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	JPRN-UMIN000015081	2014/09/08	07/09/2014	Pilot study of topical medicine of rapamycin for large diffuse plexiform neurofibroma of neurofibromatosis type 1		neurofibromatosis type1	1) Application of 0.2% rapamycin gel on one third of the tumor once a day for 2 weeks. 2) Check the skin findings, blood test results, blood level of rapamycin and adverse events after 2 weeks of treatment. If there is no problem, perform application of 0.2% rapamycin gel on two-thirds of the tumor once a day for another 2 weeks. 3) Check the skin findings, blood test results, blood level of rapamycin and adverse events after a total of 4 weeks of treatment. If there is no problem, perform application of 0.2% rapamycin gel on the whole surface of the tumor once a day for another 8 weeks.	Department of Dermatology Graduate School of Medicine, Osaka University	NULL	Recruiting	20years-old	Not applicable	Male and Female	3	Phase 2;Phase 3	Japan
2	NCT01031901 (ClinicalTrials.gov)	December 2009	10/12/2009	Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)	Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex and Neurofibromatosis 1	Tuberous Sclerosis;Neurofibromatoses;Angiofibroma;Neurofibroma	Drug: Skincerity;Drug: Skincerity plus sirolimus/rapamycin;Drug: Skinercity plus sirolimus/rapamycin	The University of Texas Health Science Center, Houston	Society for Pediatric Dermatology	Completed	13 Years	N/A	Both	52	Phase 1	United States
3	NCT00901849 (ClinicalTrials.gov)	May 2007	13/5/2009	Tarceva/Rapamycin for Children With Low-grade Gliomas With or Without Neurofibromatosis Type 1 (NF1)	Phase 1 Study of Tarceva and Rapamycin For Recurrent Low-Grad Gliomas in Children With or Without Neurofibromatosis Type 1 (NF1)	Low-grade Gliomas	Drug: Tarceva and Rapamycin	Roger Packer	Children's Research Institute	Completed	N/A	21 Years	Both	21	Phase 1	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

JPRN-UMIN000015081

2014/09/08

07/09/2014

Pilot study of topical medicine of rapamycin for large diffuse plexiform neurofibroma of neurofibromatosis type 1

neurofibromatosis type1

1) Application of 0.2% rapamycin gel on one third of the tumor once a day for 2 weeks.
2) Check the skin findings, blood test results, blood level of rapamycin and adverse events after 2 weeks of treatment.
If there is no problem, perform application of 0.2% rapamycin gel on two-thirds of the tumor once a day for another 2 weeks.
3) Check the skin findings, blood test results, blood level of rapamycin and adverse events after a total of 4 weeks of treatment.
If there is no problem, perform application of 0.2% rapamycin gel on the whole surface of the tumor once a day for another 8 weeks.

Department of Dermatology Graduate School of Medicine, Osaka University

NULL

Recruiting

20years-old

Not applicable

Male and Female

Phase 2;Phase 3

Japan

NCT01031901
(ClinicalTrials.gov)

December 2009

10/12/2009

Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)

Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex and Neurofibromatosis 1

Tuberous Sclerosis;Neurofibromatoses;Angiofibroma;Neurofibroma

Drug: Skincerity;Drug: Skincerity plus sirolimus/rapamycin;Drug: Skinercity plus sirolimus/rapamycin

The University of Texas Health Science Center, Houston

Society for Pediatric Dermatology

Completed

13 Years

N/A

Both

Phase 1

United States

NCT00901849
(ClinicalTrials.gov)

May 2007

13/5/2009

Tarceva/Rapamycin for Children With Low-grade Gliomas With or Without Neurofibromatosis Type 1 (NF1)

Phase 1 Study of Tarceva and Rapamycin For Recurrent Low-Grad Gliomas in Children With or Without Neurofibromatosis Type 1 (NF1)

Low-grade Gliomas

Drug: Tarceva and Rapamycin

Roger Packer

Children's Research Institute

Completed

N/A

21 Years

Both

Phase 1

United States