Trial of Acebilustat for the Treatment of Upper Arm Lymphedema

NCT05203835 | Active Not Recruiting Phase 2 DMC FDA Drug

• 3 other identifiers: IRB-62051NCI-2022-09343BRS0140
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

This study is designed to investigate the response of unilateral upper extremity (arm) lymphedema, during pharmacologic treatment of lymphedema with oral placebo and oral acebilustat. Participants will receive "study drug" (Acebilustat or placebo), for 9 months. For 3 of these months, the participant will receive placebo; for 6 of these months, the participant will receive active ingredient, acebilustat. The study is blinded which means that the participant will not be told which study pill they are taking.

Conditions

Lymphedema of Upper Arm

Outcome Measures

Primary Outcomes (1)

• Change in cutaneous thickness in the affected upper extremity, as measured by skin calipers.

  Cutaneous thickness will be measured by skin calipers at three designated locations in the affected upper extremity.

  Comparison of the measurement of week 0 to week 12 and of week 12 to week 36

Secondary Outcomes (4)

• Change in cutaneous dimensions and architecture in the affected upper extremity, as measured by ultrasound

  Comparison of the measurement of week 0 to week 12 and of week 12 to week 36

• Change in the volume of the affected upper extremity.

  Comparison of the measurement of week 0 to week 12 and of week 12 to week 36.

• Change in LymQoL score

  Comparison of the measurement of week 0 to week 12 and of week 12 to week 36.

• Change in LymVAS score

  Comparison of the measurement of week 0 to week 12 and of week 12 to week 36.

Study Arms (1)

Placebo and acebilustat

EXPERIMENTAL

Participants will take acebilustat and placebo over a period of 9 months.

Drug: Acebilustat; Drug: Placebo

Interventions

Acebilustat DRUG

By mouth, once a day, 100 mg capsule

Placebo and acebilustat

Placebo DRUG

Placebo to match acebilustat by mouth, once a day

Placebo and acebilustat

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Upper arm lymphedema, single arm, stage 2, greater than 6 months duration
• Male or female.
• Ages 18-75.
• Prior imaging by lymphoscintigraphy or magnetic resonance lymphangiography that confirms the presence of lymphedema in the affected limb OR, at screening, an affected:unaffected limb volume ratio of ≥1.1 with significant history of lymphedema
• Consistent use of an appropriately sized compression garment for daytime use.
• Willing to maintain a stable regimen of self-care from screening to end-of-study.
• If a potential participant has undergone prior microvascular (vascular lymph node transfer, lymphaticovenous anastomosis) or debulking surgical intervention, at least one year must have elapsed prior to screening AND, at screening, an affected: unaffected limb volume ratio of ≥1.1.
• Lymphedema therapy must be completed at least 8 weeks prior to screening.
• Has received Covid-19 vaccine (Pfizer, Moderna or Johnson \& Johnson)
• Ability to understand and the willingness to sign a written informed consent document.
• If the possibility of conception exits, agrees to use a medically acceptable method of contraception (both male and female) from the signing of the informed consent form through the entire study period; men or women who are surgically sterile (\> 6 months after surgery) or women who have been postmenopausal for at least 1 year are not considered to be of childbearing potential.

You may not qualify if:

• Concurrent participation in a clinical trial of any other investigational drug or therapy
• Other medical condition that could lead to acute limb edema (e.g. acute blood clot) or other medical condition that could result in symptoms overlapping those of lymphedema (e.g. frozen shoulder).
• History of clotting disorder.
• Chronic (persistent) infection in the affected limb.
• Active cancer treatment or history of cancer treatment within the past 2 years, except for non-melanoma skin cancer or cervical cancer in-situ.
• Chronic kidney disease
• Liver disease
• Pregnancy or nursing.
• Substance abuse (e.g., alcohol or drug abuse) within 6 months prior to screening.
• Any current use of non-steroidal anti-inflammatory drug (NSAID), e.g. ibuprofen, ketoprofen) or prior therapeutic use of ketoprofen.
• Any current use of immunosuppressive or immunomodulatory drugs (e.g., immunosuppressants, anticancer drugs, interleukins, interleukin antagonists or interleukin receptor blockers) or leukotriene pathway inhibitor (zileuton), leukotriene receptor antagonist (e.g montelukast).
• Personal or family history of prolonged QT syndrome
• Any reason (in addition to those listed above) that, in the opinion of the investigator, precludes full participation in the study.
• Any current use of statin drugs. The use of any statin drug should be discontinued at least 2 weeks prior to the trial enrollment

Sponsors & Collaborators

• Stanford University: lead
• Celltaxis LLC: collaborator

Study Sites (1)

Stanford University

Palo Alto, California, 94305, United States

Location

MeSH Terms

Interventions

acebilustat

Study Officials

• Stanley Rockson, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 10, 2022
• First Posted: January 24, 2022
• Study Start: July 27, 2022
• Primary Completion: October 21, 2025
• Study Completion: March 31, 2026
• Last Updated: November 25, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)