BDB001-101: Clinical Study of BDB001 as a Mono-therapy or in Combination With Pembrolizumab

NCT03486301 | Completed Phase 1 FDA Drug

• 2 other identifiers: BDB001-101KEYNOTE-914
• Study Type: interventional
• Target: 87
• Locations: 1 country
• Sites: 5

Brief Summary

Phase 1 Open-Label Dose Escalation Study of BDB001 as a Single Agent and in combination with Pembrolizumab in Subjects with Advanced Solid Tumors

Conditions

Tumor, Solid

Keywords

TLR7/8; Immuno-oncology; EIK1001

Outcome Measures

Primary Outcomes (1)

• Safety and Tolerability: incidence of adverse events and any dose limiting toxicity

  Safety and tolerability of BDB001 as a single agent and in combination with pembrolizumab as measured by the incidence of adverse events and any dose limiting toxicity

  Up to 30 months

Secondary Outcomes (2)

• Determine Maximum Tolerated Dose

  From first dose to 21 days after first dose for each patient (cycle 1)

• Radiographic Determination of Tumor Response after BDB001 Dosing

  Every 63 days (3 cycles) up to 30 months after the first dose for each patient (each cycle is 21 days)

Study Arms (2)

Single Agent BDB001 (EIK1001)

EXPERIMENTAL

A single subject will be enrolled at each dose level in the single agent arm until any ≥ Grade 2 treatment-emergent adverse event (TEAE) is observed in the first cycle. Then dosage escalation will follow a traditional 3+3 dose escalation design. Each successive group of patients will be enrolled at an incrementally higher dosage until the Maximum Tolerable Dose (MTD) or Recommended Phase 2 Dose (RP2D) of single agent BDB001 is reached.

Drug: BDB001 (EIK1001)

BDB001 in Combination with Pembrolizumab

EXPERIMENTAL

In the combination arm of the study, a standard 3+3 dose escalation design will be utilized for all dose levels. When the MTD or RP2D of single agent BDB001 is reached, the first dose level cohort of the combination arm will begin. Once the MTD or RP2D in combination has been determined, twenty additional subjects will be enrolled in the expansion phase of the study.

Drug: BDB001 (EIK1001); Drug: Pembrolizumab

Interventions

BDB001 (EIK1001) DRUG

BDB001 (EIK1001) is an immunotherapy agent.

BDB001 in Combination with Pembrolizumab; Single Agent BDB001 (EIK1001)

Pembrolizumab DRUG

Pembrolizumab is a potent humanized monoclonal antibody with high specificity of binding to the PD 1 receptor. Pembrolizumab has an acceptable preclinical safety profile and is approved as an IV therapy for a variety of advanced malignancies.

BDB001 in Combination with Pembrolizumab

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants are eligible to be included in the study only if all of the following criteria apply:
• Be 18 years of age on day of signing informed consent
• Subjects with histologically or cytologically confirmed advanced or metastatic solid tumors who have disease progression after treatment with all available therapies for metastatic disease that are known to confer clinical benefit, or are intolerant to treatment, or refuse standard treatment. Note: there is no limit to the number of prior treatment regimens
• A male participant must agree to use contraception during the treatment period and for at least 120 days after the last dose of study treatment and refrain from donating sperm during this period
• A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies:
• Not a woman of childbearing potential (WOCBP)
• A WOCBP who agrees to follow contraceptive guidance during the treatment period and for at least 120 days after the last dose of study treatment • A highly effective method of contraception is defined as one that results in a low failure rate (i.e., less than 1% per year, when used consistently and correctly)
• Evidence of progressive disease (PD) within 3 months of signing the informed consent form
• Have measurable disease per irRECIST as assessed by the local site investigator/radiology. Lesions situated in a previously irradiated area are considered measurable if progression has been demonstrated in such lesions
• Eastern Cooperative Oncology Group (ECOG) score of 0 - 2
• Minimum life expectancy of 3 months
• Have adequate organ function as defined by the protocol. Specimens must be collected within 10 days prior to the start of study treatment.

