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	EUCTR2016-005022-10-DE (EUCTR)	13/02/2018	09/11/2017	Improvement of synaptic plasticity and cognitive function in RAS pathway disorders	Improvement of synaptic plasticity and cognitive function in RAS pathway disorders	Noonan Syndrom and Neurofibromatosis Type 1 MedDRA version: 20.0;Level: PT;Classification code 10029748;Term: Noonan syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Classification code 10029268;Term: Neurofibromatosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Not possible to specify	Product Name: Lovastatin INN or Proposed INN: Lovastatin Other descriptive name: LOVASTATIN Product Name: Lamotrigin beta INN or Proposed INN: Lamotrigin	Technische Universität München, Fakultät für Medizin	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	30	Phase 2	Germany

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

EUCTR2016-005022-10-DE
(EUCTR)

13/02/2018

09/11/2017

Improvement of synaptic plasticity and cognitive function in RAS pathway disorders

Noonan Syndrom and Neurofibromatosis Type 1
MedDRA version: 20.0;Level: PT;Classification code 10029748;Term: Noonan syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Classification code 10029268;Term: Neurofibromatosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Not possible to specify

Product Name: Lovastatin
INN or Proposed INN: Lovastatin
Other descriptive name: LOVASTATIN
Product Name: Lamotrigin beta
INN or Proposed INN: Lamotrigin

Technische Universität München, Fakultät für Medizin

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

Germany