Phase 2
N=7
IGF-1 Treatment for Individuals With Short Stature Due to PAPP-A2 Deficiency
Short Stature
Bottom Line
View on ClinicalTrials.gov: NCT02636270 ↗Enrolled (actual)
7
Serious AEs
14.3%
Results posted
Feb 2024
Primary outcome: Primary: Height Velocity — 3.0; 4.3; 6.2; 4.8 cm/y
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Increlex (Drug)
- Age
- Pediatric, Adult, Older Adult · 10+ yrs
- Sex
- All
- Sponsor
- Children's Hospital Medical Center, Cincinnati
- Primary completion
- Oct 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Height Velocity |
3.0; 4.3; 6.2; 4.8; 5.0; 5.3 | — |
| SECONDARY Height Standard Deviation Score |
-2.86; -2.92; -2.53; -2.99; -2.54; -2.84 | — |
| SECONDARY Pharmacokinetic/Pharmacodynamic (PK/PD) Relationship |
0.79; 546.8; 10000; 0.110; 1.17; 709.1 | — |
Summary
With this study we want to investigate the pharmacokinetic (PK) effect of a single injection of rhIGF-1 in patients with PAPP-A2 mutations compared to heterozygous carriers and healthy controls. This will be followed by treatment of PAPP-A2 deficient patients with IGF-1 for a period of one-year to assess growth velocity. Additionally, we want to further describe the phenotypic characteristics of patients with PAPP-A2 deficiency.
Eligibility Criteria
PAPP-A2 deficient
Inclusion Criteria
- Defect in PAPP-A2 (heterozygous or homozygous mutation)
Exclusion Criteria
- None
Healthy Volunteers
Inclusion Criteria
- Between the ages of 18 and 30
- In general good health
Exclusion Criteria
- Any medications (with the exception of contraceptives)
- Pregnancy
Data sourced from ClinicalTrials.gov (NCT02636270). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.