Trial · NCT01678898
Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients
NCT01678898, Phase 1, Phase 2, Completed, Protalix; results first posted 2020-01-27
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients. Trials, record NCT01678898. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01678898
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients
- NCT number
- NCT01678898
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 18 participants, actual
- Lead sponsor
- Protalix
- Conditions
- Fabry Disease
- Interventions
- PRX-102
- First posted
- 2012-09-05
- Last updated
- 2023-09-13
- Results first posted
- 2020-01-27
Source ClinicalTrials.gov
Registered outcomes24
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Adverse Events | 12 months | Reportings of adverse events reported by the patient and from monitoring with clinical laboratory, physical examination and ECG. Results represent the number of AEs that were considered possibly, probably, or definitely related to treatment. |
| Other | Plasma Gb3 Concentrations | Plasma Gb3 concentration (ug/mL) was measured at baseline and every 3 months up to 12 months. | Results are presented as mean percent change from baseline (visit 1) to 12 months +/- standard error. |
| Other | Kidney Function - Change in eGFR | eGFR is performed at baseline (day 1) weeks 4, 8, 12, 26, 38 and 52 (12 months) | Estimated glomerular filtration rate (eGFR) was calculated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation. Levels of eGFR calculated by the CKD-EPI equation, based on measured serum creatinine on day 1, weeks 4, 8, 12, 26, 38 and 52 are used to determine the annualized slope of eGFR per patient over a 12 months period. The absolute mean change from baseline (visit 1) to 12 months is then derived from the eGFR slope. |
| Other | Plasma Lyso-Gb3 Levels | Plasma Lyso-Gb3 concentration (ng/mL) was measured at baseline and every 3 months up to 12 months. | Results are presented as mean percent change from baseline (visit 1) to 12 months +/- standard error. |
| Other | Change in Kidney Gb3 Accumulation | Visit 1 (day 1) in PB-102-F01 study and after a total of 6 months of treatment (i.e., at 3 months into study PB-102-F02). | Kidney biopsy was performed at baseline of study PB-102-F01 and following 6 months treatment with PRX-102. Approximately 300 capillaries were scored in each specimen. A quantitative Barisoni Lipid Inclusion Scoring System (BLISS) was used for scoring Gb3 inclusions in kidney peritubular capillary (PTC) biopsy samples.The scoring system was implemented by 3 blinded pathologists/readers.The BLISS scoring methodology consists of counting the number of Gb-3 inclusions per capillary previously annotated.The final score of each biopsy was the average number of inclusions per capillary. A decrease in scoring from baseline to 6 Month is considered an indication for clinical improvement. |
| Other | Cardiac Fibrosis Per MRI | Cardiac MRI was performed in order to assess myocardial fibrosis at baseline, 6 months and 12 months visit. | Cardiac MRI was performed to estimate the percentage and mass of the myocardial fibrotic area. Results represent the number of subjects with fibrosis after 1 year of treatment. |
| Other | Cardiac MRI - Ejection Fraction | Cardiac MRI was performed 3 times: at baseline (visit 1), 6 months and 12 months. | Results are presented as mean percent change from baseline (visit 1) to 12 months. |
| Other | Cardiac MRI - LVM | Cardiac MRI was performed 3 times: at baseline (visit 1), 6 months and 12 months. | Results are presented as mean percent change from baseline (visit 1) to 12 months. |
| Other | Cardiac MRI - LVMI | Cardiac MRI was performed 3 times: at baseline (visit 1), 6 months and 12 months. | Cardiac MRI with computer-calculated left ventricular mass index were conducted at baseline, 6 month, and 12 months. Results are presented as mean percent change from baseline (visit 1) to 12 months. LVMI is calculated by dividing the left ventricular mass by body surface area. |
| Other | Pharmacokinetics - AUC | PK parameters were determined on Day 1 and 3, 6, and 12 months at these time points: pre-infusion (baseline); 1 h after the beginning of the infusion; at the end of the infusion; 1, 4, 8, 24, 48±3, 72±3, 96±3 h and 2 weeks ± 3 days post-infusion. | PK parameters were derived from the plasma concentration versus time profiles. Results reported represent the averaged values following a single dosing of the study drug. |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 registered outcomesOutcomes24
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Adverse Events | 12 months | adverse events | NUMBER | |
| OTHER_PRE_SPECIFIED | Plasma Gb3 Concentrations | Plasma Gb3 concentration (ug/mL) was measured at baseline and every 3 months up to 12 months. | mean percent change | MEAN | |
| OTHER_PRE_SPECIFIED | Kidney Function - Change in eGFR | eGFR is performed at baseline (day 1) weeks 4, 8, 12, 26, 38 and 52 (12 months) | mL/min/1.73 m2 | MEAN | |
| OTHER_PRE_SPECIFIED | Plasma Lyso-Gb3 Levels | Plasma Lyso-Gb3 concentration (ng/mL) was measured at baseline and every 3 months up to 12 months. | mean percent change | MEAN | |
