Chronic Granulomatous Diseases (CGD) and Liver LesionsPhase 1NCT01851460
Radiofrequency Ablation for Liver Abscesses From Chronic Granulomatous Disease
A Phase 1 study testing Cool-tip RF Ablation System in people with Chronic Granulomatous Diseases (CGD) and Liver Lesions, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.
Trial summary
Background: Abscesses are a pocket of infection in an organ or tissue. Patients with a disease called chronic granulomatous disease (CGD) often develop these abscesses. CGD is an inherited disorder that affects how white blood cells function. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Chronic Granulomatous Diseases (CGD) and Liver Lesions
- What is being tested?
- Cool-tip RF Ablation System
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 8 May 2013to 24 Mar 2020
- Phase
- Phase 1
- 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
- A patient will be included if he or she meets all of the following criteria:
- Has documented chronic granulomatous disease
- Age 18 - 75
- Has a liver abscess infected with Staphylococcus aureus or other microorganism susceptible to RFA, but is not an optimal candidate for curative surgical resection either due to location of disease, multiplicity of disease, or previous surgery or other comorbidities, such as pulmonary insufficiency, or has other contraindications to general anesthesia or perioperative management or refuses surgery.
- Is willing to return to NIH for imaging scans
- Is willing to undergo testing or procedures associated with this protocol
- Has failed long term antibiotic treatment and abscess drainage if applicable.
Who cannot join
- A patient will be excluded if he or she satisfies 1 or more of the following criteria:
- Positive results for toxin-producing bacteria obtained from liver biopsy in the pertinent abscess.
- Is a good candidate for liver-curative open surgical resection and does not refuse the surgery.
- Is not a candidate for RFA therapy due to lesion size or location.
- Has a prothrombin time (PT) or partial thromboplastin time (PTT) >1.5 times normal (except in patients who have a known lupus anticoagulant or other condition which a hematologist deems will not cause excessive bleeding despite the abnormal coagulation parameters).
- Has a platelet count <50,000/mm(3) which cannot be maintained despite platelet transfusions.
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- If you are pregnant.
- Any condition that, in the investigator s opinion, places the patient at undue risk by participating in the study
- Please Note: Co-morbidities in critically ill patients will not themselves constitute exclusion criteria because the cause of their illness/condition may require the use of RFA as a less invasive treatment than surgery.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
Official title: Radiofrequency Ablation (RFA) for the Treatment of Liver Abscesses in Patients With Chronic Granulomatous Disease
Background:
- Abscesses are a pocket of infection in an organ or tissue. Patients with a disease called chronic granulomatous disease (CGD) often develop these abscesses. CGD is an inherited disorder that affects how white blood cells function. Liver abscesses in people with CGD often require surgery to remove them and treat the infection. However, some people with CGD cannot have full surgery because it would be too risky. Researchers want to try a procedure called radiofrequency ablation (RFA) to treat these liver abscesses. RFA can usually be done without a major operation. This study will see if RFA is a safe and effective treatment for liver abscesses in patients with CGD.
Read more
Objectives:
- To see if RFA is a safe and effective treatment for CGD-related liver abscesses.
Eligibility:
- Individuals between 18 and 75 years of age with CGD who have liver abscesses that cannot be treated with surgery.
Design:
- Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected. Imaging studies will be performed on the liver.
- Participants will have RFA for the abscesses. RFA is an image-guided technique that heats and destroys specific tissue, such as tumor tissue. It will target any abscesses on the liver.
- After the procedure, participants will stay in the hospital for monitoring before being released.
- Participants will have regular follow-up visits for up to 1 year after treatment. Blood and urine samples will be collected. Additional imaging studies will be performed.
Read the detailed description
Radiofrequency Ablation (RFA) has become an increasingly common therapeutic treatment for neoplasms in the liver. A number of devices are now Food and Drug Administration cleared for this indication, and a growing body of literature supports this technique as a therapeutic option for patients with primary or metastatic hepatic malignancies. In vivo animal studies have also shown that this technique can also be used to treat infections. Insertion of the thermal energy delivery probe into an infected liver abscess destroys the bacteria while preserving surrounding tissue. Off-label use of RFA was successfully used to treat 22 abscesses in 4 patients with chronic granulomatous disease who had inoperable liver abscesses. The proposed clinical trial will specifically evaluate the feasibility, safety, and to a lesser extent, efficacy of RFA to treat liver abscesses in subjects with previously diagnosed chronic granulomatous disease. This will be a non-randomized case study conducted at the Clinical Center at the National Institutes of Health. One RFA device will be used. Ten subjects will be enrolled. If the method proves to be both feasible and safe, detailed analysis on efficacy will be performed. RFA eventually could play an important clinical role in patients with chronic granulomatous disease and liver abscesses that are not amenable to surgical management and are without other effective therapeutic options, or might otherwise be incompletely treated with surgical resection and debridement alone.
