An extension study for patients with PROS or Proteus Syndrome currently treated with miransertib.

Phase 1

• Conditions: PIK3CA-related overgrowth spectrum (PROS) and Proteus Syndrome (PS); MedDRA version: 21.1Level: PTClassification code 10081236Term: PIK3CA related overgrowth spectrumSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 20.0Level: PTClassification code 10074067Term: Proteus syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

• Registration Number: EUCTR2021-001369-19-IT
• Lead Sponsor: MERCK SHARP & DOHME CORP. UNA SUSSIDIARIA DI MERCK & CO. INC.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-10-18
• Last Update Posted: 2 March 2022

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

1. Has PROS or PS and is currently being actively treated with miransertib as part of Study MK-7075-002 (NCT03094832) or ArQule's CU/EAP (NCT03317366)
2. Is male or female, from 2 years to 120 years of age, inclusive
3. The participant (or legally acceptable representative) has provided documented informed consent/assent for the study. There is no future biomedical research planned for this study
4. A WOCBP must have a negative highly sensitive pregnancy test (urine, as required by local regulations) within 72 hours before the first dose of study intervention. If a urine test cannot be confirmed as negative (eg, an ambiguous result), a serum pregnancy test is required. In such cases, the participant must be excluded from participation if the serum pregnancy result is positive
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 6
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Has previously discontinued miransertib due to related SAEs or other intolerance of miransertib
2. Other investigational agents, if any, that were administered between leaving Study MK-7075-02 or ArQule's CU/EAP and entering this trial
3. Inhibitors of the mTOR pathway (eg, sirolimus, everolimus)
4. Immunosuppressive therapies
5. Continuous high dose steroids (topical or low dose steroids equivalent to =10 mg prednisone daily are permitted)

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified