Neurofibromatosis Type 1 (NF1)PROCEEDNCT07888517
Observational Study of the Real-world Effectiveness and Safety of Selumetinib Treating Adult Patients With Neurofibromatosis Type 1 (NF1) Who Have Symptomatic, Inoperable Plexiform Neurofibroma (PN)
An observational study of Neurofibromatosis Type 1 (NF1), run by AstraZeneca at 26 sites in China.
Trial summary
This is a prospective, multicentre, observational registry study with primary data collection designed to evaluate the real-world effectiveness and safety of selumetinib in adult patients with NF1 who have symptomatic, inoperable PN. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Neurofibromatosis Type 1 (NF1)
- How many people
- 800 participants
- Where
- 26 sitesChina
- Study dates
- 28 Sep 2026to 31 Dec 2030
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: The target population of interest in this study is adult patients (≥18 years of age) with NF1 who have symptomatic, inoperable PN and intend to use selumetinib after enrolment. 800 patients with selumetinib treatment will be enrolled in the study.
Who may be able to join
- Willing and able to provide written informed consent to participate in the study.
- Participants must be ≥ 18 years at the time of screening.
- All participants must have a diagnosis of NF1 with symptomatic, inoperable PN where:
- a)Participants must have PN and at least one other diagnostic criterion for NF1 [18]: (i)Six or more café-au-lait spots (> 5 mm in greatest diameter in pre-pubertal participants or > 15 mm in greatest diameter in post-pubertal participants) (ii)Freckling in the axillary or inguinal region
- At least one of the two pigmentary findings (cafe-au-lait macules or freckling) should be bilateral (iii)Two or more iris Lisch nodules identified by slit lamp examination or 2 or more choroidal abnormalities-defined as bright, patchy nodules imaged by optical coherence tomography/near-infrared reflectance imaging (iv)Optic pathway glioma (v)A distinctive osseous lesion such as sphenoid dysplasia, anterolateral bowing of the tibia, or pseudoarthrosis of a long bone
- Sphenoid wing dysplasia is not a separate criterion in case of an ipsilateral orbital PN.
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- (vi)A NF1 heterozygous pathogenic variant with a variant allele fraction of 50% in apparently normal tissue such as white blood cells (vii)A parent with NF1 by the above criteria b)Target PN [15] is defined as a PN that is measurable (i.e. a PN of at least 3cm measured in one dimension, which can be seen on at least 3 imaging slices, and has a reasonably well-defined contour), symptomatic, and considered inoperable. Regular MRI/ Contrast-enhanced MRI/DCE-MRI/WB-MRI analysis might be performed on the target PN for volumetric analysis. If multiple PNs are present, the investigator will select the PN that is most clinically relevant (e.g., causing the predominant symptom) as the target PN.
- c)Inoperable is defined as a PN that cannot be completely surgically removed without a risk of substantial morbidity (including significant bleeding or damage to nerves and/or surrounding vital structures) due to encasement of, or close proximity to, vital structures, invasiveness, or high vascularity of the PN; or unacceptable risk from the general anesthetic as assessed by the investigator.
- d)Symptomatic is defined as clinically significant symptoms caused by the PN, as judged by the investigator; symptoms may include, but are not limited to, pain, motor dysfunction, and disfigurement.
- Have a documented treatment plan or prescription for selumetinib in the medical records as assessed by the investigator, indicating the decision to initiate selumetinib treatment.
- The first dose of selumetinib is administered within 28 days after ICF.
Who cannot join
- Participants with any evidence of a life-threatening illness, medical condition, or organ system dysfunction which, in the investigator's opinion, makes it undesirable for the participants to participate in the study.
- Participants with a confirmed or suspected malignant glioma or MPNST, or with a prior malignancy or other cancer requiring treatment with chemotherapy or radiation therapy.
- Inability to undergo MRI and/or contraindication for MRI examinations.
- Have had prior treatment with a MEKi, Ras or Raf inhibitor.
- Currently enrolled in any interventional clinical trial at the time of enrolment or initiation of selumetinib.
- Involvement in the planning and/or conduct of the study (applies to both AstraZeneca staff and/or staff at the study site).
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- Judgment by the investigator that the participant will not be able to adequately understand or consistently complete ePRO instruments.
- Pregnant or breastfeeding women.
- Male participants or female participants of childbearing potential who have childbearing plans (currently or within the next 3 months).
Trial locations and contacts
26 sites in 1 country. Contact the study team or a site near you to ask about taking part.
