X4P-001 and Pembrolizumab in Patients With Advanced Melanoma

NCT02823405 | Completed Phase 1 FDA Drug

• 1 other identifier: X4P-001-MELA
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 4

Brief Summary

The goals of this protocol are 1) to investigate the safety and tolerability of X4P-001 in combination with Keytruda® (pembrolizumab) in patients with advanced melanoma, and 2) to assess serial biopsies of melanoma tumor lesions obtained throughout the study for inflammatory and tumor cell infiltrates. After completion of study treatment, participants with resectable disease will undergo surgery, unresectable participants may continue on pembrolizumab as standard of care.

Conditions

Melanoma

Keywords

Advance malignancy

Outcome Measures

Primary Outcomes (2)

• Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability)

  Up to 13 weeks, from time of enrollment through study completion or early termination.

• Histology Characterization in Sequential Biopsies of Melanoma Lesions

  Up to 13 weeks, from time of enrollment through study completion or early termination.

Secondary Outcomes (3)

• Blood Biomarker Changes

  Up to 17 weeks, from time of screening through study completion or early termination.

• Minimum Plasma Concentration (Cmin)

  Up to 9 weeks, from time of enrollment through end of treatment.

• Clinical Tumor Response

  Up to 17 weeks, from time of screening through study completion or early termination.

Study Arms (1)

X4P-001 + Pembrolizumab

EXPERIMENTAL

X4P-001 alone, then adding pembrolizumab

Drug: X4P-001; Drug: Pembrolizumab

Interventions

X4P-001 DRUG

X4P-001 100 mg capsules, administered orally, continuous daily dosing

Also known as: AMD11070

X4P-001 + Pembrolizumab

Pembrolizumab DRUG

Pembrolizumab 2 mg/kg, administered by IV infusion every 3 weeks

Also known as: Keytruda

X4P-001 + Pembrolizumab

Eligibility Criteria

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

You may qualify if:

• Be at least 18 years of age.
• Has signed the current approved informed consent form.
• Has a histologically confirmed diagnosis of malignant melanoma.
• Has at least two separate cutaneous lesions suitable for punch biopsies (at least 3 mm diameter).
• For women of childbearing potential and men, agree to use a highly effective method of contraceptive from screening, through the study, and for at least 4 weeks after the last dose of study drug.
• For women of childbearing potential, must have a negative pregnancy test (serum or urine) on Day 1 prior to initiating study treatment, and are not nursing.
• Be willing and able to comply with the schedule, treatment, and biopsies specified by this protocol.

You may not qualify if:

• Patients with any of the following will be excluded from participation in the study:
• Has performance status Grade 2 or higher (Eastern Cooperative Oncology Group \[ECOG\] criteria).
• Has ongoing acute clinical adverse events NCI CTCAE Grade 2 or greater resulting from prior cancer therapies (except alopecia).
• Has had within the past 6 months the occurrence or persistence of one or more of the following medical conditions that could not be controlled with usual medical care (e.g., required emergency care or hospitalization): angina, congestive heart failure, diabetes, seizure disorder.
• Has had within the past 6 months the occurrence of one or more of the following events: myocardial infarction, cerebrovascular accident, hemorrhage (CTC Grade 3 or 4), chronic liver disease (meeting criteria for Child-Pugh Class B or C), a second active malignancy requiring ongoing treatment during the trial, organ transplantation.
• Has had within the 4 weeks prior to initiation of study drug, or is expected to have during the study period, surgery requiring general anesthesia
• Has, at screening, serologic laboratory tests meeting one or more of the following criteria:
• An indeterminate or positive test for antibody to human immunodeficiency virus (HIV-1 or -2).
• An indeterminate or positive test for antibody to hepatitis C virus (HCV), unless documented to have no detectable viral load on two independent samples.
• A positive test for hepatitis B surface antigen (HBsAg).
• Has, at screening, safety laboratory tests meeting one or more of the following criteria:
• Hemoglobin \<9.0 g/dL
• Absolute neutrophil count (ANC) \<1,500/μL
• Platelets \<100,000/μL
• Creatinine \>2.0x ULN

Sponsors & Collaborators

• X4 Pharmaceuticals: lead

Study Sites (4)

Clinical Site

Atlanta, Georgia, 30322, United States

Location

Clinical Site

Iowa City, Iowa, 52242, United States

Location

Clinical Site

Houston, Texas, 77030, United States

Location

Clinical Site

Salt Lake City, Utah, 84112, United States

Location

Related Publications (1)

• Andtbacka RHI, Wang Y, Pierce RH, Campbell JS, Yushak M, Milhem M, Ross M, Niland K, Arbeit RD, Parasuraman S, Bickley K, Yeung CC, Aicher LD, Smythe KS, Gan L. Mavorixafor, an Orally Bioavailable CXCR4 Antagonist, Increases Immune Cell Infiltration and Inflammatory Status of Tumor Microenvironment in Patients with Melanoma. Cancer Res Commun. 2022 Aug 31;2(8):904-913. doi: 10.1158/2767-9764.CRC-22-0090. eCollection 2022 Aug.

  PMID: 36923305 DERIVED

MeSH Terms

Conditions

Melanoma

Interventions

mavorixafor; pembrolizumab

Condition Hierarchy (Ancestors)

Neuroendocrine Tumors; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Nerve Tissue; Nevi and Melanomas; Skin Neoplasms; Neoplasms by Site; Skin Diseases; Skin and Connective Tissue Diseases

Study Officials

• Lu Gan, MD, PhD

  X4 Pharmaceuticals, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 1, 2016
• First Posted: July 6, 2016
• Study Start: September 15, 2016
• Primary Completion: March 15, 2018
• Study Completion: March 15, 2018
• Last Updated: July 7, 2020

Record last verified: 2020-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)