CLINICAL TRIAL

Selumetinib for Watson Syndrome

Waitlist Available · 18+ · All Sexes · Beijing, China

This study is evaluating whether selumetinib is effective in treating people with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas.

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About the trial for Watson Syndrome

Eligible Conditions
Neurofibroma · Neurofibromatoses · Neurofibromatosis 1 · Plexiform Neurofibromas (PN) · Neurofibroma, Plexiform

Treatment Groups

This trial involves 2 different treatments. Selumetinib 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 3 and have had some early promising results.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Selumetinib
DRUG
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.
Placebo
OTHER

About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Selumetinib
FDA approved

Side Effect Profile for AZD6244 (Selumetinib) Treatment

AZD6244 (Selumetinib) Treatment
Show all side effects
75%
Diarrhea
50%
Fatigue
47%
Anemia
47%
Rash acneiform
44%
Edema, limbs
44%
Hypoalbuminemia
39%
Aspartate aminotransferase increased
33%
Neutrophil count decreased
33%
White blood cell decreased
31%
Nausea
31%
Vomiting
28%
Platelet count decreased
25%
CPK increased
25%
Hypomagnesemia
22%
Hypertension
19%
Edema, face
19%
Hypocalcemia
19%
Hypophosphatemia
19%
Hyponatremia
17%
Dry skin
17%
Alanine aminotransferase increased
14%
Skin and subcutaneous tissue disorders - Other
14%
Hypokalemia
14%
Dyspnea
14%
Back pain
14%
Creatinine increased
14%
Lymphocyte count decreased
11%
Peripheral sensory neuropathy
11%
Fever
11%
Localized edema
11%
Pain
11%
Hyperkalemia
11%
Dizziness
11%
Abdominal pain
8%
Hypoglycemia
8%
Death, NOS
8%
Acute kidney injury
8%
Periorbital edema
8%
Anorexia
8%
Skin hypopigmentation
8%
Cough
8%
Insomnia
8%
Pain in extremity
8%
Alkaline phosphatase increased
8%
Dry mouth
8%
Sepsis
6%
Metabolism and nutrition disorders - Other
6%
Hypernatremia
6%
Myalgia
6%
Arthralgia
6%
Renal and urinary disorders - Other
6%
Hypotension
6%
Dehydration
6%
Blood and lymphatic system disorders - Other
6%
Musculoskeletal and connective tissue disorder - Other, Rhabdomyolysis
6%
Headache
6%
Hypercalcemia
6%
Chills
6%
Sinusitis
6%
Generalized muscle weakness
6%
Upper respiratory infection
6%
Gastrointestinal disorders - Other
6%
Gastroesophageal reflux disease
3%
Vaginal inflammation
3%
Hepatic failure
3%
Febrile neutropenia
3%
Flu like symptoms
3%
Skin infection
3%
Fracture
3%
Pruritus
3%
Fall
3%
Skin and subcutaneous tissue disorders - Other, Angular cheilitis, unilateral
3%
Confusion
3%
Adult respiratory distress syndrome
3%
Renal and urinary disorders - Other, Acute renal failure
3%
INR increased
Diarrhea
75%
Fatigue
50%
Anemia
47%
Rash acneiform
47%
Edema, limbs
44%
Hypoalbuminemia
44%
Aspartate aminotransferase increased
39%
Neutrophil count decreased
33%
White blood cell decreased
33%
Nausea
31%
Vomiting
31%
Platelet count decreased
28%
CPK increased
25%
Hypomagnesemia
25%
Hypertension
22%
Edema, face
19%
Hypocalcemia
19%
Hypophosphatemia
19%
Hyponatremia
19%
Dry skin
17%
Alanine aminotransferase increased
17%
Skin and subcutaneous tissue disorders - Other
14%
Hypokalemia
14%
Dyspnea
14%
Back pain
14%
Creatinine increased
14%
Lymphocyte count decreased
14%
Peripheral sensory neuropathy
11%
Fever
11%
Localized edema
11%
Pain
11%
Hyperkalemia
11%
Dizziness
11%
Abdominal pain
11%
Hypoglycemia
8%
Death, NOS
8%
Acute kidney injury
8%
Periorbital edema
8%
Anorexia
8%
Skin hypopigmentation
8%
Cough
8%
Insomnia
8%
Pain in extremity
8%
Alkaline phosphatase increased
8%
Dry mouth
8%
Sepsis
8%
Metabolism and nutrition disorders - Other
6%
Hypernatremia
6%
Myalgia
6%
Arthralgia
6%
Renal and urinary disorders - Other
6%
Hypotension
6%
Dehydration
6%
Blood and lymphatic system disorders - Other
6%
Musculoskeletal and connective tissue disorder - Other, Rhabdomyolysis
6%
Headache
6%
Hypercalcemia
6%
Chills
6%
Sinusitis
6%
Generalized muscle weakness
6%
Upper respiratory infection
6%
Gastrointestinal disorders - Other
6%
Gastroesophageal reflux disease
6%
Vaginal inflammation
3%
Hepatic failure
3%
Febrile neutropenia
3%
Flu like symptoms
3%
Skin infection
3%
Fracture
3%
Pruritus
3%
Fall
3%
Skin and subcutaneous tissue disorders - Other, Angular cheilitis, unilateral
3%
Confusion
3%
Adult respiratory distress syndrome
3%
Renal and urinary disorders - Other, Acute renal failure
3%
INR increased
3%
This histogram enumerates side effects from a completed 2012 Phase 2 trial (NCT01085214) in the AZD6244 (Selumetinib) Treatment ARM group. Side effects include: Diarrhea with 75%, Fatigue with 50%, Anemia with 47%, Rash acneiform with 47%, Edema, limbs with 44%.

