Phase 2
Completed N=7
IGF-1 Treatment for Individuals With Short Stature Due to PAPP-A2 Deficiency
Source: ClinicalTrials.gov NCT02636270 ↗Enrolled (actual)
7
Serious AEs
14.3%
Results posted
Feb 2024
Primary outcomePrimary: Height Velocity — 3.0; 4.3; 6.2; 4.8 cm/y
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.
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 | — |
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. Informational only — not medical advice.