NCT02963350

Brief Summary

  • To provide access to BMN 190 to patients with CLN2 disease who cannot participate in a clinical trial.
  • To collect additional information on the safety and tolerability of BMN 190 administration in patients with CLN2 disease.

Trial Health

60
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Geographic Reach
4 countries

5 active sites

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 9, 2016

Completed
6 days until next milestone

First Posted

Study publicly available on registry

November 15, 2016

Completed
Last Updated

July 24, 2017

Status Verified

July 1, 2017

First QC Date

November 9, 2016

Last Update Submit

July 21, 2017

Conditions

CLN2 Disease

Interventions

BMN190, recombinant human tripeptidyl peptidase-1 (rhTPP1)DRUG

Eligibility Criteria

Age2 Years+
Sexall
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Diagnosed with CLN2 disease as confirmed by deficient TPP1 enzyme activity in leukocytes or molecular analysis by identifying 2 known pathogenic mutations. If enzyme analysis is performed by dried blood spot, diagnosis must be confirmed with molecular testing
  • Age ≥2 old at the time of informed consent
  • Is willing and able to provide written, signed informed consent. Or, in the case of patients under the age of 18 (or other age as defined by regional law or regulation), provide written assent (if required) and have written informed consent, signed by a legally authorized representative, after the nature of the program has been explained, and prior to any program assessments.
  • If sexually active, must be willing to use 2 forms of acceptable methods of contraception while participating in the program.
  • If female of childbearing potential, must have a negative pregnancy test at Baseline and be willing to have additional pregnancy tests during the program.
  • Willing and able to comply with all program procedures.

You may not qualify if:

  • Another inherited neurologic disease, e.g., other forms of CLN or seizures unrelated to TPPI deficiency/CLN2 disease (patients with febrile seizures may be eligible).
  • Has received stem cell, gene therapy, or ERT for CLN2 disease.
  • Contraindications for neurosurgery (e.g., congenital heart disease, severe respiratory impairment, or clotting abnormalities).
  • Contraindications for MRI scans (e.g., cardiac pacemaker, metal fragment or chip in the eye, aneurysm clip in the brain).
  • Episode of generalized motor status epilepticus within 4 weeks before the first infusion.
  • Presence of ventricular abnormality (hydrocephalus, malformation).
  • Presence of ventricular shunt.
  • Has known hypersensitivity to any of the components of BMN 190.
  • Currently enrolled or previously enrolled in a clinical study with BMN 190.
  • Use of any investigational product or investigational medical device within 30 days prior to Baseline, or requirement for any investigational agent prior to completion of all scheduled program assessments.
  • Have travel plans that may interfere with dosing regimen, scheduled program visits and safety monitoring.
  • Has a medical condition or extenuating circumstance that, in the opinion of the physician, might compromise the patient's ability to comply with the protocol required testing or procedures or compromise the patient's wellbeing, safety, or clinical interpretability.
  • Pregnancy any time during the program; a female patient judged by the physician to be of childbearing potential will be tested for pregnancy.
  • A CLN2 combined motor/language score of less than 1 (apply to US only)
  • Asymptomatic (symptomatic is defined as having any evidence of neurological involvement attributed to CLN2 disease irrespective of the CLN2 score, including clinical signs and symptoms of disease such as seizures, ataxia, language delay or other developmental delays) (apply to US only)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

Unknown Facility

Orange, California, United States

Location

Unknown Facility

Columbus, Ohio, United States

Location

Unknown Facility

Hamburg, Germany

Location

Unknown Facility

Rome, Italy

Location

Unknown Facility

London, United Kingdom

Location

Study Officials

  • Jessica Cohen Pfeffer, MD

    Medical Monitor

    STUDY DIRECTOR

Study Design

Study Type
expanded access
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 9, 2016

First Posted

November 15, 2016

Last Updated

July 24, 2017

Record last verified: 2017-07

Locations

IT(1)US(2)DE(1)GB(1)