Clinical Trial of Pirfenidone in Adult Patients With Neurofibromatosis 1

Status Completed

Clinical Trial Summary

The study is a phase II, open label trial of oral Pirfenidone in 24 adult patients with neurofibromatosis type 1.

Pirfenidone is a new, broad-spectrum anti-fibrotic drug, with proven in vitro and in vivo negative effects on fibroblast growth and collagen matrix synthesis. Human studies indicate promising therapeutic effects in arresting and reversing fibrosis in a variety of different conditions, where the excessive formation of fibrous tissue is a major pathogenic mechanism. Since the fibrous tissue is a significant component of neurofibroma, reduction of fibrosis could diminish tumor progression and lead to tumor shrinkage. Therefore, Pirfenidone is an excellent candidate for the treatment of plexiform neurofibromas and surgically unresectable tumors in patients with NF1.

Clinical Trial Description

Specific aims of this study are:

1. To evaluate efficacy of Pirfenidone in NF1 patients with disfiguring or disabling plexiform neurofibroma (PN) and spinal neurofibromas (SN)

2. To determine the acute, subacute and chronic toxicity of Pirfenidone in patients with NF1. ;

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT00754780
Study type	Interventional
Source	Mayo Clinic
Contact
Status	Completed
Phase	Phase 2
Start date	September 2000

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT00589784` - Phase II Trial of Sunitinib (SU011248) in Patients With Recurrent or Inoperable Meningioma	Phase 2
Recruiting	`NCT04463316` - GROWing Up With Rare GENEtic Syndromes
Completed	`NCT01673009` - Phase II Study of Gleevec/Imatinib Mesylate (STI-571, NCS 716051) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas	Phase 2
Recruiting	`NCT00598351` - Natural History Study of Patients With Neurofibromatosis Type 2
Completed	`NCT02298270` - Resiliency Training for Patients With Neurofibromatosis Via Videoconferencing With Skype	N/A
Terminated	`NCT01402817` - Study of Sutent®/Sunitinib (SU11248) in Subjects With NF-1 Plexiform Neurofibromas	Phase 2
Completed	`NCT02101736` - Cabozantinib for Plexiform Neurofibromas (PN) in Subjects With NF1 in Children and Adults	Phase 2
Completed	`NCT01633008` - Acceptance and Commitment Therapy for Adolescents and Young Adults With Neurofibromatosis and Chronic Pain	Early Phase 1
Terminated	`NCT04461886` - A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I	Phase 3
Completed	`NCT01275586` - Study of Tasigna®/Nilotinib (AMN107) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas	Early Phase 1
Completed	`NCT02435628` - Relationship Between Psychosocial Factors, Health Literacy, Quality of Life and Satisfaction With Medical Visits in Adults With NF	N/A
Active, not recruiting	`NCT01140360` - Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform Neurofibromas	Phase 1/Phase 2
Completed	`NCT02211768` - Transformation of Plexiform Neurofibromas to Malignant Peripheral Nerve Sheath Tumors in Neurofibromatosis Type 1	Phase 1
Completed	`NCT00508235` - Quality of Friendships in Children With Neurofibromatosis	N/A
Completed	`NCT01639950` - Validating Pain Scales in Children and Young Adults