Treatment for Watson Syndrome

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Memorial Sloan Kettering Cancer Center, New York, NY
Watson Syndrome+3 More
Eligibility
18+
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether a topical gel can reduce the size of neurofibromas in people with neurofibromatosis type 1.

See full description

Eligible Conditions

  • Watson Syndrome
  • Cutaneous Neurofibromas

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether Treatment will improve 2 primary outcomes, 4 secondary outcomes, and 1 other outcome in patients with Watson Syndrome. Measurement will happen over the course of 182 days.

182 days
Patient Reported Outcome Measure to assess Target cNF symptoms over 182 days
Percent change in cNF volume over 182 days
Percent of subjects with at least 50% Target cNF volume reduction after 182 days
Physician's Tumor Assessment of Target cNF severity over 182 days
Safety and effectiveness of once daily NFX-179 gel treatment for 182 days measured by local tolerability assessments, safety laboratory tests, and target cNF volume reduction
Safety of once daily NFX-179 gel treatment for 182 days by adverse events and serious adverse events assessments
Subject's Self-Assessment of Target cNF severity over 182 days

Trial Safety

Safety Estimate

2 of 3
This is better than 68% of similar trials

Trial Design

3 Treatment Groups

0.5% NFX-179 gel
1 of 3
1.5% NFX-179 gel
1 of 3
Vehicle gel
1 of 3
Active Control
Non-Treatment Group

This trial requires 168 total participants across 3 different treatment groups

This trial involves 3 different treatments. Treatment is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 2 and have already been tested with other people.

0.5% NFX-179 gel
Drug
Topical gel applied once daily to target cNFs
1.5% NFX-179 gel
Drug
Topical gel applied once daily to target cNFs
Vehicle gel
Drug
Topical gel applied once daily to target cNFs

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 182 days
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 182 days for reporting.

Closest Location

Memorial Sloan Kettering Cancer Center - New York, NY

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Subject is at least 18 years of age
Subject must provide written informed consent prior to any study procedures
Subject must have a clinical diagnosis of NF1
Has, in the investigator's opinion, a clinically typical appearance
Is not within 1 cm of the orbital rim
Is not covered with hair that might, in the investigator's opinion, interfere with obtaining photographs or impair evaluation of the cNF
Has a Physician's Tumor Assessment grade ≥2
Is dome shaped
Is not pedunculated
The dimensions can be measured

Patient Q&A Section

How many people get neurofibroma a year in the United States?

"Data from a recent study from this study suggest that AFNB is a rare disease in the U.S. population, especially among children and young adults. More data are needed to confirm these findings before making public health recommendations." - Anonymous Online Contributor

Unverified Answer

What is the survival rate for neurofibroma?

"The overall 5-year survival rate for patients with neurofibromas was 96% at our institution. The 5-year survival rate for patients with dysembryomas (a benign subtype of neurofibromas) was 98%. Survival was not statistically different between the two groups. Patients with neurofibromas need to be aware of their own risks and may benefit from being able to speak with an expert on this subject." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets neurofibroma?

"The average age of diagnosis of neurofibromas among children was 11.5 yr, and patients were more likely to be male than female. We found no significant differences in terms of the site of occurrence of neurofibromas between Caucasian patients with neurofibromas and non-Caucasian patients with neurofibromas." - Anonymous Online Contributor

Unverified Answer

What are the chances of developing neurofibroma?

"Neurofibromas are rare tumors in children and adolescents. The incidence is higher in black children than in white children and correlates with race-specific genetic alterations in the NF1 gene. The overall incidence of malignant neurofibromas in adults has been estimated at 0.2%. A prospective cohort study is needed to determine the risk factors for developing neurofibromas and the natural history of these lesions in the adult population." - Anonymous Online Contributor

Unverified Answer

Have there been any new discoveries for treating neurofibroma?

"At present, there are only four drugs approved for neurofibroma as part of an FDA-approved drug label. The Src family kinase inhibitor dasatinib has shown efficacy in some randomized controlled trials with neurofibroma, but this therapy must still be evaluated in larger studies. Until then, we recommend continuing to follow the current standards of care." - Anonymous Online Contributor

Unverified Answer

How does treatment work?

"The theoretical basis behind chemotherapy is that the drug interferes with cell growth. This is substantiated by the fact that many different drugs have been demonstrated to kill tumor cells. Chemotherapy is effective because it destroys rapidly dividing tumor cells as well as stromal tumor cells (areas around the tumor where tumor cells grow) as they divide. In addition, chemotherapy kills cancer stem cells, the cells that cause tumors to form. While some treatments do not target cancer stem cells very effectively, others such as gemcitabine are very specific against them. Gemcitabine's effectiveness seems to depend on protein kinase inhibitors, which were developed specifically to block cancer stem cells." - Anonymous Online Contributor

Unverified Answer

Can neurofibroma be cured?

"A review of the literature revealed no cure for neurofibromas. The need for a prospective study was indicated. The aim of this study would be to develop a setup for a multicenter prospective study to assess the effect of various treatments on neurofibroma." - Anonymous Online Contributor

Unverified Answer

What is treatment?

"The most common surgeries performed for patients with neurofibromas are resection and removal of tumors (85%). Facial nerve blocks are also utilized frequently (49%) for pain relief after surgery or radiation treatment of facial nerves. In terms of complications that occur following surgery or radiation, wound dehiscence is the most prevalent (11%), followed by hematoma formation (4%), dysphagia (4%), and pneumothorax (3%). In the event of postoperative complications, surgical intervention can be undertaken at any time if needed. Patients should not hesitate to seek medical advice immediately." - Anonymous Online Contributor

Unverified Answer

Is treatment typically used in combination with any other treatments?

"It was concluded that surgical removal of neurofibromas has been shown to result in long-term benefit in patients' quality of life and cosmetic outcome, but not in overall survival. Considering the risks associated with surgery, chemotherapy appears to be an effective treatment modality in combination with surgeries." - Anonymous Online Contributor

Unverified Answer

What does treatment usually treat?

"Treatment options for neurofibromas include surgery (debridement), radiation therapy, and chemotherapy. There is limited evidence to support the use of topical agents such as interferons, imiquimod, and retinoids, although these treatments may slightly improve pain and function. In addition, there is little evidence to support the use of oral agents such as bisphosphonates, teriparatide, or strontium ranelate. The current most common treatment for neurofibromas is surgical debridement, which reduces swelling around the tumor. Some patients do experience recurrence of symptoms after surgery; therefore, for some patients, additional treatment may be needed." - Anonymous Online Contributor

Unverified Answer

What causes neurofibroma?

"The cause of neurofibroma remains unknown. It may be related to a genetic mutation though more research is needed. The majority of cases are diagnosed before the age of 15. This suggests that the tumor develops in childhood but it is unclear whether this occurs at birth or if there is a latent period.\n" - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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