Trial · NCT02485899
An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease
NCT02485899, Phase 1, Phase 2, Completed, BioMarin Pharmaceutical; results first posted 2022-08-24
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease. Trials, record NCT02485899. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02485899
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease
- NCT number
- NCT02485899
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 23 participants, actual
- Lead sponsor
- BioMarin Pharmaceutical
- Conditions
- Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease, CLN2 Disorder
- Interventions
- BMN 190, Intracerebroventricular (ICV) access device
- First posted
- 2015-06-30
- Last updated
- 2022-08-24
- Results first posted
- 2022-08-24
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Probability of Unreversed 2-point Decline in Motor-language (ML) Score or Score of 0 | Up to Week 289 | Motor and Language are each 0-3 point subscales in which 3 represents best function and 0 represents loss of function. Thus, the 0-6 point ML score was used as the primary mode of evaluation of loss of function. In 190-201, the primary endpoint was a responder endpoint: unreversed ML 2-point decline or score of 0 at Week 48 compared to the 50% rate expected in natural history using the 1-sample binomial test. For 190-202, the primary endpoint was revised to assess response over the full duration of follow-up. It is not clear, given the variable follow-up much greater than 48 weeks, what response rate is expected in natural history. For this reason, the assessment of the primary endpoint is based on comparing to natural history data and using the Kaplan-Meier method and Cox proportional hazards model with covariate adjustment (i.e., baseline ML score and age as continuous covariates, and genotype \[common alleles\] and sex as categorical covariates). |
| Primary | Probability of Unreversed Motor-language (ML) Score of Zero. | Up to Week 289 | Motor and Language are each 0-3 point subscales in which 3 represents best function and 0 represents loss of function. Thus, the 0-6 point ML score was used as the primary mode of evaluation of loss of function. In 190-201, the primary endpoint was a responder endpoint: unreversed ML 2-point decline or score of 0 at Week 48 compared to the 50% rate expected in natural history using the 1-sample binomial test. For 190-202, the primary endpoint was revised to assess response over the full duration of follow-up. It is not clear, given the variable follow-up much greater than 48 weeks, what response rate is expected in natural history. For this reason, the assessment of the primary endpoint is based on comparing to natural history data and using the Kaplan-Meier method and Cox proportional hazards model with covariate adjustment (baseline ML score, age, genotype \[common alleles\], and sex). |
| Secondary | Whole Brain Volume | Baseline (Week 1 of BMN 190-201) to Week 289 / Last observation | Percentage change from Baseline to Last Observation: Whole Brain volume |
| Secondary | Volume of Cerebrospinal Fluid | Baseline (Week 1 of BMN 190-201) to Week 289 / Last observation | Percentage Change from Baseline to last observation: Volume of cerebrospinal fluid |
| Secondary | Volume of Total Cortical Gray Matter | Baseline (Week 1 of BMN 190-201) to Week 289 / Last observation | Percentage Change from Baseline to last observation: Volume of total cortical gray matter |
| Secondary | Total White Matter Volume | Baseline (Week 1 of BMN 190-201) to Week 289 / Last observation | Percentage Change from Baseline to last observation: Total white matter volume |
