Primary outcomeNumber· Yearly until participant on treatment stops growing, or discontinues treatment (up to 6 years)
Height Velocity
| Group | Value (cm/y) |
|---|---|
| Treatment With rhIGF-1 | 3.0 |
| Untreated Comparison | 4.3 |
Trial · Interventional
CompletedPhase 1/2Results postedRegistry dataIGF-1 Treatment for Individuals With Short Stature Due to PAPP-A2 Deficiency
NCTNCT02636270 (opens ClinicalTrials.gov in a new tab)Other id2015-6218
Overview
- Status
- Completed
- Phase
- Phase 1/2
- Enrollment
- 7 participants (actual)
- Sponsor
- Children's Hospital Medical Center, Cincinnati · Other
- Design
- Allocation not applicable · Open label (no masking)
- Countries
- Not stated
- Start
- 21 Dec 2015
- Primary completion
- 14 Oct 2022
- Completion
- 1 Dec 2022
- First posted
- 21 Dec 2015 (estimated)
- Last update posted
- 28 Feb 2024
- Conditions
- Short Stature
Timeline
1 trial · 1 completed · 1 with posted results
- Completed
- Ongoing
- Not yet recruiting
- Stopped
- Estimated dates (est.)
- Results posted
2016201820202022
- Ph 1/2
IGF-1 Treatment for Individuals With Short Stature Due to PAPP-A2 Deficiency
NCT0263627021 Dec 2015 to 1 Dec 2022
Registry summary
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
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.
Interventions
- IncrelexDrug
Arms1
Experimental
PAPP-A2 deficient patients
Patients deficient in PAPP-A2 with short stature will be treated with Increlex (rhIGF-1)
Primary outcomes1
Outcomes the sponsor listed as primary.
Height Velocity
Time frame · Yearly until participant on treatment stops growing, or discontinues treatment (up to 6 years)
Registry description
Height velocity in a patient with PAPP-A2 deficiency treated with rhIGF-1 for five years (when the patient elected to discontinue treatment after reviewing growth velocity and skeletal maturation). Ultimately only one patient was treated for the study duration with results reported, as the other recruited participant (sibling of the treated patient) experienced pseudotumor cerebri and discontinued treatment after 51 days. He nevertheless was followed, with height velocity also reported.
Secondary outcomes(2)
Height Standard Deviation Score
Time frame · Annually until completion of study, up to 6 years
Registry description
Height Standard Deviation Score is the standard deviation above or below the mean the height is for age and gender. Values were obtained by plotting heights on Centers for Disease Control and Prevention growth charts. An increase in Height Standard Deviation Score correlates with increase in height. Results are reported for the participant with PAPP-A2 deficiency treated with rhIGF-1, as well as sibling who did not continue treatment with rhIGF-1.
Pharmacokinetic/Pharmacodynamic (PK/PD) Relationship
Time frame · Yearly until completion of the study, up to 6 years
Registry description
Assess the PK/PD relationship (PD marker being IGFBP-3) annually while on treatment with rhIGF-1
Posted results
Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.
Primary outcome results
Secondary and other outcome results(7)
Secondary outcomeNumber· Annually until completion of study, up to 6 years
Height Standard Deviation Score
| Group | Value (standard deviation score) |
|---|---|
| Treatment With rhIGF-1 | -2.86 |
| Untreated Comparison | -2.92 |
Secondary outcomeNumber· Yearly until completion of the study, up to 6 years
Pharmacokinetic/Pharmacodynamic (PK/PD) Relationship
| Group | Value (ng/mL) |
|---|---|
| Free IGF-I | 0.79 |
| IGF-I | 546.8 |
| IGFBP-3 | 10000 |
| PAPP-A2 | 0.110 |
Other pre specified outcomeNumber· Yearly until completion of the study, up to 6 years
Glucose Pre- and Post-treatment With Recombinant Human IGF-I
| Group | Value (mg/dL) |
|---|---|
| OGTT - 0 Minutes | 111.4 |
| OGTT - 30 Minutes | 193.9 |
| OGTT - 60 Minutes | 203.9 |
| OGTT - 90 Minutes | 156.6 |
| OGTT - 120 Minutes | 127.5 |
Other pre specified outcomeNumber· Annually through completion of the study, up to 6 years
Insulin Metabolism Pre- and Post-treatment With Recombinant Human IGF-I
| Group | Value (mcIU/mL) |
|---|---|
| OGTT - 0 Minutes | 18.3 |
| OGTT - 30 Minutes | 88.1 |
| OGTT - 60 Minutes | 113.2 |
| OGTT - 90 Minutes | 131.9 |
| OGTT - 120 Minutes | 107 |
Other pre specified outcomeNumber· Annually until completion of the study, up to 6 years
Body Mass Index at Baseline and on Treatment With rhIGF-I
| Group | Value (percentile) |
|---|---|
| BMI Percentile | 12.96 |
Other pre specified outcomeNumber· Annually until completion of the study, up to 6 years
Body Composition at Baseline and on Treatment With rhIGF-I
| Group | Value (g) |
|---|---|
| Total Lean Body Mass | 15274 |
| Total Fat Mass | 6543 |
Other pre specified outcomeNumber· Annually until completion of the study, up to 6 years
Total Body Fat Percentage at Baseline and on Treatment With rhIGF-I
| Group | Value (percentage of total body fat) |
|---|---|
| Total Body Fat Percentage | 29.0 |
Adverse events, as posted
| Group | Deaths | Serious events | Other events |
|---|---|---|---|
| PAPP-A2 Deficient PatientsPatients deficient in PAPP-A2 will participate in the 24-hour pharmacokinetic study, and those who have not completed growth will be treated with Increlex (recombinant human IGF-1). | 0 / 3 | 1 / 3 | 2 / 3 |
| PAPP-A2 Heterozygous RelativesUnaffected heterozygous PAPP-A2 deficient relatives (2 parents). | 0 / 2 | 0 / 2 | 0 / 2 |
| Healthy Control ParticipantsHealthy controls between the ages of 18-30. | 0 / 2 | 0 / 2 | 0 / 2 |
Each cell reads participants affected / participants at risk, as the registry posted them.
Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works
Studies1
Studies that cite this trial.
More studies
1 study on this page
PubMed tag: none · 2023
Five-Year Therapy with Recombinant Human Insulin-Like Growth Factor-1 in a Patient with PAPP-A2 Deficiency.
Muthuvel et al. 2023Hormone research in paediatricscited by 1
PMID 36646053DOI 10.1159/000529071PUB-36646053
Full text at the publisher
Other registry references
References listed at the registry that are not in PepBank. They link to PubMed.
- ResultCabrera-Salcedo C, Mizuno T, Tyzinski L, Andrew M, Vinks AA, Frystyk J, Wasserman H, Gordon CM, Hwa V, Backeljauw P, Dauber A. Pharmacokinetics of IGF-1 in PAPP-A2-Deficient Patients, Growth Response, and Effects on Glucose and Bone Density. J Clin Endocrinol Metab. 2017 Dec 1;102(12):4568-4577. doi: 10.1210/jc.2017-01411. PMID 29029190
Eligibility
Who could join, as posted.
- Sex
- All
- Age
- from 10 Years
- Healthy volunteers
- Accepted
Inclusion and exclusion criteria, as posted
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
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