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IGF-1 Treatment for Individuals With Short Stature Due to PAPP-A2 Deficiency

NCT02636270 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 7

Last updated 2024-02-28

Study results available
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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.

Conditions

  • Short Stature

Interventions

DRUG

Increlex

Treat PAPP-A2 deficient patients with Increlex

Sponsors & Collaborators

  • Children's Hospital Medical Center, Cincinnati

    lead OTHER

Principal Investigators

  • Philippe Backeljauw, MD · Cincinnati Childrens Hospital

  • Gajanathan Muthuvel, MD · Cincinnati Childrens Hospital Medical Center

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
10 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2015-12-21
Primary Completion
2022-10-14
Completion
2022-12-01

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02636270 on ClinicalTrials.gov