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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Merck Sharp & Dohme LLC

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Phenomena and Processes [G] - Genetic Phenomena [G05]

Trial duration

5 Nov 2021 → ongoing

Decision date (initial)

2022-06-28

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc.

External identifiers

EU CT number

2022-500689-87-00

EudraCT number

2021-001369-19

WHO UTN

U1111-1273-8003

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Therapy

To evaluate the safety and tolerability of miransertib administered as monotherapy

Conditions and MedDRA coding

PIK3CA-related overgrowth spectrum (PROS) and Proteus Syndrome (PS)

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Has phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha (PIK3CA)-related overgrowth spectrum (PROS) or Proteus Syndrome (PS) and has been screened in Study MK-7075-002 (or has been approved by the Sponsor to screen for MK-7075-002) or is currently being treated with miransertib as part of Study MK-7075-002 (NCT03094832) or ArQule’s CU/EAP (NCT03317366)
2. Is an individual of any sex/gender, from 2 to 120 years of age, inclusive
3. For males, agrees to be abstinent from heterosexual intercourse or use contraception unless confirmed to be azoospermic during the study period and for ≥90 days after the last dose of study intervention
4. For females, is not pregnant or breastfeeding, and is either not a participant of childbearing potential (POCBP) or is a POCBP and is abstinent or uses a highly effective method of contraception

Exclusion criteria 5

1. Has previously discontinued miransertib due to related SAEs or other intolerance of miransertib
2. Has received other investigational agents, if any, that were administered between leaving Study MK-7075-002 or ArQule’s CU/EAP and entering this trial
3. Is receiving systemic inhibitors of the mechanistic target of rapamycin (mTOR) pathway (eg, sirolimus, everolimus)
4. Is receiving immunosuppressive therapies
5. Is receiving continuous high dose steroids

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Number of participants experiencing a Serious Adverse Event (SAE)
2. Number of participants discontinuing study treatment due to an Adverse Event (AE)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Merck Sharp & Dohme LLC

Sponsor organisation

Merck Sharp & Dohme LLC

Address

126 East Lincoln Avenue, P. O. Box 2000 P. O. Box 2000

City

Rahway

Postcode

07065-4607

Country

United States

Scientific contact point

Organisation

Merck Sharp & Dohme Corp.

Contact name

Danny Liaw

Public contact point

Organisation

Merck Sharp & Dohme Corp.

Contact name

Danny Liaw

Third parties 2

Locations

1 EU/EEA country · 2 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 13 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

8 submissions — initial application plus substantial / non-substantial modifications