Lymphedema of Upper ArmPhase 2HEALNCT05203835
A Placebo Controlled Trial of Acebilustat (CTX 4430) for the Treatment of Human Upper Extremity Lymphedema
A Phase 2 study testing Acebilustat in people with Lymphedema of Upper Arm, run by Stanford University at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Lymphedema of Upper Arm
- What is being tested?
- Acebilustat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 1 siteUnited States
- Study dates
- 27 Jul 2022to 1 Dec 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase II Placebo-Controlled Acebilustat (CTX 4430) in Treatment of Human Upper Extremity Lymphedema
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.
Read the detailed description
To enter this study, participants will have to meet requirements that will be evaluated during screening, Visit 1. If eligible, and if they choose to continue, Visit 2, treatment start, will take place 2-7 days later. Study visits are every (approximately) 90 days. Physical exam, including limb measurements and skin measurements (with tape measure and dermal ultrasound) are performed. Phlebotomy for research samples and clinical labs will be performed at study start and end of study.
- Conditions
- Lymphedema of Upper Arm
- Being tested
- Acebilustat, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AcebilustatDrug
By mouth, once a day, 100 mg capsule
PlaceboDrug
Placebo to match acebilustat by mouth, once a day
| Study group | Role | What they receive |
|---|---|---|
| Placebo and acebilustat | Experimental | Participants will take acebilustat and placebo over a period of 9 months. Receives: Drug: Acebilustat; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Cutaneous measurements will be derived from serial ultrasonographic examination at three designated locations in the affected upper extremity using a Terason 3200T device
Measured: 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.
Volume changes in the affected upper extremity will be calculated through circumferential measurements of the limb and quantitated with the truncated cone approximation.
Measured: Comparison of the measurement of week 0 to week 12 and of week 12 to week 36.
Change in LymQoL score
Change in the LymQoL (Lymphedema Quality of Life) questionnaire score will be used to assess the impact of lymphedema on patients' quality of life. The LymQoL is a validated, disease-specific tool measuring physical, psychological, appearance, and functional domains (range 0-100; higher scores indicate worse QoL).
Measured: Comparison of the measurement of week 0 to week 12 and of week 12 to week 36.
Change in LymVAS score
Change in the LymVAS (Lymphedema Visual Analog Scale) questionnaire score will be used to assess patient-perceived lymphedema severity (0 = none, 10 = worst).
Measured: Comparison of the measurement of week 0 to week 12 and of week 12 to week 36.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jan 2022
- Study start27 Jul 2022
- Primary completion21 Oct 2025
- Study completion1 Dec 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lymphedema of Upper ArmStudies of the same condition, open and under way.
- More trials run by Stanford UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Collaborators
- Celltaxis LLC
- Responsible party
- Sponsor
- Investigators
- Stanley Rockson, MD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05203835
- Sponsor's protocol ID
- IRB-62051
- Other IDs
- NCI-2022-09343, BRS0140
- Acronym
- HEAL
- Status last verified
- May 2026
- First submitted
- 10 Jan 2022
- Last updated
- 28 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Placebo-Controlled Acebilustat (CTX 4430) in Treatment of Human Upper Extremity Lymphedema
- ClinicalTrials.gov ID
- NCT05203835
- Acronym / sponsor ID
- HEAL / IRB-62051
- Conditions
- Lymphedema of Upper Arm
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Collaborators
- Celltaxis LLC
- Study start
- 27 Jul 2022 (actual)
- Primary completion
- 21 Oct 2025 (actual)
- Study completion
- 1 Dec 2025 (actual)
- First posted
- 24 Jan 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
To enter this study, participants will have to meet requirements that will be evaluated during screening, Visit 1. If eligible, and if they choose to continue, Visit 2, treatment start, will take place 2-7 days later. Study visits are every (approximately) 90 days. Physical exam, including limb measurements and skin measurements (with tape measure and dermal ultrasound) are performed. Phlebotomy for research samples and clinical labs will be performed at study start and end of study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Placebo and acebilustat | Experimental | Drug: Acebilustat, Drug: PlaceboParticipants will take acebilustat and placebo over a period of 9 months. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- 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.
Key exclusion criteria (14 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AcebilustatBy mouth, once a day, 100 mg capsule | Drug | Placebo and acebilustat |
| PlaceboPlacebo to match acebilustat by mouth, once a day | Drug | Placebo and acebilustat |
Primary outcome
Change in cutaneous thickness in the affected upper extremity, as measured by skin calipers.
Time frame: Comparison of the measurement of week 0 to week 12 and of week 12 to week 36
Cutaneous thickness will be measured by skin calipers at three designated locations in the affected upper extremity.
4 secondary outcomes
Change in cutaneous dimensions and architecture in the affected upper extremity, as measured by ultrasound
Time frame: 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.
Time frame: Comparison of the measurement of week 0 to week 12 and of week 12 to week 36.
Change in LymQoL score
Time frame: Comparison of the measurement of week 0 to week 12 and of week 12 to week 36.
Change in LymVAS score
Time frame: Comparison of the measurement of week 0 to week 12 and of week 12 to week 36.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 May 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Stanley Rockson, MD principal investigator
Stanford University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Acebilustat in people with Lymphedema of Upper Arm, run by Stanford University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in cutaneous thickness in the affected upper extremity, as measured by skin calipers. (time frame: Comparison of the measurement of week 0 to week 12 and of week 12 to week 36). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 May 2026.