| Secondary | Whole Brain Apparent Diffusion Coefficient | Baseline (Week 1 of BMN 190-201) to Week 289 / Last observation | Change from Baseline to last observation Whole brain apparent diffusion coefficient |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Probability of Unreversed 2-point Decline in Motor-language (ML) Score or Score of 0 | Up to Week 289 | BMN 190-202: 24 subjects were enrolled in 190-201 at 5 clinic sites worldwide. One subject terminated from 190-201 after a single infusion and is not included in the Intent-to-Treat (ITT) population for 190-202. Study 190-202 final efficacy results include pooled data from the complete dataset from Study 190-201 \& Study 190-202 for all subjects who received \>1 dose (N=23).~NH: 42 subjects from DEM-CHILD database satisfying the criteria listed in the Arm/Group description below. | Probability of decline | NUMBER |
| PRIMARY | Probability of Unreversed Motor-language (ML) Score of Zero. | Up to Week 289 | BMN 190-202: 24 subjects were enrolled in 190-201 at 5 clinic sites worldwide. One subject terminated from 190-201 after a single infusion and is not included in the Intent-to-Treat (ITT) population for 190-202. Study 190-202 final efficacy results include pooled data from the complete dataset from Study 190-201 \& Study 190-202 for all subjects who received \>1 dose (N=23).~NH: 42 subjects from DEM-CHILD database satisfying the criteria listed in the Arm/Group description below. | Probability of decline | NUMBER |
| SECONDARY | Whole Brain Volume | Baseline (Week 1 of BMN 190-201) to Week 289 / Last observation | BMN 190-202: 24 subjects were enrolled in 190-201 at 5 clinic sites worldwide. One subject terminated from 190-201 after a single infusion and is not included in the Intent-to-Treat (ITT) population for 190-202. Study 190-202 final efficacy results include pooled data from the complete dataset from Study 190-201 \& Study 190-202 for all subjects who received \>1 dose (N=23). | percentage change | MEAN |
| SECONDARY | Volume of Cerebrospinal Fluid | Baseline (Week 1 of BMN 190-201) to Week 289 / Last observation | BMN 190-202: 24 subjects were enrolled in 190-201 at 5 clinic sites worldwide. One subject terminated from 190-201 after a single infusion and is not included in the Intent-to-Treat (ITT) population for 190-202. Study 190-202 final efficacy results include pooled data from the complete dataset from Study 190-201 \& Study 190-202 for all subjects who received \>1 dose (N=23). | percentage change | MEAN |
| SECONDARY | Volume of Total Cortical Gray Matter | Baseline (Week 1 of BMN 190-201) to Week 289 / Last observation | BMN 190-202: 24 subjects were enrolled in 190-201 at 5 clinic sites worldwide. One subject terminated from 190-201 after a single infusion and is not included in the Intent-to-Treat (ITT) population for 190-202. Study 190-202 final efficacy results include pooled data from the complete dataset from Study 190-201 \& Study 190-202 for all subjects who received \>1 dose (N=23). | percentage change | MEAN |
| SECONDARY | Total White Matter Volume | Baseline (Week 1 of BMN 190-201) to Week 289 / Last observation | BMN 190-202: 24 subjects were enrolled in 190-201 at 5 clinic sites worldwide. One subject terminated from 190-201 after a single infusion and is not included in the Intent-to-Treat (ITT) population for 190-202. Study 190-202 final efficacy results include pooled data from the complete dataset from Study 190-201 \& Study 190-202 for all subjects who received \>1 dose (N=23). | percentage change | MEAN |
| SECONDARY | Whole Brain Apparent Diffusion Coefficient | Baseline (Week 1 of BMN 190-201) to Week 289 / Last observation | BMN 190-202: 24 subjects were enrolled in 190-201 at 5 clinic sites worldwide. One subject terminated from 190-201 after a single infusion and is not included in the Intent-to-Treat (ITT) population for 190-202. Study 190-202 final efficacy results include pooled data from the complete dataset from Study 190-201 \& Study 190-202 for all subjects who received \>1 dose (N=23). | mm^2/s | MEAN |
Source ClinicalTrials.gov
Results by group29
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Probability of Unreversed 2-point Decline in Motor-language (ML) Score or Score of 0 | BMN 190-202 (300 mg) | Probability of decline: Week 49 | 0.13 | 95% Confidence Interval 0.04 to 0.35 | Probability of decline |
