Trial · NCT05559801
Registered outcomes20
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 20 of 20
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Recruitment rate of participants | Up to 24 months post-intervention | Number of participants consented at the end of the trial |
| Primary | Number of participants with correctly collected data for the study outcomes | Up to 24 months post-intervention | Number of participants with data correctly collected for the study outcomes |
| Primary | Total number of visits with protocol deviation | Up to 24 months post-intervention | Total number of visits with protocol deviations at the end of the trial |
| Primary | Visit attendance by participants | Up to 1 year post last infusion intervention | Percentage of visits completed by participants |
| Primary | Patient retention rate | Up to 1 year post last infusion intervention | Percentage of patients that complete the study intervention and all follow up study visits |
| Primary | Patient Primary Clinical Outcome Retention Rate | Up to 1 year post last infusion intervention | Percentage of participants that completed the primary clinical outcome measurements |
| Primary | Change in acceptability from baseline | Baseline, Up to 36 months post-intervention | Acceptability will be evaluated with an end of study structured, verbal interview. This interview will consist of a mix of closed ended (scaled questions from 1\[worst\]-5 \[best\]) and open-ended questions giving subjective measurements. Open-ended questions will address overall experience during the study and prompt expression of thoughts or perspectives after completing the study. The total possible score range is 18 to 90. Higher score correlates with better outcome. |
| Primary | Number of unexpected adverse events | Up to 1 year post last infusion intervention | Unexpected adverse events that occur from first study intervention to one year after last infusion. |
| Primary | Number of participants with change in vital signs from baseline | Baseline, 10 minutes pre-intervention, 15 minutes post-intervention, 30 minutes post-intervention, 1 hour post-intervention, 1 hour post completion of the infusion | Number of participants with blood pressure, oxygen saturation, heart rate, respiratory rate, and temperature below or above each participants accepted mean reference range. |
| Primary | Number of participants with changes in laboratory panels from baseline (CBC, CMP) | Baseline, every 4 months post-intervention | Number of patients with changes in CBC or CMP values from baseline CBC or CMP values. Any evidence of thrombocytopenia, anemia, neutropenia, or elevations in white blood counts will require consideration of stopping or pausing infusions by the PI. Slight changes in the ALT, AST, and calcium values are expected due to standard medication uses and will be monitored by PI. |
| Primary | Change in number of participants with changes in radiological parameters | Baseline, 8, 20 and 32 months post-intervention | Number of participants with changes in radiological parameters (xrays and DXA scan) from baseline will be monitored during the study follow up time. DXA scans and x-rays used to assess for any semi-triggers that may require considerations of stopping or pausing infusions. |
| Primary | Change in annualized linear growth velocity | Baseline, every 4 months until 1 year after last infusion intervention | This study will assess the annualized linear growth velocity at baseline and during cell therapy protocol in children with Type 3 OI (3-10 years of age and pre-pubertal at time of enrollment). Growth charts will be generated using growth measurements obtained every 4 months. Growth charts will be referenced against sex- and age-specific Centers for Disease Control and Prevention (CDC) childhood OI growth reference charts. |
| Primary | Change in fracture rate from baseline | Baseline, every 4 months post-intervention until 1 year after last infusion intervention | Overt and covert fracture rate will be measured in each child at baseline and at regular intervals during MSC therapy by using a caretaker events diary, limb films, and spine films to tally more objectively new and healing fractures. |
| Primary | Change in bone mineral density from baseline | Baseline, yearly until 1 year after last infusion intervention | Bone mineral density will be determined by DXA (dual-energy absorptiometry) analysis for TBLH (total body less head), spine and distal radius sites. |
| Primary | Change in bone age | Baseline, yearly until 1 year after last infusion intervention | A bone age assessment is done using left hand radiograph. Bone age will be followed yearly in children of 8 years of age and older at time of enrollment in conjunction with exam and laboratory assessments to determine potential effects of puberty on bone density. |
| Primary | Change in Vertebral Compression Fractures (VCFs) | Baseline, every 18 months until one year after last infusion intervention | Due to abnormal collagen and the associated decrease in BMD, VCFs are common in Type 3 OI. These can be 'silent' and/or associated with back pain. PA TL film and LAT TL film will be obtained to assess VCF's that cannot be assessed by DXA in children. Spine radiographs are within standard of care for treatment of OI. |
| Secondary | Number of participants with changes in bone metabolism markers | Baseline, every 12 months post-intervention, and at the conclusion of the study (up to 36 months post-intervention) | Markers including osteocalcin, BAP, CTX, PICP, Calcium/Creatinine Ratio (urine), and NTX/Creatinine Ratio (urine) will be assessed for both bone health as well as bone formation/resorption. |
| Secondary | Change in Limb Deformity Modified EOS Questionnaire (LD-EOSQ-22) from baseline | Baseline, annually until 1 year after last infusion | This is a validated survey that will serve as a patient-reported outcome measure evaluating health status for these patients and the burden of their caregivers. The scores can range from 0-100 with a higher score correlating with a better outcome. |
| Secondary | Change in BAMF (Brief Assessment of Motor Function) from baseline | Baseline, annually until 1 year after last infusion | This validated survey contains 10 point ordinal scales that outline rapid descriptions for gross and fine motor performance. The higher you score on the BAMF survey, the more developed your gross and fine motor skills are. |
| Secondary | Change in Pediatric Orthopedic Data Collection Instrument (PODCI) from baseline | Baseline, annually until 1 year after last intervention | The PODCI questionnaire will used to quantify function abilities in our studied population. The scores can range from 0-100, with higher scores indicating higher levels of disability, and lower scores indicating better functioning. |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
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