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	EUCTR2012-003005-10-DK (EUCTR)	06/06/2013	08/05/2013	LOW-GRADE GLIOMA	PHASE I-II STUDY OF VINBLASTINE IN COMBINATION WITH NILOTINIB IN CHILDREN, ADOLESCENTS, AND YOUNG ADULTS WITH REFRACTORY OR RECURRENT LOW-GRADE GLIOMA - Vinilo	Children, adolescents and young adults with refractory or recurrent low-grade gliomas, and children, adolescents and young adults with neurofibromatosis type 1 and previously untreated low-grade gliomas MedDRA version: 20.0;Level: PT;Classification code 10065443;Term: Malignant glioma;System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) MedDRA version: 20.0;Classification code 10038111;Term: Recurrent cancer;Classification code 10070308;Term: Refractory cancer;System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps);Therapeutic area: Diseases [C] - Cancer [C04]	Product Name: Nilotinib Product Code: AMN107 Other descriptive name: NILOTINIB HYDROCHLORIDE MONOHYDRATE Trade Name: Tasigna 150mg Product Name: Nilotinib INN or Proposed INN: NILOTINIB Trade Name: Tasigna 200mg Product Name: Nilotinib INN or Proposed INN: NILOTINIB Product Name: Vinblastine Other descriptive name: VINBLASTINE SULPHATE Trade Name: Tasigna 50 mg Product Name: Nilotinib INN or Proposed INN: NILOTINIB	Gustave Roussy	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	160	Phase 2	France;Spain;Denmark;Netherlands;Switzerland
2	NCT00352495 (ClinicalTrials.gov)	June 2006	13/7/2006	Vinblastine and Carboplatin in Treating Young Patients With Newly Diagnosed or Recurrent Low-Grade Glioma	A Phase I Study of Vinblastine in Combination With Carboplatin for Children With Newly Diagnosed and Recurrent Low-Grade Gliomas	Brain and Central Nervous System Tumors;Neurofibromatosis Type 1	Drug: carboplatin;Drug: vinblastine sulfate	Children's Oncology Group	National Cancer Institute (NCI)	Completed	N/A	21 Years	Both	26	Phase 1	United States;Canada
3	NCT00030264 (ClinicalTrials.gov)	February 2001	14/2/2002	Combination Chemotherapy in Treating Patients With Neurofibromatosis and Progressive Plexiform Neurofibromas	Vinblastine/Methotrexate For Severe Progressive Plexiform Neurofibromas: A Phase II Study	Neurofibromatosis Type 1;Precancerous Condition	Drug: Methotrexate;Drug: Vinblastine	Children's Hospital of Philadelphia	NULL	Completed	N/A	25 Years	All	23	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

EUCTR2012-003005-10-DK
(EUCTR)

06/06/2013

08/05/2013

LOW-GRADE GLIOMA

PHASE I-II STUDY OF VINBLASTINE IN COMBINATION WITH NILOTINIB IN CHILDREN, ADOLESCENTS, AND YOUNG ADULTS WITH REFRACTORY OR RECURRENT LOW-GRADE GLIOMA - Vinilo

Children, adolescents and young adults with refractory or recurrent low-grade gliomas, and children, adolescents and young adults with neurofibromatosis type 1 and previously untreated low-grade gliomas
MedDRA version: 20.0;Level: PT;Classification code 10065443;Term: Malignant glioma;System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA version: 20.0;Classification code 10038111;Term: Recurrent cancer;Classification code 10070308;Term: Refractory cancer;System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps);Therapeutic area: Diseases [C] - Cancer [C04]

Product Name: Nilotinib
Product Code: AMN107
Other descriptive name: NILOTINIB HYDROCHLORIDE MONOHYDRATE
Trade Name: Tasigna 150mg
Product Name: Nilotinib
INN or Proposed INN: NILOTINIB
Trade Name: Tasigna 200mg
Product Name: Nilotinib
INN or Proposed INN: NILOTINIB
Product Name: Vinblastine
Other descriptive name: VINBLASTINE SULPHATE
Trade Name: Tasigna 50 mg
Product Name: Nilotinib
INN or Proposed INN: NILOTINIB

Gustave Roussy

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

160

Phase 2

France;Spain;Denmark;Netherlands;Switzerland

NCT00352495
(ClinicalTrials.gov)

June 2006

13/7/2006

Vinblastine and Carboplatin in Treating Young Patients With Newly Diagnosed or Recurrent Low-Grade Glioma

A Phase I Study of Vinblastine in Combination With Carboplatin for Children With Newly Diagnosed and Recurrent Low-Grade Gliomas

Brain and Central Nervous System Tumors;Neurofibromatosis Type 1

Drug: carboplatin;Drug: vinblastine sulfate

Children's Oncology Group

National Cancer Institute (NCI)

Completed

N/A

21 Years

Both

Phase 1

United States;Canada

NCT00030264
(ClinicalTrials.gov)

February 2001

14/2/2002

Combination Chemotherapy in Treating Patients With Neurofibromatosis and Progressive Plexiform Neurofibromas

Vinblastine/Methotrexate For Severe Progressive Plexiform Neurofibromas: A Phase II Study

Neurofibromatosis Type 1;Precancerous Condition

Drug: Methotrexate;Drug: Vinblastine

Children's Hospital of Philadelphia

NULL

Completed

N/A

25 Years

All

Phase 2

United States