| Probability of Unreversed 2-point Decline in Motor-language (ML) Score or Score of 0 | Natural History Comparator | Probability of decline: Week 49 | 0.48 | 95% Confidence Interval 0.34 to 0.64 | Probability of decline |
| Probability of Unreversed 2-point Decline in Motor-language (ML) Score or Score of 0 | BMN 190-202 (300 mg) | Probability of decline: Week 97 | 0.22 | 95% Confidence Interval 0.10 to 0.45 | Probability of decline |
| Probability of Unreversed 2-point Decline in Motor-language (ML) Score or Score of 0 | Natural History Comparator | Probability of decline: Week 97 | 0.91 | 95% Confidence Interval 0.79 to 0.98 | Probability of decline |
| Probability of Unreversed 2-point Decline in Motor-language (ML) Score or Score of 0 | BMN 190-202 (300 mg) | Probability of decline: Week 145 | 0.22 | 95% Confidence Interval 0.10 to 0.45 | Probability of decline |
| Probability of Unreversed 2-point Decline in Motor-language (ML) Score or Score of 0 | Natural History Comparator | Probability of decline: Week 145 | 0.97 | 95% Confidence Interval 0.87 to 1.00 | Probability of decline |
| Probability of Unreversed 2-point Decline in Motor-language (ML) Score or Score of 0 | BMN 190-202 (300 mg) | Probability of decline: Week 193 | 0.26 | 95% Confidence Interval 0.13 to 0.50 | Probability of decline |
| Probability of Unreversed 2-point Decline in Motor-language (ML) Score or Score of 0 | Natural History Comparator | Probability of decline: Week 193 | 0.97 | 95% Confidence Interval 0.87 to 1.00 | Probability of decline |
| Probability of Unreversed 2-point Decline in Motor-language (ML) Score or Score of 0 | BMN 190-202 (300 mg) | Probability of decline: Week 241 | 0.40 | 95% Confidence Interval 0.23 to 0.63 | Probability of decline |
| Probability of Unreversed 2-point Decline in Motor-language (ML) Score or Score of 0 | Natural History Comparator | Probability of decline: Week 241 | 1.00 | 95% Confidence Interval 0.87 to 1.00 | Probability of decline |
1 to 10 of 29Source ClinicalTrials.gov
See all 29 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | 190-202 (300 mg) | 24 | |
| Overall Study | STARTED | Natural History Comparator | 42 | |
| Overall Study | COMPLETED | 190-202 (300 mg) | 17 | |
| Overall Study | COMPLETED | Natural History Comparator | 42 | |
| Overall Study | NOT COMPLETED | 190-202 (300 mg) | 7 | |
| Overall Study | NOT COMPLETED | Natural History Comparator | 0 |
Source ClinicalTrials.gov
Adverse events178
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Hypotension | Vascular disorders | BMN 190-202 (300 mg) | 3 | 24 | 3 |
| other | Haematoma | Vascular disorders | BMN 190-202 (300 mg) | 2 | 24 | 2 |
| other | Tooth extraction | Surgical and medical procedures | BMN 190-202 (300 mg) | 3 | 24 | 3 |
| other | Skin papilloma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | BMN 190-202 (300 mg) | 2 | 24 | 2 |
| other | Hypersensitivity | Immune system disorders | BMN 190-202 (300 mg) | 10 | 24 | 16 |
| other | Seasonal allergy | Immune system disorders | BMN 190-202 (300 mg) | 3 | 24 | 4 |
| other | Pyrexia | General disorders | BMN 190-202 (300 mg) | 20 | 24 | 219 |
| other | Gait disturbance | General disorders | BMN 190-202 (300 mg) | 12 | 24 | 15 |
| other | Abasia | General disorders | BMN 190-202 (300 mg) | 3 | 24 | 3 |
| other | Complication associated with device | General disorders | BMN 190-202 (300 mg) | 3 | 24 | 3 |
1 to 10 of 178Source ClinicalTrials.gov
See all 178 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | BMN 190-202 (300 mg) | 0 | 24 |
| Total, other adverse events | BMN 190-202 (300 mg) | 24 | 24 |
| Total, serious adverse events | BMN 190-202 (300 mg) | 21 | 24 |
Source ClinicalTrials.gov
Papers4
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02485899
GET https://conductscience.org/api/trials/v1/NCT02485899/results?page=2