Expanded Access to Provide ARQ 092 for the Treatment of Overgrowth Diseases and/or Vascular Anomalies
Primary Purpose
Proteus Syndrome, PIK3CA-Related Overgrowth Spectrum (PROS), Growth Disorders
Status
No longer available
Phase
Locations
Study Type
Expanded Access
Intervention
ARQ 092
Sponsored by
About this trial
This is an expanded access trial for Proteus Syndrome focused on measuring ARQ 092, ArQule, AKT, PIK3CA, Overgrowth, Congenital malformations
Eligibility Criteria
Inclusion Criteria:
- Severe overgrowth diseases and/or vascular anomalies with confirmed somatic genetic alterations of PIK3CA or AKT
- Are unable to participate in an ongoing ARQ 092 clinical trial
- Willing and able to provide written, signed informed consent. In the case of a minor, a parent or legal guardian must sign an informed consent form.
- Medically suitable for treatment with ARQ 092
- Not eligible for any other available therapy for the diagnosed overgrowth disease and/or vascular anomaly with confirmed somatic genetic alterations of PIK3CA or AKT
Exclusion Criteria:
- Currently enrolled in an ongoing clinical study of ARQ 092 or other investigational drug
- Currently being treated with any inhibitor of the PI3K/AKT/mTOR pathway
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT03317366
First Posted
October 17, 2017
Last Updated
July 31, 2021
Sponsor
ArQule, Inc. (a wholly owned subsidiary of Merck Sharp and Dohme, a subsidiary of Merck & Co., Inc.)
1. Study Identification
Unique Protocol Identification Number
NCT03317366
Brief Title
Expanded Access to Provide ARQ 092 for the Treatment of Overgrowth Diseases and/or Vascular Anomalies
Official Title
Expanded Access to Provide ARQ 092 for the Treatment of Overgrowth Diseases and/or Vascular Anomalies With Genetic Alterations of the PI3K/AKT Pathway
Study Type
Expanded Access
2. Study Status
Record Verification Date
July 2021
Overall Recruitment Status
No longer available
Study Start Date
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Primary Completion Date
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Study Completion Date
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3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
ArQule, Inc. (a wholly owned subsidiary of Merck Sharp and Dohme, a subsidiary of Merck & Co., Inc.)
4. Oversight
5. Study Description
Brief Summary
ARQ 092 is being investigated for patients with overgrowth diseases and/or vascular anomalies with genetic alterations of the PI3K/AKT pathway and may be available for patients who are ineligible for an ongoing ARQ 092 clinical trial or have other considerations that prevent access to ARQ 092 through an existing clinical trial.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Proteus Syndrome, PIK3CA-Related Overgrowth Spectrum (PROS), Growth Disorders
Keywords
ARQ 092, ArQule, AKT, PIK3CA, Overgrowth, Congenital malformations
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
ARQ 092
Intervention Description
Open-label expanded access for ARQ 092 capsules
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Eligibility Criteria
Inclusion Criteria:
Severe overgrowth diseases and/or vascular anomalies with confirmed somatic genetic alterations of PIK3CA or AKT
Are unable to participate in an ongoing ARQ 092 clinical trial
Willing and able to provide written, signed informed consent. In the case of a minor, a parent or legal guardian must sign an informed consent form.
Medically suitable for treatment with ARQ 092
Not eligible for any other available therapy for the diagnosed overgrowth disease and/or vascular anomaly with confirmed somatic genetic alterations of PIK3CA or AKT
Exclusion Criteria:
Currently enrolled in an ongoing clinical study of ARQ 092 or other investigational drug
Currently being treated with any inhibitor of the PI3K/AKT/mTOR pathway
12. IPD Sharing Statement
Learn more about this trial
Expanded Access to Provide ARQ 092 for the Treatment of Overgrowth Diseases and/or Vascular Anomalies
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