| OTHER_PRE_SPECIFIED | Change in Kidney Gb3 Accumulation | Visit 1 (day 1) in PB-102-F01 study and after a total of 6 months of treatment (i.e., at 3 months into study PB-102-F02). | 3 patients were not included in the renal biopsies Gb3 analysis. 1 male patient's biopsies were mixed up by the lab; 1 female patient's baseline biopsy didn't include cortex tissue making sample unreadable; and 1 male patient carries a cardiac GLA variant (p.N215S) associated with rare renal manifestation and had a low BLISS baseline score | Percent change | MEAN |
| OTHER_PRE_SPECIFIED | Cardiac Fibrosis Per MRI | Cardiac MRI was performed in order to assess myocardial fibrosis at baseline, 6 months and 12 months visit. | number of subjects | NUMBER | |
| OTHER_PRE_SPECIFIED | Cardiac MRI - Ejection Fraction | Cardiac MRI was performed 3 times: at baseline (visit 1), 6 months and 12 months. | Percent Change | MEAN | |
| OTHER_PRE_SPECIFIED | Cardiac MRI - LVM | Cardiac MRI was performed 3 times: at baseline (visit 1), 6 months and 12 months. | Percent change | MEAN | |
| OTHER_PRE_SPECIFIED | Cardiac MRI - LVMI | Cardiac MRI was performed 3 times: at baseline (visit 1), 6 months and 12 months. | Percent Change | MEAN | |
| OTHER_PRE_SPECIFIED | Pharmacokinetics - AUC | PK parameters were determined on Day 1 and 3, 6, and 12 months at these time points: pre-infusion (baseline); 1 h after the beginning of the infusion; at the end of the infusion; 1, 4, 8, 24, 48±3, 72±3, 96±3 h and 2 weeks ± 3 days post-infusion. | ng*hr/ml | MEAN |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 outcomesResults by group73
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Adverse Events | Safety Group | 54 | adverse events | ||
| Adverse Events | 0.2 mg/kg | 11 | adverse events | ||
| Adverse Events | 1.0 mg/kg | 29 | adverse events | ||
| Adverse Events | 2.0 mg/kg | 14 | adverse events | ||
| Plasma Gb3 Concentrations | 0.2 mg/kg | -16.8 | Standard Error 8.6 | mean percent change | |
| Plasma Gb3 Concentrations | 1 mg/kg | -30.7 | Standard Error 11.2 | mean percent change | |
| Plasma Gb3 Concentrations | 2 mg/kg | -16.2 | Standard Error 12.7 | mean percent change | |
| Plasma Gb3 Concentrations | Efficacy Group | -22.2 | Standard Error 6.1 | mean percent change | |
| Plasma Gb3 Concentrations | Phenotypically Classic Fabry Patients | -33.3 | Standard Error 7.6 | mean percent change | |
| Kidney Function - Change in eGFR | Pegunigalsidase Alfa Reporting Group | -0.8 | Standard Error 1.9 | mL/min/1.73 m2 |
1 to 10 of 73Source ClinicalTrials.gov
See all 73 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| PB-102-F01 | STARTED | 0.2 mg/kg | 6 | |
| PB-102-F01 | STARTED | 1 mg/kg | 8 | |
| PB-102-F01 | STARTED | 2 mg/kg | 4 | |
| PB-102-F01 | COMPLETED | 0.2 mg/kg | 6 | |
| PB-102-F01 | COMPLETED | 1 mg/kg | 6 | |
| PB-102-F01 | COMPLETED | 2 mg/kg | 4 | |
| PB-102-F01 | NOT COMPLETED | 0.2 mg/kg | 0 | |
| PB-102-F01 | NOT COMPLETED | 1 mg/kg | 2 | |
| PB-102-F01 | NOT COMPLETED | 2 mg/kg | 0 | |
| PB-102-F02 | STARTED | 0.2 mg/kg | 6 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events99
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 | 0.2 mg/kg | 0 | 6 | 0 |
| other | Hypotension | Vascular disorders | 1 mg/kg | 1 | 8 | 3 |
| other | Hypotension | Vascular disorders | 2 mg/kg | 0 | 4 | 0 |
| other | Sneezing | Respiratory, thoracic and mediastinal disorders | 0.2 mg/kg | 1 | 6 | 1 |
| other | Sneezing | Respiratory, thoracic and mediastinal disorders | 1 mg/kg | 0 | 8 | 0 |
| other | Sneezing | Respiratory, thoracic and mediastinal disorders | 2 mg/kg | 0 | 4 | 0 |
| other | Dyspnoea | Respiratory, thoracic and mediastinal disorders | 0.2 mg/kg | 0 | 6 | 0 |
| other | Dyspnoea | Respiratory, thoracic and mediastinal disorders | 1 mg/kg | 1 | 8 | 1 |
| other | Dyspnoea | Respiratory, thoracic and mediastinal disorders | 2 mg/kg | 0 | 4 | 0 |
| other | Chest pain | Respiratory, thoracic and mediastinal disorders | 0.2 mg/kg | 0 | 6 | 0 |
1 to 10 of 99Source ClinicalTrials.gov
See all 99 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | 0.2 mg/kg | 0 | 6 |
| Total, all-cause mortality | 1 mg/kg | 0 | 8 |
| Total, all-cause mortality | 2 mg/kg | 0 | 4 |
| Total, other adverse events | 0.2 mg/kg | 2 | 6 |
| Total, other adverse events | 1 mg/kg | 7 | 8 |
| Total, other adverse events | 2 mg/kg | 2 | 4 |
| Total, serious adverse events | 0.2 mg/kg | 0 | 6 |
| Total, serious adverse events | 1 mg/kg | 1 | 8 |
| Total, serious adverse events | 2 mg/kg | 1 | 4 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 30834538 | RESULT |
Source PubMed
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 comparisons3
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Pegunigalsidase Alfa Reporting Group vs Phenotypically Classic Fabry Patients: Gastrointestinal Symptoms Questionnaire - Severity of Abdominal Pain | Trial comparisons |
| Pegunigalsidase Alfa Reporting Group vs Phenotypically Classic Fabry Patients: Gastrointestinal Symptoms Questionnaire - Frequency of Abdominal Pain | Trial comparisons |
| Pegunigalsidase Alfa Reporting Group vs Phenotypically Classic Fabry Patients: Gastrointestinal Symptoms Questionnaire - Frequency of Diarrhea | Trial comparisons |
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/NCT01678898
GET https://conductscience.org/api/trials/v1/NCT01678898/registered?page=2