You may not qualify if:

• Participants are excluded from the study if any of the following criteria apply:
• A woman of child bearing potential who has a positive urine pregnancy test (e.g. within 72 hours) prior to treatment. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required
• Prior exposure to TLR 7 agonists (e.g., GS-9620, imiquimod, TMX 101, resiquimod, MEDI9197, 825A) and TLR 9 agonists (e.g., SD-101, IMO2125, MGN1703, GNKG168, DUK-CPG-001 and CMP-001) for treatment of the solid tumor the patient is currently being evaluated for treatment with BDB001.
• Has received prior therapy with an anti-PD-1, anti-PD-L1, or anti PD L2 agent or with an agent directed to another stimulatory or co-inhibitory T-cell receptor (e.g., CTLA-4, OX 40, CD137) and was discontinued from that treatment due to a Grade 3 or higher irAE
• Received previous therapy for malignancy within 21 days prior to administration of study drug, including any investigational agents (other than BDB001), chemotherapy, immunotherapy, biological or hormonal therapy (6 weeks for nitrosoureas or mitomycin C)
• Major surgery within 4 weeks of first dose of study drug
• Has received prior radiotherapy within 2 weeks of start of study treatment. Participants must have recovered from all radiation-related toxicities, not require corticosteroids, and not have had radiation pneumonitis. A 1-week washout is permitted for palliative radiation (≤2 weeks of radiotherapy) to non-CNS disease
• Has received a live vaccine within 30 days prior to the first dose of study drug. Examples of live vaccines include, but are not limited to, the following: measles, mumps, rubella, varicella/zoster (chicken pox), yellow fever, rabies, Bacillus Calmette-Guérin (BCG), and typhoid vaccine. Seasonal influenza vaccines for injection are generally killed virus vaccines and are allowed; however, intranasal influenza vaccines (e.g., FluMist) are live attenuated vaccines and are not allowed
• Currently receiving medications known to be strong inhibitors of CYP1A and CYP3A and strong/moderate inducers of CYP1A and CYP3A
• Receiving systemic steroid therapy or any other form of immunosuppressive therapy within 7 days prior to the first dose of study drug. The use of physiologic doses of corticosteroids may be approved after consultation with the Sponsor.
• Is currently participating in or has participated in a study of an investigational agent or has used an investigational device within 21 days prior to the first dose of study treatment. Note: Participants who have entered the follow-up phase of an investigational study may participate as long as it has been 4 weeks after the last dose of the previous investigational agent
• History within last 6 months of New York Heart Association Class III or IV heart failure, acute myocardial infarction, angina pectoris, uncontrolled arrhythmia, acute coronary syndromes, stent placement, uncontrolled hypertension
• QTc interval value \> 470 msec (using Fridericia's Correction)
• Left ventricular ejection fraction (LVEF) \< 50% by echocardiogram (ECHO) or multigated acquisition (MUGA) scan
• Has known active CNS metastases and/or carcinomatous meningitis. Participants with previously treated brain metastases may participate provided they are radiologically stable, i.e., without evidence of progression for at least 4 weeks by repeat imaging (note that the repeat imaging should be performed during study screening), clinically stable and without requirement of steroid treatment for at least 14 days prior to first dose of study treatment (Kim 2017).

Sponsors & Collaborators

• Eikon Therapeutics: lead
• Merck Sharp & Dohme LLC: collaborator

Study Sites (5)

Florida Cancer Specialists

Sarasota, Florida, 34230, United States

Location

Atlantic Health

Morristown, New Jersey, 07960, United States

Location

Tennessee Oncology

Nashville, Tennessee, 37203, United States

Location

NEXT Oncology

San Antonio, Texas, 78229, United States

Location

South Texas Accelerated Research Therapeutics (START)

San Antonio, Texas, 78229, United States

Location

MeSH Terms

Conditions

Neoplasms

Interventions

BDB001; pembrolizumab

Study Officials

• Harry Raftopoulos, MD

  Eikon Therapeutics

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: BDB001 followed by combination treatment with KEYTRUDA®

Study Record Dates

• First Submitted: February 28, 2018
• First Posted: April 3, 2018
• Study Start: February 15, 2018
• Primary Completion: March 31, 2024
• Study Completion: April 30, 2024
• Last Updated: November 12, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)