A Phase 2 interventional study of GDC-0449 in Basal Cell Nevus Syndrome and Gorlin Syndrome, sponsored by UCSF Benioff Children's Hospital Oakland. Completed at 2 sites in United States. Open to participants aged 35 Years to 75 Years. Per ClinicalTrials.gov, last updated 2021-01-11.
Sponsored by UCSF Benioff Children's Hospital Oakland · Phase 2, Interventional, and Treatment
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Among 20 studies in Basal Cell Nevus Syndrome with a site in United States — 1 open to participants now, 1 under way, none of them phase 3. Basal Cell Nevus Syndrome · United States.
Among the 20 studies in Basal Cell Nevus Syndrome with a site in United States, this one is the 6th largest by enrollment of the 18 interventional ones that give a count, is one of 2 that started in 2009, is one of 10 in phase 2, and was the 3rd registered.
35 studies on the registry are indexed under Basal Cell Nevus Syndrome; 4 are open to participants now.
Browse Basal Cell Nevus Syndrome studies →1 of the 20 studies in Basal Cell Nevus Syndrome with a site in United States is open now. Recruiting first, the newest first:
St. Jude Children's Research Hospital · Sites in United States
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 3 Basal Cell Nevus Syndrome studies recruiting now, 3 set no upper age limit and none stop at 75 or younger; 3 take children under 18; none take healthy volunteers.
This study: 35 Years to 75 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
20 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 3 · Phase 2 11 · Phase 3 3
First in 2004; this study’s year is marked. 2 started in 2009.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 41 is above the median of 24 across 25 interventional studies indexed under Basal Cell Nevus Syndrome.
Median enrollment 35 across the 18 interventional studies, 765 across the 2 observational. Of the 18 that completed or were terminated, 12 (67%) have results posted.
This tab as a report to print or save as PDF → With an account.
8 of 20 have an industry lead sponsor. The 10 most frequent:
UCSF Benioff Children's Hospital Oakland is the lead sponsor of 55 studies on the registry; none are open to participants now.
UCSF Benioff Children's Hospital Oakland has 6 studies filed under Basal Cell Nevus Syndrome — fewer than ten, so there is no comparison to draw.
None of the studies under way reaches primary completion within a year.
Among the 20 studies in Basal Cell Nevus Syndrome with a site in United States, from their registry updates:
Nothing recorded among them since 2026-09-25.
This tab as a report to print or save as PDF → With an account.
The purpose of this study is to reduce the number of new surgically eligible BCCs by 50% appearing during month 3-18 of medication ingestion.
This is a Phase II, 18 month, double blind, randomized placebo-controlled, two arm multicenter clinical study design. During the 18-month treatment period, the safety and chemopreventive efficacy of 150 mg/day GDC-0449 versus placebo will be assessed, and include evaluations of the skin at monthly intervals for the first three months and then every 3 months for the next 15 months. Removal of new surgically eligible BCCs (SEBs) will be done by primary skin care physicians (PSCPs) or at Study Centers. A Data Safety Monitoring Board (DSMB) will review unblinded results for an interim analysis when 20 subjects have completed 12 months of drug. This review will focus on adverse events and efficacy results. Subjects will be monitored for the development of new SEBs after they discontinue study treatment. At the end of the 18 months, given that the observed adverse events are minimal, patients on placebo will be offered the opportunity to take GDC-0449 for 18 months in an open label continuation, followed by six months observation, and patients on GDC449 will be monitored for the next 24 months for assessment of the duration of benefit after stopping the drug.
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.
The subject:
Exclusion Criteria:
The subject:
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.
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 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.
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.
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.
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.
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.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
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.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
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.
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.
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.
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.
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.
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.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
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.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
placebo pill by mouth once daily
Drug: GDC-0449
vismodegib 150MG by mouth once daily
Drug: GDC-0449
capsule, 150 mg, one pill daily, 18 months
Also known as: Hedgehog Inhibitor, Hedgehog Antagonist
Number of New Surgically Eligible Basal-cell Carcinomas
Number of new surgically eligible basal-cell carcinomas per patient per year
Time frame: 3 months of receiving study drug
Change in Size of the Existing Carcinomas, Expressed as the Sum of Cumulative Diameters in Millimeters Over an 18 Month Period
Time frame: Baseline and 18 months
Number of New Surgically Eligible Basal-cell Carcinomas After Stopping Vismodegib Treatment
The number of new surgically eligible basal-cell carcinomas per patient per month is reported.
