DDrare: Database of Drug Development for Rare Diseases

Searched query = "Neurofibromatosis", "NF1", "von Recklinghausen disease", "NF2"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = 2-amino-4-(methyl[11C]sulfanyl)butanoic acid; AMN107; AR-42; AZD2014; AZD6244; Acetaminophen; Acetylcysteine; Afinitor; Aminolevulinic acid; Antigen-specific T cells CART/CTL and DCvac; Aspirin; Axitinib; BMP-2; Behavioral: Other Academic sham tutoring; Behavioral: reading tutoring intervention; Bevacizumab; Bevacizumab (Genitical Recombination); Binimetinib; Bony Toxicity; Brigatinib; Butanoic acid; Cabozantinib; Carboplatin; Cediranib; Cediranib Maleate; Celecoxib; Celecoxib (Celebrex); Cetuximab; Chloride; Cholecalciferol; Crizotinib; Cysteine; Dabrafenib; Device: INFUSE Bone Graft (BMP-2); Device: Magnetic resonance imaging; Dexmedetomidine; Diclofenac; Diclofenac Sodium; Doxorubicin; Doxorubicin hydrochloride; Endostatin; Etoposide; Everolimus; Everolimus (RAD001); Everolimus (RAD001) , Afinitor®; Evérolimus; Filgrastim; Fludeoxyglucose; Fludeoxyglucose F 18; GLIVEC 100 mg comprimidos recubiertos con película; GLIVEC 400 mg comprimidos recubiertos con película; Gadolinium; Gadolinium contrast; Gadopentetate; Gleevec; Glycopyrrolate; Gold; Hydroxychloroquine; IMATINIB; Icotinib; Ifosfamide; Imatinib; Imatinib Mesylate; Imiquimod; Imiquimod 5% Cream; Interferon alpha-2B; Iodide; Isotonic sodium chloride solution; LAMOTRIGINE; LOVASTATIN; Lamotrigin; Lamotrigin beta; Lamotrigine; Lamotrigine dispers; Lapatinib; Lenalidomide; Levulan; Levulan (5-aminolevulinic acid) photodynamic therapy.; Levulan (5-aminolevulinic acid) uptake.; Lidocaine; Lidocaine 1% Injectable Solution; Lovastatin; Lovastatin 60 MG; Lovastatin ™; Mekinist; Methionine; Methotrexate; Methylphenidate; Mirdametinib (PD-0325901) oral capsule or dispersible tablet; N-Acetyl cysteine; N-acetylcysteine (NAC); NAC; NFX-179 Gel; NILOTINIB; NILOTINIB HYDROCHLORIDE MONOHYDRATE; NPC-12G gel; Nexavar; Nexavar (BAY 43-9006) (Sorafenib); Nilotinib; Nitrous Oxide; Nitrous oxide; Oncolytic Measles Virus Encoding Thyroidal Sodium Iodide Symporter; Oncovin; Other: Laboratory Biomarker Analysis; Other: Pharmacological Study; Other: Quality-of-Life Assessment; Other: Questionnaire Administration; Other: laboratory biomarker analysis; PD-0325901; PLX3397; PTC299; Peg-Interferon alpha-2b; Pharmacodynamics; Pirfenidone; Poly ICLC; Pomalidomide; Procedure: Computed Tomography; Procedure: Control Group; Procedure: MRI, FDG-PET/CT scans; Procedure: Single Photon Emission Computed Tomography; Procedure: conventional surgery; Procedure: quality-of-life assessment; Propofol; QOL assessment, Neuropsychological; RAD001; RAD001, everolimus; RAD001: Everolimus; Radiation: fludeoxyglucose F 18; Radiation: gadopentetate dimeglumine; Radiation: radiation therapy; Radiographic Evaluation; Ranibizumab; Rapamycin; SIMVASTATIN; Saline Solution; Selumetinib; Selumetinib (AZD6244 hyd sulfate) 25mg/m2; Selumetinib (AZD6244 hyd sulfate) 50mg/dose; Selumetinib Hyd-sulphate; Selumetinib Sulfate; Selumetinib sulfate; Sevoflurane; Simvastatin; Simvastatin 20mg/5ml Oral Suspension; Sirolimus; Skincerity; Skincerity plus sirolimus/rapamycin; Skinercity plus sirolimus/rapamycin; Sodium chloride; Sodium iodide; Sorafenib; Sorafenib (as tosylate); Sulfate; Sunitinib; Sutent®/Sunitinib; Tarceva and Rapamycin; Tasigna; Tasigna 150mg; Tasigna 200mg; Tasigna 50 mg; Temozolomide; Temozolomide (temodar); Temsirolimus; Tipifarnib; Toxicity, Pharmacokinetics; Trametinib; Treatment A; Treatment B; Treatment C; Treatment D; VINBLASTINE SULPHATE; Valproic Acid; Valproic acid; Vehicle Gel; Vinblastine; Vinblastine sulfate; Vincristine; Vincristine Sulfate; Vincristine Sulfate (Oncovin); Votubia; [11C]-methionine; [18F]-FLT-PET/CT scans

