Trial · NCT06975618
Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of CYH33 in Patients With PIK3CA-related Overgrowth Spectrum (PROS) and PIK3CA-related Vascular Malformations (PRVM)
NCT06975618, Phase 1, Phase 2, Recruiting, Haihe Biopharma Co., Ltd.
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of CYH33 in Patients With PIK3CA-related Overgrowth Spectrum (PROS) and PIK3CA-related Vascular Malformations (PRVM). Trials, record NCT06975618. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT06975618
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of CYH33 in Patients With PIK3CA-related Overgrowth Spectrum (PROS) and PIK3CA-related Vascular Malformations (PRVM)
- NCT number
- NCT06975618
- Status
- Recruiting
- Phases
- Phase 1, Phase 2
- Enrollment
- 141 participants, estimated
- Lead sponsor
- Haihe Biopharma Co., Ltd.
- Conditions
- PIK3CA-Related Overgrowth Spectrum (PROS), PIK3CA-related Vascular Malformations (PRVM)
- Interventions
- CYH33, Placebo
- First posted
- 2025-05-16
- Last updated
- 2026-06-29
Source ClinicalTrials.gov
Registered outcomes21
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Phase I: The maximum tolerated dose (MTD) and/or phase II recommended dose (RP2D) | 27 weeks | To evaluate the safety and tolerability of CYH33 and determine the maximum tolerated dose (MTD) and/or phase II recommended dose (RP2D) of CYH33 in adult and adolescent patients |
| Primary | Phase II PROS Cohort: BIRC-assessed objective response rate (ORR) at Week 24 | Baseline to 24weeks | Proportion of patients achieving ≥20% reduction from baseline in the sum of target lesion volumes, with no progression of non-target lesions and no new lesions, as assessed by blinded independent review committee (BIRC). |
| Primary | Phase II PRVM Cohort: BIRC-assessed objective response rate (ORR) at Week 24 | Baseline to 24weeks | Proportion of patients achieving ≥20% reduction from baseline in the sum of target lesion volumes, with no progression of non-target lesions and no new lesions, as assessed by blinded independent review committee (BIRC). |
| Secondary | Phase I: Pharmacokinetics of CYH33 and its metabolite I27 in the study population: Area Under the Curve from 0 to 24 hours (AUC0-24h) | Pre-dose and at 1, 2, 4, 6, 8, and 24 hours post-dose on Day 1 and Day 29. | AUC0-24h of CYH33 and its metabolite I27 following drug administration will be assessed. |
| Secondary | Phase I: Pharmacokinetics of CYH33 and its metabolite I27 in the study population: Maximum Concentration (Cmax) | Pre-dose and at 1, 2, 4, 6, 8, and 24 hours post-dose on Day 1 and Day 29. | Cmax of CYH33 and its metabolite I27 following drug administration will be assessed. |
| Secondary | Phase I: Pharmacokinetics of CYH33 and its metabolite I27 in the study population: Minimum Concentration (Cmin) | Pre-dose on Day 29. | Cmin of CYH33 and its metabolite I27 following drug administration will be assessed. |
| Secondary | Phase I: Pharmacokinetics of CYH33 and its metabolites in the study population: Time to Maximum Concentration (Tmax) | Pre-dose and at 1, 2, 4, 6, 8, and 24 hours post-dose on Day 1 and Day 29. | Tmax of CYH33 and its metabolite I27 following drug administration will be assessed. |
| Secondary | Phase I: Pharmacokinetics of CYH33 in the study population: Steady-State Apparent Clearance (CLss/F) | Pre-dose and at 1, 2, 4, 6, 8, and 24 hours post-dose on Day 1 and Day 29. | CLss/F of CYH33 following drug administration will be assessed. |
| Secondary | Phase I: The response rate and target lesion volume reduction rate as assessed by the investigators at each dose level | week 27 | A responder is defined as a ≥ 20% reduction in target lesion volume from baseline and in absence of progression of non-target lesions and without new lesions. The proportion of patients with reduced target lesion volume compared to baseline will also be assessed. |
| Secondary | Phase I: The changes from baseline in the Brief Pain Inventory (BPI) Worst Pain Intensity Numerical Rating score at each dose level, based on the patient-reported outcome (PRO) diary | Up to approximately 48 months | Pain is categorized into 11 levels from 0 to 10, where 0 indicates no pain at all and 10 indicates the most severe pain imaginable. Patients should assess and record their pain levels over the past 24 hours in the patient diary at each scheduled assessment visit. |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT06975618
GET https://conductscience.org/api/trials/v1/NCT06975618/registered?page=2