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	NCT01345136 (ClinicalTrials.gov)	July 1, 2015	21/4/2011	Study of RAD001 for Treatment of NF2-related Vestibular Schwannoma	A Single Arm, Monocenter Phase II Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis Type 2 - Related Vestibular Schwannoma	Neurofibromatosis Type 2;Neuroma, Acoustic	Drug: RAD001, everolimus	Jonsson Comprehensive Cancer Center	Novartis Pharmaceuticals	Active, not recruiting	16 Years	65 Years	All	4	Phase 2	United States
2	NCT02332902 (ClinicalTrials.gov)	February 2015	12/12/2014	Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232T	Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1- CRAD001CUS232T	Neurofibromatosis 1	Drug: Everolimus	The University of Texas Health Science Center, Houston	Texas Neurofibromatosis Foundation	Completed	18 Years	N/A	All	24	Phase 2	United States
3	NCT01661283 (ClinicalTrials.gov)	September 2012	7/8/2012	SARC016: Study of Everolimus With Bevacizumab to Treat Refractory Malignant Peripheral Nerve Sheath Tumors	Phase 2 Study of the mTOR Inhibitor Everolimus in Combination With Bevacizumab in Patients With Sporadic and Neurofibromatosis Type 1 (NF1) Related Refractory Malignant Peripheral Nerve Sheath Tumors	Malignant Peripheral Nerve Sheath Tumors;MPNST;Sarcoma	Drug: everolimus;Drug: bevacizumab	Sarcoma Alliance for Research through Collaboration	Novartis Pharmaceuticals;Genentech, Inc.;United States Department of Defense	Completed	18 Years	N/A	All	25	Phase 2	United States
4	NCT01365468 (ClinicalTrials.gov)	April 2012	27/5/2011	Efficacy and Safety of RAD001 in Treating Plexiform Neurofibromas (PN) Associated With Neurofibromatosis (NF1)	A Phase II Study of RAD001 in the Treatment of Patients With Plexiform Neurofibromas (PN) Associated With Neurofibromatosis Type 1 (NF1)	Plexiform Neurofibroma Associated With Neurofibromatosis Type 1	Drug: Everolimus (RAD001)	Novartis Pharmaceuticals	NULL	Terminated	6 Years	N/A	All	9	Phase 2	Israel
5	NCT01419639 (ClinicalTrials.gov)	October 2011	17/8/2011	Phase II Study of Everolimus (RAD001) in Children and Adults With Neurofibromatosis Type 2	Phase II Study of Everolimus (RAD001) in Children and Adults With Neurofibromatosis Type 2	Neurofibromatosis Type II	Drug: Everolimus (RAD001) , Afinitor®	New York University School of Medicine	Novartis Pharmaceuticals;The Children's Tumor Foundation	Completed	3 Years	N/A	All	10	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
6	NCT01412892 (ClinicalTrials.gov)	April 2011	8/8/2011	Use of RAD001 as Monotherapy in the Treatment of Neurofibromatosis 1 Related Internal Plexiform Neurofibromas	A Single Arm, Multicenter Phase II a Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis 1 Related Internal Plexiform Neurofibromas That Cannot be Removed by Surgery	Neurofibromatosis Type 1;Plexiform Neurofibroma;Neurofibromatoses	Drug: RAD001: Everolimus	Assistance Publique - Hôpitaux de Paris	Novartis	Completed	18 Years	60 Years	Both	30	Phase 2	France
7	EUCTR2016-001563-36-Outside-EU/EEA (EUCTR)		12/05/2016	Efficacy and Safety of RAD001 in Treating Plexiform Neurofibromas (PN) Associated With Neurofibromatosis (NF1)	A Phase II Study of RAD001 in the Treatment of Patients With Plexiform Neurofibromas (PN) Associated With Neurofibromatosis Type 1 (NF1)	Plexiform Neurofibroma Associated With Neurofibromatosis Type 1;Therapeutic area: Diseases [C] - Cancer [C04]	Trade Name: Votubia Product Name: Everolimus Product Code: RAD001	Novartis Pharmaceuticals	NULL	NA			Female: yes Male: yes	26	Phase 4	Israel