- Being tested
- Cool-tip RF Ablation System
- Keywords
- Non-surgical Liver Lesions, Chronic Granulomatous Disease (CGD), Microorganisms, Bacteria, Liver Lesions
- Medical subject headings
- Granulomatous Disease, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cool-tip RF Ablation SystemDevice
RFA therapy will be administered according to the instructions of the RFA device manufacturer. After infiltration of the skin (for percutaneous approach) with a local anesthetic, the needle probe will be directed into the lesion. For all methods, the needle placement is confirmed to be within the lesion by ultrasound or other imaging modality as appropriate. After confirming needle placement, the lesion will be heated to a target temperature of >75 (Infinite)c for up to 15 minutes. The exposure time may be varied depending on the temperatures achieved. Depending on the size of the lesion to be ablated, multiple passes of the probe as well as multiple ablation cycles may be performed. Adequacy of ablation will be monitored by ultrasound throughout the procedure. Depending on the size of the lesions and the time required to complete the ablation, multiple lesions may be treated as staged procedures so as to improve safety.
| Study group | Role | What they receive |
|---|---|---|
| RFA | Experimental | All subjects enrolled onto this study will receive treatment of their liver abscess (es) by RFA ablation Receives: Device: Cool-tip RF Ablation System |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of RFA in the treatment of liver abscesses in CGD subjects
The primary objective of this study is to determine the safety of radiofrequency ablation (RFA) in the treatment of liver abscesses in subjects with chronic granulomatous disease (CGD).
Measured: Fater each patient completes the study
Secondary outcomes (2)
Determine if RFA is a reasonable treatment options
To determine if RFA is a reasonable treatment option for patients with liver abscesses who are not suitable candidates for completely curative hepatic surgery
Measured: After last patient completes the study
Compare recovery outcomes
To compare the recovery outcomes of patients undergoing RFA versus historical controls for patients undergoing surgery for treatment of liver abscesses
Measured: After last patient completes the study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 May 2013 (estimated)
- Study start8 May 2013
- Primary completion24 Mar 2020
- Study completion24 Mar 2020
Why it stopped: PI choose to close the study
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Chronic Granulomatous Diseases (CGD) and Liver LesionsStudies of the same condition, open and under way.
- More trials run by National Institute of Allergy and Infectious Diseases (NIAID)The 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Elizabeth M Kang, M.D., Principal investigator, National Institute of Allergy and Infectious Diseases (NIAID)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01851460
- Sponsor's protocol ID
- 130117
- Other IDs
- 13-I-0117
- Status last verified
- Oct 2019
- First submitted
- 8 May 2013
- Last updated
- 26 Mar 2020
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Livraghi T. Radiofrequency ablation, PEIT, and TACE for hepatocellular carcinoma. J Hepatobiliary Pancreat Surg. 2003;10(1):67-76. doi: 10.1007/s10534-002-0714-y.
PubMed 12918460Lublin M, Bartlett DL, Danforth DN, Kauffman H, Gallin JI, Malech HL, Shawker T, Choyke P, Kleiner DE, Schwartzentruber DJ, Chang R, DeCarlo ES, Holland SM. Hepatic abscess in patients with chronic granulomatous disease. Ann Surg. 2002 Mar;235(3):383-91. doi: 10.1097/00000658-200203000-00010.
PubMed 11882760
Study at a glance
From the registry record, last updated by the sponsor on 26 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Radiofrequency Ablation (RFA) for the Treatment of Liver Abscesses in Patients With Chronic Granulomatous Disease
- ClinicalTrials.gov ID
- NCT01851460
- Acronym / sponsor ID
- 130117
- Keywords
- Non-surgical Liver Lesions, Chronic Granulomatous Disease (CGD), Microorganisms, Bacteria, Liver Lesions
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: PI choose to close the study
- Enrolment
- 0 participants (actual)
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)US National Institutes of Health
- Study start
- 8 May 2013 (actual)
- Primary completion
- 24 Mar 2020 (actual)
- Study completion
- 24 Mar 2020 (actual)
- First posted
- 10 May 2013 (estimated)
- 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.
Background:
- Abscesses are a pocket of infection in an organ or tissue. Patients with a disease called chronic granulomatous disease (CGD) often develop these abscesses. CGD is an inherited disorder that affects how white blood cells function. Liver abscesses in people with CGD often require surgery to remove them and treat the infection. However, some people with CGD cannot have full surgery because it would be too risky. Researchers want to try a procedure called radiofrequency ablation (RFA) to treat these liver abscesses. RFA can usually be done without a major operation. This study will see if RFA is a safe and effective treatment for liver abscesses in patients with CGD.
Objectives:
- To see if RFA is a safe and effective treatment for CGD-related liver abscesses.
Eligibility:
- Individuals between 18 and 75 years of age with CGD who have liver abscesses that cannot be treated with surgery.
Design:
- Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected. Imaging studies will be performed on the liver.
- Participants will have RFA for the abscesses. RFA is an image-guided technique that heats and destroys specific tissue, such as tumor tissue. It will target any abscesses on the liver.
- After the procedure, participants will stay in the hospital for monitoring before being released.
- Participants will have regular follow-up visits for up to 1 year after treatment. Blood and urine samples will be collected. Additional imaging studies will be performed.
