Trial · NCT05890794
Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia
NCT05890794, Phase 1, Phase 2, Completed, AM-Pharma; results first posted 2025-02-27
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia. Trials, record NCT05890794. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05890794
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia
- NCT number
- NCT05890794
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 12 participants, actual
- Lead sponsor
- AM-Pharma
- Conditions
- Hypophosphatasia
- Interventions
- Ilofotase Alfa, 0.8 mg/kg, Ilofotase Alfa, 3.2 mg/kg
- First posted
- 2023-06-06
- Last updated
- 2025-02-27
- Results first posted
- 2025-02-27
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Maximum Percent Change From Baseline in Extracellular Inorganic Pyrophosphate (PPi) | Day 1 to Day 10 | Percent Change from BaseLine (PCBL) in PPi serum concentration is calculated for all post-dose PPi measures recorded from Day 1 to Day 10. Per patient, 12 measurements were done: 3 at Day 1 and 1 each day from Day 2 to Day 10. The Percent Change from BaseLine for measurement x (x=1,…12) is calculated: PCBL(x)= \[(PPi( x)-PPi(baseline))/PPi(baseline)\]\*100. The subject's maximum percent change from baseline is defined as the Max (PCBL(1), …, PCBL(12)). |
| Primary | Maximum Percent Change From Baseline in Pyridoxal 5'-Phosphate (PLP) | Day 1 to Day 10 | Percent Change from BaseLine (PCBL) in PLP serum concentration is calculated for all post-dose PLP measures recorded from Day 1 to Day 10. Per patient, 12 measurements were done: 3 at Day 1 and 1 each day from Day 2 to Day 10. The Percent Change from BaseLine for measurement x (x=1,…12) is calculated: PCBL(x)= \[(PLP( x)-PLP(baseline))/PLP(baseline)\]\*100. The subject's maximum percent change from baseline is defined as the Max (PCBL(1), …, PCBL(12)). |
| Other | Maximum Percent Change From Baseline in Alkaline Phosphatase (ALP) Activity | Day 1 to Day 10 | Percent Change from BaseLine (PCBL) in ALP Activity is calculated for all post-dose ALP measures recorded from Day 2 to Day 10. Per patient, 9 measurements were done: 1 each day from Day 2 to Day 10. The Percent Change from BaseLine for measurement x (x=1,…9) is calculated: PCBL(x)= (ALP(x)-ALP(baseline))/ALP(baseline)\*100, \[x=1,...,9\]. The subject's maximum percent change from baseline is defined as the Max (PCBL(1), …, PCBL(9)) |
| Other | Maximum Percent Change From Baseline in Urine Phosphoethanolamine (PEA) | Day 1 to Day 10 | Percent Change from BaseLine (PCBL) in PEA concentration is calculated for all post-dose PEA measures recorded from Day 2 to Day 10. Per patient, 9 measurements were done: 1 each day from Day 2 to Day 10. The Percent Change from BaseLine for measurement x (x=1,…9) is calculated: PCBL(x)= \[(PEA( x)-PEA(baseline))/PEA(baseline)\]\*100. The subject's maximum percent change from baseline is defined as the Max (PCBL(1), …, PCBL(9)). |
| Other | Maximum Percent Change From Baseline in Pyridoxal (PL). | Day 1 to Day 10 | Percent Change from BaseLine (PCBL) in PL serum concentration is calculated for all post-dose PL measures recorded from Day 1 to Day 10. Per patient, 12 measurements were done: 3 at Day 1 and 1 each day from Day 2 to Day 10. The Percent Change from BaseLine for measurement x (x=1,…12) is calculated: PCBL(x)= \[(PL( x)-PL(baseline))/PL(baseline)\]\*100. The subject's maximum percent change from baseline is defined as the Max (PCBL(1), …, PCBL(12)). |
| Other | Maximum Fold Change From Baseline in PL/PLP Ratio | Day 1 to Day 10 | Fold Change from BaseLine (FCBL) in PL/PLP ratio is calculated for all post-dose PL/PLP ratio measures recorded from Day 1 to Day 10. Per patient, 12 measurements were done: 3 at Day 1 and 1 each day from Day 2 to Day 10. The Fold Change from BaseLine for measurement x (x=1,…12) is calculated: FCBL(x)= (PL/PLPratio( x) / PL/PLPratio(baseline)). The subject's maximum fold change from baseline is defined as the Max (FCBL(1), …, FCBL(12)). |
| Other | Treatment-emergent Adverse Events (TEAEs) | Day 1 to Day 15 | Any untoward medical occurrence in a patient enrolled and treated in the clinical trial regardless of its causal relationship to the trial medication. |
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 | Maximum Percent Change From Baseline in Extracellular Inorganic Pyrophosphate (PPi) | Day 1 to Day 10 | All randomized patients. | percent change | MEAN |
| PRIMARY | Maximum Percent Change From Baseline in Pyridoxal 5'-Phosphate (PLP) | Day 1 to Day 10 | All randomized patients. | percent change | MEAN |
