Trial Outcomes & Findings for Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia (NCT NCT05890794)
NCT ID: NCT05890794
Last Updated: 2025-02-27
Results Overview
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)).
COMPLETED
PHASE1/PHASE2
12 participants
Day 1 to Day 10
2025-02-27
Participant Flow
12 patients were enrolled, screened and randomized (6 to the 0.8 mg ilofotase alfa dose group, 6 to the 3.2 mg ilofotase alfa dose group)
Participant milestones
| Measure |
0.8 mg/kg Ilofotase Alfa
Single, 1-hour intravenous infusion of 0.8 mg/kg ilofotase alfa
|
3.2 mg/kg Ilofotase Alfa
Single, 1-hour intravenous infusion of 3.2 mg/kg ilofotase alfa
|
|---|---|---|
|
Overall Study
STARTED
|
6
|
6
|
|
Overall Study
COMPLETED
|
6
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
0.8 mg/kg Ilofotase Alfa
n=6 Participants
Single, 1-hour intravenous infusion of 0.8 mg/kg ilofotase alfa
|
3.2 mg/kg Ilofotase Alfa
n=6 Participants
Single, 1-hour intravenous infusion of 3.2 mg/kg ilofotase alfa
|
Total
n=12 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
49.5 years
n=6 Participants
|
51.0 years
n=6 Participants
|
51.0 years
n=12 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=6 Participants
|
5 Participants
n=6 Participants
|
8 Participants
n=12 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=6 Participants
|
1 Participants
n=6 Participants
|
4 Participants
n=12 Participants
|
|
Race and Ethnicity Not Collected
|
—
|
—
|
0 Participants
Race and Ethnicity were not collected from any participant.
|
PRIMARY outcome
Timeframe: Day 1 to Day 10Population: All randomized patients.
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)).
Outcome measures
| Measure |
0.8 mg/kg Ilofotase Alfa
n=6 Participants
Single, 1-hour intravenous infusion of 0.8 mg/kg ilofotase alfa
|
3.2 mg/kg Ilofotase Alfa
n=6 Participants
Single, 1-hour intravenous infusion of 3.2 mg/kg ilofotase alfa
|
Overall
n=12 Participants
Single, 1-hour intravenous infusion of 0.8 mg/kg or 3.2 mg/kg ilofotase alfa
|
|---|---|---|---|
|
Maximum Percent Change From Baseline in Extracellular Inorganic Pyrophosphate (PPi)
|
-36.46 percent change
Standard Deviation 11.502
|
-77.31 percent change
Standard Deviation 16.113
|
-56.88 percent change
Standard Deviation 25.165
|
PRIMARY outcome
Timeframe: Day 1 to Day 10Population: All randomized patients.
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)).
Outcome measures
| Measure |
0.8 mg/kg Ilofotase Alfa
n=6 Participants
Single, 1-hour intravenous infusion of 0.8 mg/kg ilofotase alfa
|
3.2 mg/kg Ilofotase Alfa
n=6 Participants
Single, 1-hour intravenous infusion of 3.2 mg/kg ilofotase alfa
|
Overall
n=12 Participants
Single, 1-hour intravenous infusion of 0.8 mg/kg or 3.2 mg/kg ilofotase alfa
|
|---|---|---|---|
|
Maximum Percent Change From Baseline in Pyridoxal 5'-Phosphate (PLP)
|
-34.68 percent change
Standard Deviation 8.898
|
-65.56 percent change
Standard Deviation 6.559
|
-50.12 percent change
Standard Deviation 17.768
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 1 to Day 10Population: All randomized patients.
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))
Outcome measures
| Measure |
0.8 mg/kg Ilofotase Alfa
n=6 Participants
Single, 1-hour intravenous infusion of 0.8 mg/kg ilofotase alfa
|
3.2 mg/kg Ilofotase Alfa
n=6 Participants
Single, 1-hour intravenous infusion of 3.2 mg/kg ilofotase alfa
|
Overall
n=12 Participants
Single, 1-hour intravenous infusion of 0.8 mg/kg or 3.2 mg/kg ilofotase alfa
|
|---|---|---|---|
|
Maximum Percent Change From Baseline in Alkaline Phosphatase (ALP) Activity
|
2685.57 percent change
Standard Deviation 1492.991
|
7021.23 percent change
Standard Deviation 2063.878
|
4853.40 percent change
Standard Deviation 2841.847
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 1 to Day 10Population: All randomized patients.
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)).
Outcome measures
| Measure |
0.8 mg/kg Ilofotase Alfa
n=6 Participants
Single, 1-hour intravenous infusion of 0.8 mg/kg ilofotase alfa
|
3.2 mg/kg Ilofotase Alfa
n=6 Participants
Single, 1-hour intravenous infusion of 3.2 mg/kg ilofotase alfa
|
Overall
n=12 Participants
Single, 1-hour intravenous infusion of 0.8 mg/kg or 3.2 mg/kg ilofotase alfa
|
|---|---|---|---|
|
Maximum Percent Change From Baseline in Urine Phosphoethanolamine (PEA)
|
-28.58 percent change
Standard Deviation 15.161
|
-51.82 percent change
Standard Deviation 19.614
|
-40.20 percent change
Standard Deviation 20.573
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 1 to Day 10Population: All randomized patients.
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)).
