A Phase 1/2 interventional study of Increlex in Short Stature, sponsored by Children's Hospital Medical Center, Cincinnati. Completed. Open to participants aged 10 Years and older, including healthy volunteers. Per ClinicalTrials.gov, last updated 2024-02-28.
Sponsored by Children's Hospital Medical Center, Cincinnati · Phase 1/2, Interventional, and Treatment
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.
The 24-hour pharmacokinetic response of free and total IGF-1 and IGF binding protein-3 (IGFBP-3) to a single dose of rhIGF-1 (120 mcg/kg) in three patients with PAPP-A2 mutation compared to up to four unaffected heterozygous relatives and 2 healthy adult controls.
One-year trial of rhIGF-1 at standard dose given to the two youngest males with PAPP-A2 mutation. The primary end point of this trial will be first year height velocity. Secondary outcomes will include height standard deviation score (SDS), height velocity, and whole body and lumbar spine bone mineral density assessment. The study was amended to extend the treatment period to continue until the subject has stopped growing (or elects to withdraw). All study procedures remain the same. Important note: the treatment phase continues to follow the youngest affected male. The older affected male developed an adverse event that resulted in discontinuation of treatment.
A post-treatment follow up visit (either in-person or remote) will be completed for the study participant who remained on Increlex approximately one-year after their discontinuation of therapy.
Description of additional phenotypic characteristics of patients with PAPP-A2 mutation will be studied by collecting information on glucose and insulin metabolism, body composition, bone geometry and bone density before and after treatment with rhIGF-1. These measures will be collected at the 12-month time period, and every year thereafter until the completion of the study. All three affected siblings will take part in the phenotyping activities.
99 studies on the registry are indexed under Dwarfism; 12 are open to participants now.
This study's enrollment of 7 is below the median of 60 across 66 interventional studies indexed under Dwarfism.
Browse Dwarfism studies →Children's Hospital Medical Center, Cincinnati is the lead sponsor of 673 studies on the registry; 130 are open to participants now.
Of its 57 completed or terminated interventional studies of FDA-regulated products, 31 (54%) have results posted.
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PAPP-A2 deficient
Inclusion Criteria:
Exclusion Criteria:
Healthy Volunteers
Inclusion Criteria:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients deficient in PAPP-A2 with short stature will be treated with Increlex (rhIGF-1)
Drug: Increlex
Treat PAPP-A2 deficient patients with Increlex
Also known as: rhIGF-1
Height Velocity
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.
Time frame: Yearly until participant on treatment stops growing, or discontinues treatment (up to 6 years)
Height Standard Deviation Score
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.
Time frame: Annually until completion of study, up to 6 years
Pharmacokinetic/Pharmacodynamic (PK/PD) Relationship
Assess the PK/PD relationship (PD marker being IGFBP-3) annually while on treatment with rhIGF-1
Time frame: Yearly until completion of the study, up to 6 years
Glucose Pre- and Post-treatment With Recombinant Human IGF-I
Observe nonparametric measures of glucose pre and post ongoing treatment with rhIGF-1. Oral glucose tolerance tests (OGTT) were performed annually during the five-year treatment period at pre-treatment (baseline) and post-initiation of treatment (after 12 months, 24 months, 36 months, 48 months, and 60 months).
Time frame: Yearly until completion of the study, up to 6 years
Insulin Metabolism Pre- and Post-treatment With Recombinant Human IGF-I
Observe nonparametric measures of insulin metabolism in each individual pre (baseline) and post ongoing treatment with rhIGF-1. Oral glucose tolerance testing (OGTT) was performed pre-treatment (baseline) and post-treatment (after 12 months, 24 months, 36 months, 48 months, and 60 months).
