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Neurofibromatosis Type 1

11
Pipeline Programs
11
Companies
38
Clinical Trials
2 recruiting
1
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
2
2
5
0
0
2
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Small Molecule
5100%
+ 10 programs with unclassified modality

On Market (1)

Approved therapies currently available

AstraZeneca
KOSELUGOApproved
selumetinib
AstraZeneca
Kinase Inhibitor [EPC]oral2025
15M Part D

Competitive Landscape

11 companies ranked by most advanced pipeline stage

AstraZeneca
AstraZenecaCAMBRIDGE, United Kingdom
1 program
1
KOSELUGO(selumetinib)APPROVED_FOR_MARKETINGSmall Molecule5 trials
Active Trials
NCT03259633Approved For Marketing
NCT06360406Recruiting200Est. Sep 2031
NCT05891847Active Not Recruiting150Est. Mar 2026
+2 more trials
Human BioSciences
Human BioSciencesWV - Martinsburg
2 programs
1
LovastatinPhase 2Small Molecule1 trial
Adaptation and Quality of Life Among Adults With Neurofibromatosis Type IN/A1 trial
Active Trials
NCT00684398Completed645Est. Nov 2015
NCT02964884Active Not Recruiting120Est. Jan 2027
Utah Medical
Utah MedicalIreland - Dublin
2 programs
2
Lovastatin ™Phase 21 trial
SirolimusPhase 21 trial
Active Trials
NCT00853580Completed146Est. Dec 2016
NCT00634270Completed58Est. Dec 2015
Sandoz
SandozAustria - Kundl
1 program
1
RAD001: EverolimusPhase 21 trial
Active Trials
NCT01412892Completed30Est. Oct 2014
Alliance Pharmaceuticals
1 program
1
filgrastimPhase 21 trial
Active Trials
NCT00304083Completed48Est. Jun 2014
Sharp Therapeutics
Sharp TherapeuticsPA - Pittsburgh
2 programs
1
1
Selumetinib granule formulationPhase 1/2
SelumetinibPhase 1Small Molecule
MSD
MSDIreland - Ballydine
2 programs
1
1
Selumetinib granule formulationPhase 1/21 trial
SelumetinibPhase 1Small Molecule1 trial
Active Trials
NCT05101148Active Not Recruiting24Est. Apr 2028
NCT05309668Active Not Recruiting36Est. Apr 2028
Genome & Company
Genome & CompanyKorea - Suwon
1 program
Adaptation and Quality of Life Among Adults With Neurofibromatosis Type IN/A
Angeles Therapeutics
Angeles TherapeuticsCA - Los Angeles
1 program
CogmedRMN/A1 trial
Active Trials
NCT02944032Completed103Est. Sep 2021
Merck & Co.
Merck & Co.RAHWAY, NJ
1 program
SelumetinibPHASE_1Small Molecule
Healx
HealxUK - Cambridge
1 program
HLX-1502PHASE_21 trial
Active Trials
NCT06541847Recruiting25Est. Jan 2028

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
HealxHLX-1502
AstraZenecaselumetinib
AstraZenecaselumetinib
AstraZenecaselumetinib
Human BioSciencesLovastatin
AstraZenecaselumetinib
AstraZenecaselumetinib
SandozRAD001: Everolimus
Utah MedicalLovastatin ™
Utah MedicalSirolimus
Alliance Pharmaceuticalsfilgrastim
MSDSelumetinib granule formulation
AstraZenecaselumetinib
AstraZenecaselumetinib
AstraZenecaselumetinib

Showing 15 of 37 trials with date data

Clinical Trials (38)

Total enrollment: 3,114 patients across 38 trials

A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1

Start: Feb 2025Est. completion: Jan 202825 patients
Phase 2Recruiting

Maintenance With Olaparib or Selumetinib + Durvaluma for m-PDAC Guided by BRCAness and KRAS Status.

Start: Dec 2020Est. completion: Dec 2025307 patients
Phase 2Active Not Recruiting

MEDI4736 With Selumetinib for KRAS Mutant Non-Small Cell Lung Cancer (NSCLC)

Start: May 2018Est. completion: May 20240
Phase 2Withdrawn

Trial of Selumetinib in Patients With Neurofibromatosis Type II Related Tumors

Start: May 2017Est. completion: May 202410 patients
Phase 2Terminated

Interventions for Reading Disabilities in NF1

Start: Nov 2016Est. completion: Jan 2027120 patients
Phase 2Active Not Recruiting

Selumetinib in Patients Receiving Pemetrexed and Platinum-based Chemotherapy in Advanced or Metastatic KRAS Wildtype or Unknown Non-Squamous NSCLC

Start: May 2015Est. completion: Jun 201962 patients
Phase 2Completed

Immune-Modulated Study of Selected Small Molecules (Gefitinib, AZD9291, or Selumetinib + Docetaxel) or a 1st Immune-Mediated Therapy (IMT; Tremelimumab) With a Sequential Switch to a 2nd IMT (MEDI4736) in Patients With Locally Advanced or Metastatic Non-Small-Cell Lung Cancer

Start: Jul 2014Est. completion: Jun 201632 patients
Phase 2Completed
NCT01412892SandozRAD001: Everolimus

Use of RAD001 as Monotherapy in the Treatment of Neurofibromatosis 1 Related Internal Plexiform Neurofibromas

Start: Apr 2011Est. completion: Oct 201430 patients
Phase 2Completed

A Randomized Placebo-Controlled Study of Lovastatin in Children With Neurofibromatosis Type 1

