Last updated on September 2018

A Phase 2 clinical study for patients using Lenalidomide

Brief description of study

To determine the activity of lenalidomide in the treatment of pediatric subjects with relapsed/refractory AML (with second or greater relapse or refractory to at least 2 prior induction attempts) measured by morphological complete response defined as either a CR or CRi within the first 4 cycles of treatment.

Detailed Study Description

This is a multicenter, open-label, single-arm, Phase 2, Simon's Optimal two-stage design study, with an Optional Extension Phase (OEP), that will assess the activity, safety and Pharmacokinetics (PK) of lenalidomide in pediatric subjects from 1 to 18 years of age with second or greater Relapsed or Refractory Acute Myeloid Leukemia (rrAML). A total of 43 evaluable subjects (18 subjects in Stage 1 and an additional 25 subjects in Stage 2) are required for assessment of the primary endpoint. To allow for subjects found to be unevaluable for the primary endpoint due to an incorrect diagnosis, not having a disease assessment post screening, or who discontinued prior to receiving lenalidomide, up to 4 additional subjects may be enrolled for a maximum of 47 evaluable subjects across approximately 70 sites. Approximately 50% of enrolled subjects will be younger than 12 years of age to provide adequate PK data for this age subset.

If during Stage 1, at least 3 of 18 subjects achieve a morphologic complete response (either CR or CRi) within the first 4 cycles of study treatment, then the study will proceed to Stage 2; otherwise, the study will be terminated. Similarly, if at the final analysis, at least 8 of 43 evaluable subjects across Stages 1 and 2 achieve a response (CR/CRi) within the first 4 cycles of study treatment, it will be concluded that lenalidomide has sufficient activity in pediatric Acute Myeloid Leukemia (AML) to warrant subsequent study.

The optional extension phase (OEP) will allow subjects who demonstrate clinical benefit, as assessed by the Investigator at the completion of 12 cycles of lenalidomide therapy, to continue receiving oral lenalidomide until they meet the criteria for study discontinuation. In the OEP, only safety, dosing, concomitant medications/procedures, and second primary malignancies (SPMs) will be monitored.

Clinical Study Identifier: NCT02538965

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Children's Hospital
Birmingham, AL United States

Phoenix Childrens Hospital
Phoenix, AZ United States

Arkansas Children's Hospital
Little Rock, AR United States

Miller Children's Hospital
Long Beach, CA United States

Children's Hospital of Los Angeles
Los Angeles, CA United States

Southern California Permanente Medical Group
Los Angeles, CA United States

Valley Children's Hospital
Madera, CA United States

Children's Hospital of Orange County
Orange, CA United States

Lucile Salter Packard Children's Hospital at Stanford
Palo Alto, CA United States

Loma Linda University
San Bernardino, CA United States

UCSF Children's Hospital
San Francisco, CA United States

Colorado Children's Hospital
Aurora, CO United States

Connecticut Children's Medical Center
Hartford, CT United States

Alfred I Dupont Hospital For Children
Wilmington, DE United States

Golisano Children's Hospital of Southwest Florida
Fort Myers, FL United States

Nemours Children's Clinic
Jacksonville, FL United States

All Children's Hospital
Saint Petersburg, FL United States

Children's Healthcare of Atlanta
Atlanta, GA United States

Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, IL United States

Advocate Chilldren's Hospital
Oak Lawn, IL United States

Riley Hospital For Children at IU Health
Indianapolis, IN United States

Kosair Children's Hospital
Louisville, KY United States

Children's Hospital New Orleans
New Orleans, LA United States

Johns Hopkins University
Baltimore, MD United States

University of Michigan
Ann Arbor, MI United States

Children's Hospitals and Clinics of Minnesota
Minneapolis, MN United States

University of Minnesota
Minneapolis, MN United States

University of Mississippi Medical Center
Jackson, MS United States

Children's Mercy Hospital
Kansas City, MO United States

Washington University School of Medicine
Saint Louis, MO United States

Children's Specialty Center of Nevada
Las Vegas, NV United States

Hackensack University Medical Center
Hackensack, NJ United States

Morristown Memorial Hosp
Morristown, NJ United States

Cancer Institute of New Jersey
New Brunswick, NJ United States

Roswell Park Cancer Inst
Buffalo, NY United States

Columbia University Medical Center
New York, NY United States

University of Rochester Medical Center
Rochester, NY United States

New York Medical College
Valhalla, NY United States

University of North Carolina
Chapel Hill, NC United States

Cincinnati Children's Hospital Medical Center
Cincinnati, OH United States

Nationwide Children's Hospital
Columbus, OH United States

Legacy Emanuel Hospital and Health Center
Portland, OR United States

Penn State Milton S Hershey Medical Center
Hershey, PA United States

UPMC Childrens Hospital of Pittsburgh
Pittsburgh, PA United States

Carolinas Healthcare System
Charleston, SC United States

Greenville Health System
Greenville, SC United States

Vanderbilt University Medical Center
Nashville, TN United States

Dell Children's Medical Center of Central Texas
Austin, TX United States

University of Texas Southwestern Medical Center
Dallas, TX United States

Texas Children's Cancer Center
Houston, TX United States

Methodist Hospital
San Antonio, TX United States

Primary Children's Medical Center
Salt Lake City, UT United States

Children's Hospital of The King's Daughters
Norfolk, VA United States

Seattle Children's Hospital
Seattle, WA United States

Midwest Children's Cancer Center
Milwaukee, WI United States

Alberta Childrens Hospital
Calgary, AB Canada

British Columbia Children's Hospital
Vancouver, BC Canada

IWK Health Center
Halifax, NS Canada

Childrens Hospital of Eastern Ontario
Ottawa, ON Canada

McGill University Health Center
Montreal, QC Canada

Hospital For Sick Children
Torento, Canada

Children's Hospital National Medical Center
Washington, WA United States