Time frame: Annually through completion of the study, up to 6 years
Body Mass Index at Baseline and on Treatment With rhIGF-I
Observe nonparametric measures of body composition pre (baseline) and post ongoing treatment with rhIGF-1 (assessed after 12 months, 24 months, 36 months, 48 months, and 60 months of rhIGF-1 treatment). BMI percentiles were determined utilizing Centers for Disease Control and Prevention growth charts.
Time frame: Annually until completion of the study, up to 6 years
Body Composition at Baseline and on Treatment With rhIGF-I
Observe nonparametric measures of body composition pre (baseline) and post ongoing treatment with rhIGF-1 (assessed after 12 months, 24 months, 36 months, 48 months, and 60 months of rhIGF-1 treatment). Body fat content and lean body mass were evaluated with dual energy x-ray absorptiometry.
Time frame: Annually until completion of the study, up to 6 years
Total Body Fat Percentage at Baseline and on Treatment With rhIGF-I
Observe nonparametric measures of body composition pre (baseline) and post ongoing treatment with rhIGF-1 (assessed after 12 months, 24 months, 36 months, 48 months, and 60 months of rhIGF-1 treatment). Body fat content and lean body mass were evaluated with dual energy x-ray absorptiometry.
Time frame: Annually until completion of the study, up to 6 years
C-telopeptide and Osteocalcin Concentrations at Baseline (Pre-treatment) and While on rhIGF-1.
Observe nonparametric measures of bone turnover pre-treatment (baseline) and post initiation of ongoing treatment (at 12 months, 24 months, 36 months, 48 months, and 60 months) while on rhIGF-1
Time frame: Yearly until completion of the study, up to 6 years
Bone Density Pre-treatment (Baseline) and on Treatment With rhIGF-1
Observe nonparametric measures of bone density at baseline (pre-treatment) and post initiation of ongoing treatment with rhIGF-1. Dual energy x-ray absorptiometry (DXA) was performed of total body less head, lumbar spine, hip, and forearm. Results are reported as height adjusted z-scores for age and gender, commonly done for bone density. A z-score of 0 is equivalent to population mean, positive above the mean, and negative below the mean. Z-scores within 2 standard deviations of the mean (Z-score 2 to -2) are generally considered normal, however are not sufficient to comment on presence or absence of osteoporosis in the pediatric population.
Time frame: Annually until completion of therapy, up to 6 years
Pharmacokinetic Description After Receiving Recombinant Human Insulin Like Growth Factor 1 (rhIGF-1): Maximum Corrected Total IGF-I and Free IGF-I
This was a pharmacokinetic assessment in regards to rhIGF-1 completed in siblings with PAPP-A2 deficiency, Participants received a 120 mcg/kg dose of rhIGF-1 (Increlex). Pharmacokinetic measurements were obtained over 24 hours. To isolate the effect of injected rhIGF-1, baseline-corrected concentrations were included by subtracting baseline concentration from measured concentrations. Results reported include maximum corrected total IGF-I and free IGF-I. Due to constraints of reporting platform, time to maximum values and area under the curve (AUC) 12 hours after the dose are reported as separate outcomes.
Time frame: At baseline, prior to the ongoing treatment phase with rhIGF-1
Pharmacokinetic Description After Receiving Recombinant Human Insulin Like Growth Factor 1 (rhIGF-1): Time to Maximum Corrected Total IGF-I and Free IGF-I
This was a pharmacokinetic assessment in regards to rhIGF-1 completed in siblings with PAPP-A2 deficiency, Participants received a 120 mcg/kg dose of rhIGF-1 (Increlex). Pharmacokinetic measurements were obtained over 24 hours. To isolate the effect of injected rhIGF-1, baseline-corrected concentrations were included by subtracting baseline concentration from measured concentrations. Results reported include time to maximum corrected total IGF-I and free IGF-I. Due to constraints of reporting platform, maximum corrected total IGF-I and free IGF-I values and area under the curve (AUC) 12 hours after the dose are reported as separate outcomes.
