An Open-label Treatment Protocol for VTS-270 in Patients With Neurologic Manifestations of NPC



Status:Available
Conditions:Neurology
Therapuetic Areas:Neurology
Healthy:No
Age Range:4 - Any
Updated:3/1/2019
Contact:Christopher Hitchens
Email:clinicaltrials@mnk.com
Phone:800-556-3314

Use our guide to learn which trials are right for you!

An Open-label Expanded Access Treatment Protocol for VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Patients With Neurologic Manifestations of Niemann-Pick Type C Disease (NPC)

Prospective, open-label, treatment protocol to provide expanded access to VTS-270 to eligible
Niemann-Pick type C disease (NPC) patient

This is a prospective, open-label, treatment protocol to provide expanded access to VTS-270
to eligible Niemann-Pick type C disease (NPC) patients. Eligible patients will receive
VTS-270 via intrathecal (IT) lumbar puncture (LP) every 2 weeks, and assessments of safety
and exploratory efficacy measures will be performed at prescribed intervals. Patients who
have not previously been treated with VTS-270 will start at a dose of 900 mg, and patients
who have been previously receiving VTS-270 will receive their current stable dose of VTS-270.
Treatment will continue until the clinician considers VTS-270 to be no longer beneficial, the
patient withdraws from the treatment protocol, VTS-270 receives marketing authorization in
the US, or the development program is discontinued. Patients participating in this treatment
protocol should not expect additional access to VTS-270 beyond this treatment protocol.

Inclusion Criteria:

1. Patient is male or female and at least 4 years of age at time of screening.

2. Patient has a confirmed diagnosis of NPC.

3. Patient or parent/guardian must provide written informed consent to participate in the
treatment protocol. In addition to parental consent, assent to participate must also
be sought from minor children.

4. Patient has the ability to undergo LP and IT drug administration.

5. If taking miglustat (Zavesca®), must have been on a stable dose for the past 6 weeks
and be willing to remain on a stable dose for the duration of participation in the
treatment protocol. Alternatively, patients may elect to discontinue miglustat use and
be eligible for entry into the treatment protocol after undergoing a minimum 6-week
washout period prior to study Day 1.

6. If a patient has a history of seizures, the condition must be adequately controlled,
i.e., the pattern of seizure activity must be stable and the patient must be on a
stable dose and regimen of antiepileptic medication during the 1 month prior to
screening, with no change in dose or regimen up to and including the day of the first
dose of VTS-270 (Day 1).

7. Patients not currently receiving VTS-270 agree to discontinue any other
investigational treatment for at least 1 month prior to first dose of VTS-270 (Day 1).

8. Patients who are females of childbearing potential (not surgically sterile) must use a
medically acceptable method of contraception and must agree to continue use of this
method for the duration of the treatment protocol and for 30 days after participation
in the treatment protocol. Acceptable methods of contraception include barrier method
with spermicide, intrauterine device, steroidal contraceptive in conjunction with a
barrier method, abstinence, or same-sex partner.

9. Patient or parent/guardian must possess the ability, in the clinician's opinion, to
understand and comply with treatment protocol requirements, including clinical outcome
measurements and instructions, for the entire duration of the treatment protocol.

10. Caregiver, parent, guardian, or responsible adult must be able and willing to
accompany the patient to treatment protocol visits.

Exclusion Criteria:

1. Patient weighs less than 15 kg.

2. Patient has uncontrolled psychosis.

3. Patient has a history of hypersensitivity reactions to any product containing HP-β-CD
or has a history of hypersensitivity reactions or allergy to anesthesia/sedation.

4. Patient has received treatment with any investigational product (other than VTS-270)
within 1 month prior to Day 1 of treatment.

5. Patient is a female who is pregnant or nursing.

6. Patient has suspected infection of the CNS or any systemic infection.

7. Patient has a spinal deformity that, in the opinion of the clinician, could impact the
ability to perform repeated LPs.

8. Patient, in the opinion of the clinician, has a serious skin infection in the lumbar
region within 2 months of entry into the treatment protocol.