Detailed description
Radiofrequency Ablation (RFA) has become an increasingly common therapeutic treatment for neoplasms in the liver. A number of devices are now Food and Drug Administration cleared for this indication, and a growing body of literature supports this technique as a therapeutic option for patients with primary or metastatic hepatic malignancies. In vivo animal studies have also shown that this technique can also be used to treat infections. Insertion of the thermal energy delivery probe into an infected liver abscess destroys the bacteria while preserving surrounding tissue. Off-label use of RFA was successfully used to treat 22 abscesses in 4 patients with chronic granulomatous disease who had inoperable liver abscesses. The proposed clinical trial will specifically evaluate the feasibility, safety, and to a lesser extent, efficacy of RFA to treat liver abscesses in subjects with previously diagnosed chronic granulomatous disease. This will be a non-randomized case study conducted at the Clinical Center at the National Institutes of Health. One RFA device will be used. Ten subjects will be enrolled. If the method proves to be both feasible and safe, detailed analysis on efficacy will be performed. RFA eventually could play an important clinical role in patients with chronic granulomatous disease and liver abscesses that are not amenable to surgical management and are without other effective therapeutic options, or might otherwise be incompletely treated with surgical resection and debridement alone.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| RFA | Experimental | Device: Cool-tip RF Ablation SystemAll subjects enrolled onto this study will receive treatment of their liver abscess (es) by RFA ablation |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Has documented chronic granulomatous disease
- Age 18 - 75
- Has a liver abscess infected with Staphylococcus aureus or other microorganism susceptible to RFA, but is not an optimal candidate for curative surgical resection either due to location of disease, multiplicity of disease, or previous surgery or other comorbidities, such as pulmonary insufficiency, or has other contraindications to general anesthesia or perioperative management or refuses surgery.
- Is willing to return to NIH for imaging scans
- Is willing to undergo testing or procedures associated with this protocol
Key exclusion criteria (9 listed)
- Positive results for toxin-producing bacteria obtained from liver biopsy in the pertinent abscess.
- Is a good candidate for liver-curative open surgical resection and does not refuse the surgery.
- Is not a candidate for RFA therapy due to lesion size or location.
- Has a prothrombin time (PT) or partial thromboplastin time (PTT) >1.5 times normal (except in patients who have a known lupus anticoagulant or other condition which a hematologist deems will not cause excessive bleeding despite the abnormal coagulation parameters).
- Has a platelet count <50,000/mm(3) which cannot be maintained despite platelet transfusions.
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 |
|---|---|---|
| Cool-tip RF Ablation SystemRFA therapy will be administered according to the instructions of the RFA device manufacturer. After infiltration of the skin (for percutaneous approach) with a local anesthetic, the needle probe will be directed into the lesion. For all methods, the needle placement is confirmed to be within the lesion by ultrasound or other imaging modality as appropriate. After confirming needle placement, the lesion will be heated to a target temperature of >75 (Infinite)c for up to 15 minutes. The exposure time may be varied depending on the temperatures achieved. Depending on the size of the lesion to be ablated, multiple passes of the probe as well as multiple ablation cycles may be performed. Adequacy of ablation will be monitored by ultrasound throughout the procedure. Depending on the size of the lesions and the time required to complete the ablation, multiple lesions may be treated as staged procedures so as to improve safety. | Device | RFA |
Primary outcome
Safety of RFA in the treatment of liver abscesses in CGD subjects
Time frame: Fater each patient completes the study
The primary objective of this study is to determine the safety of radiofrequency ablation (RFA) in the treatment of liver abscesses in subjects with chronic granulomatous disease (CGD).
2 secondary outcomes
Determine if RFA is a reasonable treatment options
Time frame: After last patient completes the study
Compare recovery outcomes
Time frame: After last patient completes the study
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Mar 2020.
- Background: PubMed 12918460Livraghi T. Radiofrequency ablation, PEIT, and TACE for hepatocellular carcinoma. J Hepatobiliary Pancreat Surg. 2003;10(1):67-76. doi: 10.1007/s10534-002-0714-y.
- Background: PubMed 11882760Lublin M, Bartlett DL, Danforth DN, Kauffman H, Gallin JI, Malech HL, Shawker T, Choyke P, Kleiner DE, Schwartzentruber DJ, Chang R, DeCarlo ES, Holland SM. Hepatic abscess in patients with chronic granulomatous disease. Ann Surg. 2002 Mar;235(3):383-91. doi: 10.1097/00000658-200203000-00010.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
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.
Elizabeth M Kang, M.D. principal investigator
National Institute of Allergy and Infectious Diseases (NIAID)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Cool-tip RF Ablation System in people with Chronic Granulomatous Diseases (CGD) and Liver Lesions, run by National Institute of Allergy and Infectious Diseases (NIAID) 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 7 inclusion and 9 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: withdrawn, as of the sponsor's last update on 26 Mar 2020. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 was withdrawn before enrolling participants, so there are no results.
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?
Safety of RFA in the treatment of liver abscesses in CGD subjects (time frame: Fater each patient completes the study). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01851460, which also lists 2 linked publications.
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 26 Mar 2020.