Time frame: At baseline, prior to the ongoing treatment phase with rhIGF-1
Pharmacokinetic Description After Receiving Recombinant Human Insulin Like Growth Factor 1 (rhIGF-1): Area Under the Curve
This was a pharmacokinetic assessment in regards to rhIGF-1 completed in siblings with PAPP-A2 deficiency, Participants received a 120 mcg/kg dose of rhIGF-1 (Increlex). Pharmacokinetic measurements were obtained over 24 hours. To isolate the effect of injected rhIGF-1, baseline-corrected concentrations were included by subtracting baseline concentration from measured concentrations. Results reported include area under the curve (AUC) 12 hours after the dose. Due to constraints of reporting platform, maximum corrected total IGF-I and free IGF-I, as well as time to maximum values separate outcomes.
Time frame: At baseline, prior to the ongoing treatment phase with rhIGF-1
This is an n=7 study. A total of seven individuals will be recruited to participate in this study. Among the participants, we will have the three affected siblings with Pappalysin-2 (PAPP-A2) mutation (two males and one female), their heterozygous relatives (2 parents) and 2 healthy adult controls.
| Milestone | PAPP-A2 Deficient Patients | Healthy Control Participants | PAPP-A2 Heterozygous Relatives |
|---|---|---|---|
| Started | 3 | 2 | 2 |
| Completed | 1 | 2 | 2 |
| Not completed | 2 | 0 | 0 |
| Withdrew: Adverse event | 1 | 0 | 0 |
| Withdrew: Subject had completed growth prior to enrollment, so never started on recombinant igf-1 | 1 | 0 | 0 |
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.
| cm/y | Treatment With rhIGF-1 | Untreated Comparison |
|---|---|---|
| Baseline | 3.0 | 4.3 |
| Year 1 | 6.2 | 4.8 |
| Year 2 | 5.0 | 5.3 |
| Year 3 | 5.3 | 3.4 |
| Year 4 | 7.6 | 1.4 |
| Year 5 | 6.5 | — |
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.
| standard deviation score | Treatment With rhIGF-1 | Untreated Comparison |
|---|---|---|
| Baseline | -2.86 | -2.92 |
| Year 1 | -2.53 | -2.99 |
| Year 2 | -2.54 | -2.84 |
| Year 3 | -2.61 | -2.69 |
| Year 4 | -2.43 | -2.63 |
| Year 5 | -2.27 | — |
Assess the PK/PD relationship (PD marker being IGFBP-3) annually while on treatment with rhIGF-1
| ng/mL | Free IGF-I | IGF-I | IGFBP-3 | PAPP-A2 |
|---|---|---|---|---|
| Baseline | 0.79 | 546.8 | 10000 | 0.110 |
| Year 1 | 1.17 | 709.1 | 6362 | 0.190 |
| Year 2 | 4.03 | 814.4 | 4001 | 0.122 |
| Year 3 | 5.13 | 961.7 | 6953 | 0.077 |
| Year 4 | 6.75 | 1200 | 6956 | 0.050 |
| Year 5 | 16.80 | 1200 | 6053 | 0 |
Observe nonparametric measures of glucose pre and post ongoing treatment with rhIGF-1. Oral glucose tolerance tests (OGTT) were performed annually during the five-year treatment period at pre-treatment (baseline) and post-initiation of treatment (after 12 months, 24 months, 36 months, 48 months, and 60 months).
| mg/dL | OGTT - 0 Minutes | OGTT - 30 Minutes | OGTT - 60 Minutes | OGTT - 90 Minutes | OGTT - 120 Minutes |
|---|---|---|---|---|---|
| Baseline | 111.4 | 193.9 | 203.9 | 156.6 | 127.5 |
| 12 Months | 84.1 | 144.1 | 78.8 | 96.9 | 90.6 |
| 24 Months | 103.5 | 183.4 | 132.0 | 122.7 | 116.2 |
| 36 Months | 103.9 | 185.6 | 153.8 | 126.0 | 104.3 |
| 48 Months | 97.3 | 203.1 | 249.3 | 198.7 | 169.7 |
| 60 Months | 93.1 | 198.9 | 224.5 | 199.3 | 151.6 |
Observe nonparametric measures of insulin metabolism in each individual pre (baseline) and post ongoing treatment with rhIGF-1. Oral glucose tolerance testing (OGTT) was performed pre-treatment (baseline) and post-treatment (after 12 months, 24 months, 36 months, 48 months, and 60 months).
