A Phase 1/2 interventional study of Sirolimus in Desmoid Tumor, sponsored by MaineHealth. Completed at 8 sites in United States. Open to participants aged Up to 29 Years. Per ClinicalTrials.gov, last updated 2023-06-26.
Sponsored by MaineHealth · Phase 1/2, Interventional, and Treatment
Among 33 studies in Desmoid Tumors with a site in United States — 7 open to participants now, 10 under way, 1 of them phase 3. Desmoid Tumors · United States.
Among the 33 studies in Desmoid Tumors with a site in United States, this one is the 21st largest by enrollment of the 24 interventional ones, is one of 3 that started in 2014, is one of 4 in phase 1/2, and was the 8th registered.
71 studies on the registry are indexed under Desmoid Tumors; 21 are open to participants now.
This study's enrollment of 9 is below the median of 44 across 47 interventional studies indexed under Desmoid Tumors.
Browse Desmoid Tumors studies →7 of the 33 studies in Desmoid Tumors with a site in United States are open now, in 7 countries. Each count lists the studies.
Of the 17 Desmoid Tumors studies recruiting now, 13 (76%) set no upper age limit and 4 (24%) stop at 75 or younger; 6 take children under 18; 0 take healthy volunteers.
This study: Up to 29 Years · all sexes · no healthy volunteers.
The full table on the hub →33 studies; the count open now beside each. This study's row is marked.
First in 1991; this study’s year is marked. 3 started in 2014.
Median enrollment 43 across the 24 interventional studies, 150 across the 7 observational. Of the 20 that completed or were terminated, 9 (45%) have results posted.
6 of 33 have an industry lead sponsor. The 10 most frequent:
MaineHealth is the lead sponsor of 36 studies on the registry; 5 are open to participants now.
MaineHealth has 1 study filed under Desmoid Tumors — fewer than ten, so there is no comparison to draw.
2 of the studies in Desmoid Tumors with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT04281381Memorial Sloan Kettering Cancer CenterObserving People With Desmoid-Type Fibromatosis | 2027-02-19 |
| NCT05919264Parabilis Medicines, Inc.FOG-001 in Locally Advanced or Metastatic Solid Tumors | 2027-04-01 |
Among the 33 studies in Desmoid Tumors with a site in United States, from their registry updates:
Nothing recorded among them since 2026-09-25.
Desmoid-type fibromatosis (or desmoid tumor) represents an intermediate grade neoplasm with a striking predilection for locally invasive growth and recurrence following resection. It occurs in children as well as young adults. As a typically localized disease, the historical standard of care for treatment has been surgical resection, with or without ionizing radiation. In some cases where surgical resection or radiation is not feasible, chemotherapy has been used. Two clinical trials conducted in the Pediatric Oncology Group (POG) and the Children's Oncology Group (COG) evaluated the role for either low intensity or non-cytotoxic chemotherapy for children with desmoid tumor that is not amenable to standard therapy. These were largely empirical treatment strategies or based on somewhat anecdotal observations. By better understanding desmoid tumor biology, even more effective therapy targeting a particular protein that is central to the disease can be developed.
Desmoid tumor is well-known to be associated with deregulation of the Adenomatous Polyposis Cell/beta-catenin (APC/β-catenin pathway). This is true of familial cases associated with Gardner's Syndrome and also in sporadic desmoid tumor, nearly all of which display histological or molecular evidence of Adenomatous Polyposis Cell/beta-catenin (APC β-catenin) pathway activation (Alman et al., 1997; Lips et al., 2009). Several new pieces of evidence support the concept that deregulation of the mammalian target of rapamycin (mTOR) cell proliferation/survival pathway may play an important role in tumor biology when the APC/β-catenin pathway is disrupted. Sirolimus, a drug that inhibits mammalian target of rapamycin (mTOR), is currently being evaluated as an anti-cancer agent in a variety of tumor types, but it has not been previously studied in desmoid tumor.
