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

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ClinicalTrials.gov Identifier: NCT04085653
Expanded Access Status : Available
First Posted : September 11, 2019
Last Update Posted : May 27, 2020
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

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

Condition or disease Intervention/treatment
PIK3CA-Related Overgrowth Spectrum (PROS) Drug: alpelisib

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Study Type : Expanded Access
Expanded Access Type : Treatment IND/Protocol
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: Managed Access Program (MAP) to Provide Alpelisib (BYL719) for Patients With PIK3CA-Related Overgrowth Spectrum (PROS)



Intervention Details:
  • Drug: alpelisib

    Alpelisib will be provided as 50 mg and 200 mg film coated tablets as individual patient supply and will be dosed on a flat scale of mg/day.

    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 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.


Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 confirmed diagnosis of PROS and documented evidence of a mutation in the PIK3CA gene, as determined by a local laboratory.
  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.

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 II).
  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 Steven Johnson's syndrome or toxic epidermal necrolysis.
  5. 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

    1. Sexually active males unless they are sterilized (at least 6 months prior to screening) or 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.
    2. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for at least 4 weeks after stopping alpelisib.

    Highly effective contraception methods include:

    • Total abstinence when this is in line with the preferred and usual lifestyle of the patient. Periodic abstinence (e.g. calendar, ovulation, symptothermal, 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 tubal ligation at least 6 weeks before taking program treatment. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment.
    • Male partner sterilization (at least 6 months prior to screening). For female patients in the program the vasectomized male partner should be the sole partner for that patient.
    • Placement of an intrauterine device (IUD).

    Note: Use of oral (estrogen and progesterone), transdermal, injected or implanted hormonal methods of contraception as well as hormonal replacement therapy is not allowed in this program; barrier method is not considered an effective method.

  6. Patient is pregnant or lactating, where pregnancy is defined as state of a female after conception and until the termination of gestation, confirmed by a positive human chorionic gonadotrophin (hCG) lab test.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04085653


Contacts
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Contact: Novartis Pharmaceuticals 1-888-669-6682 novartis.email@novartis.com
Contact: Novartis Pharmaceuticals +41613241111

Locations
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United States, Arkansas
Arkansas Children's Hospital Available
Little Rock, Arkansas, United States, 72202
Contact    501-320-3755      
United States, California
Children's Hospital Los Angeles Available
Los Angeles, California, United States, 90027
Contact    323-660-2450      
Principal Investigator: Janna Journeycake         
Children's Hospital Los Angeles Available
Los Angeles, California, United States, 90027
Contact    323-669-2501      
United States, New Jersey
Atlantic Medical Group Available
Morristown, New Jersey, United States, 07960
United States, North Carolina
Mission Hospital Available
Asheville, North Carolina, United States, 28801
Principal Investigator: Janna Journeycake         
United States, Ohio
Cincinnati Children's Hospital Medical Center Available
Cincinnati, Ohio, United States, 45229-3039
Principal Investigator: Janna Journeycake         
United States, Oklahoma
University of Oklahoma Health Sciences Center Available
Oklahoma City, Oklahoma, United States, 73104
Contact    405-271-5311      
Principal Investigator: Janna Journeycake         
United States, Tennessee
Vanderbilt University Medical Center Available
Nashville, Tennessee, United States, 37232
Contact    615-322-5000      
Principal Investigator: Janna Journeycake         
United States, Virginia
Children's Hospital of Richmond at VCU Available
Richmond, Virginia, United States, 23219
Contact    804-628-3093      
United States, Washington
Virginia Mason Medical Center Available
Seattle, Washington, United States, 98101
Contact    206-223-6193      
Principal Investigator: Janna Journeycake         
Netherlands
Novartis Investigative Site Available
Nijmegen, Netherlands, 6525EX
Spain
Novartis Investigative Site Available
Oviedo, Asturias, Spain, 33011
Novartis Investigative Site Available
Madrid, Spain, 28046
Sponsors and Collaborators
Novartis Pharmaceuticals
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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04085653    
Other Study ID Numbers: CBYL719F12001M
First Posted: September 11, 2019    Key Record Dates
Last Update Posted: May 27, 2020
Last Verified: May 2020
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
adult, pediatric, BYL719,PIK3CA-Related Overgrowth Spectrum, PIK3CA, Related Overgrowth Spectrum,PROS