| mcIU/mL | OGTT - 0 Minutes | OGTT - 30 Minutes | OGTT - 60 Minutes | OGTT - 90 Minutes | OGTT - 120 Minutes |
|---|---|---|---|---|---|
| Baseline | 18.3 | 88.1 | 113.2 | 131.9 | 107 |
| 12 Months | 4.5 | 38.1 | 11.2 | 22.6 | 22.3 |
| 24 Months | 8.7 | 51.2 | 33.8 | 26.9 | 25.8 |
| 36 Months | 20.4 | 120.6 | 67.6 | 58.9 | 50.3 |
| 48 Months | 12.3 | 102.3 | 116.3 | 114.1 | 134.4 |
| 60 Months | 14.9 | 84.2 | 98.5 | 116.9 | 94.4 |
Observe nonparametric measures of body composition pre (baseline) and post ongoing treatment with rhIGF-1 (assessed after 12 months, 24 months, 36 months, 48 months, and 60 months of rhIGF-1 treatment). BMI percentiles were determined utilizing Centers for Disease Control and Prevention growth charts.
| percentile | BMI Percentile |
|---|---|
| Baseline | 12.96 |
| Year 1 | 30.83 |
| Year 2 | 56.60 |
| Year 3 | 42.76 |
| Year 4 | 54.20 |
| Year 5 | 70.42 |
Observe nonparametric measures of body composition pre (baseline) and post ongoing treatment with rhIGF-1 (assessed after 12 months, 24 months, 36 months, 48 months, and 60 months of rhIGF-1 treatment). Body fat content and lean body mass were evaluated with dual energy x-ray absorptiometry.
| g | Total Lean Body Mass | Total Fat Mass |
|---|---|---|
| Baseline | 15274 | 6543 |
| Year 1 | 18961 | 7390 |
| Year 2 | 21366 | 10501 |
| Year 3 | 23876 | 10084 |
| Year 4 | 29779 | 12060 |
| Year 5 | 36398 | 13998 |
Observe nonparametric measures of body composition pre (baseline) and post ongoing treatment with rhIGF-1 (assessed after 12 months, 24 months, 36 months, 48 months, and 60 months of rhIGF-1 treatment). Body fat content and lean body mass were evaluated with dual energy x-ray absorptiometry.
| percentage of total body fat | Total Body Fat Percentage |
|---|---|
| Baseline | 29.0 |
| Year 1 | 27.1 |
| Year 2 | 32.0 |
| Year 3 | 28.7 |
| Year 4 | 28.0 |
| Year 5 | 27.0 |
Observe nonparametric measures of bone turnover pre-treatment (baseline) and post initiation of ongoing treatment (at 12 months, 24 months, 36 months, 48 months, and 60 months) while on rhIGF-1
| ng/mL | C-telopeptide | Osteocalcin |
|---|---|---|
| Baseline | 1744 | 86 |
| Year 1 | 2262 | 123 |
| Year 2 | 1762 | 88 |
| Year 3 | 2098 | 110 |
| Year 4 | 3216 | 152 |
| Year 5 | 2729 | 126 |
Observe nonparametric measures of bone density at baseline (pre-treatment) and post initiation of ongoing treatment with rhIGF-1. Dual energy x-ray absorptiometry (DXA) was performed of total body less head, lumbar spine, hip, and forearm. Results are reported as height adjusted z-scores for age and gender, commonly done for bone density. A z-score of 0 is equivalent to population mean, positive above the mean, and negative below the mean. Z-scores within 2 standard deviations of the mean (Z-score 2 to -2) are generally considered normal, however are not sufficient to comment on presence or absence of osteoporosis in the pediatric population.