Time frame: These five patients were given placebo for a mean of 7.4 months (SD 2.3). After receiving vismodegib for a mean of 13.8 months (SD 6.8) and then discontinuing vismodegib for a mean of 11.8 months (SD 7.9) the new basal-cell carcinoma rate is reported.
| Milestone | Sugar Pill | GDC-0449 |
|---|---|---|
| Started | 15 | 26 |
| Completed | 14 | 23 |
| Not completed | 1 | 3 |
| Withdrew: Adverse event | 0 | 3 |
| Withdrew: Disease progression | 1 | 0 |
| Milestone | Sugar Pill | GDC-0449 |
|---|---|---|
| Started | 14 | 23 |
| Completed | 11 | 21 |
| Not completed | 3 | 2 |
| Withdrew: Death | 1 | 1 |
| Withdrew: Adverse event | 2 | 1 |
Number of new surgically eligible basal-cell carcinomas per patient per year
| events per patient year | Sugar Pill | GDC-0449 |
|---|---|---|
| Number of New Surgically Eligible Basal-cell Carcinomas | 34 ± 1.32 | 2 ± 0.12 |
| Millimeters | Sugar Pill | GDC-0449 |
|---|---|---|
| Change in Size of the Existing Carcinomas, Expressed as the Sum of Cumulative Diameters in Millimeters Over an 18 Month Period | 55 (NA to NA) | -60 (NA to NA) |
The number of new surgically eligible basal-cell carcinomas per patient per month is reported.
| carcinomas per patient per month | Sugar Pill | GDC-0449 |
|---|---|---|
| Number of New Surgically Eligible Basal-cell Carcinomas After Stopping Vismodegib Treatment | 1.7 ± 1.5 | 0.06 ± 0.12 |
Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Sugar Pill | 0/15 (0%) | 2/15 (13.3%) | 12/15 (80%) |
| GDC-0449 | 2/40 (5%) | 21/40 (52.5%) | 40/40 (100%) |
| Event | Sugar Pill | GDC-0449 |
|---|---|---|
| Weight lossMetabolism and nutrition disorders | 0/15 | 6/40 |
| Cardiac stent for blocked arteryCardiac disorders | 1/15 | 0/40 |
| HysterectomyReproductive system and breast disorders | 1/15 | 0/40 |
| Muscle crampsMusculoskeletal and connective tissue disorders | 0/15 | 2/40 |
| PneumoniaInfections and infestations | 0/15 | 2/40 |
| Reactions to antibioticInjury, poisoning and procedural complications | 0/15 | 2/40 |
| Chest painCardiac disorders | 0/15 | 2/40 |
| Atrial flutterCardiac disorders | 0/15 | 1/40 |
| ColitisGastrointestinal disorders | 0/15 | 1/40 |
| OesophagitisGastrointestinal disorders | 0/15 | 1/40 |
| Event | Sugar Pill | GDC-0449 |
|---|---|---|
| Hair lossSkin and subcutaneous tissue disorders | 1/15 | 40/40 |
| Muscle crampsMusculoskeletal and connective tissue disorders | 1/15 | 40/40 |
| DysgeusiaGastrointestinal disorders | 1/15 | 37/40 |
| Gastrointestinal upsetGastrointestinal disorders | 2/15 | 26/40 |
| Weight lossMetabolism and nutrition disorders | 1/15 | 25/40 |
| FatigueGeneral disorders | 0/15 | 19/40 |
| Common coldRespiratory, thoracic and mediastinal disorders | 3/15 | 8/40 |
| AcneSkin and subcutaneous tissue disorders | 2/15 | 7/40 |
| Runny noseRespiratory, thoracic and mediastinal disorders | 0/15 | 7/40 |
| RashSkin and subcutaneous tissue disorders | 0/15 | 5/40 |
| Age, Continuous(years) | Sugar Pill | GDC-0449 | Total |
|---|---|---|---|
| Mean | 53 ± 8 | 54 ± 8 | 54 ± 8 |
| Sex: Female, Male(Participants) | Sugar Pill | GDC-0449 | Total |
|---|---|---|---|
| Female | 6 | 8 | 14 |
| Male | 9 | 18 | 27 |
| Region of Enrollment(participants) | Sugar Pill | GDC-0449 | Total |
|---|---|---|---|
| United States | 15 | 26 | 41 |
Plan to share: No
The record gives a start of Aug 2009, a primary completion of Jan 2014 and a completion of Jan 2014. It does not say whether these dates are actual or estimated.
Results were first posted in Jan 2021, 7 years after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Dec 2020. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: HhAntag691
UCSF Benioff Children's Hospital Oakland