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	EUCTR2012-005742-38-GB (EUCTR)	02/05/2013	05/04/2013	Early phase triple blind placebo controlled RCT of simvastatin treatment for autism in young children with Neurofibromatosis Type 1	Early phase triple blind placebo controlled RCT of simvastatin treatment for autism in young children with Neurofibromatosis Type 1 - SimvAstatin in Neurofibromatosis Type 1-Autism (SANTA)	Neurofibromatosis Type 1 MedDRA version: 14.1;Level: LLT;Classification code 10029270;Term: Neurofibromatosis, type 1 (von Recklinghausen's disease);System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]	Trade Name: Simvastatin 20mg/5ml Oral Suspension Product Name: Simvastatin INN or Proposed INN: Simvastatin	Central Manchester University Hospitals NHS Foundation Trust	NULL	Not Recruiting			Female: yes Male: yes	0	Phase 2	United Kingdom
2	EUCTR2009-010965-22-NL (EUCTR)	29/09/2009	21/09/2009	The effect of long-term simvastatin treatment on cognitive function and daily life in children with Neurofibromatosis 1: a one year randomized controlled trial	The effect of long-term simvastatin treatment on cognitive function and daily life in children with Neurofibromatosis 1: a one year randomized controlled trial	Neurofibromatosis 1	Product Name: SIMVASTATIN Product Code: SIMVASTATIN INN or Proposed INN: SIMVASTATIN	Erasmus MC - Department of Pediatrics	NULL	Not Recruiting			Female: yes Male: yes	106		Netherlands

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Public_title

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Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
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EUCTR2012-005742-38-GB
(EUCTR)

02/05/2013

05/04/2013

Early phase triple blind placebo controlled RCT of simvastatin treatment for autism in young children with Neurofibromatosis Type 1

Early phase triple blind placebo controlled RCT of simvastatin treatment for autism in young children with Neurofibromatosis Type 1 - SimvAstatin in Neurofibromatosis Type 1-Autism (SANTA)

Neurofibromatosis Type 1
MedDRA version: 14.1;Level: LLT;Classification code 10029270;Term: Neurofibromatosis, type 1 (von Recklinghausen's disease);System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

Trade Name: Simvastatin 20mg/5ml Oral Suspension
Product Name: Simvastatin
INN or Proposed INN: Simvastatin

Central Manchester University Hospitals NHS Foundation Trust

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

United Kingdom

EUCTR2009-010965-22-NL
(EUCTR)

29/09/2009

21/09/2009

The effect of long-term simvastatin treatment on cognitive function and daily life in children with Neurofibromatosis 1: a one year randomized controlled trial

Neurofibromatosis 1

Product Name: SIMVASTATIN
Product Code: SIMVASTATIN
INN or Proposed INN: SIMVASTATIN

Erasmus MC - Department of Pediatrics

NULL

Not Recruiting

Female: yes
Male: yes

106

Netherlands