ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 14 of 25 for:    Child | "congenital neuronal ceroid lipofuscinosis" OR "Neuronal Ceroid-Lipofuscinoses"

A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01907087
Recruitment Status : Completed
First Posted : July 24, 2013
Results First Posted : June 11, 2018
Last Update Posted : June 11, 2018
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions: Jansky-Bielschowsky Disease
Batten Disease
Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
CLN2 Disease
Intervention: Biological: BMN 190

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
300 mg BMN 190

Intracerebroventricular (ICV) infusion every two weeks. This is a study of a single cohort followed for at least 48 weeks at dosing of 300 mg.

Subjects 1, 2, and 3 were initially assigned to the 30 mg dose, then escalated to 100 mg (and subsequently 300 mg) after data review. Subjects 4, 5, and 6 were initially assigned to the 100 mg dose, then escalated to 300 mg after data review. Subjects 7, 8 and 9 were initially assigned to the 300 mg dose. One patient withdrew after one dose due to unwillingness to comply with study procedures, requiring the addition of a 10th patient to this group. DMC approved full recruitment at the 300 mg dose (n=24) after data review.


Participant Flow:   Overall Study
    300 mg BMN 190
STARTED   24 
COMPLETED   23 
NOT COMPLETED   1 
Withdrawal by Subject                1 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
300 mg BMN 190 Intracerebroventricular (ICV) infusion every two weeks.

Baseline Measures
   300 mg BMN 190 
Overall Participants Analyzed 
[Units: Participants]
 24 
Age 
[Units: Years]
Mean (Standard Deviation)
 4.3  (1.24) 
Age, Customized 
[Units: Participants]
Count of Participants
 
<=5 years   20 
>5 years   4 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      15  62.5% 
Male      9  37.5% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
 
Hispanic or Latino      1   4.2% 
Not Hispanic or Latino      23  95.8% 
Unknown or Not Reported      0   0.0% 
Race/Ethnicity, Customized 
[Units: Participants]
Count of Participants
 
American Indian or Alaska Native   0 
Asian   1 
Native Hawaiian or Other Pacific Islander   0 
Black or African American   0 
White   23 
Other   0 


  Outcome Measures

1.  Primary:   Motor-Language (ML) Scale Score During 300 mg Dosing Period   [ Time Frame: Baseline, Week 49/Last Assessment ]

2.  Secondary:   Percentage Change From Baseline of Magnetic Resonance Imaging (MRI) at Week 49 During 300 mg Dosing Period: Whole Brain Volume   [ Time Frame: Baseline, Week 49 ]

3.  Secondary:   Percentage Change From Baseline of Magnetic Resonance Imaging (MRI) at Week 49 During 300 mg Dosing Period: Volume of Total Grey Matter   [ Time Frame: Baseline, Week 49 ]

4.  Secondary:   Percentage Change From Baseline of Magnetic Resonance Imaging (MRI) at Week 49 During 300 mg Dosing Period: Total White Matter Volume   [ Time Frame: Baseline, Week 49 ]

5.  Secondary:   Percentage Change From Baseline of Magnetic Resonance Imaging (MRI) at Week 49 During 300 mg Dosing Period: Volume of Cerebrospinal Fluid   [ Time Frame: Baseline, Week 49 ]

6.  Secondary:   Percentage Change From Baseline of Magnetic Resonance Imaging (MRI) at Week 49 During 300 mg Dosing Period: Whole Brain Apparent Diffusion Coefficient   [ Time Frame: Baseline, Week 49 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
10 subjects were assigned in 1 of 3 cohorts in Dose Escalation Period for a 4-22 weeks treatment of 30, 100, and/or 300 mg. 9 subjects completing the Dose Escalation Period, plus 14 new subjects, were administered a stable dose of 300mg for 48 weeks.


  More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Peter Slasor/Sr Director, Biostatistics, Global Clinical Sciences
Organization: BioMarin Pharmaceutical Inc.
phone: 415-506-6765
e-mail: PSlasor@bmrn.com


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT01907087     History of Changes
Other Study ID Numbers: 190-201
First Submitted: July 19, 2013
First Posted: July 24, 2013
Results First Submitted: February 15, 2018
Results First Posted: June 11, 2018
Last Update Posted: June 11, 2018