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A Study to Evaluate DAY101 in Pediatric and Young Adult Patients With Relapsed or Progressive Low-Grade Glioma

Study Purpose

FIREFLY-1 is a Phase 2, multi center, open-label study to evaluate the safety and efficacy of oral pan-RAF inhibitor DAY101 in pediatric, adolescent, and young adult patients with recurrent or progressive low-grade glioma harboring a known BRAF alteration.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages 6 Months - 25 Years
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria:

  • - Age 6 months to 25 years with a relapsed or progressive LGG with known activating BRAF alteration.
  • - Confirmation of histopathologic diagnosis of LGG and molecular diagnosis of activating BRAF alteration.
  • - Must have received at least one line of systemic therapy and have evidence of radiographic progression.
  • - Must have at least 1 measurable lesion as defined by RANO criteria.

Exclusion Criteria:

  • - Patient's tumor has additional previously-known activating molecular alterations.
  • - Patient has symptoms of clinical progression in the absence of radiographic progression.
- Known or suspected diagnosis of neurofibromatosis type 1 (NF-1) - Other inclusion/exclusion criteria as stipulated by protocol may apply

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT04775485
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 2
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Day One Biopharmaceuticals, Inc.
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

N/A
Principal Investigator Affiliation N/A
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

IndustryOther
Overall Status Recruiting
Countries Australia, Canada, Korea, Republic of, Switzerland, United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Low-grade Glioma
Additional Details

Approximately 60 pediatric patients will be treated with DAY101, an oral pan-RAF inhibitor, for a planned period of 26 cycles will be treated with DAY101 for a planned period of 26 cycles (approximately 24 months). DAY101 will be administered at the recommended Phase 2 dose (RP2D) of 420 mg/m2 (not to exceed 600 mg) orally once weekly (QW) for each 28-day treatment cycle. Treatment cycles will repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients will undergo radiographic evaluation of their disease at the end of every third cycle. Patients will continue on DAY101 until radiographic evidence of disease progression by RANO criteria as determined by treating investigator, unacceptable toxicity, patient withdrawal of consent, or death. Patients who have radiographic evidence of disease progression may be allowed to continue DAY101 if, in the opinion of the investigator and approval by the Sponsor, the patient is deriving clinical benefit from continuing study treatment. Disease assessments for patients being treated beyond progression should continue as per regular schedule. DAY101 is an oral pan-RAF inhibitor administered as an oral tablet at 420 mg/m2 (not to exceed 600 mg).

Arms & Interventions

Arms

Experimental: Single Arm

DAY101, an oral pan-RAF inhibitor in immediate-release tablet form in 2 strengths, 20 mg and 100 mg.

Interventions

Drug: - DAY101

DAY101 is an oral pan-RAF inhibitor provided as a white to off-white crystalline powder as an immediate-release tablet in 2 strengths, 20 mg and 100 mg.

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Children's National Medical Center, Washington, District of Columbia

Status

Recruiting

Address

Children's National Medical Center

Washington, District of Columbia, 20010

Site Contact

braintumorresearch@childrensnational.org

firefly-1@dayonebio.com

202-476-5000

Johns Hopkins Hospital, Baltimore, Maryland

Status

Recruiting

Address

Johns Hopkins Hospital

Baltimore, Maryland, 21231

CS Mott Children's Hospital, Ann Arbor, Michigan

Status

Recruiting

Address

CS Mott Children's Hospital

Ann Arbor, Michigan, 48109

St. Louis Children's Hospital, Saint Louis, Missouri

Status

Recruiting

Address

St. Louis Children's Hospital

Saint Louis, Missouri, 63110

Duke Univ. Medical Center, Durham, North Carolina

Status

Recruiting

Address

Duke Univ. Medical Center

Durham, North Carolina, 27710

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania

Status

Recruiting

Address

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104

Texas Children's Hospital, Houston, Texas

Status

Recruiting

Address

Texas Children's Hospital

Houston, Texas, 77030

Seattle Children's Hospital, Seattle, Washington

Status

Recruiting

Address

Seattle Children's Hospital

Seattle, Washington, 98105

International Sites

Melbourne - Royal Children's, Parkville, Australia

Status

Recruiting

Address

Melbourne - Royal Children's

Parkville, , 3052

Perth Children's Hospital, Perth, Australia

Status

Recruiting

Address

Perth Children's Hospital

Perth, , WA 6009

Sydney Children's Hospital, Randwick, Australia

Status

Recruiting

Address

Sydney Children's Hospital

Randwick, , NSW 2031

CHU-Sainte Justine, Montreal, Quebec, Canada

Status

Recruiting

Address

CHU-Sainte Justine

Montreal, Quebec, H3T 1C5

McGill University, Montreal, Quebec, Canada

Status

Recruiting

Address

McGill University

Montreal, Quebec, H4A 3J1

Centre Mère-Enfant Soleil du CHU, Québec, Quebec, Canada

Status

Recruiting

Address

Centre Mère-Enfant Soleil du CHU

Québec, Quebec, G1V 4G2

Seoul National University Hospital, Seoul, Korea, Republic of

Status

Recruiting

Address

Seoul National University Hospital

Seoul, , 3080

Severance Hospital - Yonsei University, Seoul, Korea, Republic of

Status

Recruiting

Address

Severance Hospital - Yonsei University

Seoul, , 3722

Zürich, Switzerland

Status

Recruiting

Address

Universitäts-Kinderspital Zürich - Eleonorenstiftung

Zürich, , 8032