Clinical Trial Results:
Open label clinical study to evaluate the safety and efficacy of ProvayBlueTM (methylene blue injection USP) for the treatment of acquired methemoglobinemia
Summary
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EudraCT number |
2017-000290-37 |
Trial protocol |
FR GB |
Global end of trial date |
09 Dec 2021
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Results information
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Results version number |
v1(current) |
This version publication date |
21 Jul 2022
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First version publication date |
21 Jul 2022
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Other versions |
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
PVP-2016003
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT03395223 | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
Provepharm SAS
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Sponsor organisation address |
22 rue Marc Donadille, Marseille, France, 13013
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Public contact |
Clinical Trials Information, Provepharm SAS, +33 491086930,
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Scientific contact |
Clinical Trials Information, Provepharm SAS, +33 491086930,
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
09 Dec 2021
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Is this the analysis of the primary completion data? |
No
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Global end of trial reached? |
Yes
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Global end of trial date |
09 Dec 2021
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
The primary objective was to confirm that ProvayBlue is efficacious in patients with acquired methemoglobinemia.
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Protection of trial subjects |
NA
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
19 Oct 2017
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
France: 6
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Country: Number of subjects enrolled |
United States: 25
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Worldwide total number of subjects |
31
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EEA total number of subjects |
6
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
29
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From 65 to 84 years |
2
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85 years and over |
0
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Recruitment
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Recruitment details |
Pediatric or adult patients (males and females of all ages) diagnosed with acquired methemoglobinemia and receiving treatment with ProvayBlue per the treating physician’s diagnosis and hospital/urgent care/acute care facility’s standard care. Acquired methemoglobinemia was defined as a level of metHb >30% or ≤30% with associated clinical symptoms. | ||||||
Pre-assignment
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Screening details |
Subjects who presented in a hospital/urgent care setting with acquired methemoglobinemia, defined as a metHb level of 30% or higher and/or symptoms referable to reduced oxygen-carrying capacity and resulting respiratory distress, alteration in mental status, sleepiness, cyanosis, dizziness, etc. | ||||||
Pre-assignment period milestones
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Number of subjects started |
31 | ||||||
Number of subjects completed |
31 | ||||||
Period 1
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Period 1 title |
Overall period
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Is this the baseline period? |
Yes | ||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||
Arms
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Arm title
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Overall Period | ||||||
Arm description |
Pediatric or adult patients (males and females of all ages) diagnosed with acquired methemoglobinemia and receiving treatment with ProvayBlue per the treating physician’s diagnosis and hospital/urgent care/acute care facility’s standard care. Acquired methemoglobinemia was defined as a level of metHb >30% or ≤30% with associated clinical symptoms. | ||||||
Arm type |
Experimental | ||||||
Investigational medicinal product name |
ProvayBlue (Methylthioninium Chloride Proveblue)
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Solution for injection
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Routes of administration |
Intravenous use
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Dosage and administration details |
Subjects were administered ProvayBlue IV over 5 to 30 minutes, usually at a dose of 1 mg/kg, and, if the metHb level remained above 30% or clinical symptoms persisted, a repeat dose of up to 1 mg/kg was to be administered 1 hour after the first dose
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Baseline characteristics reporting groups
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Reporting group title |
Overall period
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Reporting group description |
- | |||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Overall Period
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Reporting group description |
Pediatric or adult patients (males and females of all ages) diagnosed with acquired methemoglobinemia and receiving treatment with ProvayBlue per the treating physician’s diagnosis and hospital/urgent care/acute care facility’s standard care. Acquired methemoglobinemia was defined as a level of metHb >30% or ≤30% with associated clinical symptoms. |
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End point title |
Efficacy endpoints: changes from baseline in metHb levels [1] | ||||||
End point description |
The primary efficacy endpoint was the number (%) of subjects with ≥50% reduction in metHb based on the first available posttreatment assessment when the first posttreatment measurement was taken within 2 hours of completion of first infusion.
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End point type |
Primary
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End point timeframe |
Before and after ProvayBlue infusion
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Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: The results are presented as a series of individual patient narratives with limited statistical analysis, befitting the observational nature of most of the data. Test Hypothesis and P Value Justification is not applicable since no inferential statistics are provided for this study. |
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Notes [2] - 9 subjects had MethHb level assessed withinb 2 hours of the end of the first ProvayBlue treatment |
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No statistical analyses for this end point |
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Adverse events information
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Timeframe for reporting adverse events |
Prevalence and nature of TEAEs, defined as all adverse events (AEs) with an onset date after the first dose of ProvayBlue infusion and within 10 days of the last dose of ProvayBlue infusion
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
23
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Reporting groups
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Reporting group title |
Safety analysis set
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Reporting group description |
All 31 enrolled subjects received at least 1 ProvayBlue infusion and were included in the Safety Analysis Set. | ||||||||||||||||||||||||||||||||||||||||||||||||
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Frequency threshold for reporting non-serious adverse events: 5% | |||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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Were there any global substantial amendments to the protocol? No | |||||||
Interruptions (globally) |
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Were there any global interruptions to the trial? Yes | |||||||
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Limitations and caveats |
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Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||||||
None reported |