Height Standard Deviation Score
| Group | Value (standard deviation score) |
|---|---|
| Growth Hormone Treatment for Participants With ACAN Deficiency | -2.52 |
Trial · Interventional
CompletedPhase 1/2Results postedRegistry dataUnited States
1 trial · 1 completed · 1 with posted results
Growth Hormone Treatment in Patients With Aggrecan (ACAN) Deficiency
NCT032881031 Feb 2018 to 31 Aug 2023
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
This is an open-label, single-arm prospective pilot study to study the effects of a single dose regimen of daily growth hormone medication (Norditropin) on pre-pubertal children with Aggrecan deficiency. The growth response will be tracked over a 12 month period. A protocol extension has been approved to continue subjects on treatment for an additional 2 years.
Experimental
Growth Hormone Treatment for Participants with ACAN Deficiency
Pre-pubertal children with ACAN deficiency on daily growth hormone (Norditropin) regimen for a 3 year period.
Outcomes the sponsor listed as primary.
Height Standard Deviation Score
Time frame · Annually through three years of treatment
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 10 patients treated with recombinant human growth hormone.
Height Velocity After Three Years of Treatment With Recombinant Human Growth Hormone (rhGH)
Time frame · Annually through three years of treatment
A participants calculated height velocity derived from height measurements taken over a period of 36 months (baseline visit to 36 month visit). Only those in the treatment arm were treated with growth hormone and observed for response.
Number of Participants With Clinical Features of ACAN Deficiency - Osteochondritis Dissecans
Time frame · Baseline
Evidence of osteochondritis dissecans on MRI (in those who could cooperate to perform such unsedated between the ages of 6 years-old and 20 years-old) or radiograph examination of affected participant's knees.
Number of Participants With Clinical Features of ACAN Deficiency - Osteoarthritis
Time frame · Baseline
Evidence of early joint pathology evident (osteoarthritis) on MRI (in those who could cooperate to perform such unsedated between the ages of 6 years-old and 20 years-old) or radiograph examination of affected participant's knees, performed in those who were age appropriate and would cooperate.
Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.
| Group | Value (standard deviation score) |
|---|---|
| Growth Hormone Treatment for Participants With ACAN Deficiency | -2.52 |
| Group | Value (cm/year) |
|---|---|
| Growth Hormone Treatment for Participants With ACAN Deficiency | 5.2 |
| Group | Value (participants) |
|---|---|
| Pediatric Phenotype Group | 3 |
| Adult Phenotype Group | 1 |
| Group | Value (participants) |
|---|---|
| Pediatric Phenotype Group | 0 |
| Adult Phenotype Group | 8 |
| Group | Deaths | Serious events | Other events |
|---|---|---|---|
| Treatment GroupPatients with Aggrecan (ACAN) deficiency who have been treatment with human growth hormone (rHGH) for 3 years. | 0 / 10 | 0 / 10 | 10 / 10 |
| Phenotype GroupFirst degree relatives of patients in the treatment group who also have Aggrecan (ACAN) deficiency but did not participate in the treatment trial. | 0 / 12 | 0 / 12 | 0 / 12 |
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
Studies that cite this trial.
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References listed at the registry that are not in PepBank. They link to PubMed.
Who could join, as posted.
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