| z-score | DXA Total Body Less Head | DXA Lumbar Spine | DXA Hip | DXA Forearm |
|---|---|---|---|---|
| Baseline | -0.9 | -0.2 | -0.6 | -0.6 |
| Year 1 | -0.2 | 0.3 | -0.5 | -0.1 |
| Year 2 | -0.2 | 0.7 | -0.4 | -0.4 |
| Year 3 | -0.3 | 0.5 | -0.3 | -1.0 |
| Year 4 | -0.5 | 0.4 | -0.7 | -1.0 |
| Year 5 | -0.9 | 0.2 | -0.9 | -0.8 |
This was a pharmacokinetic assessment in regards to rhIGF-1 completed in siblings with PAPP-A2 deficiency, Participants received a 120 mcg/kg dose of rhIGF-1 (Increlex). Pharmacokinetic measurements were obtained over 24 hours. To isolate the effect of injected rhIGF-1, baseline-corrected concentrations were included by subtracting baseline concentration from measured concentrations. Results reported include maximum corrected total IGF-I and free IGF-I. Due to constraints of reporting platform, time to maximum values and area under the curve (AUC) 12 hours after the dose are reported as separate outcomes.
| mcg/L | PAPP-A2 Deficient Patients | PAPP-A2 Heterozygous Relatives | Healthy Controls |
|---|---|---|---|
| Maximum corrected total IGF-I | 225 (186 to 254) | 243 (203 to 283) | 284.5 (278 to 291) |
| Maximum corrected free IGF-I | 32 (26.6 to 35) | 30.5 (28.6 to 32.4) | 33.5 (10.9 to 56) |
This was a pharmacokinetic assessment in regards to rhIGF-1 completed in siblings with PAPP-A2 deficiency, Participants received a 120 mcg/kg dose of rhIGF-1 (Increlex). Pharmacokinetic measurements were obtained over 24 hours. To isolate the effect of injected rhIGF-1, baseline-corrected concentrations were included by subtracting baseline concentration from measured concentrations. Results reported include time to maximum corrected total IGF-I and free IGF-I. Due to constraints of reporting platform, maximum corrected total IGF-I and free IGF-I values and area under the curve (AUC) 12 hours after the dose are reported as separate outcomes.
| h | PAPP-A2 Deficient Patients | PAPP-A2 Heterozygous Relatives | Healthy Controls |
|---|---|---|---|
| Time to maximum corrected total IGF-I | 4.2 (3.5 to 5) | 3.3 (3 to 3.5) | 5.5 (5 to 6) |
| Time to maximum corrected free IGF-I | 2.0 (1 to 3.5) | 2.3 (2 to 2.5) | 2.3 (2 to 2.5) |
This was a pharmacokinetic assessment in regards to rhIGF-1 completed in siblings with PAPP-A2 deficiency, Participants received a 120 mcg/kg dose of rhIGF-1 (Increlex). Pharmacokinetic measurements were obtained over 24 hours. To isolate the effect of injected rhIGF-1, baseline-corrected concentrations were included by subtracting baseline concentration from measured concentrations. Results reported include area under the curve (AUC) 12 hours after the dose. Due to constraints of reporting platform, maximum corrected total IGF-I and free IGF-I, as well as time to maximum values separate outcomes.
