Alpelisib for Overgrowth Spectrum

Phase-Based Progress Estimates

Effectiveness

Safety

UCLA Medical Center, Los Angeles, CAOvergrowth SpectrumAlpelisib - Drug

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Eligibility

Any Age

All Sexes

What conditions do you have?

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Eligibility

Any Age

All Sexes

What conditions do you have?

Select

Study Summary

This trial is testing a new drug to see if it's effective and safe for kids and adults with a growth disorder.

Treatment Effectiveness

Effectiveness Progress

1 of 3

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Study Objectives

2 Primary · 31 Secondary · Reporting Duration: Up to approximately 5 years

Baseline to Week 16

Change from baseline to Week 16 in Brief Pain Inventory (BPI) Worst Pain intensity for alpelisib compared to placebo treatment arm in pediatric and adult populations

Change from baseline to Week 16 in Brief Pain Inventory (BPI) Worst Pain intensity in Group 1 and 2

Change from baseline to Week 16 in Patient Global Impression of Symptom Severity (PGIS) for alpelisib compared to placebo treatment arm in pediatric and adult populations

Change from baseline to Week 16 in Patient Global Impression of Symptom Severity (PGIS) in Group 1 and 2

Week 16

Changes in symptoms and complications/comorbidities up to Week 16 on treatment with alpelisib as compared to placebo in Group 1 and Group 2

Year 5

Changes in symptoms and complications/comorbidities associated with PROS over time in Group 1 and Group 2

Year 5

Change from Baseline in patient-reported pain assessed by Brief Pain Inventory (BPI) Worst Pain intensity item or Wong-Baker Faces Scale (age appropriate) in pediatric and adult populations

Change from Baseline until End of Treatment in patient-reported pain assessed by Brief Pain Inventory (BPI) Worst Pain intensity item or Wong-Baker Faces Scale (age appropriate) in Group 1 and Group 2

Changes from Baseline in patient-reported health-related quality of life assessed by PROMIS-profile (Patient Reported Outcome Measurement Information System) in pediatric and adult populations

Changes from Baseline in patient-reported overall impression of symptoms assessed by Patient Global Impression of Symptom Severity (PGIS) in pediatric and adult populations

Metals, Alkali

Changes from Baseline until End of Treatment in patient-reported overall impression of symptoms assessed by Patient Global Impression of Symptom Severity (PGIS) for Group 1 and Group 2

Magnetic Resonance Imaging

Proportion of participants requiring rescue surgery due to PROS in Group 1 and Group 2

Proportion of participants with changes from baseline in other non-target lesions in Group 1 and Group 2

Proportion of participants with healthcare visit/hospitalized due to PROS in Group 1 and Group 2

Proportion of participants with new lesions in Group 1 and Group 2

Time to treatment failure in participants who received alpelisib in Group 1 and Group 2

Time to treatment failure in pediatric and adult populations

Year 5

Duration of response (DOR) in participants who received alpelisib in Group 1 and Group 2

Up to 24 weeks

Proportion of participants randomized to alpelisib with a confirmed objective response by BIRC in Group 1 and Group 2

Up to Week 16

Frequency and severity of adverse events in Groups 1 and 2 up to Week 16

Year 5

Frequency and severity of adverse events in Group 1 and Group 2 participants with PROS over time

Frequency and severity of adverse events in all Groups of participants over time

Week 16

Proportion of participants with response at Week 16 by BIRC in Group 1 and Group 2

Proportion of participants with response at Week 16 in Group 1 and Group 2

Week 48

Overall clinical response rate as assessed by investigator in Group 1 and Group 2

Week 48

Overall clinical response rate as assessed by investigator in participants who received alpelisib in Group 1 and Group 2

Week 28

Pharmacokinetics (PK) of alpelisib in Group 1 and Group 2: Trough concentration (Ctrough)

Week 28

Pharmacokinetics (PK) of alpelisib in Group 1 and Group 2: Maximum Concentration (Cmax)

Week 24

Proportion of participants randomized to alpelisib with a response at Week 24 in Group 1 and Group 2

Proportion of participants with a response at Week 24 (by BIRC) in Groups 1 and 2

Week 40

Proportion of participants with response during the extension period in Group 1 and Group 2

Trial Safety

Safety Progress

2 of 3

This is further along than 68% of similar trials

Similar Trials

MBL949 Arm 4

ritonavir

Setanaxib

Trial Design

8 Treatment Groups

Pediatric cohort (group 3: 2 to 5 years old)- Alpelisib

1 of 8

Pediatric cohort (group 2: 6 to 17 years old) -Alpelisib

1 of 8

Pediatric cohort (group 3: 2 to 5 years old)- Alpelisib granules

1 of 8

Pediatric cohort (group 4: 2 to 5 years old)- Alpelisib FCT

1 of 8

Pediatric cohort (group 5: 6-17 years old)-Alpelisib FCT

1 of 8

Adult cohort (group 1)- Alpelisib

1 of 8

Adult cohort (group 1)- Placebo

1 of 8

Pediatric cohort (group 2: 6 to 17 years old)-Placebo

1 of 8

Experimental Treatment

Non-Treatment Group

189 Total Participants · 8 Treatment Groups

Primary Treatment: Alpelisib · Has Placebo Group · Phase 2

Pediatric cohort (group 3: 2 to 5 years old)- Alpelisib

Drug