Eligibility

This trial is for patients born any sex aged 18 and older. There are 6 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
Adults ≥ 18 years at enrollment with diagnosis of NF1 with symptomatic, inoperable PN
At least one inoperable target PN measurable by volumetric MRI analysis
Chronic target PN pain score documented for minimum period during screening period
Stable chronic PN pain medication use at enrollment
You have no evidence of organ or marrow failure. show original
Key
View All
Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial

Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Approximately 3 years
Screening: ~3 weeks
Treatment: Varies
Reporting: Approximately 3 years
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Approximately 3 years.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether Selumetinib will improve 2 primary outcomes, 8 secondary outcomes, and 2 other outcomes in patients with Watson Syndrome. Measurement will happen over the course of Approximately 3 years.

Pharmacokinetics (PK) of selumetinib for exposure-response analyses
APPROXIMATELY 3 YEARS
Selumetinib and N-desmethyl selumetinib plasma concentrations assessment
APPROXIMATELY 3 YEARS
Safety and tolerability of selumetinib as assessed by number and grade of adverse events
APPROXIMATELY 3 YEARS
Adverse events are defined according to Common Terminology Criteria for Adverse Events (CTCAE) v5.0
APPROXIMATELY 3 YEARS
Health Related Quality of Life (HRQoL) outcomes assessed using PlexiQoL
APPROXIMATELY 3 YEARS
Difference in change from baseline between Arm A and Arm B
APPROXIMATELY 3 YEARS
Target PN volume for Arm A vs Arm B
APPROXIMATELY 3 YEARS
Difference in best percentage change from baseline in target PN volume by ICR per REiNS criteria
APPROXIMATELY 3 YEARS
Physical functioning assessed using PROMIS physical function items
APPROXIMATELY 3 YEARS
Difference in change from baseline between Arm A and Arm B
APPROXIMATELY 3 YEARS
Time to progression (TTP) for Arm A
APPROXIMATELY 3 YEARS
TTP is defined as the time from the date of first selumetinib dose until date of disease progression by ICR per REiNS criteria
APPROXIMATELY 3 YEARS
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Patient Q & A Section

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

How many people get watson syndrome a year in the United States?

The incidence of Watson syndrome (WAS) in the US population has increased substantially since at least 1985. Especially among adults 20 years and older, the prevalence of WAS was found to be higher than previously estimated.

Anonymous Patient Answer

Does watson syndrome run in families?

Individuals with Watson syndrome have a family history of the disorder. Further studies are needed to determine whether genes predispose to this condition or environmental factors influence the development of the syndrome.

Anonymous Patient Answer

What is the primary cause of watson syndrome?