| mcg*h/L | PAPP-A2 Deficient Patients | PAPP-A2 Heterozygous Relatives | Healthy Controls |
|---|---|---|---|
| AUC_total IGF-1_0-12h | 2118 (1602 to 2406) | 2405 (2068 to 2742) | 2878.5 (2755 to 3002) |
| AUC_free IGF-I_0-12h | 115.7 (95 to 133) | 135 (118 to 152) | 155.5 (64 to 247) |
Collected over The study adverse event data was collected over a period of 6 years (from baseline visit to one year post-treatment phone follow up).. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| PAPP-A2 Deficient Patients | 0/3 (0%) | 1/3 (33.3%) | 2/3 (66.7%) |
| PAPP-A2 Heterozygous Relatives | 0/2 (0%) | 0/2 (0%) | 0/2 (0%) |
| Healthy Control Participants | 0/2 (0%) | 0/2 (0%) | 0/2 (0%) |
| Event | PAPP-A2 Deficient Patients | PAPP-A2 Heterozygous Relatives | Healthy Control Participants |
|---|---|---|---|
| Pseudotumor cerebri in subjectNervous system disorders | 1/3 | 0/2 | 0/2 |
| Event | PAPP-A2 Deficient Patients | PAPP-A2 Heterozygous Relatives | Healthy Control Participants |
|---|---|---|---|
| Walking pneumoniaRespiratory, thoracic and mediastinal disorders | 1/3 | 0/2 | 0/2 |
| HeadachesNervous system disorders | 1/3 | 0/2 | 0/2 |
| Hair lossSkin and subcutaneous tissue disorders | 1/3 | 0/2 | 0/2 |
| FeverInfections and infestations | 1/3 | 0/2 | 0/2 |
| VomitingGastrointestinal disorders | 1/3 | 0/2 | 0/2 |
| Body aches and chillsInfections and infestations | 1/3 | 0/2 | 0/2 |
| Mild lipohypertrophySkin and subcutaneous tissue disorders | 1/3 | 0/2 | 0/2 |
| Flu type AInfections and infestations | 1/3 | 0/2 | 0/2 |
| Viral strep throatInfections and infestations | 1/3 | 0/2 | 0/2 |
| DiarrheaGastrointestinal disorders | 1/3 | 0/2 | 0/2 |
| Age, Categorical(Participants) | PAPP-A2 Deficient Patients | PAPP-A2 Heterozygous Relatives | Health Control Participants | Total |
|---|---|---|---|---|
| <=18 years | 2 | 0 | 0 | 2 |
| Between 18 and 65 years | 1 | 2 | 2 | 5 |
| >=65 years | 0 | 0 | 0 | 0 |
| Sex: Female, Male(Participants) | PAPP-A2 Deficient Patients | PAPP-A2 Heterozygous Relatives | Health Control Participants | Total |
|---|---|---|---|---|
| Female | 1 | 1 | 1 | 3 |
| Male | 2 | 1 | 1 | 4 |
| Race (NIH/OMB)(Participants) | PAPP-A2 Deficient Patients | PAPP-A2 Heterozygous Relatives | Health Control Participants | Total |
|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 |
| Asian | 3 | 2 | 0 | 5 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 | 0 |
| White | 0 | 0 | 1 | 1 |
| More than one race | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 1 | 1 |
| Region of Enrollment(Participants) | PAPP-A2 Deficient Patients | PAPP-A2 Heterozygous Relatives | Health Control Participants | Total |
|---|---|---|---|---|
| United States | 3 | 2 | 2 | 7 |
No study locations are listed for this record.
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — De-identifiable data may be shared with physician that is treating the only other two patients in the world with this genetic mutation.
Supporting information: Clinical study report
Time frame: The participant data will be available when the treatment trial is actively ongoing. Per study protocol, the study will provide clinical updates to the participants primary Endocrinologist. Should the family request any further information be shared once the study is closed, it can be given to a physician specified by the family.
Access criteria: De-identifiable data may be shared with physician that is treating the only other two patients in the world with this genetic mutation.
Ran from Dec 2015 to primary completion in Oct 2022, 81 months.
Results were first posted in Feb 2024, 1 year 4 months after primary completion.
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Studies of the drug tested here: Mecasermin
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