Outcome measures
| Measure |
0.8 mg/kg Ilofotase Alfa
n=6 Participants
Single, 1-hour intravenous infusion of 0.8 mg/kg ilofotase alfa
|
3.2 mg/kg Ilofotase Alfa
n=6 Participants
Single, 1-hour intravenous infusion of 3.2 mg/kg ilofotase alfa
|
Overall
n=12 Participants
Single, 1-hour intravenous infusion of 0.8 mg/kg or 3.2 mg/kg ilofotase alfa
|
|---|---|---|---|
|
Maximum Percent Change From Baseline in Pyridoxal (PL).
|
409.76 percent change
Standard Deviation 138.504
|
222.33 percent change
Standard Deviation 91.332
|
316.05 percent change
Standard Deviation 148.635
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 1 to Day 10Population: All randomized patients.
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)).
Outcome measures
| Measure |
0.8 mg/kg Ilofotase Alfa
n=6 Participants
Single, 1-hour intravenous infusion of 0.8 mg/kg ilofotase alfa
|
3.2 mg/kg Ilofotase Alfa
n=6 Participants
Single, 1-hour intravenous infusion of 3.2 mg/kg ilofotase alfa
|
Overall
n=12 Participants
Single, 1-hour intravenous infusion of 0.8 mg/kg or 3.2 mg/kg ilofotase alfa
|
|---|---|---|---|
|
Maximum Fold Change From Baseline in PL/PLP Ratio
|
6.9145 fold change
Standard Deviation 1.83957
|
7.8062 fold change
Standard Deviation 2.70554
|
7.3604 fold change
Standard Deviation 2.25440
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 1 to Day 15Population: All patients who received trial medication were included in the population for the safety analysis.
Any untoward medical occurrence in a patient enrolled and treated in the clinical trial regardless of its causal relationship to the trial medication.
Outcome measures
| Measure |
0.8 mg/kg Ilofotase Alfa
n=6 Participants
Single, 1-hour intravenous infusion of 0.8 mg/kg ilofotase alfa
|
3.2 mg/kg Ilofotase Alfa
n=6 Participants
Single, 1-hour intravenous infusion of 3.2 mg/kg ilofotase alfa
|
Overall
n=12 Participants
Single, 1-hour intravenous infusion of 0.8 mg/kg or 3.2 mg/kg ilofotase alfa
|
|---|---|---|---|
|
Treatment-emergent Adverse Events (TEAEs)
|
1 Participants
|
3 Participants
|
4 Participants
|
Adverse Events
0.8 mg/kg Ilofotase Alfa
3.2 mg/kg Ilofotase Alfa
Overall
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
0.8 mg/kg Ilofotase Alfa
n=6 participants at risk
Single dose administered intravenously over 1 hour
Ilofotase Alfa, 0.8 mg/kg: Biological: single 1-hour intravenous infusion of 0.8 mg/kg ilofotase alfa
|
3.2 mg/kg Ilofotase Alfa
n=6 participants at risk
Single dose administered intravenously over 1 hour
Ilofotase Alfa, 3.2 mg/kg: Biological: single 1-hour intravenous infusion of 3.2 mg/kg ilofotase alfa
|
Overall
n=12 participants at risk
Single dose administered intravenously over 1 hour
Ilofotase Alfa, 0.8 or 3.2 mg/kg: Biological: single 1-hour intravenous infusion of 0.8 or 3.2 mg/kg ilofotase alfa
|
|---|---|---|---|
|
Nervous system disorders
Dysgeusia
|
0.00%
0/6 • Day 1 to Day 15
|
33.3%
2/6 • Number of events 2 • Day 1 to Day 15
|
16.7%
2/12 • Number of events 2 • Day 1 to Day 15
|
|
Nervous system disorders
Headache
|
0.00%
0/6 • Day 1 to Day 15
|
16.7%
1/6 • Number of events 1 • Day 1 to Day 15
|
8.3%
1/12 • Number of events 1 • Day 1 to Day 15
|
|
Nervous system disorders
Restless arm syndrome
|
0.00%
0/6 • Day 1 to Day 15
|
16.7%
1/6 • Number of events 1 • Day 1 to Day 15
|
8.3%
1/12 • Number of events 1 • Day 1 to Day 15
|
|
Nervous system disorders
Restless legs syndrome
|
0.00%
0/6 • Day 1 to Day 15
|
16.7%
1/6 • Number of events 1 • Day 1 to Day 15
|
8.3%
1/12 • Number of events 1 • Day 1 to Day 15
|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.00%
0/6 • Day 1 to Day 15
|
16.7%
1/6 • Number of events 1 • Day 1 to Day 15
|
8.3%
1/12 • Number of events 1 • Day 1 to Day 15
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/6 • Day 1 to Day 15
|
16.7%
1/6 • Number of events 1 • Day 1 to Day 15
|
8.3%
1/12 • Number of events 1 • Day 1 to Day 15
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
16.7%
1/6 • Number of events 1 • Day 1 to Day 15
|
0.00%
0/6 • Day 1 to Day 15
|
8.3%
1/12 • Number of events 1 • Day 1 to Day 15
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The PI may publish the study results but must submit the publication to the sponsor for review at least 60 days prior to publication. The sponsor may request changes/removals of sponsor information. The PI should remove such information if the correct presentation of the results is still ensured. The PI must consider the sponsor's other comments but must not adopt them. If publication could affect the patentability of the invention, the sponsor may request an additional period of up to 120 days.
- Publication restrictions are in place
Restriction type: OTHER