AstraZeneca Clinical Study Information Center contact
26 sites in 1 country
China26 sites, 26 recruiting
- Not yet recruiting
Research site
Beijing
- Not yet recruiting
Research site
Changchun
- Not yet recruiting
Research site
Changsha
- Not yet recruiting
Research site
Chengdu
- Not yet recruiting
Research site
Chongqing
- Not yet recruiting
Research site
Fuzhou
- Not yet recruiting
Research site
Guangzhou
- Not yet recruiting
Research site
Guiyang
- Not yet recruiting
Research site
Hangzhou
- Not yet recruiting
Research site
Hefei
- Not yet recruiting
Research site
Jinan
- Not yet recruiting
Research site
Kunming
- Not yet recruiting
Research site
Lanzhou
- Not yet recruiting
Research site
Nanchang
- Not yet recruiting
Research site
Nanjing
- Not yet recruiting
Research site
Nanning
- Not yet recruiting
Research site
Shanghai
- Not yet recruiting
Research site
Shenyang
- Not yet recruiting
Research site
Shenzhen
- Not yet recruiting
Research site
Shijiazhuang
- Not yet recruiting
Research site
Suzhou
- Not yet recruiting
Research site
Ürümqi
- Recruiting
Research site
Wuhan
- Not yet recruiting
Research site
Wuhan
- Not yet recruiting
Research site
Xi'an
- Not yet recruiting
Research site
Zhengzhou
About this study
This is a prospective, multicentre, observational registry study with primary data collection designed to evaluate the real-world effectiveness and safety of selumetinib in adult patients with NF1 who have symptomatic, inoperable PN. A total of approximately 800 participants who initiate selumetinib treatment will be enrolled across about 45 investigational sites in China. Sites will be selected based on their diagnostic and treatment expertise in NF1-PN, research capability and sufficient patient pool. Patients who are eligible and willing to participate will be enrolled into the study.
The study includes an approximately 25-month enrolment period and a 96-week observation period. Patients will be followed up until the end of the 96-weeks observation period after first dose of selumetinib, or patient death, lost to follow-up, withdrawal of consent, whichever occurs first. Patients will be followed within 96 weeks period (starting after first dose received) in the study even if selumetinib is discontinued. The study will end until the last participant has the opportunity to complete 96 weeks' assessment.
- Conditions
- Neurofibromatosis Type 1 (NF1)
- Medical subject headings
- Neurofibromatosis 1
What is being measured
The primary outcome is the main question the study is designed to answer.
ORR of PN by REiNS criteria.
To describe the objective response rate (ORR) of PN as assessed by the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) criteria
Measured: 96 weeks
QoL change from baseline by INF1-QoL(IMPACT OF NEUROFIBROMATOSIS 1 ON QUALITY-OF-LIFE) questionnaire.
To assess the change from baseline in quality of life (QoL) using the impact of neurofibromatosis 1 on quality-of-life questionnaire (INF1-QoL) questionnaire. The QoL score ranges from 0 to 42. Higher scores indicate worse quality of life. Therefore, a greater decrease from baseline in the QoL score represents an improvement in quality of life and a better outcome.
Measured: 96 weeks
Secondary outcomes (7)
The median best tumour volume change of PN from baseline
To assess the median best percentage change in PN tumour volume.
Measured: 96 weeks
The change of the diameter of cNF from baseline.
To assess the change in the diameter of cutaneous neurofibromas (cNF).
Measured: 96 weeks
AE/ADRs/SAE.
To characterize the safety profile of selumetinib by assessing AEs, adverse drug reactions (ADRs), and serious adverse events (SAEs).
Measured: 96 weeks
Percentage of patients with prior PN surgeries
Percentage of Chinese adult NF1-PN patients who underwent PN surgery prior to initiation of selumetinib treatment. Unit of Measure: Participants (%)
Measured: 96 weeks
Number of prior PN surgeries
Number of PN surgeries undergone by patients before initiation of selumetinib treatment. Unit of Measure: Number
Measured: 96 weeks
Percentage of patients undergoing PN surgery during selumetinib treatment
Percentage of Chinese adult NF1-PN patients who underwent PN surgery while receiving selumetinib treatment. Unit of Measure: Participants (%)
Measured: 96 weeks
Interval between PN surgery and initiation of selumetinib treatment
Time interval between PN surgery and initiation of selumetinib treatment. Unit of Measure: Days.
Measured: 96 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Oct 2026
- Study start28 Sep 2026
- Primary completion31 Dec 2030 (estimated)
- Study completion31 Dec 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurofibromatosis Type 1 (NF1)Studies of the same condition, open and under way.
- More trials run by AstraZenecaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 800 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AstraZeneca
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Zhichao Wang, Principal investigator, Shanghai Ninth People's Hospital Affiliated to Shanghai Jiao Tong UniversityPinan Liu, Principal investigator, Beijing Tiantan Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07888517
- Sponsor's protocol ID
- D134BR00012
- Acronym
- PROCEED
- Status last verified
- Oct 2026
- First submitted
- 2 Oct 2026
- Last updated
- 22 Oct 2026
- Results posted
- No
- Individual data sharing
- Yes: Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.