Phase 2 N=13 Treatment

Hematopoietic Stem Cell Transplant in Devic's Disease

Devic's Disease

Bottom Line

View on ClinicalTrials.gov: NCT00787722 ↗

Enrolled (actual)

Serious AEs

7.7%

Results posted

Feb 2020

Primary outcome: Primary: Survival — 13; 12; 12; 11 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Hematopoietic Stem Cell Transplantation (Procedure); Cyclophosphamide (Drug); G-CSF (Drug); rATG (Drug); Mesna (Drug); Rituximab (Drug); Methylprednisolone (Drug)
Age: Pediatric, Adult, Older Adult · 16+ yrs
Sex: All
Sponsor: Northwestern University
Primary completion: Nov 2018

Outcome Measures

Outcome	Result	p-value
PRIMARY Survival	13; 12; 12; 11; 11; 11	—
SECONDARY Quality of Life (QOL) Short Form - 36 (SF-36)	30.83; 52.69; 61.63	—
SECONDARY Post HSCT Immune -Modulating Medication and Relapse	12; 1; 1; 3; 0; 1	—
SECONDARY Number of Patients Who Require No Device Assistance for Ambulation	6; 9; 10; 9; 8; 8	—
SECONDARY Disability Score: Expanded Disability Status Scale (EDSS)	4.4; 2.8; 3.3	—
SECONDARY NMO-IgG Aquaporin- 4 Autoantibody Titer	11; 2	—

Summary

This study is designed to examine whether treating Devic's disease patients with high dose cyclophosphamide together with rabbit antithymocyte globulin (rATG)/rituximab (drugs which reduce the function of the immune system), followed by return of previously collected patient's stem cells will result in improvement in Devic's disease. Stem cells are undeveloped cells that have the capacity to grow into mature blood cells, which normally circulate in the blood stream. The purpose of the intense chemotherapy is to destroy the cells in patient's immune system, which may be causing his/her disease. The purpose of the stem cell infusion is to produce a normal immune system that will no longer attack patient's body. The purpose of study is to examine the safety and efficacy of this treatment. The drugs used in this study treatment are drugs for commonly used for immune suppression.

Eligibility Criteria

Inclusion Criteria

Age between 16-65, at the time of pretransplant evaluation
An established diagnosis of Devic's disease (more than one acute attack)
NMO- IgG aquaporin-4 autoantibody positive

Exclusion Criteria

Paraplegia or quadriplegia and legal blindness (defined as visual acuity of 20/200 or less in the better eye with the best correction possible)
Any illness that in the opinion of the investigators would jeopardize the ability of the patient to tolerate aggressive chemotherapy
Prior history of malignancy except localized basal cell, squamous skin cancer or carcinoma in situ of the cervix. Other malignancies for which the patient is judged to be cured, such as head and neck cancer, or breast cancer will be considered on an individual basis
Positive pregnancy test
Inability or unwillingness to pursue effective means of birth control. Effective birth control is defined as 1) refraining from all acts of vaginal intercourse (ABSTINENCE); 2) consistent use of birth control pills; 3) injectable birth control methods (Depo-provera, Norplant); 4) tubal sterilization or male partner who has undergone vasectomy; 5) placement of an intrauterine device (IUD); or 6) use, with every act of intercourse, of diaphragm with contraceptive jelly and/or condoms with contraceptive foam
Failure to willingly accept or comprehend irreversible sterility as a side effect of therapy
forced expiratory volume at one (FEV1) / forced vital capacity (FVC) 2.0 mg/dl
Known hypersensitivity to mouse, rabbit, or E. Coli derived proteins
Presence of metallic objects implanted in the body that would preclude the ability of the patient to safely have MRI exams
Bilirubin > 2.0 mg/dl
Platelet count 3x of normal limits, liver cirrhosis

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00787722). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.