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Treatment of Rett Syndrome With rhIGF-1 (Mecasermin [rDNA]Injection)
Plain-language guide to NCT01253317: phase 1 interventional trial of IGF-1 LR3 and Mecasermin (rhIGF-1), 12 participants. Who took part, design and what was measured.
Key Facts
- Registry ID
- NCT01253317
- Phase
- phase 1
- Status
- completed
- Start
- 2010-12
- Completion (planned or actual)
- 2012-09
- Participants
- 12 (actual)
- Design
- na, open label (everyone knows who gets what), treatment
- Conditions studied
- Rett Syndrome
- Results posted
- yes
What This Study Did, in Plain Language
What Was Tested
In this phase 1 trial, researchers set out to test IGF-1 LR3 and Mecasermin (rhIGF-1).
As you may know, there is no treatment for this illness.
It had 1 arm (groups): rhIGF-1 (experimental).
The registry classes the interventions as drug.
More on the Plan
There are two treatment periods in the trial.
During in-patient sessions, an IV line will be placed for frequent blood samples.
A lumbar puncture will be performed by a physician at the beginning and again at the end of the MAD.
The primary goal of the MAD is to determine the safety of IGF-1 therapy for girls with RTT.
Who Took Part
Enrollment: 12 actual participants took part.
It was open to women only aged from 2 years up to 12 years.
Healthy volunteers were not accepted; participants needed the condition being studied.
Examples of who could join: Genetically defined mutation or deletion of the MECP2 gene. Chronological age must be 2 years or older.
The full record lists 5 inclusion and 4 exclusion criteria; among the exclusions: prior therapeutic use of igf-1, growth hormone, lupron® or sex steroids; allergy to the trial product; severe scoliosis (defined as a spinal curve of 70 degrees or more as measured on clinical and radiological examination).
How the Study Was Run
The set-up was single-group and open-label.
Being open-label, the IGF-1 LR3 study did not mask who received what.
Everyone received the same intervention, so there was no separate comparison group inside the trial.
It started in 2010-12 with completion listed for 2012-09.
It ran at 1 site in United States.
Its stated primary purpose is treatment.
What Was Measured
Primary outcome: Adverse Events (time frame: biweekly during the MAD and every five weeks during the OLE).
It also tracked 2 secondary outcomes, for example Change From Pre-MAD Apnea Index at Post-OLE and Change in Social Avoidance Subscale Scores on the ADAMS From Pre-OLE to Post-OLE.
What This Means in Plain Language
Put simply, this is a phase 1 trial of IGF-1 LR3 and Mecasermin (rhIGF-1) in 12 people.
Its size is small, so on its own it can only give an early look.
The trial is marked completed; results are posted on the registry, and this page does not restate them.
More context: the IGF-1 LR3 compound page and the Mecasermin (rhIGF-1) compound page, all human trials.
Our Notes on the Compounds
IGF-1 LR3: A modified IGF-1 analog that escapes binding proteins, so it stays active ≥24 h.
Mecasermin (rhIGF-1): FDA-approved recombinant human IGF-1 for severe primary IGF-1 deficiency in children; distinct from IGF-1 LR3 and IGF-1 DES.
Related Pages
Source: ClinicalTrials.gov record. This explanation is written by PeptideWhiz from the public record. For research and education only. Not medical advice; no dosing information is given.