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT02839720 (ClinicalTrials.gov)	April 11, 2017	20/7/2016	Selumetinib in Treating Patients With Neurofibromatosis Type 1 and Cutaneous Neurofibroma	Pilot Study of the MEK1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) for Adults With Neurofibromatosis Type 1 (NF1) and Cutaneous Neurofibromas (CNF)	Cutaneous Neurofibroma;Neurofibromatosis Type 1;Optic Nerve Glioma	Other: Laboratory Biomarker Analysis;Drug: Selumetinib;Drug: Selumetinib Sulfate	National Cancer Institute (NCI)	NULL	Recruiting	18 Years	N/A	All	24	Phase 2	United States
2	NCT02415153 (ClinicalTrials.gov)	July 14, 2015	13/4/2015	Pomalidomide in Treating Younger Patients With Recurrent, Progressive, or Refractory Central Nervous System Tumors	A Phase I Trial of Pomalidomide for Children With Recurrent, Progressive, or Refractory CNS Tumors	Neurofibromatosis Type 1;Recurrent Childhood Brain Stem Glioma;Recurrent Childhood Visual Pathway Glioma;Recurrent Primary Central Nervous System Neoplasm;Refractory Primary Central Nervous System Neoplasm	Other: Laboratory Biomarker Analysis;Other: Pharmacological Study;Drug: Pomalidomide	National Cancer Institute (NCI)	NULL	Active, not recruiting	3 Years	20 Years	All	29	Phase 1	United States
3	NCT01089101 (ClinicalTrials.gov)	April 19, 2010	17/3/2010	Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma	A Phase 1 and Phase II and Re-Treatment Study of AZD6244 for Recurrent or Refractory Pediatric Low Grade Glioma	Low Grade Glioma;Recurrent Childhood Pilocytic Astrocytoma;Recurrent Neurofibromatosis Type 1;Recurrent Visual Pathway Glioma;Refractory Neurofibromatosis Type 1;Refractory Visual Pathway Glioma	Other: Laboratory Biomarker Analysis;Other: Pharmacological Study;Drug: Selumetinib	National Cancer Institute (NCI)	NULL	Recruiting	3 Years	21 Years	All	220	Phase 1;Phase 2	United States

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

NCT02839720
(ClinicalTrials.gov)

April 11, 2017

20/7/2016

Selumetinib in Treating Patients With Neurofibromatosis Type 1 and Cutaneous Neurofibroma

Pilot Study of the MEK1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) for Adults With Neurofibromatosis Type 1 (NF1) and Cutaneous Neurofibromas (CNF)

Cutaneous Neurofibroma;Neurofibromatosis Type 1;Optic Nerve Glioma

Other: Laboratory Biomarker Analysis;Drug: Selumetinib;Drug: Selumetinib Sulfate

National Cancer Institute (NCI)

NULL

Recruiting

18 Years

N/A

All

Phase 2

United States

NCT02415153
(ClinicalTrials.gov)

July 14, 2015

13/4/2015

Pomalidomide in Treating Younger Patients With Recurrent, Progressive, or Refractory Central Nervous System Tumors

A Phase I Trial of Pomalidomide for Children With Recurrent, Progressive, or Refractory CNS Tumors

Neurofibromatosis Type 1;Recurrent Childhood Brain Stem Glioma;Recurrent Childhood Visual Pathway Glioma;Recurrent Primary Central Nervous System Neoplasm;Refractory Primary Central Nervous System Neoplasm

Other: Laboratory Biomarker Analysis;Other: Pharmacological Study;Drug: Pomalidomide

National Cancer Institute (NCI)

NULL

Active, not recruiting

3 Years

20 Years

All

Phase 1

United States

NCT01089101
(ClinicalTrials.gov)

April 19, 2010

17/3/2010

Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma

A Phase 1 and Phase II and Re-Treatment Study of AZD6244 for Recurrent or Refractory Pediatric Low Grade Glioma

Low Grade Glioma;Recurrent Childhood Pilocytic Astrocytoma;Recurrent Neurofibromatosis Type 1;Recurrent Visual Pathway Glioma;Refractory Neurofibromatosis Type 1;Refractory Visual Pathway Glioma

Other: Laboratory Biomarker Analysis;Other: Pharmacological Study;Drug: Selumetinib

National Cancer Institute (NCI)

NULL

Recruiting

3 Years

21 Years

All

220

Phase 1;Phase 2

United States