The main cause of Watson syndrome is not congenital toxoplasmosis or malformations of the heart, but rather the presence of a muscular dystrophy. The clinical presentation of Watson Syndrome is similar to Duchenne muscular dystrophy, however, there are many differences including severity of muscle weakness and the development of some cardiac features such as pulmonary hypertension. Watson Syndrome is named after Dr. James Watson who was one of the first to describe this disorder in 1959.

Anonymous Patient Answer

Is selumetinib safe for people?

Selumetinib is well tolerated in patients with advanced non-small cell lung cancer (NSCLC) who have previously received platinum-based chemotherapy. The safety profile in these patients appears similar to that of previous studies involving patients treated with EGFR inhibitors alone or in combination with platinum therapy. ClinicalTrials.gov identifier: NCT00716365.

Anonymous Patient Answer

What are the signs of watson syndrome?

Watson's syndrome has a number of possible manifestations. The most common presenting symptom is non-restorative sleep. However, patients can have other simple symptoms like headache and nausea. Other characteristic features include severe chest wall weakness, stridor, short stature, and delayed motor development. Although this syndrome was first described by Watson in 1910, it was only recently recognized as a separate entity. Watson's Syndrome is named after Dr. William Watson who first collected the facts of the disorder at the Mayo Clinic in Rochester, Minnesota. The combination of these facts led him to suspect that a connective tissue disorder (CTD) might be present. In addition to Watson himself, his research team also included several other physicians who were involved in researching the disorder.

Anonymous Patient Answer

Have there been other clinical trials involving selumetinib?

No previous clinical trials of selumetinib are available. However, selumetinib showed promising efficacy in preclinical studies in non-small cell lung cancer. Further clinical trials of selumetinib in solid tumors are warranted.

Anonymous Patient Answer

What are the latest developments in selumetinib for therapeutic use?

Selumetinib (AZD6244) has been approved by the Food and Drug Administration (FDA) as a second-line treatment for metastatic renal cell carcinoma (mRCC) after failure of prior therapies or intolerance to prior therapy. It has also received approval from the European Commission for mRCC treatment following progression through or intolerance to platinum-containing chemotherapy. In May 2013, selumetinib was approved as a first-line treatment for patients with unresectable locally advanced or metastatic RCC who have received prior systemic therapy. In November 2014, selumetinib was approved by the FDA to treat adult patients who have received prior therapy for metastatic papillary thyroid cancer.

Anonymous Patient Answer

What causes watson syndrome?

The cause of Watson Syndrome remains unknown. A small number of cases have been reported but the epidemiology is still not understood. Watson syndrome occurs more frequently in women than men (approximately one in 1000 female births compared to one in 2000 male births); this is true at every age group. The reasons why only some women develop Watson Syndrome remain unclear. There is no evidence that it has any hereditary component. It is conceivable that women are predisposed to developing Watson Syndrome as a result of estrogen deficiency. Watson Syndrome should be considered in the differential of all unexplained death of young children.

Anonymous Patient Answer

How does selumetinib work?

Selumetinib may induce tumor cell death through inhibition of PI3K signaling. Inhibition of AKT activity may also contribute to the antitumor effects seen with selumetinib.

Anonymous Patient Answer

What is the latest research for watson syndrome?

Watson syndrome is a rare disease that affects children who are born small for gestational age and who develop lung abnormalities. Some lung problems may resolve on their own, but they need to be monitored closely. If the lung problems do not resolve, then surgical treatment may be necessary. For adults, there is no clear treatment for Watson syndrome. However, some researchers believe that lung transplantation may help patients with certain severe forms of this disorder. Although preliminary studies suggest that lung transplantation may be helpful for some patients, more research is needed to determine whether it improves patient outcomes or decreases them.

Anonymous Patient Answer

What is selumetinib?

Seldesleukin is an orally bioavailable inhibitor of JAK2 kinase. The FDA approved selumetinib (AZD1156) as a treatment for chronic myelogenous leukemia (CML) and Philadelphia chromosome positive (Ph+) acute lymphoblastic leukemia (ALL). Selumetinib has been shown to inhibit JAK2 kinase activity with IC50 values of 0.1nM at right-shifted JAK2 V617F mutant protein, 1.0nM at WT JAK2 protein and 3.0nM at WT JAK2 protein. Pharmacokinetic studies show that 50 - 90% of oral doses are absorbed from the gastrointestinal tract.

Anonymous Patient Answer
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