Trial · NCT01981551
Phase II Trial of the Gamma-Secretase Inhibitor PF-03084014 in Adults With Desmoid Tumors/Aggressive Fibromatosis
NCT01981551, Phase 2, Completed, National Cancer Institute (NCI); results first posted 2020-02-05
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Phase II Trial of the Gamma-Secretase Inhibitor PF-03084014 in Adults With Desmoid Tumors/Aggressive Fibromatosis. Trials, record NCT01981551. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01981551
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase II Trial of the Gamma-Secretase Inhibitor PF-03084014 in Adults With Desmoid Tumors/Aggressive Fibromatosis
- NCT number
- NCT01981551
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 17 participants, actual
- Lead sponsor
- National Cancer Institute (NCI)
- Conditions
- Desmoid Tumors, Aggressive Fibromatosis
- Interventions
- PF-03084014
- First posted
- 2013-11-11
- Last updated
- 2024-02-06
- Results first posted
- 2020-02-05
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With a Complete Response (CR) + Partial Response (PR) | 20 months | Complete Response + Partial Response was determined by the Response Evaluation Criteria in Solid Tumors (RECIST). Complete Response is disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm. Partial Response is at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. Progressive Disease (PD) is at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). The sum must also demonstrate an absolute increase of at least 5 mm. The appearance of one or more new lesions is also considered progressions. Stable Disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study. |
| Secondary | Number of Participants With Serious and Non-serious Adverse Events | Date treatment consent signed to date off study, approximately 66 months and 27 days. | Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. |
| Secondary | Number of Participants With Somatic or Germline Mutations Identified in Adenomatous Polyposis Coli Gene (APC) or Catenin Beta-1 (CTNNB1) Genes | Baseline | Tumor and blood samples were obtained from participants and genotyped for somatic and germline mutations. |
| Secondary | Mean MD Anderson Symptom Inventory Scores After Treatment | At baseline prior to drug administration and at least every 6 cycles of drug (18 weeks) up to 20 months of treatment. | The MD Anderson Symptoms Inventory (MDSAI) questionnaire includes questions regarding 13 symptoms commonly experienced by patients with cancer and 6 additional items that assess the extent to which these symptoms interfered with how patients felt and were able to function. The 0-10 scale (0=none, 1-4=mild, 5-6=moderate, and 7-10=severe) assesses how patients felt and were able to function over the previous 24 hours. A component score representing symptom severity is obtained by taking the average of the 13 symptom items (e.g., pain, fatigue, etc.) together. A component score representing symptom distress is obtaining by averaging the 6 symptom interference items (e.g., general activity, mood, etc.). |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With a Complete Response (CR) + Partial Response (PR) | 20 months | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Number of Participants With Serious and Non-serious Adverse Events | Date treatment consent signed to date off study, approximately 66 months and 27 days. | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Number of Participants With Somatic or Germline Mutations Identified in Adenomatous Polyposis Coli Gene (APC) or Catenin Beta-1 (CTNNB1) Genes | Baseline | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Mean MD Anderson Symptom Inventory Scores After Treatment | At baseline prior to drug administration and at least every 6 cycles of drug (18 weeks) up to 20 months of treatment. | Scores on a scale | MEAN |
Source ClinicalTrials.gov
Results by group9
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With a Complete Response (CR) + Partial Response (PR) | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | Complete Response | 0 | Participants | |
| Number of Participants With a Complete Response (CR) + Partial Response (PR) | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | Partial Response | 5 | Participants | |
| Number of Participants With a Complete Response (CR) + Partial Response (PR) | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | Not Evaluable | 1 | Participants | |
| Number of Participants With a Complete Response (CR) + Partial Response (PR) | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | Stable Disease | 11 | Participants | |
| Number of Participants With a Complete Response (CR) + Partial Response (PR) | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | Progressive Disease | 0 | Participants | |
| Number of Participants With Serious and Non-serious Adverse Events | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | 17 | Participants | ||
| Number of Participants With Somatic or Germline Mutations Identified in Adenomatous Polyposis Coli Gene (APC) or Catenin Beta-1 (CTNNB1) Genes | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | 15 | Participants | ||
| Mean MD Anderson Symptom Inventory Scores After Treatment | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | Symptom Severity Score | 1.37 | Standard Deviation 1.46 | Scores on a scale |
| Mean MD Anderson Symptom Inventory Scores After Treatment | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | Symptom Interference Score | 1.57 | Standard Deviation 2.69 | Scores on a scale |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | 17 | |
| Overall Study | COMPLETED | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | 8 | |
| Overall Study | NOT COMPLETED | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | 9 |
Source ClinicalTrials.gov
Adverse events230
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Abdominal pain | Gastrointestinal disorders | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | 8 | 17 | 11 |
| other | Activated partial thromboplastin time prolonged | Investigations | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | 4 | 17 | 6 |
| other | Alanine aminotransferase increased | Investigations | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | 9 | 17 | 35 |
| other | Alkaline phosphatase increased | Investigations | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | 5 | 17 | 16 |
| other | Allergic rhinitis | Immune system disorders | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | 2 | 17 | 2 |
| other | Alopecia | Skin and subcutaneous tissue disorders | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | 2 | 17 | 2 |
| other | Amnesia | Nervous system disorders | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | 1 | 17 | 1 |
| other | Anemia | Blood and lymphatic system disorders | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | 9 | 17 | 23 |
| other | Anorexia | Metabolism and nutrition disorders | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | 3 | 17 | 3 |
| other | Anxiety | Psychiatric disorders | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | 3 | 17 | 4 |
1 to 10 of 230Source ClinicalTrials.gov
See all 230 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | 1 | 17 |
| Total, other adverse events | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | 17 | 17 |
| Total, serious adverse events | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | 9 | 17 |
Source ClinicalTrials.gov
Papers4
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01981551
GET https://conductscience.org/api/trials/v1/NCT01981551/events?page=2