The investigators are conducting this pilot study to begin to explore whether mTOR inhibition may be beneficial for children and young adults with desmoid tumor.
We propose a translational research project that will directly test the hypothesis that mTOR is active in desmoid tumor in children and young adults. Activity will be assessed by clinical and histological studies following a course of pre-operative chemotherapy using sirolimus. Clinical response will be measured using validated pain assessment scales because desmoid tumor size is unlikely to change during the course of pre-operative chemotherapy in this study. Histological response will be based on quantifying the phosphorylation of following mTOR targets: thr389p-p70S6K, p-4E-BP1, and ser473p-AKT.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Patients must have surgery planned to remove the desmoid tumor and either:
Concomitant medication restrictions:
Patients must have recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study
Patients must have normal laboratory values as defined below:
Hepatic: Adequate liver function is defined as:
Hematologic function: Adequate bone marrow function is defined as:
Exclusion Criteria:
Concomitant medication restrictions
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Life expectancy
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GVHD (graft-versus-host disease)
After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Preoperative sirolimus: * loading dose of 12 milligrams/meter2; Per Os (PO), by mouth day 1 (Max dose 12 milligram) * starting 24 hours after the initial loading dose, subjects will receive a dose of 4 milligram/meters2 daily; Per Os (PO), by mouth days 2 through 28
Drug: Sirolimus
* Loading dose of 12 milligrams/meter2; Per Os (PO), by mouth day 1 (Max dose 12 milligrams) * Starting 24 hours after the initial loading dose, patients will receive a dose of 4 milligrams/meter2 daily; Per Os (PO), by mouth days 2 through 28 (Max dose 4 milligram/day)
Also known as: Rapamune®, Rapamycin
Immunohistochemical Immunoreactive Score Results After 4 Weeks of Sirolimus Compared to Control Specimens
Changes to the mTOR pathway were determined using an immunohistochemical immunoreactive score (IRS). The components of the IRS for each phosphoprotein (p4EPB and pS706K) are as follows: the percentage of positive cells were scored as: 0 (0%); 1 (\<10%); 2 (11-50%); 3 (51-80%); 4(\>80%). The staining intensity were scored as: 0 (negative), 1 (weak), 2 (moderate), and 3 (strong). To derive the IRS, the percentage of positive cells and staining intensity were multiplied together, resulting in a value from 0 to 12. The score for patients after 4 weeks of sirolimus was compared to a group of control desmoid tumor samples from the UCLA tumor bank (n=68). Lower scores for patients following 4 weeks of sirolimus compared to control sample scores indicate a better outcome.
Time frame: 4 weeks
Pain Levels After 4 Weeks of Sirolimus
Pain assessments were performed using the validated numeric (age ≥ 10 years) and Wong-Baker FACES (≥3 and \< 10 years) pain rating scales at specified study time points including baseline, at week 1 and after 4 weeks of treatment (just prior to surgery). Both pain scales range from a value of 0 to 10 with 0 being equal to no pain and 10 being equal to the worst pain. The median pain score and pain score range for study participants at Week 1 and Prior to Surgery (Week 4) timepoints are provided. Lower pain scale values (median and upper limit of the range) at the Prior to Surrgery timepoint are considered a better outcome.
Time frame: 4 weeks
Number of Participants Without Tumor Recurrence
We evaluated the number of study participants whose tumor did not recur by 3 years from the date of surgery.
Time frame: 3 years from the end of therapy, a total duration of 3 years and 4 weeks
Number of Participants With Grade 3 or Higher Toxicity Per CTCAE Definitions
The safety and tolerability of patients receiving sirolimus was evaluated using Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 grading criteria. All reported grades of toxicity (1-5) were collected.
