IGF-1 Treatment for Individuals With Short Stature Due to PAPP-A2 Deficiency (NCT02636270) | Clinical Trial Compass
CompletedPhase 1/2
IGF-1 Treatment for Individuals With Short Stature Due to PAPP-A2 Deficiency
7 participantsStarted 2015-12-21
Plain-language 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.
Who can participate
Age range
10 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since this was a Phase 1/2 trial testing IGF-1 treatment for a very specific genetic cause of short stature called PAPP-A2 deficiency, can you test whether my child actually has a PAPP-A2 deficiency before we consider this as a relevant option?
2The trial measured height velocity as its primary outcome — can you tell me what the results showed about how much faster children grew, and whether those gains were clinically meaningful compared to not treating?
3Because this was a Phase 1/2 study, the safety data is still relatively early — what side effects were observed, and are there any longer-term risks from IGF-1 treatment that I should understand before discussing this further?
4Since this trial is now completed, has the research led to any approved treatment or next-phase study that might be more accessible for my child, or is IGF-1 for PAPP-A2 deficiency still considered experimental?
5How does IGF-1 treatment for this specific condition compare to other options like growth hormone therapy — and would standard treatments even work if the underlying cause is PAPP-A2 deficiency rather than a growth hormone problem?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Height Velocity
Timeframe: Yearly until participant on treatment stops growing, or discontinues treatment (up to 6 years)
Trial details
NCT IDNCT02636270
SponsorChildren's Hospital Medical Center, Cincinnati