| OTHER_PRE_SPECIFIED | Maximum Percent Change From Baseline in Alkaline Phosphatase (ALP) Activity | Day 1 to Day 10 | All randomized patients. | percent change | MEAN |
| OTHER_PRE_SPECIFIED | Maximum Percent Change From Baseline in Urine Phosphoethanolamine (PEA) | Day 1 to Day 10 | All randomized patients. | percent change | MEAN |
| OTHER_PRE_SPECIFIED | Maximum Percent Change From Baseline in Pyridoxal (PL). | Day 1 to Day 10 | All randomized patients. | percent change | MEAN |
| OTHER_PRE_SPECIFIED | Maximum Fold Change From Baseline in PL/PLP Ratio | Day 1 to Day 10 | All randomized patients. | fold change | MEAN |
| OTHER_PRE_SPECIFIED | Treatment-emergent Adverse Events (TEAEs) | Day 1 to Day 15 | All patients who received trial medication were included in the population for the safety analysis. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group21
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Maximum Percent Change From Baseline in Extracellular Inorganic Pyrophosphate (PPi) | 0.8 mg/kg Ilofotase Alfa | -36.46 | Standard Deviation 11.502 | percent change | |
| Maximum Percent Change From Baseline in Extracellular Inorganic Pyrophosphate (PPi) | 3.2 mg/kg Ilofotase Alfa | -77.31 | Standard Deviation 16.113 | percent change | |
| Maximum Percent Change From Baseline in Extracellular Inorganic Pyrophosphate (PPi) | Overall | -56.88 | Standard Deviation 25.165 | percent change | |
| Maximum Percent Change From Baseline in Pyridoxal 5'-Phosphate (PLP) | 0.8 mg/kg Ilofotase Alfa | -34.68 | Standard Deviation 8.898 | percent change | |
| Maximum Percent Change From Baseline in Pyridoxal 5'-Phosphate (PLP) | 3.2 mg/kg Ilofotase Alfa | -65.56 | Standard Deviation 6.559 | percent change | |
| Maximum Percent Change From Baseline in Pyridoxal 5'-Phosphate (PLP) | Overall | -50.12 | Standard Deviation 17.768 | percent change | |
| Maximum Percent Change From Baseline in Alkaline Phosphatase (ALP) Activity | 0.8 mg/kg Ilofotase Alfa | 2685.57 | Standard Deviation 1492.991 | percent change | |
| Maximum Percent Change From Baseline in Alkaline Phosphatase (ALP) Activity | 3.2 mg/kg Ilofotase Alfa | 7021.23 | Standard Deviation 2063.878 | percent change | |
| Maximum Percent Change From Baseline in Alkaline Phosphatase (ALP) Activity | Overall | 4853.40 | Standard Deviation 2841.847 | percent change | |
| Maximum Percent Change From Baseline in Urine Phosphoethanolamine (PEA) | 0.8 mg/kg Ilofotase Alfa | -28.58 | Standard Deviation 15.161 | percent change |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | 0.8 mg/kg Ilofotase Alfa | 6 | |
| Overall Study | STARTED | 3.2 mg/kg Ilofotase Alfa | 6 | |
| Overall Study | COMPLETED | 0.8 mg/kg Ilofotase Alfa | 6 | |
| Overall Study | COMPLETED | 3.2 mg/kg Ilofotase Alfa | 6 | |
| Overall Study | NOT COMPLETED | 0.8 mg/kg Ilofotase Alfa | 0 | |
| Overall Study | NOT COMPLETED | 3.2 mg/kg Ilofotase Alfa | 0 |
Source ClinicalTrials.gov
Adverse events21
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 | Dysgeusia | Nervous system disorders | 0.8 mg/kg Ilofotase Alfa | 0 | 6 | 0 |
| other | Dysgeusia | Nervous system disorders | 3.2 mg/kg Ilofotase Alfa | 2 | 6 | 2 |
| other | Dysgeusia | Nervous system disorders | Overall | 2 | 12 | 2 |
| other | Headache | Nervous system disorders | 0.8 mg/kg Ilofotase Alfa | 0 | 6 | 0 |
| other | Headache | Nervous system disorders | 3.2 mg/kg Ilofotase Alfa | 1 | 6 | 1 |
| other | Headache | Nervous system disorders | Overall | 1 | 12 | 1 |
| other | Restless arm syndrome | Nervous system disorders | 0.8 mg/kg Ilofotase Alfa | 0 | 6 | 0 |
| other | Restless arm syndrome | Nervous system disorders | 3.2 mg/kg Ilofotase Alfa | 1 | 6 | 1 |
| other | Restless arm syndrome | Nervous system disorders | Overall | 1 | 12 | 1 |
| other | Restless legs syndrome | Nervous system disorders | 0.8 mg/kg Ilofotase Alfa | 0 | 6 | 0 |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | 0.8 mg/kg Ilofotase Alfa | 0 | 6 |
| Total, all-cause mortality | 3.2 mg/kg Ilofotase Alfa | 0 | 6 |
| Total, all-cause mortality | Overall | 0 | 12 |
| Total, other adverse events | 0.8 mg/kg Ilofotase Alfa | 1 | 6 |
| Total, other adverse events | 3.2 mg/kg Ilofotase Alfa | 3 | 6 |
| Total, other adverse events | Overall | 4 | 12 |
| Total, serious adverse events | 0.8 mg/kg Ilofotase Alfa | 0 | 6 |
| Total, serious adverse events | 3.2 mg/kg Ilofotase Alfa | 0 | 6 |
| Total, serious adverse events | Overall | 0 | 12 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 41378916 | DERIVED |
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 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/NCT05890794
GET https://conductscience.org/api/trials/v1/NCT05890794/results?page=2