Juvenile Neuronal Ceroid Lipofuscinosis Clinical Trial
— JUMPOfficial title:
Phase II, Randomized, Placebo Controlled Trial of the Safety and Tolerability of Mycophenolate in Children With Juvenile Neuronal Ceroid Lipofuscinosis
NCT number | NCT01399047 |
Other study ID # | 3908 |
Secondary ID | |
Status | Completed |
Phase | Phase 2 |
First received | |
Last updated | |
Start date | July 2011 |
Est. completion date | November 2015 |
Verified date | May 2019 |
Source | University of Rochester |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
The primary objective of this trial is to establish the safety and tolerability of short-term
(8 weeks) administration of mycophenolate mofetil in ambulatory children with JNCL. The
secondary objective is to gather preliminary evidence of the short-term (8 week) impact of
mycophenolate mofetil on clinically relevant features of JNCL as measured by the Unified
Batten Disease Rating Scale (UBDRS), including motor features, seizures, behavior, cognitive
and functional measures.
Funding source-FDA Office of Orphan Product Development (OOPD).
Status | Completed |
Enrollment | 19 |
Est. completion date | November 2015 |
Est. primary completion date | November 2015 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 6 Years to 25 Years |
Eligibility |
Inclusion Criteria: - JNCL as determined by a characteristic clinical presentation and confirmatory genetic evidence. - Able to walk 10 feet without assistance beyond that required due to vision impairment. - Subjects with local treating clinician (pediatrician or neurologist) willing to conduct the trial according to the protocol, good clinical practice, and applicable regulations. - Subjects with a parent/legal guardian willing to accompany them to all study visits, oversee study drug compliance, and monitor and report to local treating clinician/investigator and the URBC investigative personnel any signs of adversity. Exclusion Criteria: - Inability to tolerate oral administration of medications - Concomitant medical condition, which, in the opinion of the local treating clinician, the parent(s)/guardian, or the URBC study investigator would place the child at greater than acceptable risk from: 1) travel by plane or car to the URBC on four occasions over the course of 22 weeks, 2) exposure to mycophenolate mofetil at protocol defined dosages for periods up to 8 weeks. - Anticipated inability of the child (on the part of the investigator, parent/guardian, or URBC study personnel) to comply with the rigors of the protocol.. - Use of disallowed concomitant medications. - Administration of immunosuppressive medications - History of any prior exposure to mycophenolate mofetil - History of hypersensitivity to mycophenolate mofetil, or any other component of the product - History of frank gastrointestinal hemorrhage, ulceration, or melena - White blood cell count < 3000/µL, absolute neutrophil count (ANC) < 1500/µL, hemoglobin < 10g/dL, or thrombocytopenia <100,000/µL. - Abnormal liver function (aspartate aminotransferase (AST) or alanine aminotransferase (ALT) or bilirubin greater than 3 times the upper limit of normal) - Pregnancy or vulnerability to engage in sexual intercourse based on report of the parent/guardian, judgment of the local treating clinician/investigator or judgment of the URBC study personnel. - Positive Tuberculosis test - Immunizations not up to date for age according to Centers for Disease Control guidelines |
Country | Name | City | State |
---|---|---|---|
United States | University of Rochester | Rochester | New York |
Lead Sponsor | Collaborator |
---|---|
University of Rochester | Batten Disease Support and Research Assocation (BDSRA) |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Tolerability - Number of Participants Who Completed Each Arm on Assigned Study Drug Dose | The primary outcome measure is tolerability, defined as the completion of 8 weeks on the assigned dosage of study drug. | Baseline to 8 weeks | |
Secondary | Unified Batten Disease Rating Scale Physical Subscale Change | The Unified Batten Disease Rating Scale (UBDRS) is a disease-specific clinical assessment. Motor impairment was measured by the physical subscale of the UBDRS with a range of 0-112 with zero indicating better outcome. The overall treatment effect was determined - mean difference in physical subscale change (Mycophenolate minus placebo periods) in outcome, adjusted for baseline value and period effects. |
Baseline to 8 weeks | |
Secondary | Unified Batten Disease Rating Scale Seizure Subscale Change | The Unified Batten Disease Rating Scale (UBDRS) is a disease-specific clinical assessment. Seizure severity was measured by the seizure subscale of the UBDRS with a range of 0-54 with zero indicating better outcome. The overall treatment effect was determined - mean difference in seizure subscale change (Mycophenolate minus placebo periods) in outcome, adjusted for baseline value and period effects. |
Baseline to 8 weeks | |
Secondary | Unified Batten Disease Rating Scale Behavior Subscale Change | The Unified Batten Disease Rating Scale (UBDRS) is a disease-specific clinical assessment. Mood and behavior severity was measured by the behavior subscale of the UBDRS with a range of 0-55 with zero indicating better outcome. The overall treatment effect was determined - mean difference in behavior subscale change (Mycophenolate minus placebo periods) in outcome, adjusted for baseline value and period effects. |
Baseline to 8 weeks | |
Secondary | Unified Batten Disease Rating Scale Capability Subscale Change | The Unified Batten Disease Rating Scale (UBDRS) is a disease-specific clinical assessment. Capability severity was measured by the capability subscale of the UBDRS with a range of 0-14 with higher scores indicating better outcome. The overall treatment effect was determined - mean difference in capability subscale change (Mycophenolate minus placebo periods) in outcome, adjusted for baseline value and period effects. |
Baseline to 8 weeks |
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