BXQ-350 Pharmacokinetic/Pharmacodynamic Study in Cancer Patients

NCT05291286 | Completed Early Phase 1 DMC FDA Drug

• 1 other identifier: BXQ-350.AH
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 3

Brief Summary

This study will assess pharmacokinetic (PK)/pharmacodynamic (PD) relationships and whether BXQ-350 may decrease the intensity and/or duration of chemotherapy induced peripheral neuropathy (CIPN) thereby improving quality of life (QoL) in cancer patients who have been exposed to oxaliplatin and/or taxane-based chemotherapy. This study includes two randomized, placebo controlled, blinded treatment cycles of BXQ-350/placebo, an optional open-label BXQ-350 treatment period, and an unblinded Post-Treatment Follow-up period.

Conditions

Neuropathy;Peripheral; Chemotherapy-induced Peripheral Neuropathy

Outcome Measures

Primary Outcomes (7)

• Peak Plasma Concentration (Cmax)

  To evaluate the Cmax of BXQ-350.

  6 months

• Ceramide

  To evaluate ceramide levels following administration of BXQ-350.

  6 months

• S1P levels

  To evaluate S1P levels following administration of BXQ-350.

  6 months

• Cytokine levels

  To evaluate cytokine levels following administration of BXQ-350.

  6 months

• Quality of Life (QoL)

  To evaluate QoL in patients with neuropathy receiving BXQ-350. QoL will be measured utilizing the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30).

  6 months

• Total Sensory Neuropathy

  To evaluate neuropathy symptoms in patients with neuropathy receiving BXQ-350. Total sensory neuropathy scores will be obtained from the EORTC QLQ-CIPN20 questionnaire.

  6 months

• CIPN Assessment

  To evaluate CIPN symptoms in patients with neuropathy receiving BXQ-350 utilizing the CIPN Assessment Tool questionnaire.

  6 months

Secondary Outcomes (1)

• Incidence of Treatment Emergent Adverse Events as Assessed by CTCAE v5.0

  6 months

Study Arms (2)

BXQ-350

EXPERIMENTAL

BXQ-350 will be administered by IV infusion

Drug: BXQ-350

Placebo

PLACEBO COMPARATOR

Placebo (0.9% normal saline) will be administered by IV infusion

Other: Placebo

Interventions

BXQ-350 DRUG

BXQ-350 is a novel anti-neoplastic therapeutic agent configured from two components: Saposin C (SapC), an expressed (human) lysosomal protein, and the phospholipid dioleoylphosphatidyl-serine (DOPS), a phospholipid located on cell membranes (clinical formulation BXQ-350). BXQ-350 will be administered by intravenous (IV) infusion

Also known as: SapC-DOPS

BXQ-350

Placebo OTHER

Placebo will be 0.9% normal saline of matching volume to BXQ-350 administered by intravenous (IV) infusion

Placebo

Eligibility Criteria

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

You may qualify if:

• Participants who meet the following criteria will be considered eligible to participate in the clinical study:
• Age ≥ 18 years of age at the time of signing the informed consent.
• Have a diagnosis of cancer.
• Have symptoms of CIPN persisting ≥6 months and determined by the participant's treating physician to be caused by prior exposure to oxaliplatin or taxane-based chemotherapy.
• Have an EORTC QLQ-CIPN20 score of 3 (quite a bit) or 4 (very much) on at least 1 of the 6 questions pertaining to numbness, tingling, or pain in the fingers/hands or toes/feet.
• Have a life expectancy \> 12 months.
• Have ECOG Performance Status of 0 or 1.
• Have acceptable liver function defined as:
• Total serum bilirubin ≤ 1.5 x upper limit of normal (ULN) for the study site. In participants with known Gilbert Syndrome, total bilirubin ≤ 3 x ULN, with direct bilirubin ≤ 1.5 x ULN).
• Aspartate transaminase (AST), serum glutamic oxaloacetic transaminase (SGOT), alanine transaminase (ALT), serum glutamic pyruvic transaminase (SGPT) ≤ 3 x ULN (if liver metastases are present, then ≤ 5 x ULN is allowed).
• Serum albumin ≥ 3 g/ dL.
• Have acceptable renal function defined as:
• Creatinine clearance ≥ 50 mL/minute calculated using the Cockcroft-Gault formula (Cockcroft 1976):
• CCr = {((140 - age) x weight kg) / (72 x SCr)} x 0.85 (if female).
• Urine dipstick protein ≤ 1 + (30 - 70 mg/dL) OR urine protein/creatinine ratio of ≤ 1, OR 24 hour urine protein \< 1g/24 hours.

You may not qualify if:

• Participants must not meet any of the following criteria:
• Have received chemotherapy known to cause CIPN in the last 12 months.
• Currently receiving or expected to initiate chemotherapy for the treatment of an active cancer during the study period; cancer therapies utilized to maintain remission that are not known to cause or exacerbate peripheral neuropathy, as well as maintenance endocrine/hormonal/immune therapy for cancer are allowed. Continuation of polyadenosine diphosphate-ribose polymerase (PARP) inhibitors or other targeted therapies not associated with peripheral neuropathy is permitted.
• Have Type 1 or 2 diabetes mellitus.
• Have a family history of a genetic/familial neuropathy.
• Have pre-existing clinical neuropathy ≥ Grade 2 per CTCAE v5.0 from any cause.
• Currently taking daily oral steroids exceeding prednisone 10 mg daily or its equivalent.
• Participants with brain metastases may participate provided they are clinically stable for at least 4 weeks prior to study entry, have no evidence of new or enlarging metastases and are off steroids for at least 7 days.
• Have had major surgery within 28 days prior to randomization or have not recovered from major side effects of the surgery if more than 4 weeks have elapsed since surgery. Minor outpatient procedures are allowed.
• Have poorly controlled hypertension defined as blood pressure \> 150/90 mmHg on at least 2 repeated determinations prior to screening or on day of screening.
• Have a history of cardiac dysfunction including:
• Myocardial infarction within 6 months prior to initiation of screening.
• History of documented congestive heart failure (New York Heart Association functional classification III-IV) within 6 months prior to initiation of screening.
• Active cardiomyopathy.
• Electrocardiogram with QTc \> 470 milliseconds at screening.

Sponsors & Collaborators

• Bexion Pharmaceuticals, Inc.: lead
• CTI Clinical Trial and Consulting Services: collaborator

Study Sites (3)

CTI Clinical Research Center

Cincinnati, Ohio, 45212, United States

Location

The Ohio State Unviersity

Columbus, Ohio, 43210, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

MeSH Terms

Conditions

Neuritis

Condition Hierarchy (Ancestors)

Peripheral Nervous System Diseases; Neuromuscular Diseases; Nervous System Diseases

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: randomized, placebo controlled, double blind
• Purpose: SUPPORTIVE CARE
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Participants will be assigned to 1 of 2 treatment arms (BXQ-350 or placebo) in a 1:1 ratio for the first two treatment cycles. At the end of the second treatment cycle, participants will be unblinded to their individual treatment assignment. Participants who received placebo will be offered 2 cycles of open-label BXQ-350. Participants who received BXQ-350 in Cycles 1 and 2 will continue to the Post-Treatment Follow-Up. If placebo participants elect to forego BXQ-350 at the time of unblinding, their participation in the study will end following an end of study visit.

Study Record Dates

• First Submitted: March 11, 2022
• First Posted: March 22, 2022
• Study Start: October 17, 2022
• Primary Completion: December 20, 2024
• Study Completion: December 20, 2024
• Last Updated: January 27, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)