Proteus SyndromeNCT03317366
Expanded Access to Provide ARQ 092 for the Treatment of Overgrowth Diseases and/or Vascular Anomalies
A study testing ARQ 092 in people with Proteus Syndrome and PIK3CA-Related Overgrowth Spectrum (PROS), run by ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA).
Trial 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… Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Proteus Syndrome, PIK3CA-Related Overgrowth Spectrum (PROS), Growth Disorders
- What is being tested?
- ARQ 092
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
About this study
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
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.
- Being tested
- ARQ 092
- Keywords
- ARQ 092, ArQule, AKT, PIK3CA, Overgrowth, Congenital malformations
- Medical subject headings
- Proteus Syndrome, Growth Disorders, Hereditary Sensory and Autonomic Neuropathies, Congenital Abnormalities
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ARQ 092Drug
Open-label expanded access for ARQ 092 capsules
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Oct 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Proteus SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03317366
- Sponsor's protocol ID
- ARQ 092 Expanded Access
- Status last verified
- Jul 2021
- First submitted
- 17 Oct 2017
- Last updated
- 6 Aug 2021
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03317366
- Acronym / sponsor ID
- ARQ 092 Expanded Access
- Keywords
- ARQ 092, ArQule, AKT, PIK3CA, Overgrowth, Congenital malformations
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)Industry (pharma, biotech, devices)
- First posted
- 23 Oct 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (2 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ARQ 092Open-label expanded access for ARQ 092 capsules | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Aug 2021.
Related trials for Proteus Syndrome
RecruitingObservational study
Study of Proteus Syndrome and Related Congenital Disorders
Proteus Syndrome, PIK3CA Related Overgrowth Spectrum
Active, not recruitingPhase 2
MK-7075 (Miransertib) in Proteus Syndrome
Proteus Syndrome
Active, not recruitingPhase 2
A Study of the Safety and Tolerability in Participants With PIK3CA-related Overgrowth Spectrum or Proteus Syndrome Who Are Being Treated With Miransertib (MK-7075) in Other Studies (MK-7075-006)
PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing ARQ 092 in people with Proteus Syndrome and PIK3CA-Related Overgrowth Spectrum (PROS), run by ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA).
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 6 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Aug 2021.