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, 96826

Available

Contact: 808-522-4536

University of Iowa Health Care Dept of Pediatrics & Pulmonary
Iowa City, Iowa, 52242

Available

Contact: 319-356-7883

Investigator: Theodosia Thoma

Boston Childrens Hospital Pediatric Heamatology Oncology
Boston, Massachusetts, 02215

Available

Contact: 267-425-3010

Investigator: Whitney Eng

Children's Hospital and Clinics of Minnesota Childrens Hosp/Clinic
Minneapolis, Minnesota, 55404

Available

Atlantic Medical Group
Morristown, New Jersey, 07960

Available

Investigator: Michael Scola

Roswell Park Cancer Institute Pediatrics
Buffalo, New York, 14263

Available

Investigator: Steven Ambrusko

Cohen Children's Medical Center of New York Oncology
New Hyde Park, New York, 11040

Available

Contact: 9015956411

Investigator: Mark Peter Atlas

NYU Langone Health
New York, New York, 10016

Available

Investigator: Elisabeth A. Raetz

NYU Laura and Isaac Perlmutter Cancer Center
New York, New York, 10016

Available

Contact: 212-263-9959

Mission Hospital
Asheville, North Carolina, 28801

Available

Investigator: Krystal Bottom

Cincinnati Children's Hospital Medical Center Cancer & Blood Disease Inst.
Cincinnati, Ohio, 45229-3039

Available

Contact: 513-803-1140

Investigator: Adrienne Hammill

Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229-3039

Available

Investigator: Philippe Backeljauw

Children's Hospital Medical Center
Cincinnati, Ohio, 45229

Available

Contact: 513-636-4955

Investigator: Adrienne Hammill

Oklahoma State University Health Sciences Center Pediatric Hem/Onc
Oklahoma City, Oklahoma, 73104

Available

Contact: 405-271-5311

Investigator: Janna Journeycake

University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104

Available

Contact: 405-271-5311

Investigator: Janna Journeycake

Doernbecher Children's Hospital
Portland, Oregon, 97239

Available

Contact: 503-418-5336

Children's Hospital of Philadelphia Pediatric Oncology Clinic
Philadelphia, Pennsylvania, 19104

Available

Contact: 215-590-1000

Investigator: Denise Adams

The Childrens Hospital of Philadelphia Children's Hosp Philadelphia
Philadelphia, Pennsylvania, 19104

Available

Contact: 215-590-7399

Investigator: Denise Adams

The Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104

Available

Investigator: Denise Adams

LeBonheur Children's Medical Center
Memphis, Tennessee, 38103

Available

Investigator: Lucas Elijovich

Monroe Carell Jr Childrens Hospital at Vanderbilt
Nashville, Tennessee, 37232

Available

Contact: 615-321-5247

Investigator: Alexandra Borst

Vanderbilt University Medical Center
Nashville, Tennessee, 37232

Available

Contact: 615-322-5000

Investigator: Alexandra Borst

Vanderbilt University Medical Center
Nashville, Tennessee, 37232

Available

Investigator: Alexandra Borst

Vanderbilt University Medical Center
Nashville, Tennessee, 37232

Available

Contact: 615-936-0219

Investigator: Alexandra Borst

Vanderbilt University Medical Center
Nashville, Tennessee, 37232

Available

Dell Childrens Medical Center of Central Texas
Austin, Texas, 78723

Available

Investigator: Donald Thomas Wells

Texas Cancer Center ( Medical City Dallas Hospital)
Dallas, Texas, 75230

Available

Contact: 972-566-7790

University of Texas Southwestern Medical Center
Dallas, Texas, 75235-8858

Available

Contact: 214-648-2017

Investigator: Kathleen Ludwig

University of Texas Southwestern Medical Center UT Southwestern
Dallas, Texas, 75390-8852

Available

Contact: 214-645-4673

Investigator: Kathleen Ludwig

University of Texas Southwestern Medical Center Regulatory
Dallas, Texas, 75390

Available

Investigator: Kathleen Ludwig

UT Southwestern Medical Center Pediatric Hematology/Onc
Dallas, Texas, 75390

Available

Contact: 214-648-8594

Investigator: Kathleen Ludwig

Texas Childrens Hospital CFTY720D2311
Houston, Texas, 77030

Available

Contact: 832-822-1804

Investigator: Ionela Iacobas

Texas Childrens Hospital Oncology Department
Houston, Texas, 77030

Available

Contact: 832-824-4635

Investigator: Ionela Iacobas

Texas Childrens Hospital
Houston, Texas, 77030

Available

Contact: 713-798-1204

Investigator: Ionela Iacobas

The University of Vermont Medical Center
Burlington, Vermont, 05401

Available

Contact: 802-847-4746

Investigator: Julian Sprague

UVA Children s Hospital
Charlottesville, Virginia, 22903

Available

Investigator: William Petersen

Children's Hospital of Richmond at VCU Pediatric Hematology Oncology
Richmond, Virginia, 23219

Available

Contact: 804-828-9605

Investigator: Kelly K Lastrapes

Children's Hospital of Richmond at VCU
Richmond, Virginia, 23219

Available

Contact: 804-628-3093

Investigator: Janna Journeycake

Virginia Mason Medical Center Buck 2
Seattle, Washington, 98101

Available

Investigator: Andrew Friedman

Virginia Mason Medical Center
Seattle, Washington, 98101

Available

Contact: 206-223-6193

Investigator: Andrew Friedman

Children's Hospital and Regional Medical Center
Seattle, Washington, 98105-0371

Available

Contact: 206-528-5134

Investigator: Jonathan A Perkins

Children's Hospital and Regional Medical Center
Seattle, Washington, 98105

Available

Contact: 206-526-2100

Investigator: Jonathan A Perkins

Seattle Childrens Hospital
Seattle, Washington, 98105

Available

Contact: 206-884-1480

Investigator: Jonathan A A Perkins

Childrens Hospital of Wisconsin
Milwaukee, Wisconsin, 53226

Available

Investigator: Nathan Schloemer

Medical College of Wisconsin
Milwaukee, Wisconsin, 53226

Available

Investigator: Nathan Schloemer

Midwest Children's Cancer Center
Milwaukee, Wisconsin, 53226

Available

Investigator: Nathan Schloemer

Contacts

Novartis Pharmaceuticals
1-888-669-6682
novartis.email@novartis.com

Novartis Pharmaceuticals
+41613241111

Novartis Pharmaceuticals
NCT Number
Keywords
Adult
Pediatric
byl719
PIK3CA
Overgrowth Spectrum
PROS
alpelisib