A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)

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Brief Title

A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)

Official Title

A Phase 1 Open Label Study to Assess the Safety, Tolerability, Pharmacokinetics and Clinical Efficacy of Selumetinib, a Selective Mitogen Activated Protein Kinase Kinase (MEK) 1 Inhibitor, in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)

Brief Summary

      A Phase 1 Open Label Study to Assess the Safety, Tolerability, Pharmacokinetics and Clinical
      Efficacy of Selumetinib, a Selective Mitogen Activated Protein Kinase Kinase (MEK) 1
      Inhibitor, in Chinese Paediatric and Adult Subjects with Neurofibromatosis Type 1 (NF1) and
      Inoperable Plexiform Neurofibromas (PN)
    

Detailed Description

      Paediatric and adult patients with Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform
      Neurofibromas (PN) will be evaluated in the screening visit to confirm eligibility.
      Approximately 16 paediatric and 16 adult qualified patients will receive oral selumetinib 25
      mg/m^2 twice a day (approximately every 12 hours) continuously until disease progression or
      unacceptable drug-related toxicity, whichever occurs first. Once a patient has discontinued
      the study treatment then the patient will be followed for specified period for safety
      assessment
    

Study Phase

Phase 1

Study Type

Interventional


Primary Outcome

Adverse events

Secondary Outcome

 objective response rate (ORR) of selumetinib in Chinese paediatric and adult subjects with Neurofibromatosis Type 1 and inoperable Plexiform Neurofibromas

Condition

Neurofibromatosis 1

Intervention

Selumetinib

Study Arms / Comparison Groups

 Selumetinib
Description:  All eligible subjects will first receive a single oral dose of selumetinib 25 mg/m^2. Then, selumetinib 25 mg/m^2 oral twice daily will be administered continuously until disease progression or unacceptable drug-related toxicity, whichever occurs first.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

32

Start Date

December 16, 2020

Completion Date

September 30, 2026

Primary Completion Date

October 31, 2023

Eligibility Criteria

        Inclusion Criteria:

          -  Paediatric cohort: Chinese subjects ≥3 years and <18 years of age

          -  Adult cohort: Chinese subjects ≥18 years of age at the time of study enrollment

          -  Subjects must be diagnosed with (i) NF1 as per NIH Consensus Development Conference
             Statement and(ii) PN is defined as a neurofibroma that has grown along the length of a
             nerve and may involve multiple fascicles and branches. (iii) inoperable PN

          -  Subjects must have at least one measurable typical or nodular PN

          -  Absolute neutrophil count ≥1.5×10^9/L, haemoglobin ≥9g/dL, and platelet count
             ≥100×10^9/L. Subject must be without growth factor support and platelet transfusion
             support 7 days before the screening assessment.

          -  Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2×upper limit of
             normal (ULN), total bilirubin ≤1.5×ULN except in the case of subjects with documented
             Gilbert's disease (≤2.5×ULN).

        Exclusion Criteria:

          -  Evidence of malignant peripheral nerve sheath tumour.

          -  Clinically significant cardiovascular disease

          -  Prior malignancy (except for adequately treated basal cell or squamous cell skin
             cancer, in situ cervical cancer, or other cancer from which the subject had been
             disease free for ≥2 years or which would not have limited survival to <2 years) or
             other cancer requiring treatment with chemotherapy or radiation therapy.

          -  Subjects with the following ophthalmological findings/conditions:

        Current or past history of retinal pigment epithelial detachment/central serous retinopathy
        or retinal vein occlusion; Intraocular pressure >21 mmHg (or ULN adjusted by age) or
        uncontrolled glaucoma (irrespective of IOP); Subjects with known glaucoma and increased IOP
        who do not have meaningful vision (light perception only or no light perception) and are
        not experiencing pain related to the glaucoma, may be eligible after discussion with the
        study physician; Any other significant abnormality on ophthalmic examination that would
        make the subject unsuitable for enrolment into the study, as assessed by the investigator.
      

Gender

All

Ages

3 Years - 99 Years

Accepts Healthy Volunteers

No

Contacts

Qingfeng Li, 1-877-240-9479, [email protected]

Location Countries

China

Location Countries

China

Administrative Informations


NCT ID

NCT04590235

Organization ID

D1346C00011


Responsible Party

Sponsor

Study Sponsor

AstraZeneca

Collaborators

 Merck Sharp & Dohme Corp.

Study Sponsor

Qingfeng Li, Principal Investigator, Shanghai Ninth People's Hospital affiliated to Shanghai JiaoTong University


Verification Date

September 2021