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PTC923 for Phenylketonuria

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
PhenylketonuriaPTC923 - Drug
Eligibility
Any Age
All Sexes
What conditions do you have?
Select

Study Summary

This trial is testing a new drug to see if it can reduce blood Phe levels in people with phenylketonuria.

Eligible Conditions
  • Phenylketonuria

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Similar Trials

1
MAD Cohorts 1 to 4: Participants receiving ECC4703
1
MAD Cohorts 1-5 Experimental Arm
1
Part 2 (MAD): Participants receiving VH4004280

Study Objectives

1 Primary · 2 Secondary · Reporting Duration: Baseline, Weeks 1 and 2, Weeks 3 and 4, and Weeks 5 and 6

Baseline to Week 6
Percentage of Participants With Baseline Phe Levels ≥600 micromoles (μmol)/liter (L) who Achieved Phe Levels <600 μmol/L at the End of the Double-Blind Treatment Period
Week 5
Change From Baseline in Mean Blood Phe Levels at Each PTC923 Dose Level (Average of the Each 2-Week Period in Part 2)
Week 5
Change From Baseline in Phe Levels at Part 2 Weeks 5 and 6

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Similar Trials

1
MAD Cohorts 1 to 4: Participants receiving ECC4703
1
MAD Cohorts 1-5 Experimental Arm
1
Part 2 (MAD): Participants receiving VH4004280

Trial Design

3 Treatment Groups

Part 2: PTC923
1 of 3
Part 1: PTC923
1 of 3
Part 2: Placebo
1 of 3

Experimental Treatment

Non-Treatment Group

178 Total Participants · 3 Treatment Groups

Primary Treatment: PTC923 · Has Placebo Group · Phase 3

Part 2: PTC923
Drug
Experimental Group · 1 Intervention: PTC923 · Intervention Types: Drug
Part 1: PTC923
Drug
Experimental Group · 1 Intervention: PTC923 · Intervention Types: Drug
Part 2: Placebo
Drug
PlaceboComparator Group · 1 Intervention: Placebo · Intervention Types: Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: baseline, weeks 1 and 2, weeks 3 and 4, and weeks 5 and 6

Who is running the clinical trial?

PTC TherapeuticsLead Sponsor
70 Previous Clinical Trials
5,871 Total Patients Enrolled

Eligibility Criteria

Age Any Age · All Participants · 5 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You are willing to continue your current diet unchanged while participating in the study.
You are male and you are sexually active with women of childbearing potential

Who else is applying?

What state do they live in?
Michigan100.0%
How old are they?
< 18100.0%
What site did they apply to?
Indiana University School of Medicine100.0%
What portion of applicants met pre-screening criteria?
Met criteria50.0%
Did not meet criteria50.0%
References

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