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

NCT01345136
(ClinicalTrials.gov)

July 1, 2015

21/4/2011

Study of RAD001 for Treatment of NF2-related Vestibular Schwannoma

A Single Arm, Monocenter Phase II Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis Type 2 - Related Vestibular Schwannoma

Neurofibromatosis Type 2;Neuroma, Acoustic

Drug: RAD001, everolimus

Jonsson Comprehensive Cancer Center

Novartis Pharmaceuticals

Active, not recruiting

16 Years

65 Years

All

Phase 2

United States

NCT02332902
(ClinicalTrials.gov)

February 2015

12/12/2014

Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232T

Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1- CRAD001CUS232T

Neurofibromatosis 1

Drug: Everolimus

The University of Texas Health Science Center, Houston

Texas Neurofibromatosis Foundation

Completed

18 Years

N/A

All

Phase 2

United States

NCT01661283
(ClinicalTrials.gov)

September 2012

7/8/2012

SARC016: Study of Everolimus With Bevacizumab to Treat Refractory Malignant Peripheral Nerve Sheath Tumors

Phase 2 Study of the mTOR Inhibitor Everolimus in Combination With Bevacizumab in Patients With Sporadic and Neurofibromatosis Type 1 (NF1) Related Refractory Malignant Peripheral Nerve Sheath Tumors

Malignant Peripheral Nerve Sheath Tumors;MPNST;Sarcoma

Drug: everolimus;Drug: bevacizumab

Sarcoma Alliance for Research through Collaboration

Novartis Pharmaceuticals;Genentech, Inc.;United States Department of Defense

Completed

18 Years

N/A

All

Phase 2

United States

NCT01365468
(ClinicalTrials.gov)

April 2012

27/5/2011

Efficacy and Safety of RAD001 in Treating Plexiform Neurofibromas (PN) Associated With Neurofibromatosis (NF1)

A Phase II Study of RAD001 in the Treatment of Patients With Plexiform Neurofibromas (PN) Associated With Neurofibromatosis Type 1 (NF1)

Plexiform Neurofibroma Associated With Neurofibromatosis Type 1

Drug: Everolimus (RAD001)

Novartis Pharmaceuticals

NULL

Terminated

6 Years

N/A

All

Phase 2

Israel

NCT01419639
(ClinicalTrials.gov)

October 2011

17/8/2011

Phase II Study of Everolimus (RAD001) in Children and Adults With Neurofibromatosis Type 2

Neurofibromatosis Type II

Drug: Everolimus (RAD001) , Afinitor®

New York University School of Medicine

Novartis Pharmaceuticals;The Children's Tumor Foundation

Completed

3 Years

N/A

All

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

NCT01412892
(ClinicalTrials.gov)

April 2011

8/8/2011

Use of RAD001 as Monotherapy in the Treatment of Neurofibromatosis 1 Related Internal Plexiform Neurofibromas

A Single Arm, Multicenter Phase II a Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis 1 Related Internal Plexiform Neurofibromas That Cannot be Removed by Surgery

Neurofibromatosis Type 1;Plexiform Neurofibroma;Neurofibromatoses

Drug: RAD001: Everolimus

Assistance Publique - Hôpitaux de Paris

Novartis

Completed

18 Years

60 Years

Both

Phase 2

France

EUCTR2016-001563-36-Outside-EU/EEA
(EUCTR)

12/05/2016

Efficacy and Safety of RAD001 in Treating Plexiform Neurofibromas (PN) Associated With Neurofibromatosis (NF1)

A Phase II Study of RAD001 in the Treatment of Patients With Plexiform Neurofibromas (PN) Associated With Neurofibromatosis Type 1 (NF1)

Plexiform Neurofibroma Associated With Neurofibromatosis Type 1;Therapeutic area: Diseases [C] - Cancer [C04]

Trade Name: Votubia
Product Name: Everolimus
Product Code: RAD001

Novartis Pharmaceuticals

NULL

Female: yes
Male: yes

Phase 4

Israel