Time frame: 4 weeks
| Milestone | Sirolimus |
|---|---|
| Started | 9 |
| Completed | 9 |
| Not completed | 0 |
Changes to the mTOR pathway were determined using an immunohistochemical immunoreactive score (IRS). The components of the IRS for each phosphoprotein (p4EPB and pS706K) are as follows: the percentage of positive cells were scored as: 0 (0%); 1 (\<10%); 2 (11-50%); 3 (51-80%); 4(\>80%). The staining intensity were scored as: 0 (negative), 1 (weak), 2 (moderate), and 3 (strong). To derive the IRS, the percentage of positive cells and staining intensity were multiplied together, resulting in a value from 0 to 12. The score for patients after 4 weeks of sirolimus was compared to a group of control desmoid tumor samples from the UCLA tumor bank (n=68). Lower scores for patients following 4 weeks of sirolimus compared to control sample scores indicate a better outcome.
| score on a scale | Immunoreactive Score Results of p4EPB After 4 Weeks of Sirolimus for the Study Patients | Immunoreactive Score Results of pS706K After 4 Weeks of Sirolimus for the Study Patients | Immunoreactive Score Results of p4EPB for the Control Tumors | Immunoreactive Score Results of pS706K for the Control Tumors |
|---|---|---|---|---|
| Immunohistochemical Immunoreactive Score Results After 4 Weeks of Sirolimus Compared to Control Specimens | 5.22 (1 to 12) | 7.44 (0 to 12) | 4.47 (0 to 12) | 9.39 (0 to 12) |
Pain assessments were performed using the validated numeric (age ≥ 10 years) and Wong-Baker FACES (≥3 and \< 10 years) pain rating scales at specified study time points including baseline, at week 1 and after 4 weeks of treatment (just prior to surgery). Both pain scales range from a value of 0 to 10 with 0 being equal to no pain and 10 being equal to the worst pain. The median pain score and pain score range for study participants at Week 1 and Prior to Surgery (Week 4) timepoints are provided. Lower pain scale values (median and upper limit of the range) at the Prior to Surrgery timepoint are considered a better outcome.
| score on a scale | Week 1 | Prior to Surgery |
|---|---|---|
| Pain Levels After 4 Weeks of Sirolimus | 1 (0 to 3) | 0 (0 to 3) |
We evaluated the number of study participants whose tumor did not recur by 3 years from the date of surgery.
| Participants | Study Patients |
|---|---|
| Number of Participants Without Tumor Recurrence | 5 |
The safety and tolerability of patients receiving sirolimus was evaluated using Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 grading criteria. All reported grades of toxicity (1-5) were collected.
| Participants | Safety |
|---|---|
| Number of Participants With Grade 3 or Higher Toxicity Per CTCAE Definitions | 0 |
Collected over Adverse events were collected for the duration of treatment (4 weeks).. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Sirolimus | 0/9 (0%) | 0/9 (0%) | 7/9 (77.8%) |
| Event | Sirolimus |
|---|---|
| Mucositis oralGastrointestinal disorders | 5/9 |
| HypotensionVascular disorders | 4/9 |
| White blood cell decreasedInvestigations | 3/9 |
| Creatinine increasedInvestigations | 3/9 |
| HeadacheNervous system disorders | 2/9 |
| FatigueGeneral disorders | 2/9 |
| Oral painGastrointestinal disorders | 2/9 |
| Neutrophil count decreasedInvestigations | 2/9 |
| HypertriglyceridemiaMetabolism and nutrition disorders | 2/9 |
| Sinus bradycardiaCardiac disorders | 2/9 |
| Age, Continuous(years) | Sirolimus |
|---|---|
| Median | 15 (5 to 28) |
| Sex: Female, Male(Participants) | Sirolimus |
|---|---|
| Female | 3 |
| Male | 6 |
| Race and Ethnicity Not Collected(Participants) | Sirolimus |
|---|
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Feb 2014 to primary completion in Dec 2021, 94 months.
Results were first posted in Jun 2023, 1 year 6 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Jun 2023. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Sirolimus
MaineHealth