Expanded Access to Provide ARQ 092 for the Treatment of Overgrowth Diseases and/or Vascular Anomalies

NCT03317366 | Unknown

• 1 other identifier: ARQ 092 Expanded Access
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Proteus Syndrome; PIK3CA-Related Overgrowth Spectrum (PROS); Growth Disorders

Keywords

ARQ 092; ArQule; AKT; PIK3CA; Overgrowth; Congenital malformations

Interventions

ARQ 092 DRUG

Open-label expanded access for ARQ 092 capsules

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA): lead

MeSH Terms

Conditions

Proteus Syndrome; Growth Disorders; Hereditary Sensory and Autonomic Neuropathies; Congenital Abnormalities

Interventions

Miransertib

Condition Hierarchy (Ancestors)

Hamartoma Syndrome, Multiple; Hamartoma; Neoplasms; Neoplasms, Multiple Primary; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Limb Deformities, Congenital; Musculoskeletal Abnormalities; Abnormalities, Multiple; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Nervous System Malformations; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 17, 2017
• First Posted: October 23, 2017
• Last Updated: August 6, 2021

Record last verified: 2021-07