Start: Jul 2009Est. completion: Dec 2016146 patients
Phase 2Completed

A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas

Start: Apr 2008Est. completion: Dec 201558 patients
Phase 2Completed

Combination Chemotherapy in Treating Patients With Stage III or Stage IV Malignant Peripheral Nerve Sheath Tumors

Start: Dec 2005Est. completion: Jun 201448 patients
Phase 2Completed
NCT05309668MSDSelumetinib granule formulation

Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN

Start: Jan 2022Est. completion: Apr 202836 patients
Phase 1/2Active Not Recruiting

Intermittent Dosing Of Selumetinib In Childhood NF1 Associated Tumours

Start: Sep 2019Est. completion: Feb 202414 patients
Phase 1/2Completed

International Trial of Selumetinib in Combination With Dexamethasone for the Treatment of Acute Lymphoblastic Leukaemia

Start: May 2018Est. completion: May 202312 patients
Phase 1/2Terminated

A Study of Selumetinib in Patients With Kaposi's Sarcoma

Start: Jun 2012Est. completion: Dec 201719 patients
Phase 1/2Terminated
NCT05101148MSDSelumetinib

Phase I Study to Assess the Effect of Food on the PK and Gastrointestinal Tolerability of Selumetinib in Adolescent Children With Neurofibromatosis Type 1 Related Plexiform Neurofibromas

Start: Jul 2021Est. completion: Apr 202824 patients
Phase 1Active Not Recruiting

Selumetinib Paediatric NF1 Japan Study

Start: Aug 2020Est. completion: Mar 202312 patients
Phase 1Completed

Selumetinib and Olaparib in Solid Tumors

Start: Aug 2017Est. completion: Aug 202690 patients
Phase 1Active Not Recruiting

Intermittent Selumetinib for Uveal Melanoma

Start: Feb 2017Est. completion: Sep 202042 patients
Phase 1Completed

A Study to Assess the Safety, Tolerability and Anti-tumour Activity of Ascending Doses of Selumetinib in Combination With MEDI4736 and Selumetinib in Combination With MEDI4736 and Tremelimumab in Patients With Advanced Solid Tumours

Start: Dec 2015Est. completion: Sep 201958 patients
Phase 1Completed

A Study to Assess the Relative Bioavailability of Process Variants of Selumetinib in Healthy Male Volunteers

Start: Feb 2015Est. completion: Apr 201548 patients
Phase 1Completed

A Study to Assess the Absolute Bioavailability of Oral Selumetinib in Healthy Male Volunteers.

Start: Oct 2014Est. completion: Nov 201421 patients
Phase 1Completed

AZD9291 in Combination With Ascending Doses of Novel Therapeutics

Start: Aug 2014Est. completion: Dec 2026344 patients
Phase 1Active Not Recruiting

To Assess the Effect of Itraconazole and Fluconazole on the Pharmacokinetics of Selumetinib in Healthy Male Volunteers

Start: Apr 2014Est. completion: Jun 201426 patients
Phase 1Completed

To Assess the Pharmacokinetics, Safety and Tolerability of Selumetinib in Patients With Hepatic Impairment and Healthy Subjects

Start: Mar 2014Est. completion: Nov 201432 patients
Phase 1Completed

To Assess the Pharmacokinetics, Safety and Tolerability of Selumetinib in Renal Impaired Subjects and Healthy Subjects

Start: Mar 2014Est. completion: Jul 201424 patients
Phase 1Completed

To Assess the Effect of Rifampicin on the Pharmacokinetics of Selumetinib in Healthy Male Volunteers

Start: Feb 2014Est. completion: Apr 201424 patients
Phase 1Completed

Dose-escalation Study to Assess Selumetinib Safety, Tolerability and PK

Start: Oct 2013Est. completion: May 2014117 patients
Phase 1Completed

Study Evaluating Absorption, Distribution, Metabolism and Excretion (ADME) of Single Dose [14C] Selumetinib in Volunteers

Start: Oct 2013Est. completion: Nov 20136 patients
Phase 1Completed

Assess Safety & Efficacy of Selumetinib When Given in Combination With Standard First Line Treatment for Advanced Non-small Cell Lung Cancer

Start: Apr 2013Est. completion: Jan 201655 patients
Phase 1Completed

ABC-04 a Study of Cisplatin, Gemcitabine and Selumetinib in Patients With Advanced Biliary Tract Cancer

Start: Feb 2012Est. completion: May 201613 patients
Phase 1Completed

An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1

N/AApproved For Marketing

Real-World Treatment Study of Koselugo (Selumetinib)

Start: Jun 2024Est. completion: Sep 2031200 patients
N/ARecruiting

Non-interventional Study of Patients With PN NF1 Starting Selumetinib in Russia

Start: Mar 2023Est. completion: Mar 2026150 patients
N/AActive Not Recruiting

PASS of Paediatric Patients Initiating Selumetinib

Start: May 2022Est. completion: May 2028124 patients
N/AActive Not Recruiting

US Selumetinib Registry

Start: Jan 2019Est. completion: Nov 202437 patients
N/ATerminated

Efficacy of Computerized Cognitive Training and Stimulant Medication in Neurofibromatosis Type 1

Start: May 2016Est. completion: Sep 2021103 patients
N/ACompleted
NCT00684398Human BioSciencesAdaptation and Quality of Life Among Adults With Neurofibromatosis Type I

Adaptation and Quality of Life Among Adults With Neurofibromatosis Type I

Start: May 2008Est. completion: Nov 2015645 patients
N/ACompleted

Related Jobs

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

2 actively recruiting trials targeting 3,114 patients
11 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.