9. Patient has neutropenia, defined as an absolute neutrophil count of less than 1.0 ×
109/L.

10. Patient has thrombocytopenia (platelet count of less than 75 × 109/L).

11. Patient has an activated partial thromboplastin time (aPTT) or prothrombin time (PT)
prolonged by greater than 1.5 times the upper limit of normal (ULN) or a known history
of a bleeding disorder.

12. Patient has evidence of obstructive hydrocephalus or normal pressure hydrocephalus.

13. Patient has recently used anticoagulants (in past 2 weeks prior to first dose of
VTS-270 [Day 1].

14. Patient, in the opinion of the clinician, is unable to comply with the treatment
protocol or has a medical condition (e.g., cardiovascular, respiratory, hematologic,
neurologic, renal) that would potentially increase the risk of participation
We found this trial at
17
sites
Atlanta, Georgia 30318
Phone: 678-883-6897
?
mi
from
Atlanta, GA
Click here to add this to my saved trials
4900 Mueller Boulevard
Austin, Texas 78723
(512) 324-0000
Phone: 512-628-1840
Dell Children's Medical Center of Central Texas Welcome to Dell Children
?
mi
from
Austin, TX
Click here to add this to my saved trials
3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
 1-513-636-4200 
Phone: 513-636-4760
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
?
mi
from
Cincinnati, OH
Click here to add this to my saved trials
529 West Markham Street
Little Rock, Arkansas 72205
(501) 686-7000
Phone: 501-364-2966
University of Arkansas for Medical Sciences The University of Arkansas for Medical Sciences (UAMS) in...
?
mi
from
Little Rock, AR
Click here to add this to my saved trials
4650 Sunset Blvd
Los Angeles, California 90027
 (323) 660-2450
Phone: 323-361-2471
Childrens Hospital Los Angeles Children's Hospital Los Angeles is a 501(c)(3) nonprofit hospital for pediatric...
?
mi
from
Los Angeles, CA
Click here to add this to my saved trials
1211 Medical Center Dr
Nashville, Tennessee 37232
(615) 322-5000
Phone: 615-322-7601
Vanderbilt Univ Med Ctr Vanderbilt University Medical Center (VUMC) is a comprehensive healthcare facility dedicated...
?
mi
from
Nashville, TN
Click here to add this to my saved trials
1201 W La Veta Ave
Orange, California 92868
(714) 997-3000
Children's Hospital of Orange County For more than 45 years, CHOC Children’s has been steadfastly...
?
mi
from
Orange, CA
Click here to add this to my saved trials
3181 Southwest Sam Jackson Park Road
Portland, Oregon 97239
503 494-8311
Phone: 503-494-7231
Oregon Health and Science University In 1887, the inaugural class of the University of Oregon...
?
mi
from
Portland, OR
Click here to add this to my saved trials
13123 E 16th Ave
Aurora, Colorado 80045
(720) 777-1234
Phone: 303-724-3632
Children's Hospital Colorado At Children's Hospital Colorado, we see more, treat more and heal more...
?
mi
from
Aurora, CO
Click here to add this to my saved trials
1653 W. Congress Parkway
Chicago, Illinois 60612
(312) 942-5000
Phone: 312-942-9841
Rush University Medical Center Rush University Medical Center encompasses a 664-bed hospital serving adults and...
?
mi
from
Chicago, IL
Click here to add this to my saved trials
Las Vegas, Nevada 89109
Phone: 702-732-1493
?
mi
from
Las Vegas, NV
Click here to add this to my saved trials
Loma Linda, California 92354
Phone: 909-558-5830
?
mi
from
Loma Linda, CA
Click here to add this to my saved trials
Nashville, Tennessee 37203
Phone: 615-342-7339
?
mi
from
Nashville, TN
Click here to add this to my saved trials
New York, New York 10016
Phone: 212-263-7744
?
mi
from
New York, NY
Click here to add this to my saved trials
Roanoke, Virginia 24014
Phone: 540-526-1265
?
mi
from
Roanoke, VA
Click here to add this to my saved trials
Saint Paul, Minnesota 55101
?
mi
from
Saint Paul, MN
Click here to add this to my saved trials
Washington, District of Columbia 20010
?
mi
from
Washington,
Click here to add this to my saved trials